Regulatory News
EU Consultation Launched to Update GMP Chapter 1 on Pharmaceutical Quality Systems
On 3rd September, the European Commission opened a stakeholders’ consultation on proposed revisions to Chapter 1 of EudraLex Volume 4, Good Manufacturing Practice (GMP) guidelines – the section covering the Pharmaceutical Quality System. The draft updates align GMP requirements with the latest ICH Q9(R1) guideline, enhancing risk management, knowledge management, and proactive mitigation of manufacturing risks, including supply‑chain vulnerabilities. The consultation is open until the 3rd of December 2025, targeting organisations and individuals involved in GMP activities.
FDA Launches Crackdown on Deceptive Drug Advertising
On 9th September, the U.S. Department of Health and Human Services and the FDA announced significant reforms to address misleading direct-to-consumer pharmaceutical advertisements. The FDA is issuing thousands of warning letters and approximately 100 cease-and-desist orders to companies with deceptive ads.
MHRA Outlines Strategic Vision and Priorities for 2030
On 16th September, the MHRA’s Chief Executive, Lawrence Tallon, shared reflections on the strategic choices facing the agency as it develops its multi-year strategy through to 2030. The MHRA aims to adapt to rapid changes in healthcare, such as personalised medicine and AI-driven diagnostics, while keeping patient safety at the core. The agency plans to balance innovation with risk-proportionate regulation, improve efficiency through automation and international collaboration, and make strategic decisions amid limited resources. Broad engagement with patients, clinicians, industry, and staff will shape the strategy moving forward.
Professor Richard FitzGerald Appointed to the Commission on Human Medicines
On 17th September 2025, the Department of Health and Social Care announced the appointment of Professor Richard FitzGerald as a commissioner to the Commission on Human Medicines (CHM). His four-year term commenced on 8 September 2025. Professor FitzGerald, an Emeritus Professor at the University of Limerick, brings extensive expertise in clinical research and pharmacology to the CHM. The role involves a commitment of approximately 22 days per year, including 11 meetings, with remuneration set at £325 per meeting.
UK Launches Route B Pilot for Faster Review of Clinical Trial Modifications
On 17th September, the MHRA Inspectorate Blog announced a pilot program for “Route B” substantial modifications under the new clinical trials regulations, which will take effect on 28th April 2026. Under Route B, eligible modifications to approved trial applications may receive automatic approval within 14 days, instead of the longer 35‑day timeframe for Type A modifications.
EMA Introduces New Variations Guidelines to Streamline Medicine Lifecycle Management
On 22nd September, the EMA announced the adoption of new Variations Guidelines, effective from 15th January 2026. Developed in partnership with the European medicines regulatory network and the European Commission, these guidelines introduce a streamlined, risk-based approach to managing post-authorisation changes to medicines. The updated framework is designed to enhance regulatory efficiency by simplifying the classification and processing of variations, addressing the growing volume driven by scientific and technological advancements. This initiative supports broader efforts to accelerate the delivery of medicines, including those combined with medical devices, to patients across the EU throughout their lifecycle.
EMA Sets Ambitious Targets to Enhance Clinical Trials in Europe
On 23rd September, the EMA, in collaboration with the European Commission and the Heads of Medicines Agencies (HMA), announced new targets to strengthen clinical trials in the European Union. Over the next five years, the aim is to increase the number of multinational clinical trials by 500 annually and ensure that two-thirds of clinical trials begin recruiting patients within 200 days of application submission, up from the current 50%. These goals are part of the Accelerating Clinical Trials in the EU (ACT EU) initiative, which focuses on improving trial design, enhancing support for non-commercial sponsors, and implementing the revised Good Clinical Practice guidelines.
EMA Appoints New Co-Chairs to Strengthen Patient and Healthcare Professional Engagement
On 23rd September, EMA elected Marco Greco as co-chair of the Patients’ and Consumers’ Working Party and Dr Piotr Szymanski as co-chair of the Healthcare Professionals’ Working Party for the 2025–2028 mandate. Together, they will help ensure patient and healthcare professional perspectives continue to shape EMA’s work.
EMA Calls for Public Input to Strengthen Patient-Centred Medicine Regulation
On 29th September, the EMA published a draft reflection paper, opening a public consultation period until 31st January 2026. The paper aims to strengthen the incorporation of patient experience data into the regulation of medicines. This initiative underscores the critical role of patients’ lived experiences in evaluating treatment benefits and risks, particularly in areas such as cancer care, where quality of life often outweighs traditional clinical metrics. The EMA is calling on medicine developers, patient organisations, and researchers to contribute feedback that will help shape the future of regulatory practices.
