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Topline Phase 3 Results of Hengrui Pharma and Kailera Therapeutics’ Dual Agonist HRS9531 Shows Promising Weight Loss: A Rising Competitor to Zepbound?

July 22, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

19 July 2025

Category: Clinical Trials & Drug Development I

Obesity & Metabolic Disorders

Written by: Pharmacally Medical News Desk

In a key event, Chinese pharmaceutical giant Hengrui Pharma and U.S.-based biotech firm Kailera Therapeutics have reported inspiring Phase 3 results for their novel weight-loss drug candidate, HRS9531. This once-weekly injectable drug, a dual GLP-1/GIP receptor agonist, demonstrated weight reduction effects that match with its closest rival of Eli Lilly’s tirzepatide (Zepbound), showcasing it as a strong potential alternative in the anti-obesity therapeutics market.

The HRS9531 program was initiated by Hengrui Pharma and is based on a GLP-1 receptor agonist platform aimed at treating metabolic disorders. In 2023, Hengrui partnered with U.S.-based Kailera Therapeutics to advance the international development of HRS9531. This collaboration supports the global clinical progression of the drug in what is considered to be the most competitive field of GLP-1-based therapies.

Trial Design and Results

The HRS9531‑301 trial (NCT06396429) is a randomized, double-blind, placebo-controlled, multicenter Phase 3 clinical study conducted across multiple sites in China. The study was sponsored by Hengrui Pharma and evaluated the efficacy and safety of HRS9531. The trial spanned 48 weeks of treatment, with an additional follow-up period to monitor long-term effects and adverse events. The trial was designed in a way that it will support regulatory approval in China and other markets.

The study enrolled 567 adults aged more than 18 years with a mean weight of 93 kg with obesity or overweight status as defined by a BMI ≥28 kg/m² or a BMI ≥24 kg/m² with at least one obesity-related comorbidity (e.g., hypertension, dyslipidemia, sleep apnea).

Key exclusion criteria included a diagnosis of type 1 or type 2 diabetes, History of significant gastrointestinal disease, prior or ongoing anti-obesity pharmacotherapy or bariatric surgery, and uncontrolled psychiatric illness or endocrine disorders.

This strict inclusion-exclusion criterion was designed to isolate the weight-loss effect of HRS9531 in a non-diabetic population; the trial will mimic the real-world use cases for first-line obesity pharmacotherapy.

Participants were randomized to receive once-weekly subcutaneous injections of HRS9531 at 2 mg, 4 mg, or 6 mg, or Placebo. All participants followed a dose-escalation schedule during the early phase to improve gastrointestinal tolerability, followed by fixed-dose maintenance through week 48. Lifestyle advice was given to all groups (diet and physical activity guidelines), in line with global obesity trial standards (similar to SURMOUNT and STEP trials).

Primary Endpoint includes percent change in body weight from baseline to week 48; key secondary endpoints include the proportion of participants achieving ≥5%, ≥10%, ≥15%, and ≥20% weight loss. Absolute weight change (kg), Changes in waist circumference, BMI, metabolic biomarkers (lipid levels, liver enzymes, blood pressure), and Patient-reported outcomes (quality of life, satiety). These endpoints reflect a comprehensive and clinically meaningful set of outcomes that regulatory bodies typically require for obesity drug approvals.

HRS9531 showed robust and dose-dependent weight loss, with top-line data showing at a 6 mg dose a mean body weight reduction of 17.7% vs baseline, Placebo-adjusted difference of 14.1%, a 4 mg dose with 14.5% weight loss, and a 2 mg dose with 9.8% weight loss.

88.4% of participants in the 6 mg arm lost ≥5% body weight, 70.9% achieved ≥10%, 51.2% achieved ≥15%, and 44.2% achieved ≥20% weight loss

These efficacy results are comparable to or even exceed benchmarks seen with established drugs like semaglutide (Wegovy) and tirzepatide (Zepbound) in similar populations.

Mechanism of Action

Like tirzepatide, HRS9531 is a dual agonist targeting GLP-1 (Glucagon-like peptide-1) receptors, which enhances insulin secretion, reduces appetite, and delays gastric emptying, along with GIP (Glucose-dependent insulinotropic polypeptide) receptors, which may enhance insulin response and contribute to weight loss synergistically with GLP-1.

This “twin hormone” approach is currently viewed as the most promising of the coming years’ obesity drugs, offering superior weight reduction compared to GLP-1-only therapies like semaglutide (Wegovy, Ozempic).

Safety

HRS9531 showed a favorable safety and tolerability profile consistent with other GLP-1/GIP receptor agonists. The most commonly reported adverse events were gastrointestinal.

Previous Phase 2 trial

In the previously reported Phase 2 clinical trial (NCT06054698), the primary analysis using the treatment policy estimand showed that participants receiving the 8 mg dose of HRS9531 experienced a mean weight loss of 22.8% at week 36, which corresponds to a placebo-adjusted reduction of 21.1%.

Key comparisons

Parameter	Tirzepatide (Zepbound)	HRS9531 (China Phase 3)
Weight Loss (Avg)	21%	18–19.2%
≥20% Weight Loss	36–40%	44%
Duration	Up to 72 weeks	48 weeks (long-term study is initiated)
Safety Profile	Gastrointestinal AEs	Gastrointestinal AEs

With HRS9531 still showing weight loss at 48 weeks, longer-duration trials in global populations may demonstrate even higher efficacy. This makes the candidate a serious challenger in upcoming global obesity markets, particularly in the U.S. and EU, where Kailera is preparing further development programs.

“The positive data from the HRS9531-301 study demonstrated meaningful, sustained weight loss. With an affirmed safety and tolerability profile, we strongly believe in its potential to help more people living with obesity reach their individual weight loss goals,” said Hong Chen, Head of Metabolism Department I of Hengrui Pharma

Ron Renaud, President and Chief Executive Officer of Kailera, added that “As Kailera prepares to advance KAI-9531 into a global clinical program, we look forward to evaluating both higher doses and longer durations of treatment to expand on KAI-9531’s best-in-class potential.”

Conclusion
The strong Phase 3 results of HRS9531 mark a significant milestone for Hengrui Pharma and Kailera Therapeutics. With near-Zepbound efficacy, a manageable safety profile, and ongoing weight loss even at 48 weeks, this dual agonist could become a highly competitive player in the anti-obesity space.

Kailera now plans to initiate global Phase 3 trials with the global name KAI-9531, exploring higher doses and extended durations, to match or exceed Zepbound’s performance. If successful, HRS9531 could become one of the first Chinese-origin obesity drugs to secure international regulatory approval.

References

Hengrui Pharma and Kailera Therapeutics Report Positive Topline Data from Phase 3 Obesity Trial in China of Dual GLP-1/GIP Receptor Agonist HRS9531, GlobNewswire, 15 July 2025, https://www.globenewswire.com/news-release/2025/07/15/3115481/0/en/Hengrui-Pharma-and-Kailera-Therapeutics-Report-Positive-Topline-Data-from-Phase-3-Obesity-Trial-in-China-of-Dual-GLP-1-GIP-Receptor-Agonist-HRS9531.html?utm_source=chatgpt.com

Hengrui Pharma and Kailera Therapeutics Report Positive Topline Data from Phase 3 Obesity Trial in China of Dual GLP-1/GIP Receptor Agonist HRS9531, BioSpace, 16 July 2025, https://www.biospace.com/press-releases/hengrui-pharma-and-kailera-therapeutics-report-positive-topline-data-from-phase-3-obesity-trial-in-china-of-dual-glp-1-gip-receptor-agonist-hrs9531

Hengrui and Kailera report positive data from Phase III obesity treatment trial, Clinical Trial Area, 16 July 2025, https://www.clinicaltrialsarena.com/news/hengrui-kailera-obesity-treatment/?cf-view&cf-closed&cf-closed

Efficacy and Safety of HRS9531 Injections in Overweight or Obese Subjects, ClinicalTrials.gov ID NCT06054698, ClinicalTrials.gov, https://clinicaltrials.gov/study/NCT06054698?term=HRS9531&rank=10#participation-criteria

To Compare the Efficacy and Safety of HRS9531 and Placebo in Subjects With Overweight or Obese, ClinicalTrials.gov ID NCT06396429, ClinicalTrials.gov, https://clinicaltrials.gov/study/NCT06396429#participation-criteria

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CDSCO Issues Advisory on Flushing 17 Expired Drugs: A Crucial Step for Public Health and Safety

July 13, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

13 July 2025

Category: Drug Regulation and Public Health Policy I

Drug Safety & Disposal I Pharmaceutical Waste Management

Written By:

Pharmacally Medical News Desk

“In past instances, when you have taken any medicine and completed the course, what did you do with the leftover medicines? Did you keep them for future use until expiry, or did you throw them in the dustbin?” Most of us either store leftover medicines for future use and forget about them altogether or discard them carelessly, unaware of the risks.

The safe disposal of medicines, whether used or expired, is important because every medicine contains various chemicals and active ingredients. Even though they are expired or unexpired, they still possess some activity. Whenever medicines are released into the environment, intentionally or unintentionally, such as by throwing them in the garbage or flushing them down the toilet, they pose a serious threat to the environment, aquatic life, wild animals, and birds. In due course, these threats loop back to humans, sometimes in the form of antibiotic resistance and other health hazards. To address this issue, strong rules and regulations are needed from national and international health regulatory bodies.

In a significant move, the Central Drugs Standard Control Organisation (CDSCO), India, under the Ministry of Health and Family Welfare, has released a comprehensive guidance document for the disposal of expired and unused medicines.

This advisory lays down detailed procedures for the safe disposal of expired drugs, specifically intended for drug manufacturers, wholesalers, and retailers. It outlines the complete process for how expired medicines should be handled and disposed of. Additionally, the advisory includes important instructions for the general public regarding the safe disposal of certain medication that may be harmful or, to some extent, fatal with just one dose, so such expired and unused medication should not be kept at a home where it is accessible to any person or pet. Such medication should be flushed down the toilet or sink to prevent danger. A total of 17 such critical expired drug categories have been identified, and specific guidelines have been provided on how to dispose of them safely, even if only one dose remains.

