A consultation document has been issued by the European Medicines Agency (EMA) ahead of a revision to EU GMP Chapter 1. The briefing reads:
‘The GMP/GDP Inspectors Working Group and the PIC/S Committee jointly recommended to amend the text of Chapter 1 in order to reflect the changes introduced in ICH Q9(R1) on Quality Risk Management.’
The chapter concerns the Pharmaceutical Quality System. The draft can be found here: https://health.ec.europa.eu/document/download/db93406b-e121-4f33-a399-9ad6b238383a_en?filename=mp_eu-gmp-chap1_draft-guidelines_en.pdf&trk=article-ssr-frontend-pulse-lite_little-text-block
The U.S. Food and Drug Administration (FDA) has issued a new draft guidance titled ‘Oncology Therapeutic Radiopharmaceuticals: Dosage Optimization During Clinical Development’.
The guidance is designed to assist sponsors in identifying an optimised dosage(s) (administered activity and schedule) for radiopharmaceutical therapies for oncology indications during clinical development. Such information is then typically used prior to submitting a marketing application, presenting a new indication and usage.
The guidance is intended to be reviewed alongside another FDA guidance titled ‘Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases (August 2024)’.
The guidance does not address the selection of the initial radiopharmaceutical therapies administered activity in first-in-human trials nor does it address other aspects of the clinical development of radiopharmaceutical therapies, for example, use of dosimetry software, use of fixed administered activity dosing for a population versus dosing determined by personalised dosimetry and theranostic co-development.
The draft can be accessed here: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/oncology-therapeutic-radiopharmaceuticals-dosage-optimization-during-clinical-development?utm_source=substack&utm_medium=email
The FDA has published a draft guidance relating to oncology clinical trials, titled ‘Approaches to Assessment of Overall Survival in Oncology Clinical Trials’.
The objective of the guidance is to provide recommendations to sponsors on the assessment of overall survival in randomised oncology clinical trials conducted to support marketing approval of drugs and biological products, with an emphasis on the analysis of overall survival as a pre-specified safety endpoint.
While the guidance discusses situations in which it is appropriate to consider overall survival for the primary endpoint, this guidance primarily focuses on statistical and design considerations when overall survival is not the primary endpoint. Additionally, this guidance focuses on the assessment of overall survival in randomised trials.
The document can be accessed here: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/approaches-assessment-overall-survival-oncology-clinical-trials?utm_source=substack&utm_medium=email
The following sterilisation standards are being developed via the International Standards Organisation (ISO):
The ISO committee TC 76 Transfusion, infusion and injection, and blood processing equipment for medical and pharmaceutical use is currently developing:
The FDA is updating its guidance on the labelling of medicines. The guidance authorises FDA to require application holders for certain drug and biological products to make labelling changes based on new safety information that becomes available after approval of the drug.
The revised guidance (draft) can be found here: https://www.fda.gov/media/188793/download
FDA has issued a final guidance for industry entitled ‘Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications’.
This guidance provides information to applicants on how FDA intends to use alternative tools to assess drug manufacturing facilities identified in a marketing application (i.e., a New Drug Application (NDA), an Abbreviated New Drug Application (ANDA), a Biologics License Application (BLA) or a supplement to any of these types of applications).
The document is available here: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/alternative-tools-assessing-drug-manufacturing-facilities-identified-pending-applications
FDA has issued a final guidance for industry entitled ‘E6(R3) Good Clinical Practice’. This revision incorporates flexible, risk-based approaches and embraces innovations in trial design, conduct and technology.
This important milestone marks a significant evolution in the global clinical trial landscape, aiming to modernise GCP principles in alignment with current scientific and technological advances while maintaining a strong focus on quality by design, participant protection and the reliability of trial results.
Key updates in ICH E6 (R3) include:
This guideline also incorporates the perspectives of academic clinical trial experts to ensure the practical relevance of its provisions. ICH E6 (R3) is intended to encourage the use of technology and innovations and it is designed to remain relevant and consistent as technology and methods evolve.
The document is available here: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/e6r3-good-clinical-practice-gcp
In September 2025, new amendments to the International Health Regulations (IHR) entered into force under the World Health Organization.
One of the changes was the introduction of a new level of global alert - a ‘pandemic emergency’ - to trigger stronger international collaboration when a health risk escalates beyond a Public Health Emergency of International Concern (PHEIC) and poses the risk of becoming, or has already become, a pandemic, with widespread impact on the health system and disruption to societies.
The amendments also introduce the establishment of National IHR Authorities by governments to coordinate IHR implementation and include provisions to strengthen access to medical products and financing based on equity and solidarity.
See: https://www.who.int/news/item/19-09-2025-amended-international-health-regulations-enter-into-force
The World Health Organization (WHO) has released updated editions of its Essential Medicines List (EML) and Essential Medicines for Children (EMLc), adding new treatments for various types of cancer and diabetes with associated comorbidities such as obesity. Medicines for cystic fibrosis, psoriasis, haemophilia and blood-related disorders are among the other additions.
The EML and EMLc include medicines for priority health needs of populations. They are adopted in over 150 countries, serving as a basis for public sector procurement, supply of medicines and health insurance and reimbursement schemes. The revisions mark the 24th edition of WHO EML and 10th edition of EMLc.
Scientists at UC San Diego have identified a new drug, ION224, that could transform the treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH). This dangerous form of fatty liver disease, tied to obesity and type 2 diabetes, can lead to cirrhosis, liver failure and even liver cancer.
The study, published in The Lancet, found that ION224 targets a liver enzyme called DGAT2, which plays a key role in how the liver produces and stores fat. By blocking this enzyme, ION224 reduces fat and inflammation, halting the root causes of liver damage.
In a year-long clinical trial, patients showed major improvements without serious side effects, offering hope to millions affected worldwide.
ISO/FDIS 23691: ‘Microbiology of the food chain - Determination and use of cardinal values’ is set to be released in October 2025. This document establishes basic principles and specifies requirements and methods to determine cardinal values of bacteria and yeast strains.
These methods are based on (1) the determination of maximum specific growth rates of the studied strain grown in a defined range of values of the intrinsic or extrinsic factor under study and (2) the use of secondary models to obtain the cardinal values. These methods can be applied to all type of bacteria and yeasts.
Finally, this document provides guidelines on the use of the determined cardinal values in growth simulation based on predictive microbiological models.
Also, in development are:
Researchers at the University of Maine Forest Bioproducts Research Institute (FBRI) have developed a new process to make (S)-3-hydroxy-γ-butyrolactone (HBL), a key ingredient in many medicines, from renewable glucose instead of petroleum. The approach not only lowers drug production costs but also reduces emissions.
The FBRI have also discovered a sustainable method to produce the key ingredient in a broad range of pharmaceuticals, which could help address high prescription drug costs in the U.S.
Among some of the most expensive medications are those that require a chiral centre - a property in which a molecule cannot be superimposed with its mirror image. Chirality can direct a drug's biological effects including efficacy, side effects and metabolisation. A large factor of the price of chiral drugs is attributed to the building blocks used during synthesis, which are costly to produce due to complex reaction and purification pathways.
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