MHRA Issues September 2025 Safety Roundup
On 30th September, the MHRA published its monthly safety update, summarising the latest guidance and alerts for users of medicines and medical devices.
Sir David Spiegelhalter Defines “Preference Zone” for Regulation in Personalised Medicine
On 2nd October 2025, Sir David Spiegelhalter published a guest article via the MHRA exploring how regulation must evolve in the era of personalised medicine. He introduces the idea of a “preference zone” where individuals may reasonably vary in how they weigh benefits and harms. He argues that regulation should account not only for scientific variation in how treatments affect different people, but also for variation in how people value outcomes. Spiegelhalter also highlights the importance of strong real‑world monitoring and risk communication, drawing on experiences from COVID‑19 vaccine risk/benefit analysis.
EMA Network Adopts Strategic Data Vision to Boost Public & Animal Health
On 3rd October, the European Medicines Regulatory Network (EMRN) unveiled a comprehensive data strategy, officially adopted in mid‑2025, to maximise the value of regulatory data for public and animal health. The strategy outlines guiding principles (e.g. “data as assets,” interoperability, secure and ethical use) and six key strategic goals, including data governance, quality management, interoperability, metadata cataloguing, knowledge exchange, and advanced analytics. The framework is designed to harmonise data practices across national regulators and the EMA, supporting smarter decision‑making, more efficient processes, and broader access to high‑quality data.
Published Guidance
Medicines and Healthcare products Regulatory Agency (MHRA)
- 5th September: Medical devices: Standardised format for the post-market surveillance report. Guidance was published detailing a standardised format for Post‑Market Surveillance Reports (PMSRs) for medical devices. This document is intended to help manufacturers understand which data to include in their PMSRs, though sections not relevant may be omitted or adapted.
US Food & Drug Administration (FDA)
- 10th September: Development of Non-Opioid Analgesics for Chronic Pain. Aims to support sponsors in the development of non-opioid analgesics for chronic pain treatment. It outlines the FDA’s current recommendations for conducting Phase 3 clinical trials of prescription non-opioid analgesic products intended for managing chronic pain.
- 18th September: Safety Labelling Changes – Implementation of Section 505(o)(4) of the FD&C Act. Outlines the implementation of the statutory provision granting the FDA authority to require application holders of certain drug and biological products to update labelling based on new safety information that emerges after approval and is deemed necessary for inclusion in the drug’s labelling.
- 24th September: Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations. Offers recommendations to sponsors planning clinical trials for cell and gene therapy (CGT) products targeting diseases or conditions that affect a small population—typically those classified as rare diseases or conditions under section 526(a)(2) of the FD&C Act (21 U.S.C. 360bb(a)(2)).
- 24th September: Expedited Programs for Regenerative Medicine Therapies for Serious Conditions. Offers recommendations to sponsors developing regenerative medicine therapies for serious or life-threatening diseases or conditions, focusing on expedited development and review processes as outlined in section 506(g) of the Federal Food, Drug, and Cosmetic Act, added by section 3033 of the 21st Century Cures Act.
- 30th September: E20 Adaptive Designs for Clinical Trials. The draft guidance aims to offer a clear and harmonised framework for clinical trials employing adaptive designs. It emphasises key principles for the planning, execution, analysis, and interpretation of such trials, to confirm treatment efficacy and support benefit-risk assessments.
European Medicines Agency (EMA)
- 30th September: Guideline on clinical investigation of medicinal products. The updated document reflects changes in clinical practice and therapeutic options since the original 2007 guideline. European Medicines Agency (EMA) Key updates include tighter recommendations on patient selection criteria, efficacy endpoints across multiple disease domains, study designs (including dose finding, comparator choice, and handling of rescue treatments), and safety monitoring, especially long-term risks and immunogenicity.
- 6th October: Scientific advice on MRL classification of ‘chemical-unlike’ biological substances. The Committee for Veterinary Medicinal Products (CVMP) of the European Medicines Agency (EMA) published scientific advice confirming that five previously assessed ‘chemical-unlike’ biological substances do not require a Maximum Residue Limit (MRL) evaluation. The CVMP’s assessment supports their classification as ‘No MRL required’ in Table 1 of the annexe to Regulation (EU) No. 37/2010, aligning with the European Commission’s request for scientific advice on this matter.
Updated Guidance
MHRA
- 2nd September: Make a payment to MHRA. Updated content for clarification that Device Registration and Certificates of Free Sale for medical device fees must be made only through the Device Online Registration system (DORS).
- 2nd September: Medicines: apply for a parallel import licence. The Tell and Do (TaD) cover sheet for parallel import (PLPI) companies has been updated.