The identified 17 drugs are

The following medicines, if found unused or expired at the home, should be immediately flushed down the toilet or sink

Sr. No.	Name of Drugs
1	Fentanyl
2	Fentanyl Citrate
3	Morphine Sulfate
4	Buprenorphine
5	Buprenorphine Hydrochloride
6	Methylphenidate
7	Meperidine Hydrochloride
8	Diazepam
9	Hydromorphone Hydrochloride
10	Methadone Hydrochloride
11	Hydrocodone Bitartrate
12	Tapentadol
13	Oxymorphone Hydrochloride
14	Oxycodone
15	Oxycodone Hydrochloride
16	Sodium Oxybate
17	Tramadol

Why Only These 17 Medicines recommended for Flushing

The CDSCO has advised that only a select list of 17 medicines should be flushed down the sink or toilet by the general public, and this recommendation is not arbitrary. It is based on the unique risks posed by these specific drugs. Here’s why they were selected:

High Risk of Accidental Exposure

These drugs include potent opioids, sedatives, and central nervous system depressants like fentanyl, oxycodone, methadone, and morphine. Even a single dose can be fatal, especially to children, pets, or individuals not prescribed the medication. Flushing eliminates the risk of these drugs being accidentally ingested if left in household trash.

Potential for Misuse and Abuse

These medicines are highly addictive and frequently misused. Retaining unused doses at home increases the risk of theft, diversion, or illicit use. Flushing ensures immediate and irreversible disposal, minimizing the chance of misuse.

Inadequate Take-Back Infrastructure

In many areas, especially rural or underserved regions, drug take-back facilities or hazardous waste facilities may not be available. Flushing offers a practical, immediate, and risk-reducing option for these specific high-risk drugs.

The most important question: Is it justifiable to flush the medicine into the environment?

While flushing most medications is generally discouraged due to the risk of environmental contamination, particularly to aquatic life, the potential danger to human life from improper retention of these 17 high-risk drugs outweighs environmental concerns. Recognizing this, agencies such as the US FDA and WHO support flushing such medicines under specific conditions. The Biomedical Waste Management Rules, 2016, also acknowledge the need for secure and immediate disposal of certain hazardous pharmaceuticals. Also, since these drugs are regulated under the NDPS Act or are Schedule H/Narcotic drugs, secure disposal becomes even more critical.

Legal Framework and Compliance

This CDSCO advisory draws legal support from some of the strong rules and regulations; these rules provide the legal foundation for the safe disposal of expired and unused medicines in India. They ensure that drugs, especially hazardous, narcotic, or radioactive ones, are handled, stored, and destroyed in a way that protects public health. These rules are

Drugs and Cosmetics Rules, 1945
Biomedical Waste Management Rules, 2016
Narcotic Drugs and Psychotropic Substances Act, 1985
Atomic Energy Act, 1962 (for radioactive drugs)

Conclusion

As one of the largest pharmaceutical markets in the world, India faces a unique responsibility in ensuring not just access to medicines but also their safe disposal. Unfortunately, public awareness around the correct methods of disposing of expired or unused drugs remains low. This becomes especially dangerous when it comes to some high-risk medicines such as opioids and controlled substances, which, if left unused at home, can lead to fatal accidental ingestion, misuse, or addiction. While flushing most medicines is generally discouraged due to environmental concerns, in the case of these specific drugs, the immediate threat to human life far outweighs potential ecological risks. Recognizing this, CDSCO’s advisory prioritizes safety over contamination fears, aligning with global practices.

References

Guidance document on disposal of expired/unused drugs (WI/01/DCC-P-25), CDSCO, Directorate General of Health Services, India, 26 May 2025

https://cdsco.gov.in/opencms/resources/UploadCDSCOWeb/2018/UploadPublic_NoticesFiles/Guidance%20document%20on%20disposal.pd

Drug Disposal: FDA’s Flush List for Certain Medicines, US Food and Drug and Administration, https://www.fda.gov/drugs/disposal-unused-medicines-what-you-should-know/drug-disposal-fdas-flush-list-certain-medicines

Guidelines for the Control of Narcotic and Psychotropic Substances, World Health Organization 1984, https://iris.who.int/bitstream/handle/10665/39299/9241541725_eng.pdf

Alphabet’s Inc. subsidiary Isomorphic Labs Initiates Human Trials of First AI-Designed Drug: the Beginning of a New Era in Precision Therapeutics

July 9, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

09 July 2025

Category: Drug Discovery and Development I

AI in Drug Development

Written By: Pharmacally Medical News Desk

Isomorphic Labs, a wholly owned subsidiary of Alphabet Inc., has announced the commencement of human trials for its first AI-designed drug. The molecule is still undisclosed for competitive and regulatory reasons. This announcement is an important step in bringing artificial intelligence (AI) and medicine together. It shows that AI is no longer just an idea for the future; it is now being tested and used in real medical situations and drug discovery processes.

This project is driven by AlphaFold 3, the newest version of a powerful AI tool that can accurately predict the shape of proteins and how they interact with other molecules. It was created by Isomorphic Labs and Google DeepMind, working together to shape the future of using computers and AI to discover new medicines.

About Isomorphic Labs

Isomorphic Labs was started in 2021 by Demis Hassabis, who is also the CEO and co-founder of Google DeepMind. The main goal of the company is to completely rethink how new medicines are discovered, using artificial intelligence (AI) from the very beginning of the process.

While DeepMind continues to focus on developing advanced AI technologies, Isomorphic Labs puts those AI tools to practical use. It applies them to real biological challenges, such as understanding how diseases work and designing new drugs to treat them.

Isomorphic Labs is working closely by connecting biology, physics, chemistry, and AI together, trying to make drug development faster and more successful. Normally, it takes 10 to 15 years to develop a new drug, and many trials fail along the way. The company’s goal is to use AI to lower failure rates and speed up this long and costly process.

Isomorphic Labs has collaborated with Novartis and Eli Lilly for drug discovery.

About AlphaFold

This breakthrough began with the launch of AlphaFold, an AI system developed by DeepMind in 2020. AlphaFold has solved a major scientific problem that had puzzled researchers for over 50 years: how to predict the 3D shape of proteins just from their amino acid sequences. This was a huge step forward because understanding protein structures is key to understanding how the human body works and how diseases develop.

AlphaFold had such a huge impact on science that its creators, John Jumper and Demis Hassabis, were awarded the 2024 Nobel Prize in Chemistry. Their work was honored for helping decode the structural code of life, how proteins are shaped, which is essential to understanding biology and disease.

First into the Human Body

The complete details are not disclosed; early reports indicate that it is designed to act on a new and previously unexplored biological pathway, which is linked to a disease that currently lacks effective treatment options. The drug has shown promising results in preclinical studies, demonstrating favorable pharmacokinetics, along with low toxicity and strong effectiveness in early testing models. The drug was entirely developed using artificial intelligence, with little or no reliance on traditional high-throughput compound screening.

The Phase 1 trial, now underway in the UK and potentially expanding to Europe and the US, will assess:

In early-stage human trials, the focus will be on evaluating the drug’s safety and tolerability in healthy volunteers. Researchers will also study its pharmacokinetics and pharmacodynamics; additionally, the trials will help determine the optimal dose to be used in future clinical studies, ensuring both effectiveness and safety in patient populations.

This trial will employ adaptive design elements where AI tools may continue to monitor biomarker responses in real time, potentially guiding dosage adjustments mid-study

Next Step

According to company officials, this is only the beginning for Isomorphic Labs. The company is actively building a pipeline of AI-designed drugs targeting a wide range of conditions, including cancer, rare diseases, and infectious diseases. It is also forming strategic partnerships with top academic institutions and biotech companies to accelerate innovation. In parallel, Isomorphic Labs is working to expand AlphaFold 3’s capabilities beyond protein prediction into areas like RNA-targeting drugs, enzyme engineering, and vaccine design, opening up new frontiers in drug discovery.

References

Jumper, J et al. Highly accurate protein structure prediction with AlphaFold. Nature (2021).

Varadi, M et al. AlphaFold Protein Structure Database in 2024: providing structure coverage for over 214 million protein sequences. Nucleic Acids Research (2024)

Google’s secret weapon against cancer might be an AI algorithm, Business Today

DeepMind AlphaFold DB (2021–2024), https://www.alphafold.ebi.ac.uk

NobelPrize.org (2024), Chemistry Prize for Protein Structure Prediction

Isomorphic Labs official website, https://www.isomorphiclabs.com

DeepMind Research Blog: https://www.deepmind.com/blog

Can the Weight Loss Indication of Semaglutide and Tirzepatide Be Revoked in India? Understanding the Recent PIL against GLP-1 Drugs

July 8, 2025

by official.ajinkya11@gmail.com with No Comment Disease & Drugs Health Tidings

08 July 2025

Category: Drug Safety & Regulation

Written by:

Pharmacally Medical News Desk

A recent Public Interest Litigation (PIL) filed by Jitendra Chouksey, founder of FITTR, in the Delhi High Court has sparked nationwide discussion over the misuse of GLP-1 receptor agonists, notably semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro, Zepbound), as an aesthetic weight loss agent in India. The court has now directed the Indian drug regulatory authority, the Central Drug Standard Control Organization (CDSCO), to reassess its approval, raising critical questions about drug safety, misuse, and the ethics of cosmetic pharmacology. The petition argues that these have been approved and marketed in India without robust local safety and efficacy trials and are being misused by gym-goers, wellness influencers, gyms, and clinics as a “magic formula” for aesthetic weight reduction.

The petition claims:

These drugs have been approved in India without robust local clinical trials.
There is rampant misuse of cosmetic weight loss, driven by social media and unqualified prescribers.
Users are being exposed to serious side effects like pancreatitis, thyroid/pancreatic cancers, cardiovascular events, and vision loss.
The phenomenon is driven more by body image trends than actual medical needs, raising a public health concern

Delhi High Court’s Response

On July 1–2, 2025, the Delhi High Court bench of Chief Justice Devendra K. Upadhyaya and Justice Tushar Rao Gedela issued key directions:

The Central Drugs Standard Control Organisation (CDSCO) and the Drug Controller General of India (DCGI) must review the PIL representation within three months, involving all stakeholders, including doctors, manufacturers, and the petitioner.
The court allowed the petitioner to submit additional scientific evidence within two weeks.
Strong remarks were made about the unauthorized promotion of these drugs by gym trainers and influencers, stating it could be a matter for criminal investigation.
Observed that if regulatory bodies fail to act, the court may entertain another PIL on similar grounds (a liberty granted to the petitioner)

Why Is This Significant for India?

India is witnessing a surge in aesthetic- and fitness-focused drug use, especially among urban youth. While GLP-1 RA drugs are proven effective for managing obesity in specific populations (e.g., those with diabetes or severe obesity), their off-label use for cosmetic slimming poses several challenges:

Indian experts say that genetic, dietary, and lifestyle differences could lead to different side‑effect profiles in Indian patients, warranting specific trials.
No Indian-specific safety data exists yet for long-term use in non-diabetic individuals.
The drug retail system lacks strict control, leading to unsupervised access.
The Indian market is highly price-sensitive, and cheaper versions or black-market imports could aggravate misuse.
The trend is amplified by social media influencers, leading to the normalization of injectable “slimming shots” among teenagers and young adults.

Furthermore, India has previously banned irrational drug combinations lacking local trial data. This PIL could establish a model for evaluating imported drug approvals through an Indian regulatory lens, balancing public demand with safety.

Next steps & possible outcomes

Review by DCGI/CDSCO
Within three months, they must evaluate:

Whether local trials and safety data justify the drugs’ weight-loss approvals
If marketing is overly permissive or misleading
Whether stricter controls, a contraindication for non-diabetics, brand-label rewriting, or pharmacovigilance systems are needed

Potential outcomes

Approvals may be maintained with added safeguards.
Labeling could be tightened, restricting use to diabetics or those with morbid obesity under supervision.
Non-diabetic use might be revoked or suspended, pending new trials.
Enforcement actions may be directed against gyms/clinics illegally prescribing or administering them.

Further legal action

If the government/regulators fail to act satisfactorily, the petitioner can file another PIL to compel stricter measures or revocation.

Summary

The PIL is a major challenge to the current GLP-1 RA drug approval, allowing them to be used for weight loss, raising critical issues such as off-label usage, aesthetic-driven misuse, lack of India-specific safety data, and commercialization without adequate oversight. The Delhi High Court’s directive to the CDSCO/DCGI to act by early October 2025 will be a crucial moment. Their decision, ranging from maintaining the status quo with safety warnings and limiting to prescription-only access, to revoking the weight-loss indication, could significantly shape the future of anti-obesity drug regulation in India.

References

Delhi HC seeks regulator’s reply after PIL questions Ozempic, Mounjaro nod, The Economic Times, 03 July 2025, https://economictimes.indiatimes.com/industry/healthcare/biotech/pharmaceuticals/delhi-hc-seeks-regulators-reply-after-pil-questions-ozempic-mounjaro-nod/articleshow/122211529.cms?utm_source=chatgpt.com

Decide within 3 months’: Delhi HC tells CDSCO on plea against use of diabetes drugs in weight management, The Healthy Indian Project (THIP), 02 July 2025, https://www.thip.media/news/decide-within-3-months-delhi-hc-tells-cdsco-on-plea-against-use-of-diabetes-drugs-in-weight-management/120177/?utm_source=chatgpt.com

Delhi HC ‘alarm’ over misuse of diabetes drug for weight loss, News Arena India, 03 July 2025, https://newsarenaindia.com/nation/delhi-hc-alarm-over-misuse-of-diabetes-drug-for-weight-loss/49182?utm_source=chatgpt.com

Wegovy vs. Mounjaro: A Head-On Comparison at the Dawn of Wegovy’s Launch in India

June 30, 2025

by official.ajinkya11@gmail.com with No Comment Disease & Drugs Health Tidings

30 June 2025

Category: Pharmaceutical Market Analysis I Drug Comparison & Reviews I

Obesity Therapeutics

Written by:

Priyanka Khamkar, MPharm, and

Vikas Londhe, MPharm

Reviewed By:

Pharmacally Editorial Team

Wegovy (Semaglutide), the widely acclaimed GLP-1 agonist for weight loss, has officially launched in India by Danish pharmaceutical giant Novo Nordisk, following its overseas success. Wegovy’s launch has set the stage for a direct face-off with Eli Lilly’s Mounjaro (Tirzepatide), which entered the Indian market just three months earlier.

India is now home to the second-largest number of adults living with diabetes; obesity is also on the rise due to interdependent metabolic pathways, poor dietary habits, and increasingly sedentary urban lifestyles. Earlier in India, obesity was considered to be a lifestyle issue, and spending money on anti-obesity drugs was never on the priority list. But as the awareness about obesity and its associated health conditions is increased due to social media, physician counseling, the rise of nutritional health experts, and increased link of obesity with other ailments, it is now being recognized as a chronic, relapsing disease with serious health consequences, including cardiovascular disease, type-2 diabetes, and certain cancers.

Mounjaro, launched earlier this year in March, has already gained a foothold due to its superior weight loss efficacy (up to 23%) and promising results in both diabetes and obesity treatment. In contrast, Wegovy, which got approval in June 2025, has earned widespread global recognition for its effectiveness in achieving 15–20% weight loss and its cardiovascular benefits.

Wegovy is now available in major Indian cities, offering a new option for patients struggling with obesity and its associated health burdens. Its entry indicated not just another medicine option apart from Mounjaro, but a broader shift in India’s healthcare arena, where obesity is finally being addressed with serious medical intervention.

Drug Profiles & Mechanisms

Wegovy (semaglutide)

Type: Once-weekly GLP-1 receptor agonist
Mechanism of Action: Mimics glucagon-like peptide-1 (GLP-1), a natural satiety hormone. It reduces appetite, slows gastric emptying, and enhances insulin secretion, leading to reduced calorie intake and improved metabolic control.
Approved Indications: Approved in India as the first obesity-specific medication with a clear indication for chronic weight management
Also approved for cardiovascular risk reduction in overweight/obese individuals with established heart disease (based on the the SELECT trial).
Clinical Trials & Efficacy: STEP trials (Semaglutide Treatment Effect in People with Obesity): Demonstrated average weight loss of 15%, with some patients reaching up to 20% at higher doses.
Indian data indicates that 1 in 3 patients may achieve 20% weight loss with consistent high-dose use.

Mounjaro (Tirzepatide)

Type: Once-weekly dual GIP and GLP-1 receptor agonist
Mechanism of Action: Acts on both glucose-dependent insulinotropic polypeptide (GIP) and GLP-1 receptors, offering synergistic metabolic effects. It improves insulin sensitivity, reduces appetite, enhances satiety, and facilitates significant fat loss.
Approved Indications: Initially approved in India for type 2 diabetes, now also approved for chronic weight management. In June 2025, India approved the Mounjaro KwikPen covering full doses (2.5–15 mg) for easier self-administration.
Clinical Trials & Efficacy: SURMOUNT trials, particularly SURMOUNT-1 and SURMOUNT-5, showed average weight loss of 20–22.5%, outperforming semaglutide in head-to-head comparisons.
Recognized for offering one of the highest weight reductions ever seen in obesity pharmacotherapy.

Launch Timeline

Mounjaro launched in India in March 2025 via vials.
Mounjaro KwikPen launched in June 2025.
Wegovy launched on June 24, 2025

Dosage and Delivery Availability

Features	Wegovy Prefilled Pen	Mounjaro Vial	Mounjaro KwikPen (Approved in June 2025)
Device Type	FlexPen (prefilled, single-use pen)	Vial for manual injection	KwikPen (prefilled, auto-injector)
Dose Range	0.25 mg – 2.4 mg	2.5 mg – 5 mg	2.5 mg – 15 mg
Dosing Frequency	Once Weekly	Once Weekly	Once Weekly

Wegovy is available in 5 different doses ranging from 0.25 mg, 0.5 mg, 1.0 mg, 1.7 mg, and 2.4 mg. It is available as a single-use FlexPen covering all step-up doses (0.25 mg to 2.4 mg), administered once a week.

The Mounjaro vial provides limited dose options available in two doses, 2.5 mg and 5 mg, which require manual preparation before injection.

Mounjaro, recently approved as a prefilled auto-injector in June 2025, named Mounjaro KwikPen, expands the dosing range up to 15 mg, enabling more convenient and auto-injector-based delivery, administered once weekly.

Pricing of Wegovy

Dose	Per Dose Pricing	Monthly Pricing
0.25mg	Rs. 4366	Rs. 17345
0.5mg	Rs. 4366	Rs. 17345
1.0mg	Rs. 4366	Rs. 17345
1.7mg	Rs. 6070	Rs. 24280
2.4mg	Rs. 6503	Rs. 26015

Pricing of Mounjaro

Dose	Per-Dose Pricing	Monthly Pricing
2.5 mg	Rs. 3500	Rs. 14000
5.0 mg	Rs. 4375	Rs. 17500

Wegovy (semaglutide) is priced uniformly at ₹4,366 per dose for the initial lower doses (0.25 mg to 1.0 mg), with prices increasing for higher doses: 1.7 mg: ₹6,070 per dose (₹24,280/month); 2.4 mg (maintenance dose): ₹6,503 per dose (₹26,015/month)

Mounjaro (Tirzepatide) is more cost-effective in comparison: 2.5 mg (starter dose): ₹3,500 per dose (₹14,000/month); 5.0 mg (maintenance dose): ₹4,375 per dose (₹17,500/month)

Mounjaro offers a significantly more affordable monthly treatment, up to ₹8,500 less per month at maintenance doses, making it a cost-attractive option in India’s growing anti-obesity drug market. However, Wegovy is looking more expensive as compared to Mounjaro, but it is backed by robust global clinical trial data and proven long-term efficacy.

Market Growth and Patient Response

As Mounjaro is having a first-mover advantage, as it is launched in March 2025, it shows explosive growth in its first three months of sales. According to Business Today, data from March to May reveals a steady month-on-month increase in both value and volume sales. Revenue grew from ₹3.46 crore in March to ₹7.87 crore in April and ₹12.60 crore in May, with a cumulative total of ₹23.94 crore over three months. Similarly, unit sales increased from 11,640 units in March to 27,650 in April and further to 42,280 in May, bringing the total to over 81,000 units sold during this period. These figures reflect a strong ascending trend in physician prescribing and patient onboarding since the drug’s market entry in March.

Wegovy, which has now entered the Indian market, is expected to benefit from the same rising demand seen with Mounjaro. Previously, Wegovy faced global supply issues with limited production and distribution due to high demand worldwide. However, those supply problems have now mostly been resolved, specifically for the Indian market, allowing a more stable and consistent availability of the drug in India. This positions Wegovy to compete effectively and meet growing patient needs without the delays or shortages experienced earlier in other countries.

Market Size & Growth

As per the report of HORIZON Grand View Research, India’s anti-obesity medication market is ready for explosive growth. In 2023, the market generated revenue of approximately ₹1,525 crore (USD 183.4 million), and it is projected to reach around ₹21,790 crore (USD 2,619 million) by 2030. This growth represents an impressive compound annual growth rate (CAGR) of 46.2% from 2024 to 2030. The market surge is typically due to rising obesity rates, increasing awareness of related health risks, and a growing demand for effective medical treatments like Mounjaro and Wegovy. As medicine availability and accessibility improve and physician adoption increases, India is emerging as a major market for next-generation weight-loss therapies.

Strategic Battle: Wegovy vs Mounjaro in India

First-Mover Advantage

Eli Lilly gained an early lead by launching Mounjaro in March 2024, capturing physician attention and onboarding patients first. In response, Novo Nordisk quickly launches Wegovy, aiming to exploit the same momentum and not lose market share.