- 3rd September: Medicines: Marketing Authorisation Holders’ submission of Nitrosamine risk evaluation, risk assessment and confirmatory testing. Updated to include lifecycle management and corrective and preventive action (CAPA) implementation.
- 12th September: Apply for manufacturer or wholesaler of medicines licences. Additional information was added under ‘Change of ownership’
- 12th September: MHRA fees. Updated the link to the ‘Apply for payment easement as an SME’ form.
- 17th September: International Recognition Procedure. Updated to align with guidance on UK-wide Licensing.
- 1st October: Project Orbis. Updated to add Voranigo to the Overview of Orbis Project approvals.
- 1st October: Clinical trials for medicines: collection, verification, & reporting of safety events. Updated to include new guidance wording and attachments.
- 1st October: Clinical trials for medicines: notifiable trials. Changed subheading from “Guidance on categorising certain clinical trials as ‘notifiable’” To “Guidance on clinical trials that are categorised as ‘notifiable’” and added new documents
- 1st October: Clinical trials for medicines: modifying a clinical trial approval. Updated to add new documents on Route A and B for Modifying a Clinical Trial Approval
FDA
- 8th September: E6(R3) Good Clinical Practice (GCP). This update adopts adaptable, risk-based strategies and integrates advancements in trial design, execution, and technology. This key milestone represents a major step forward in the global clinical trial environment, striving to modernise Good Clinical Practice principles in line with contemporary scientific and technological progress, while prioritising quality by design, participant safety, and the integrity of trial outcomes.
- 9th September: Development of Therapeutic Protein Biosimilars: Comparative Analytical Assessment and Other Quality-Related Considerations Guidance for Industry. Describes the Agency’s recommendations on the design and evaluation of comparative analytical studies intended to support a demonstration that a proposed therapeutic protein product is biosimilar to a reference product licensed under section 351(a) of the Public Health Service Act (PHS Act).
- 11th September: Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications. This final guidance informs applicants about the FDA’s approach to using alternative tools to evaluate drug manufacturing facilities referenced in marketing applications, including new drug applications (NDAs), abbreviated new drug applications (ANDAs), biologics license applications (BLAs), and their respective supplements.
- 11th September: Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications. This final guidance informs applicants about the FDA’s approach to using alternative tools to evaluate drug manufacturing facilities referenced in marketing applications, including new drug applications (NDAs), abbreviated new drug applications (ANDAs), biologics license applications (BLAs), and their respective supplements.
- 24th September: Computer Software Assurance for Production and Quality System Software. Provides guidance on computer software assurance for computers and automated data processing systems utilised in medical device production and quality systems.
Industry News
Quotient Sciences and CPI Forge Strategic Partnership to Accelerate RNA Therapy Development
On 2nd September, Quotient Sciences and the Centre for Process Innovation (CPI) announced a strategic joint venture through a newly signed Memorandum of Understanding, aiming to significantly accelerate the development of RNA-based therapeutics. The partnership combines Quotient’s Translational Pharmaceutics® platform with CPI’s cutting-edge capabilities in small-scale RNA manufacturing and lipid nanoparticle (LNP) formulation. Together, they aim to streamline the journey from plasmid DNA to clinical dosing in as little as two months – substantially shortening timelines to early-phase clinical trials.
Increased Risks Found in Profemur Co‑Chrome Modular Neck Hip Replacements
On 4th September, the MHRA published a safety update highlighting increased risks linked to certain Profemur cobalt-chrome modular neck hip replacements. These devices, used in the UK between 2009 and January 2025, have been associated with a higher likelihood of metal wear, which may lead to soft tissue reactions and, in some cases, revision surgery. The update advises healthcare providers to identify affected patients and offer follow-up assessments based on individual risk. Patients do not need to contact their hospitals, as they will be approached directly if necessary.
EMA Backs Orphan Drug Status for SOM3355 in Huntington’s Disease
On 15th September, SOM Biotech announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) had issued a positive opinion recommending Orphan Drug Designation (ODD) for SOM3355 in the treatment of Huntington’s disease. This recognition marks a significant milestone in the development of SOM3355 and reinforces SOM Biotech’s commitment to advancing innovative therapies for rare central nervous system disorders.
MHRA and Department for Education Integrate Medicine Safety into School Curriculum
On 17th September 2025, the MHRA announced that, in collaboration with the Department for Education, it has embedded medicine safety education into the statutory Relationships, Sex and Health Education (RSHE) curriculum for schools in England. This initiative, launched on World Patient Safety Day, introduces students to the importance of reporting side effects through the Yellow Card scheme. The curriculum now includes guidance on recognising side effects, understanding their significance, and submitting reports via the Yellow Card website. This move empowers the next generation to actively contribute to the safety of medicines and medical devices.