Device Convenience: Pens over Vials

Both companies now use pen devices instead of vials, which are more user-friendly, improve patient adherence, and support easy self-injection.

Efficacy Advantage

In clinical trials, Mounjaro (tirzepatide) showed up to ~23% weight loss, and Wegovy (semaglutide) showed 15–20% weight loss. This superior efficacy profile could tilt the market in favour of Mounjaro, especially for patients seeking maximum weight loss results.

Patent Expiry and Generic Entry

Wegovy (semaglutide) will lose patent protection in India by March 2026, opening the door for low-cost generics, potentially 60–90% cheaper

In contrast, Mounjaro (tirzepatide) remains patent-protected until around 2036, giving Lilly a longer pricing runway and protection from generic competition.

Final Take

India’s anti-obesity drug market is undergoing a crucial shift. Historically, obesity was not recognized as a medical condition in the country, and as a result, anti-obesity medications were often seen as non-essential or lifestyle products. Many patients were unwilling to invest in prescription-based weight loss treatments. However, for the first time, India is witnessing a competitive fight between two global pharmaceutical giants, Eli Lilly and Novo Nordisk, as they strive for dominance in this emerging therapeutic space. This competition signals a shift in perception, where obesity is increasingly considered a serious and treatable health condition, opening the door to more scientifically and medically proven weight management.

Elli Lilly’s Mounjaro currently holds a strategic edge with its superior efficacy (~23% weight loss) and longer patent protection until ~2036, making it well-suited for medically supervised, premium patients who prioritize results and can afford higher prices. Its early market entry and KwikPen device covering the full dose range further strengthen its position.

On the other hand, Novo Nordisk’s Wegovy brings a powerful mix of strong global brand equity and proven cardiovascular benefits, making it highly appealing, especially as obesity increasingly intersects with metabolic and heart health. Novo Nordisk’s accelerated India launch shows a clear intent to capture momentum, particularly in urban centers, before generics flood the market post-2026, when semaglutide’s patent expires.

References

Novo Nordisk launches weight-loss drug Wegovy in India to compete with Lilly’s Mounjaro, Reuters, June 24 2025, https://www.reuters.com/business/healthcare-pharmaceuticals/novo-nordisk-launches-blockuster-weight-loss-drug-wegovy-india-2025-06-24/

Novo Nordisk’s Wegovy debuts in India: How it works, who it’s for, and how much it costs, Times of India, June 27 2025, https://timesofindia.indiatimes.com/life-style/health-fitness/health-news/novo-nordisks-wegovy-debuts-in-india-how-it-works-who-its-for-and-how-much-it-costs/articleshow/122054770.cms

Novo Nordisk launches blockbuster weight-loss drug Wegovy in India; Check price, dosage and other details, The Economics Times, June 24 2025, https://economictimes.indiatimes.com/news/new-updates/novo-nordisk-launches-blockbuster-weight-loss-drug-wegovy-in-india-check-price-dosage-and-other-details/articleshow/122042130.cms?from=mdr

Highlights of prescribing information, Wegovy, https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215256s007lbl.pdf

Highlights of prescribing information, Mounjaro, https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215866s000lbl.pdf

Lilly to launch Mounjaro pen in India to compete with Novo’s weight-loss drug Wegovy, Reuters, June 26 2026, https://www.reuters.com/business/healthcare-pharmaceuticals/lilly-launch-mounjaro-pen-india-compete-with-novos-wegovy-2025-06-26/

Eli Lilly’s Mounjaro hits Rs 24 crore sales in 3 months as adoption grows for diabetes and obesity in India, Business Today, 08 June 2025, https://www.businesstoday.in/industry/pharma/story/eli-lillys-mounjaro-hits-rs-24-crore-sales-in-three-months-as-adoption-grows-for-diabetes-and-obesity-in-india-479483-2025-06-08

Eli Lilly launches weight-loss drug Mounjaro in India, beats Novo Nordisk to major market, March 21 2025, https://www.reuters.com/business/healthcare-pharmaceuticals/eli-lilly-launches-weight-loss-drug-mounjaro-india-after-drug-regulator-approval-2025-03-20/

India Anti-Obesity Medication Market Size & Outlook, HORIZON Grand View Research, https://www.grandviewresearch.com/horizon/outlook/anti-obesity-medication-market/india

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Rising Threat: the Latest Aspergillus fumigatus Outbreak in the U.S. and Global Precautions

June 21, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

Published on: 21 June 2025

Category: Health News I Infectious Diseases & Outbreaks I

Fungal Infection

Written By:

Dewanshee Ingale, BPharm

Reviewed and Fact-Checked By:

Pharmacally Medical News Team

Aspergillus fumigatus, a silent but very deadly fungus, is rapidly spreading at a very alarming rate throughout the United States. Aspergillus fumigatus is a prevalent environmental fungus that is gradually being identified as a threat to public health. Initially, this organism was only known to affect the immune systems of immunocompromised patients. But now it has been clear that this fungus is expanding its reach; this fungus is airborne and spreads farther than it did in previous times. Its drug-resistant strain poses a threat to global infectious disease management. Experts in this field warn that not only the USA but also the world is not prepared for the threat that will be caused by the outbreak and spread of this fungus. As the infection rate climbs, especially in areas with warm, humid climates and also in urban, high-density populations, Aspergillus fumigatus poses serious complications to healthcare systems as well as endangers the vulnerable populations across the USA and globally in coming days.

Aspergillus Fumigatus

Aspergillus fumigatus is mold found in the environment at various places like soil, decaying leaves, and compost, and it is also found in indoor environments. The exposure is common to this fungus, and it presents little threat to healthy individuals; however, it can show serious complications in individuals with immunocompromised status or with chronic lung conditions.

The spores of the fungus Aspergillus fumigatus enter the body via the respiratory tract through the air we inhale. The spores reach the nose, throat, sinuses, and lungs. When these spores enter the body of healthy individuals, the immune system usually clears the spores. When these spores enter the bodies of people with compromised immunity or pre-existing lung diseases, the fungus invades the tissues, disrupts immune cell function, forms blood clots, and may also trigger severe allergic reactions.

Major Health Risks and Types of Aspergillosis

The infection with Aspergillus fumigatus can cause a range of conditions collectively known as aspergillosis in people with weakened immune systems or lung conditions. These include:

Allergic Bronchopulmonary Aspergillosis (ABPA): The allergic reaction produced as a reaction to the inhaled spores is most common in individuals with asthma, cystic fibrosis, or bronchiectasis. The symptoms observed are wheezing, coughing, shortness of breath, fever, and, many times, brown or bloody mucus. ABPA may worsen due to the presence of an underlying lung disease and can cause serious asthma attacks.

Chronic pulmonary aspergillosis (CPA) is a progressively growing infection mainly observed in patients with chronic lung diseases like tuberculosis or emphysema. The symptoms reported are persistent cough, severe weight loss, fatigue, breathlessness, and sometimes the occurrence of blood in the cough (hemoptysis), and they may often lead to notable lung damage and scarring if they are not treated.

Aspergilloma (“Fungus Ball”): The fungus forms a tangled mass inside the lung cavity. It often stays asymptomatic but can cause coughing up blood, which may turn severe.

Invasive Aspergillosis the most serious form, seen in the people with seriously weakened immune systems for example cancer, organ transplant, prolonged steroid use etc. the symptoms observed are fever, chest pain, cough (sometimes with blood), shortness of breath, and if the infection still persists may lead to neurological symptoms or often organ dysfunction. This particular form may be life-threatening and may require urgent medications.

Fungal Sinusitis and Other Forms: Spores often infect the sinuses, which cause a stuffy or runny nose, facial pain, headaches, and a decreased sense of smell. In some rare cases, the fungus may also spread through the eyes, skin, brain, or digestive tract, which may lead to the development of additional symptoms like red eyes, blurred vision, skin ulcers, confusion, and abdominal pain.

Who is at risk?

People suffering from cancer, HIV/AIDS, or organ transplants or under the therapy of immunosuppressive drugs have weak immunity, or individuals with chronic lung diseases such as asthma, cystic fibrosis, COPD, or tuberculosis and patients who have recently recovered from serious viral infections like influenza or COVID-19 are at major risk and require mechanical ventilation. Such people are exposed to environments that have a concentration of mold spores, for example, compost heaps, damp buildings, or construction sites.

Details of the U.S. Outbreak

As of mid-2025, multiple states in the U.S., including California, Texas, and New York, have reported a spike in invasive aspergillosis cases caused by azole-resistant Aspergillus fumigatus (ARAf). The Centers for Disease Control and Prevention (CDC) has confirmed that several isolates have genetic mutations associated with environmental fungicide exposure.

Why Is the Outbreak Getting Worse?

Several factors accelerate the spread and the impact of the Aspergillus fumigatus:

Climate Change: Overall, the increasing global warming has caused increased global temperatures and increased humidity that create an environment that is more favorable for the fungus to grow and spread, allowing the fungus to bloom in new areas and increase the risk to life.

Drug Resistance: The overuse of fungicide agents in the agricultural processes leads to the production of strains that are resistant to the first-line antifungal drugs, especially azole antifungals such as voriconazole or itraconazole, which makes the infection more difficult to treat and increases the complications associated with the fungus.

Population Vulnerability: The number of people with compromised immunity is continuously increasing due to the advancements in cancer treatments, organ transplantation, and other chronic disease management.

Environmental Disturbance: The increase in environmental disturbances like construction, renovations, and the occurrence of natural disasters leads to the release of large amounts of spores in the air, which further leads to a significant increase in the risk of outbreaks, especially in the healthcare systems.

USA’s Preparedness and Response

The United States faces significant challenges in tracking and fighting Aspergillus fumigatus-affected patients,, which makes it more difficult to control the outbursts and protect the susceptible populations

The CDC Mycotic Diseases Branch is actively monitoring for azole-resistant Aspergillus fumigatus (ARAf) and promoting molecular testing to detect resistance mutations like TR34/L98H and TR46/Y121F/T289A.

The CDC has promoted antifungal stewardship in healthcare settings to prevent unnecessary use of azole antifungals, parallel to antibiotic stewardship efforts.

Advisory alerts were issued to hospitals and clinicians in affected states (e.g., California, Texas, and New York) recommending

Heightened awareness in transplant units and ICUs
Use of molecular diagnostics for suspected resistant strains
Review of fungicide usage data in surrounding agricultural areas

Recognizing that drug-resistant Aspergillus has environmental origins, U.S. agencies are advancing a One Health approach through the Environmental Protection Agency, which works on regulating fungicide use in agriculture to reduce environmental resistance pressures.

Global concerns

Fungal spores can travel long distances in the air. What starts as a localized outbreak can quickly become a global bio-risk; hence, the threat associated with Aspergillus fumigatus is not only confined to the USA but is also susceptible to global spread by expanding its range, exposing millions more people in Europe, Asia, and the Americas.

The rising number of people undergoing chemotherapy, organ transplants, or long-term corticosteroid therapy makes the global population increasingly vulnerable for infection.

How to Stay Safe from Aspergillus Fumigatus

These are the steps that one must ensure to reduce the risk of this fungus, especially if you are immunocompromised:

Avoid visiting dusty and moldy areas: Staying away from construction sites, renovation zones, compost piles, and places with visible mold would help prevent the spread of infection.

Use Protective Gear: Use protective gear like an N95 mask and gloves whenever you visit such places that pose risks.

Keep Indoor Air Clean: Using HEPA filters at home and inside the hospital rooms to reduce the entry of airborne spores.

Practice Good Hygiene: Wash your hands more often and at regular intervals, especially when you are outdoors, and also make sure to clean the cuts and wounds, if any.

Monitor Your Health: If you have weak immunity and you feel symptoms like a persistent cough, fever, or shortness of breath, immediately see a doctor and seek medical therapy.

Consult Your Doctor: People who are at high risk should communicate with their physicians about the extra preventive measures to be taken to stay safe from the fungal infection, including the antifungal therapies.

By staying informed and following these precautions, you can help protect yourself and your loved ones from this emerging fungal threat.

Conclusion

Aspergillus fumigatus is a blooming public health threat in the US and all around the globe. The rapid spread of this fungus, the increasing resistance to drugs, and the susceptibility of the increasing populations demand immediate consideration from the health organizations, various researchers, and policymakers. The use of protective measures is very crucial to minimize its impact and safeguard public health in the upcoming future.

Reference

Global pandemic warning: Aspergillus deadly fungus mirrors HBO’s ‘The Last of Us’, Times of India, https://timesofindia.indiatimes.com/life-style/health-fitness/health-news/global-pandemic-warning-aspergillus-deadly-fungus-mirrors-hbos-the-last-of-us/articleshow/121438926.cms

Deadly fungus in US threatens lives as infection rates rise in these seven states, Hindustan Times, https://www.hindustantimes.com/world-news/deadly-aspergillus-fumigatus-in-us-threatens-lives-as-infection-rates-rise-in-these-seven-states-101750078523990.html

Aspergillus: All about the fungus that could spark a pandemic like in HBO’s ‘The Last of Us’, Economic Times, https://economictimes.com/news/international/global-trends/aspergillus-all-about-the-fungus-that-could-spark-a-pandemic-like-in-hbos-the-last-of-us/articleshow/121380695.cms

Hiel SJP, Hendriks ACA, Eijkenboom JJA, et al, Aspergillus Outbreak in an Intensive Care Unit: Source Analysis with Whole Genome Sequencing and Short Tandem Repeats. J Fungi (Basel). 2024 Jan 6; 10(1):51. doi: 10.3390/jof10010051. PMID: 38248960; PMCID: PMC10817286.

Data and Statistics on Aspergillosis, US centre for disease control and prevention, https://www.cdc.gov/aspergillosis/statistics/index.html

Risk Factors and Environmental Preventive Actions for Aspergillosis in Patients with Haematological Malignancies https://pmc.ncbi.nlm.nih.gov/articles/PMC10888107/

Arastehfar A, Carvalho A, Houbraken J,et al, Aspergillus fumigatus and aspergillosis: From basics to clinics. Stud Mycol. 2021 May 10;100:100115. Doi: 10.1016/j.simyco.2021.100115. PMID: 34035866; PMCID: PMC8131930.

Rising temperatures could spread deadly fungus, experts warn, and other health stories Global Heat Health Information Network, https://ghhin.org/news/rising-temperatures-could-spread-deadly-fungus-experts-warn-and-other-health-stories/

Aspergillosis Symptoms and Diagnosis, American Lung Association http://lung.org/lung-health-diseases/lung-disease-lookup/aspergillosis/symptoms-diagnosis

Céline M. O’Gorman, Airborne Aspergillus fumigatus conidia: a risk factor for aspergillosis, Fungal Biology Reviews, Volume 25, Issue 3, 2011, Pages 151-157, https://doi.org/10.1016/j.fbr.2011.07.002.

Reducing Risk for Aspergillosis, US centre for disease control and prevention, https://www.cdc.gov/aspergillosis/prevention/index.html

Preventing Aspergillus Fumigatus: Your Guide to a Fungus-Free Environment, Ion Bar, https://auraionbar.com/preventing-aspergillus-fumigatus/

Fungal Disease Laboratories, US centre for disease control and prevention, https://www.cdc.gov/fungal/hcp/laboratories/index.html

Core Elements of Antibiotic Stewardship, US centre for disease control and prevention, https://www.cdc.gov/antibiotic-use/hcp/core-elements/index.html

EPA Finalizes Framework for Interagency Collaboration on Resistance Risks Associated with Antibacterial and Antifungal Pesticides, US Enviornment Protection Agency, https://www.epa.gov/pesticides/epa-finalizes-framework-interagency-collaboration-resistance-risks-associated

Ms. Dewanshee Ingale is a pharmacy graduate with a deep passion for medical writing and evidence-based healthcare communication. She brings a sharp scientific perspective to her work, enriched by certifications in WHO’s Research Ethics Online Learning and Artificial Intelligence in Research and Science. At Pharmacally, she combines her academic foundation and research-driven insights to translate complex medical topics into accessible and reliable health information.

“UK’s Martha’s Rule: A Mother’s Fight That Sparked a National Reform and a Global Wake-Up Call on Patient Safety”

June 14, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

Written by: Soniya Hajare, MPharm

Reviewed and Fact-Checked by: Vikas Londhe, MPharm

The UK government has recently introduced ‘Martha’s Rule,’ a new policy that allows patients and their families to directly request a rapid second medical opinion when someone’s condition is worsening or when there is disagreement about their care. This rule was created following the tragic death of Martha Mills, a a 13-year-old girl who tragically died of sepsis at King’s College Hospital in August 2021. Despite multiple warning signs—fever, rash, fluctuating vitals, and parents’ repeated concerns—the pediatric liver team failed to transfer her care to intensive support.

Martha’s Rule, implemented in NHS hospitals across England in April 2024, is a patient safety measure aimed at ensuring timely medical intervention when a patient’s condition worsens. It gives patients, their families, caregivers, or healthcare staff the right to request an urgent review by a critical care outreach team if they are worried about a patient’s health or feel their concerns are not being adequately addressed.

Martha’s Rule also serves as a cultural shift in healthcare, aiming to reduce hierarchical barriers, improve open communication, and empower patients and families to take an active role in medical decisions. Early reports from its initial rollout, shared in December 2024, suggest that the policy is already leading to better patient outcomes by preventing avoidable harm and deaths.

Background

In the summer of 2021, 13-year-old Martha Mills suffered a pancreatic injury after a bicycle accident during a family holiday in Wales, due to seriousness of injury she transferred to King’s College Hospital in London, a specialist centre for paediatric pancreatic trauma under the supervision of paediatric hepatology team. Although her health condition showed signs of worsening (high BPEWS score), she remained on a general paediatric ward and tragically succumbed to septic shock on 31 August 2021.

Investigations and Inquests

Following Martha’s death, a detailed internal investigation by King’s College Hospital found five missed opportunities to involve the intensive care unit, despite ICU beds being available at the time. In March 2022, the coroner concluded that Martha would likely have survived if she had been transferred to the intensive care ward earlier when worsening signs started appearing.

The inquest also highlighted systemic issues, such as the continued use of paper-based observation charts, poor communication between departments, and an absence of a well-defined ICU escalation protocol.

In May 2025, the General Medical Council (GMC) conducted a tribunal into the conduct of the senior consultant overseeing Martha’s care. He was found guilty of professional misconduct, including giving inaccurate and outdated information about Martha’s condition during a critical stage of her illness. Despite these findings, the tribunal decided not to impose any formal sanction, citing his long-standing career, previous good record, and the complexity of the situation.

The Five Missed Opportunities

Failure to escalate to high-dependency or ICU beds early enough
Ignoring early sepsis indicators
Communication breakdown and team culture issues
Misdiagnosis of rash on 29 August
Absence of senior consultant reviews during critical deterioration

Implementation

Martha’s mother, Merope Mills, a senior editor at The Guardian, used her platform not just to share her grief but to campaign for change. She didn’t seek blame; she wanted reform. Her stand was simple: families should have a formal right to escalate concerns if they believe something is wrong. This led to the creation and rollout of Martha’s Rule

After the heart-rending and preventable death of 13-year-old Martha Mills, a series of reforms and actions were taken in response to the findings of the investigation and public pressure from her family. The most significant and systemic change was the implementation of ‘Martha’s Rule’ across all NHS hospitals in England. Below is a detailed summary of the key implementations:

Introduction of “Martha’s Rule”

Martha’s Rule is a national policy designed to give patients and families direct access to a second clinical opinion or critical care review when they are concerned that a patient is deteriorating and not being properly heard.

Core Features

24/7 access for patients and families to request a rapid clinical review from a critical care team (separate from the patient’s current care team)

Hospitals must prominently display how and when to escalate concerns, including bedside posters and leaflets

Staff training to encourage listening to families, recognizing that they often detect subtle signs of deterioration early

Strengthening the early warning score systems and escalation pathways already in place, making them more transparent and family-accessible

Rollout of Martha’s Rule

The implementation of Martha’s Rule began as a phased rollout following strong public pressure and policy discussions initiated in late 2023. NHS England, working with patient safety experts and Martha Mills’s family, selected 143 NHS trusts to pilot the scheme in early 2024.

These pilot sites tested how effectively patients and families could escalate a rapid clinical review when they were concerned about a patient’s condition. Following positive evaluations, the NHS committed to nationwide implementation. By April 2025, Martha’s Rule was made mandatory across all acute hospitals in England, with dedicated resources, signage, and staff training provided to ensure uniform compliance.

Impact & Data from Pilot Evaluation

During the initial rollout across 143 NHS hospitals in England (September–October 2024), 573 calls were made by patients, families, carers, and staff concerning suspected deterioration. Of these:

286 calls (50%) led to an urgent clinical review by critical care outreach teams

Around 57 reviews (20%) resulted in a change in treatment; such as administration of antibiotics, oxygen, or other life-saving interventions. However the patient remained on their ward.