First Treatment for Barth Syndrome Receives Accelerated Approval
On 19th September, Stealth BioTherapeutics announced the accelerated approval of Forzinity™ (elamipretide HCl), marking the first approved therapy for Barth syndrome, a rare and progressive mitochondrial disorder. This approval was granted based on clinical trial results showing significant improvements in muscle strength among patients. Stealth BioTherapeutics is committed to expanding access to younger children weighing under 30 kilograms and continues to advance the development of therapies for Barth syndrome and other mitochondrial diseases.
MHRA Confirms Paracetamol Use During Pregnancy is Safe
On 22nd September, the MHRA reaffirmed that taking paracetamol during pregnancy remains safe and there is no evidence linking its use to autism in children. This statement follows a comprehensive review, including a 2024 Swedish study involving 2.4 million children, which found no association between prenatal paracetamol exposure and autism. The MHRA advises pregnant women to use paracetamol at the lowest effective dose and for the shortest duration, in line with NHS guidance. Healthcare professionals and the public are encouraged to report any suspected side effects via the Yellow Card scheme.
GM CRDC Launches First Studies on Vaccine Effectiveness
On 22nd September, the Greater Manchester Commercial Research Delivery Centre (GM CRDC), hosted by Manchester University NHS Foundation Trust (MFT), initiated its first three studies aimed at evaluating the real-world effectiveness of COVID-19 and Respiratory Syncytial Virus (RSV) vaccines. These studies, targeting patients hospitalised with severe acute respiratory infections (SARI), are part of the id.DRIVE initiative, a collaborative effort to assess infectious disease interventions across the UK and Europe.
FDA Initiates Approval of Leucovorin Calcium Tablets for Autism-Related Symptoms
On 22nd September, the FDA initiated the approval process for leucovorin calcium tablets to treat cerebral folate deficiency (CFD), a neurological condition associated with developmental delays and autistic features. The FDA’s review of literature and case reports supports leucovorin’s potential benefits for individuals with CFD. The agency is collaborating with GSK to update the Wellcovorin label to include information on its use in both paediatric and adult CFD patients.
Government Backs Oxfordshire Vaccine Manufacturing with £50m Boost for Life Sciences
On 25th September 2025, the UK government announced that a new Moderna Innovation and Technology Centre in Oxfordshire will produce millions of mRNA vaccines annually, supporting both seasonal and pandemic responses. This initiative is part of a wider push to stimulate the life sciences sector through a newly piloted £50 million Life Sciences Transformational R&D Investment Fund, aimed at attracting large-scale, capital-intensive projects in UK-based life sciences research and development. The Harwell facility has the potential to scale up production to 250 million doses per year during a health emergency and is expected to create roughly 150 skilled jobs locally.
UK Launches Commission to Speed Up Safe NHS Adoption of AI
On 26th September 2025, the MHRA announced the formation of a National Commission to accelerate NHS use of artificial intelligence. The commission – comprising clinicians, patient advocates, tech firms like Google and Microsoft, and regulators – will guide MHRA in rewriting the regulatory rulebook for healthcare AI, set to be published next year. It will immediately focus on areas of regulatory uncertainty, such as AI assistants for doctors and “Ambient Voice Technology,” and examine tools in radiology, pathology, and remote patient monitoring.
MHRA, ASA & GPhC Reinforce Legal Ban on Advertising Prescription-Only Weight‑Loss Medicines
On 26th September 2025, the MHRA, in partnership with the Advertising Standards Authority (ASA) and the General Pharmaceutical Council (GPhC), issued an updated Enforcement Notice to reinforce the legal ban on advertising prescription-only weight-loss medicines to the public. The notice targets pharmacies and other businesses promoting these medicines through websites, social media, and influencer content.
Essential GLP: Key Components to Ensure Compliance and Inspection Readiness
As the Good Laboratory Practice (GLP) landscape evolves with updated guidance and renewed regulatory focus, it’s crucial for facilities to stay inspection-ready. In our recent blog, Nicole explores the key areas you should be focusing on and shares practical steps, along with regulatory insights, to help your facility remain compliant and audit-ready.
Topics include the management of Test Items, ensuring equipment is fit-for-purpose and properly qualified, robust Computer System Validation (CSV), and maintaining strong Data Integrity (DI). Quality Assurance (QA) oversight and vendor/test site management also feature prominently, all within a risk-based framework designed to support data integrity and reproducibility.
Issue Number: WN00202
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