14 patients were urgently transferred to intensive care units (ICU) after the escalation, potentially prevention serious complications or death.

By March 2025

A total of over 2,000 escalations had been made under the Rule

More than 300 escalations were followed by documented improvements in care

Over 100 patients were transferred to ICU or its equivalent directly due to their concerns being flagged through Martha’s Rule

Additionally, about 75% of calls originated from family members, highlighting the vital role that caregivers play in recognising deterioration that might otherwise be missed

These figures clearly exemplify both the scale and effectiveness of Martha’s Rule: it is being used with meaningful results, not abused, and is saving lives. NHS England’s national medical director, Prof Sir Stephen Powis, described it as “one of the most significant changes in patient safety in recent years,” and England’s Patient Safety Commissioner, Dr Henrietta Hughes, confirmed it “improving safety and reducing harm”

Conclusion and Pharmacally’s Take

At Pharmacally, we believe that patient safety begins with patient empowerment and Martha’s Rule marks a powerful shift in that direction. Martha’s Rule is arises from the heartbreaking loss of 13-year-old Martha Mills, This reform is more than just a policy, It is a major step toward making sure that patients and families are listened, their concerns are taken seriously, and their role in care decisions is truly respected.

For a long time, when patients or families raised concerns, those warnings often got delayed or overlooked due to complicated hospital systems and strict hierarchies. Martha’s Rule changes that it gives patients and families the power to speak up and be taken seriously, not just with empathy, but with real action.

At Pharmacally, where our core mission is to translate cutting-edge medical insight into safer outcomes, we see Martha’s Rule as a milestone for all who believe that safety is a shared responsibility. It formalizes a patient’s right to speak up and be taken seriously, especially when every minute matters.

We urge our readers, patients, caregivers, clinicians, and health systems to view Martha’s Rule not just as a protocol, but as a cultural reset. A chance to build a health system that listens, learns, and acts faster. Patient Safety doesn’t start in the ICU it starts at the bedside, with a voice saying, “I think something is wrong.”

Martha’s Rule isn’t just Martha’s legacy. It’s a blueprint for a safer, smarter, more responsive healthcare system. And at Pharmacally, that’s exactly the kind of future we stand for

Wrong Blood, Lost Life: How a Blood Transfusion Error at SMS Hospital, Rajasthan Sparked a State-wide Medical Reckoning

June 11, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

Written and Reviewed by Vikas Londhe M.Pharm (pharmacology)

Assisted By: Shakuntala Kawhale (M.Pharm pharmacology)

On May 20, 2025, a 23-year-old pregnant woman died in Jaipur’s prestigious Sawai Man Singh (SMS) Hospital, not from the illness she was fighting but from a blood transfusion mistake that was preventable and never should have happened.

A female patient from Tonk district, who was seven months pregnant, was admitted to SMS Hospital on May 12 with severe anaemia and miliary tuberculosis. Her pregnancy was already classified as high-risk. However, she was mistakenly given A+ blood, despite her actual blood type being B+.

The error was not caught until it was too late. By the time symptoms like fever, chills, hematuria, and rapid heart rate appeared, around 350 ml of incompatible blood had already been transfused. Patient succumbed to the reaction later that night.

Timeline of Errors

The tragedy was not the result of a single misstep but a chain of preventable lapses:

No dual verification between the blood samples sent from the ward and the patient’s records.
Incorrect blood group identification during ward-level preparation.
Failure to match identifiers (yellow tag, ABHA ID, blood bag barcode) at the point of transfusion.
Inadequate monitoring in the critical first 15–30 minutes post-transfusion, when early signs of reaction generally appear.
No immediate escalation occurred when the patient developed symptoms, delaying corrective measures.
Delayed and incomplete communication with her family, who were not immediately informed of the transfusion error.

This was the third transfusion-related death involving SMS and its affiliated hospitals in just over a year, highlighting a pattern of systemic failure.

What the Internal Inquiry Found and Why It Was Rejected

SMS Hospital conducted an internal investigation and acknowledged the transfusion error. However, the report controversially claimed that the mismatched blood was not the direct cause of death, pointing towards the patient’s tuberculosis and low haemoglobin levels.

This standpoint received immediate criticism from many levels. The Rajasthan government rejected the internal findings, labelled them unsatisfactory and ordered an independent, high-level investigation. Health Minister Gajendra Singh Khimsar announced the formation of a committee comprising senior doctors, administrative officials, and public health experts, tasked with submitting a report within three days.

The case also reached the Rajasthan State Human Rights Commission, which issued a notice demanding detailed reports by June 12.

Rajasthan Government’s Official Response: a Shift toward Safety

Under the public pressure and clear protocol violations, the Rajasthan government took immediate and serious action:

Key Policy Decisions Announced

Digital Blood Group mapping

All patients in state hospitals will now have their blood type linked to their Ayushman Bharat Health Account ID (ABHA ID), reducing room for clerical or manual entry errors.

Compulsory Admission—Time Blood Typing

Every patient must now undergo blood grouping at admission, with results entered into a centralized digital lab system.

Dual Verification at Blood Banks

Before releasing any blood unit, technicians must re-verify the patient’s blood type and documentation, independent of the ward-level grouping.

Detailed Chain of Custody Documentation

Blood request forms must now contain:

Name and mobile number of the requesting resident

Attending the duty doctor’s name

Name of the staff that collected the sample and the blood bag

Standardized Monitoring Protocol during Transfusion

Clinical staff must now:

Match the patient ID, yellow wristband, and blood label

Monitor vitals at 0, 5, 15, and 30 minutes during the transfusion

Record all observations in real time

Statewide SOP Enforcement across RMES Hospitals

SMS-level safety protocols are now mandatory across all government medical colleges and district hospitals under the Rajasthan Medical Education Society (RMES).

Patient Safety Lessons: When Systems Fail, People Pay the Price

The loss of life is a staggering reminder of how even routine clinical procedures can turn deadly without robust precaution. Blood transfusions are considered high-alert medical procedures, requiring multiple fail-safe mechanisms to prevent mismatches. When these mechanisms are skipped whether due to staff shortages, lack of training, or system fatigue the consequences are often fatal.

This case illustrates the urgent need for error-proof processes, accountability tracking, and a culture where speaking up about unsafe practices is encouraged.

Pharmacally’s Take: Reform Begins with Acknowledging the Risk

While the Rajasthan government’s response has been proactive and progressive, the real test lies in enforcement. Policies on paper must translate to practice on the ground. Frontline workers residents, nurses, lab techs must be trained, audited, and supported in following these updated protocols.

At Pharmacally.com, we advocate for healthcare systems that:

Prioritize patient safety
Embed digital tools to reduce human error.
Enforce chain-of-responsibility in critical procedures, and
Promote transparent investigations when outcomes go wrong.

Final Word: From Tragedy to Transformation

This patient should have gone home with her newborn in a few months. Instead, her death has exposed deep vulnerabilities in our public health system. If Rajasthan succeeds in turning this tragedy into a safety revolution, it could become a blueprint for the rest of India.

References:

Gandhi a, görlinger k, nair sc, kapoor pm, trikha a, mehta y, handoo a, karlekar a, kotwal j, john j, apte s. Patient blood management in india-review of current practices and feasibility of applying appropriate standard of care guidelines. A position paper by an interdisciplinary expert group. Journal of anaesthesiology clinical pharmacology. 2021 jan 1;37(1):3-13.

Bisht a, singh s, marwaha n. Hemovigilance program-india. Asian journal of transfusion science. 2013 jan 1;7(1):73-4.

Mammen jj, asirvatham es, sarman cj, ranjan v, charles b. A review of legal, regulatory, and policy aspects of blood transfusion services in india: issues, challenges, and opportunities. Asian journal of transfusion science. 2021 jul 1;15(2):204-11

Rajasthan to streamline blood transfusion system in all hospitals, News Arena India, https://newsarenaindia.com/nation/rajasthan-to-tighten-hospital-blood-transfusion-rules/45595

State Gov to tightens blood safety protocols after transfusion deaths, times of India, https://timesofindia.indiatimes.com/city/jaipur/state-govt-to-tighten-blood-safety-protocols-after-transfusion-death/articleshow/121421643.cms

Rajasthan hospital accused of fatal transfusion error after pregnant woman, 23, dies, The Economic Times, https://economictimes.indiatimes.com/news/india/rajasthan-hospital-accused-of-fatal-transfusion-error-after-pregnant-woman-23-dies/articleshow/121362945.cms?from=mdr

“Deadly Saline Killed 8: Neuromelioidosis Outbreak in Tamil Nadu Linked to Contaminated Dental Saline – What Went Wrong?”

June 9, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

Written by: Dr. Arshada Fathin, PharmD (Coimbatore, Tamil Nadu)

Reviewed By: Dr. Seema Satbhai (BAMS, MPH, PhD, Public Health)

Neuromelioidosis, a central nervous system manifestation of melioidosis caused by Burkholderia pseudomallei, has emerged as a serious health concern in Vaniyambadi, Tirupattur district, Tamil Nadu, India. An unusual cluster of cases was identified between July 2022 and April 2023. Importantly, 10 out of 21 patients had undergone some dental procedure, most at the same dental clinic. Out of these 10 cases, 8 patients succumbed to death. This has raised critical questions regarding infection control practices in dental care settings. This article provides an overview of the outbreak, disease pathology, diagnostic considerations, and the role of procedural lapses in precipitating iatrogenic transmission.

Background of the Case

The outbreak of neuromelioidosis in Tamil Nadu centres on a cluster of 21 cases reported between July 2022 and April 2023. These included 11 sporadic infections and 10 distinct sub-cluster epidemiologically linked to a dental clinic in Vaniyambadi, Tirupattur district. The affected patients who visited the clinic presented with severe symptoms shortly after dental treatment.

About Neuromelioidosis

Neuromelioidosis results from infection of the central nervous system by Burkholderia pseudomallei, manifesting primarily as brainstem syndrome. Typical clinical features include facial pain, cranial nerve dysfunction, abscess formation, and necrotizing encephalitis. The infection often spreads along neuronal pathways, such as the trigeminal nerve, leading to rapid neurological decline. Adults within the age range of 26 to 44 years are predominantly affected in endemic regions. Diagnosis relies on isolation of the organism from blood, cerebrospinal fluid (CSF), or affected tissues through culture or polymerase chain reaction (PCR) assays. Molecular tools, including WGS and detection of virulence-associated genes such as bimA, aid in strain typing and epidemiological investigations. Management requires prompt initiation of intravenous antibiotics, commonly ceftazidime or meropenem, followed by prolonged oral eradication therapy, i.e., Co-Trimoxazole, for several months. Delay in recognition and treatment often results in poor clinical outcomes, sometimes death.

The Culprit: Burkholderia pseudomallei

This bacterium typically inhabits soil and water, but in this outbreak, it entered patients directly along nerve pathways by contact with mucous membranes in the mouth during dental treatments. This neurological route led to rapid brain infections rather than the more typical bloodstream spread.

Case Description and Microbiological Findings

The outbreak involved 21 patients with neuromelioidosis in Tamil Nadu, with a median age of 33 years, affecting both males and females. Clinical presentations include facial cellulitis, lymphadenopathy, brainstem involvement, and rapid neurological decline. Among the 10 patients with documented dental procedure exposure, 8 succumbed to the illness within a median period of 17 days post-exposure, indicating a higher fatality rate compared to sporadic cases. The high fatality of the outbreak is indicative of an iatrogenic source of infection. A high-level, authoritative investigation was warranted on an immediate basis.

Investigation by Researchers

It all started when a married couple presented at CMC Vellore with acute-onset fever followed by symptoms of brainstem syndrome. Radiological imaging revealed necrotizing brainstem encephalitis, a finding consistent with neuromelioidosis. The relative reported that both patients had undergone a dental procedure at the dental clinic in Vaniyambadi shortly before illness.

A highly authoritative investigation was carried out which led by researchers from CMC Vellore, ICMR-National Institute of Epidemiology, Chennai, and the Tamil Nadu Directorate of Public Health and Preventive Medicine. The primary objective of the investigation was to understand how the infection spread and confirm whether the source was iatrogenic. The outbreak investigation utilized clinical evaluation, microbiological cultures, environmental sampling and whole-genome sequencing (WGS) of bacterial strain to establish the transmission dynamics and identify the source of infection. The initial investigation pointed to the cases from Tirupattur district experiencing an outbreak after undergoing invasive dental procedures at a local dental clinic and suggested possible contamination during dental procedures.

Identified Deviations in Infection Control – Medication Error and Sterility Breach

Based on an interview with the dentist and investigation findings, the dental clinic involved in the outbreak provided a wide range of services, including fixation of partial dentures, full-mouth prophylaxis, root canal treatments, and tooth extraction. The clinic was staffed by a dentist, nursing personnel, and a receptionist; however, none of the staff had formal training in hospital infection control practices.

Normal saline was supplied in sterile 500 mL plastic bottles and used both for wound irrigation during surgical procedures and for dilution of local anesthetic for infiltration. It was observed that the sterile saline bottles were opened using a non-sterile periosteal elevator. These bottles were then loosely resealed and reused over several days until empty. There was no proper sterilization of equipment between patients.

Such practices deviated significantly from established infection control standards. The repeated use of opened saline bottles without proper aseptic technique, combined with the use of non-sterile instruments to open them, increased the risk of contamination. Indeed, an increase in failed dental procedures and complications was reported concurrent with the emergence of neuromelioidosis cases in the district.

Microbiological analysis supported these findings, as Burkholderia pseudomallei was isolated from one of the in-use saline bottles collected during environmental sampling at the clinic. These breaches in sterile technique and medication handling directly contributed to the iatrogenic transmission of the pathogen to patients during dental procedures.

Recommended Preventive Measures

Based on the outbreak findings, several corrective measures are essential to prevent similar incidents in the future:

Strict use of sterile supplies: Single-use sterile saline bottles should be opened aseptically and discarded after one use. The reuse of opened bottles must be avoided.

Adherence to aseptic technique: All dental procedures must be performed under sterile conditions, employing sterilized instruments and protective barriers such as sterile gloves.

Comprehensive staff training: Dental clinic personnel should receive ongoing training in infection prevention and control protocols, emphasizing proper handling of sterile supplies.

Environmental monitoring: Routine microbial testing of dental unit water lines and fluids should be conducted, particularly in regions endemic for B. pseudomallei.

Immediate investigation of infection clusters: Any unusual increase in post-procedural infections must prompt epidemiological investigation and suspension of implicated materials or practices.

Implementing these measures will reduce the risk of iatrogenic infection and ensure patient safety during dental procedures.

Conclusion: A Wake-Up Call for Infection Control in Outpatient Care

The tragic Neuromelioidosis outbreak in Tamil Nadu is more than just a public health incident. It is a warning about how single lapse in clinical hygiene, such as reusing a contaminated saline bottle, can escalate into a fatal outbreak of a rare and deadly infection. While melioidosis is typically associated with environmental exposure in endemic regions, this event underscores an alarming reality: modern medical settings are not immune to such threats when basic infection control protocols are neglected.

The Vaniyambadi dental clinic case highlights critical vulnerabilities in outpatient care, particularly in resource-limited settings where practices like reusing consumables or improper instrument handling may still occur. In this instance, the use of an unsterile periosteal elevator to repeatedly open a saline bottle facilitated the direct introduction of Burkholderia pseudomallei, a soil-dwelling microorganism, into the oral mucosa, bypassing traditional routes of transmission. The result was an outbreak of neuromelioidosis, a rare neurological manifestation with devastating consequences, including an 80% fatality rate among exposed patients.

The rapid identification of the outbreak source, coordinated by CMC Vellore, ICMR-NIE Chennai, and state health authorities, demonstrates the importance of multisectoral collaboration in outbreak investigation.

For healthcare systems across India and globally, this event serves as a wake-up call. It emphasizes the urgent need to:

Reinforce standard infection prevention protocols in dental and outpatient clinics

Mandate the single-use of consumables like saline bottles

Educate practitioners on rare but deadly infections like melioidosis

Improve clinical waste management and sterilization practices

Establish local microbiology networks for early pathogen detection

Ultimately, this outbreak is an earnest reminder that patient safety must never be compromised for convenience. The lives lost in Vaniyambadi are a tragic testament to what can go wrong, but also a call to action, urging the healthcare community to uphold the highest standards of hygiene, vigilance, and accountability.

This investigation and findings are published in the Lancet Regional Health – Southeast Asia

References

Angel Miraclin Thirugnanakumar, Prabu Rajkumar, Karthik Gunasekaran et al, Neuromelioidosis outbreak in Tamil Nadu, India: an investigation of transmission with genomic insights, The Lancet Regional Health – Southeast Asia 2025;37: 100602, https://doi.org/10.1016/j.lansea.2025.100602

Wiersinga WJ, Currie BJ, Peacock SJ. Melioidosis. N Engl J Med. 2012; 367(11):1035-44. Doi:10.1056/NEJMra1204699.

Kohn WG, Collins AS, Cleveland JL, Harte JA, Eklund KJ, Malvitz DM. Guidelines for infection control in dental health-care settings—2003. MMWR Recomm Rep. 2003;52(RR-17):1-61.

Meumann EM, Currie BJ. Approach to melioidosis. CMI Communications. 2024 Jun 6;100008.

White NJ. Melioidosis, Lancet, 2003 May 17;361(9370):1715-22

Chatterjee A, Saravu K, Mukhopadhyay C, Chandran V. Neurological melioidosis presenting as rhombencephalitis, optic neuritis, and scalp abscess with meningitis: a case series from Southern India. Neurol India. 2021 Mar-Apr;69(2):480-2. doi:10.4103/0028-3886.314590.

Wiersinga WJ, van der Poll T, White NJ, Day NP, Peacock SJ. Melioidosis: insights into the pathogenicity of Burkholderia pseudomallei. Nat Rev Microbiol. 2006 Apr;4(4):272-82. doi:10.1038/nrmicro1385.

Enhancing Drug Safety: The Vital Role of Real-World Data (RWD) and Real-World Evidence (RWE) in Modern Pharmacovigilance

June 6, 2025

by official.ajinkya11@gmail.com with No Comment Health Tidings

Written By: Shital Doifode, M.Pharm Pharmacology

Reviewed and Fact-Checked By: Ashish Jaydeokar (Manager, Pharmacovigilance Operations, Germany)

Pharmacovigilance is the field of detection, assessment, understanding, and prevention of adverse effects or any other drug-related problems. The field has undergone many evolutions in the 21st century, with the integration of artificial intelligence in recent times. Conventionally, it depends on randomized controlled trials (RCTs) and spontaneous adverse event reporting systems. However, these methods now fall short in capturing the complete safety profile of a drug, particularly in real-world use across diverse patient populations and disease profiles. This limitation has facilitated the integration of real-world data (RWD) and real-world evidence (RWE), active tools that offer a more inclusive, proactive approach to safety surveillance. By leveraging data from electronic health records, insurance claims, patient registries, digital health tools, and other routine clinical sources, pharmacovigilance systems can now monitor drug safety and effectiveness in real time throughout the entire life cycle of a pharmaceutical product. This shift will not only enhance signal detection and risk assessment but also support more informed regulatory and clinical decision-making in everyday healthcare practice.

What Are RWD and RWE?

Real-World Data (RWD): It refers to data related to patient health status collected from sources such as electronic health records (EHRs), insurance claims, patient registries, and even digital health tools like mobile health apps and also from social media.

Real-World Evidence (RWE) covers the clinical insights and evidence about the usage and potential safety and risk of medical products that are derived from the analysis of Real-World Data (RWD).

This change is crucial, as clinical trials during the establishment of efficacy and safety often involve highly selected populations and controlled environments, limiting their applicability to broader patient groups.

In contrast, RWD/RWE allows for the continuous and proactive assessment of medications across diverse populations, capturing long-term safety outcomes, rare adverse events, and drug performance in real-world scenarios.

Why RWD and RWE Matter in Pharmacovigilance

Clinical trials are conducted in controlled environments with carefully selected participants, generally excluding elderly patients, pregnant women, and those with multiple comorbidities. RWD and RWE help to fill these gaps by showing how drugs behave in the real world, including in these excluded populations. RWE helps to eliminate the potential bias caused in CTs. Biases are often described in 3 categories:

Selection bias, which derives from including or selectively following subsets of the population in the study in a way that distorts the relation between the exposure and the outcome.
Information bias, which derives from measurement errors.
Confounding bias: this derives from noncomparability of the intervention groups in the study (27 in ->11).

Early Detection of Adverse Drug Reactions (ADRs)

By continuously analyzing data from large and diverse patient populations, pharmacovigilance teams are able to detect rare, serious, or delayed adverse drug reactions much earlier; that would not be possible through traditional clinical trial methods alone. Traditional trials are typically limited in size, duration, and participant diversity, which can make it difficult to identify side effects that only occur infrequently or in specific subgroups. In contrast, real-world data sources such as electronic health records, insurance claims, patient registries, and post-marketing surveillance reports allow pharmacovigilance experts to monitor the safety of medications across millions of users in real time.

Improved Signal Detection and Risk Management

Real-World Evidence (RWE) enables more accurate signal detection by providing an all-inclusive view of how drugs perform across diverse, real-life patient populations. The broader dataset of RWE allows researchers and regulators to correlate adverse drug events (ADEs) not just with the drug itself, but with specific patient characteristics such as age, gender, genetic factors, or underlying co-morbidities like diabetes, hypertension, or renal impairment. RWE is generated according to a research plan and interpreted accordingly.

Furthermore, RWE supports the identification of risks associated with concurrent therapies. Many patients are on multiple medications simultaneously (polypharmacy), which can lead to drug-drug interactions that are difficult to study in traditional trials. RWE can detect patterns where certain drug combinations are consistently associated with adverse outcomes, enabling more precise risk stratification.

By uncovering these population-specific safety signals, RWE enhances pharmacovigilance efforts and allows healthcare providers and regulatory agencies to implement targeted risk management strategies.

RWE allows for more accurate signal detection by correlating adverse events with specific patient populations, comorbid conditions, or concurrent therapies. This enhances the ability to manage risks in a targeted manner.

Regulatory Decision Support

Health authorities like the FDA and EMA increasingly rely on RWE to support regulatory decisions, such as label updates, post-marketing requirements, and even new indications for existing drugs.

First-ever regulatory approval of label expansion of IBRANCE (palbociclib) for male breast cancer based on RWE, have brought in a new era in the applicability of RWE in healthcare.

Overseen by the Big Data Steering Group (BDSG), EMA and the European Medicines Regulatory Network (EMRN) are working to establish a sustainable framework to enable the use and establish the value of real-world evidence (RWE) across different regulatory use cases.

Approvals where RWE was considered:

A total of 30 FDA approvals were identified (EMA: 16).
The number of approvals is steadily increasing.
The approvals of new drugs only concern orphan drugs at both FDA and EMA; i.e. drugs against rare diseases.

Supporting Patient-Centred Outcomes

Real-World Data (RWD) captures the patient experience in a more holistic and meaningful way, as compare to traditional clinical trials. While randomized controlled trials (RCTs) focus primarily on efficacy under ideal conditions, RWD reflects how treatments perform in everyday clinical settings, where patients may have diverse backgrounds, health conditions, and behaviours.

One of the key advantages of RWD is its ability to provide insights into treatment adherence, how consistently patients follow prescribed therapies. Non-adherence is a major factor affecting treatment outcomes and safety, yet it’s often underreported in clinical trials. RWD can identify adherence patterns, uncover barriers (e.g., side effects, cost, complex regimens), and inform interventions to improve long-term medication use.

FDA recognizes the potential utility of using RWD in interventional studies; for example, to identify potential participants for a randomized controlled trial, to ascertain endpoints or outcomes (e.g., occurrence of stroke or other discrete events, hospitalization, survival) in a randomized controlled trial, or to serve as a comparator arm in an externally controlled trial, including historically controlled trials

Real-World Applications of RWD and RWE in Pharmacovigilance

Post-Marketing Surveillance: After a pharmaceutical product is approved and enters the market, continuous monitoring is essential to ensure its long-term safety and effectiveness. Companies now increasingly rely on electronic health records (EHRs) and insurance claims data to conduct this post-marketing surveillance. These real-world data sources enable the identification of safety trends, rare adverse events, and patterns of use that may not have emerged during clinical trials.

EHRs provide detailed clinical information, such as lab results, diagnoses, and physician notes, while claims data offer insights into medication usage, healthcare utilization, and treatment costs across large patient populations.

Risk Evaluation and Mitigation Strategies (REMS): Real-world evidence (RWE) plays a crucial role in both the design and assessment of REMS programs, which are mandated by regulatory agencies like the FDA to ensure that the benefits of certain high-risk medications outweigh their potential risks.

Adaptive Pharmacovigilance Systems: Advances in artificial intelligence (AI) and machine learning (ML), when applied to real-world data (RWD) such as electronic health records, claims databases, and patient-reported outcomes, are transforming traditional pharmacovigilance into a more dynamic, automated, and predictive system.

These technologies enable the development of adaptive pharmacovigilance systems, which can continuously analyze large and complex datasets to automatically detect safety signals such as unusual patterns of adverse events, drug interactions, or shifts in usage trends. Unlike traditional, passive reporting systems, these AI-driven tools can flag potential risks in near real-time, improving both the speed and sensitivity of signal detection.

Tools and Technologies Powering RWD and RWE

1. Electronic Health Records (EHR) Systems

EHRs are one of the primary sources of RWD. Tools like Allscripts collect detailed patient-level clinical information, including diagnoses, treatments, lab results, and adverse events. Integration of EHR data into pharmacovigilance platforms helps identify safety signals in near real-time.

2. Claims and Billing Databases

Administrative claims databases such as Optum offer large-scale, longitudinal patient information. These are particularly useful for tracking healthcare utilization, medication adherence, and long-term safety outcomes.

3. Patient Registries

Disease-specific and product-specific registries collect structured data over time from patients with defined characteristics. Tools like OpenClinica are commonly used to manage registry data. Registries help monitor rare adverse events and long-term safety in real-world populations.

4. Mobile Health (mHealth) and Wearables

Apps and devices like Fitbit, Apple Watch, and mobile symptom trackers generate real-time data on patient activity, heart rate, medication intake, and other health metrics. This type of continuous monitoring offers valuable insights into drug effects outside clinical settings.

5. Natural Language Processing (NLP) Tools

NLP tools can extract relevant information from unstructured data such as clinical notes, discharge summaries, and patient forums. Examples include Amazon Comprehend Medical NLP pipelines, which can help identify adverse drug reactions from text sources.

6. Artificial Intelligence and Machine Learning Platforms

AI-powered platforms like SAS, IBM Watson Health, and Google Cloud AI are used to detect patterns in large datasets, support predictive analytics, and improve the accuracy of signal detection. Machine learning algorithms can also classify and prioritize adverse events based on severity and novelty.

7. Data Integration and Analytics Platforms

Platforms like OMOP (Observational Medical Outcomes Partnership) and the Sentinel initiative by the FDA standardize and harmonize data from multiple sources. These tools enable scalable and reproducible analysis of RWD to support RWE generation.

8. Social Listening and Digital Epidemiology Tools

Mining social media and online patient communities through platforms like Brandwatch or MedWatcher Social can reveal emerging drug safety concerns from patients themselves, often before they are formally reported.

Conclusion

The integration of real-world data and real-world evidence into pharmacovigilance marks a more inclusive and responsive drug safety system. By bridging the gap between controlled clinical environments and the complexity of everyday healthcare, RWD and RWE enable a more nuanced, proactive, and patient-centric approach to monitoring drug safety. As regulatory frameworks and technological tools continue to evolve, their role in modern pharmacovigilance will only become more beneficial.

References

FDA (2018), Framework for FDA’s Real-World Evidence Program, U.S. Food and Drug Administration, https://www.fda.gov/media/120060/download

Makady, A., de Boer, A., Hillege, H., Klungel, O., & Goettsch, W. (2017). What is real-world data? A review of definitions based on literature and stakeholder interviews. Value in Health, 20(7), 858–865. https://doi.org/10.1016/j.jval.2017.03.008

Ghosh R, et al. (2022). Pharmacovigilance: A Review on Current Strategies and Future Directions. Therapeutic Advances in Drug Safety, 13, 20420986221093745

Vandenbroucke JP. (2008), Observational research, randomised trials, and two views of medical science, PLoS Medicine, 5(3), e67

Sherman RE, et al. (2016), Real-world evidence — what is it and what can it tell us? New England Journal of Medicine, 375(23), 2293–2297.

U.S. FDA, (2018), Framework for FDA’s Real-World Evidence Program, https://www.fda.gov/media/120060/download

European Medicines Agency (2021), EMA Regulatory Science to 2025 — Strategic Reflection, https://www.ema.europa.eu/en/documents/report/ema-regulatory-science-2025-strategic-reflection_en.pdf

Botsis T, et al. (2010). Secondary use of EHR: data quality issues and informatics opportunities. Summit on Translational Bioinformatics, 2010, 1–5.

Platt R, et al. (2018). The U.S. FDA’s Sentinel Initiative — A Comprehensive Approach to Medical Product Surveillance. Clinical Pharmacology & Therapeutics, 103(3), 219–222.

Hripcsak G, et al. (2015). Observational Health Data Sciences and Informatics (OHDSI): Opportunities for Observational Researchers. Studies in Health Technology and Informatics, 216, 574–578.

FDA, (2018). Framework for FDA’s Real-World Evidence Program https://www.fda.gov/media/120060/download

EMA. (2022). Data Analysis and Real World Interrogation Network (DARWIN EU). https://www.ema.europa.eu/en/darwin-eu

Franklin JM, et al. (2021). Real-world evidence for regulatory decision-making: Challenges and opportunities. Clinical Pharmacology & Therapeutics, 109(4), 816–829.

Kakkar AK, et al. (2021). Direct oral anticoagulants: real-world evidence and clinical utility. Therapeutic Advances in Cardiovascular Disease, 15, 1753944720981525.

Schneeweiss S. (2019). Real-world evidence of treatment effects: the useful and the misleading. Clinical Pharmacology & Therapeutics, 106(1), 43–44.

Corrigan-Curay J, et al. (2018). Real-world evidence and real-world data for evaluating drug safety and effectiveness, JAMA, 320(9), 867–868.

Duke-Margolis Center for Health Policy. (2017). A Framework for Regulatory Use of Real-World Evidence. https://healthpolicy.duke.edu/sites/default/files/2020-11/rwe_white_paper_2017.09.13.pdf

Wedam S, Fashoyin-Aje L, Bloomquist E et al (2020) FDA approval summary: palbociclib for male patients with metastatic breast cancer. Clin Cancer Res 26(6):1208–1212. https://doi.org/10.1158/1078-0432.CCR-19-2580

FDA: Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry, U.S. Department of Health and Human Services Food and Drug Administration, August 2023

Marc L. Berger, Harold Sox, Richard J. Willke, et al, (2017), Good practices for real world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR ISPE Special Task Force on real‐world evidence in health care decision making, Pharmacoepidemiol Drug Saf. 2017; 26:1033–1039, DOI:10.1002/pds.4297

EMA: Real-world evidence framework to support EU regulatory decision-making EMA/289699/2023), https://www.ema.europa.eu/en/documents/report/real-world-evidence-framework-support-eu-regulatory-decision-making-report-experience-gained-regulator-led-studies-september-2021-february-2023_en.pdf

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Enhancing Drug Safety: The Vital Role of Real-World Data (RWD) and Real-World Evidence (RWE) in Modern Pharmacovigilance

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