Each month, we scan the regulatory landscape to bring you the latest developments shaping pharmaceutical and biotechnology innovation. In this roundup, you’ll find key publications that caught our attention, plus a curated summary of the most important regulatory news. Our goal is to highlight what matters, cut through the noise, and keep you up to date on changes that could impact drug development and market access in the UK, Europe and the US.
Regulatory Affairs News
The EMA published the medicines approved in 2025
In 2025, EMA recommended 104 medicines for marketing authorisation, including 38 with new active substances not previously authorised in the EU. These included the first treatment for non-cystic fibrosis bronchiectasis, a first-in-class therapy to delay stage 3 type 1 diabetes and the first oral treatment for postpartum depression.
Sixteen medicines were recommended for rare diseases, including the first treatment for Wiskott-Aldrich syndrome and a topical gene therapy for dystrophic epidermolysis bullosa. The EMA also issued three positive opinions for medicines outside of the EU, including a twice-yearly injectable pre-exposure prophylaxis for human immunodeficiency virus type 1 prevention.
Additionally, 41 biosimilars were recommended, supporting cost management and access to treatment. The 2025 overview highlights key authorisations and safety-related recommendations. After European Commission authorisation, medicines are continuously monitored, with regulatory actions taken when necessary to protect public health.
The EMA created a new ‘Concept Paper to Develop a Reflection Paper on Using Bayesian Methods in Clinical Development’
The purpose of the guideline is to address key considerations for studies using Bayesian statistics in clinical development. Frequentist methods have traditionally been the standard approach to data analysis in drug development and regulatory submissions. Nevertheless, the ICH E9 guideline states that Bayesian methods may be used “when the reasons for their use are clear and when the resulting conclusions are sufficiently robust”.
The FDA increased flexibility on requirements for cell and gene therapies
The FDA announced a flexible approach to overseeing chemistry, manufacturing and control (CMC) requirements for cell and gene therapies (CGTs), aimed at expediting development and guiding evaluation ahead of Biologics License Application submissions.
Over the past decade, the FDA’s CBER has approved close to 50 CGTs. These complex, often individualised therapies require sophisticated manufacturing under tight timelines. While CBER has historically applied similar CMC expectations across products, it has used regulatory flexibilities to address the unique characteristics of CGTs while maintaining rigorous quality standards and appropriate control measures.
Recognising rapid scientific advances, the FDA is clarifying how these flexibilities are applied to remove barriers and misconceptions, supporting faster development without compromising safety, purity, potency or benefit–risk evaluation.
The FDA published guidance on modernising statistical methods for clinical trials
The FDA published draft guidance to facilitate the use of Bayesian methodologies in clinical trials for drugs and biologics. By combining study data with relevant prior information, Bayesian analyses generate updated distributions to support conclusions on safety and efficacy, helping sponsors use available data more efficiently and potentially deliver treatments sooner.
Bayesian approaches can enable earlier futility or success decisions in adaptive trials, inform dose selection, incorporate prior clinical data, real-world evidence and external controls, support subgroup analyses, and contribute to primary inference. They may be particularly valuable in rare or paediatric indications with small patient populations.
The guidance, ‘Use of Bayesian Methodology in Clinical Trials of Drugs and Biologics Guidance for Industry’, fulfils the FDA’s Prescription Drug User Fee Act VII commitment to enhance review capacity for complex and innovative trial designs.
The FDA published a draft guidance on the use of the plausible mechanism framework
This guidance outlines key considerations for generating substantial evidence of safety and effectiveness for individualised therapies that target specific genetic conditions with known biological causes, using the plausible mechanism framework. It provides recommendations to help developers produce sufficient clinical safety and efficacy data to demonstrate that a drug or biological product is safe, effective for its intended use, and can be manufactured to regulatory quality standards. These data are intended to support approval or licensure for a specific indication and include careful evaluation of nonclinical and clinical findings, as well as chemistry, manufacturing, and controls (CMC) data necessary to ensure product quality.
The MHRA published a press release: boosting clinical trial attractiveness with faster assessments
The UK is strengthening its position as a leading destination for clinical trials, with 2025 data showing rising activity and upcoming reforms to accelerate study start-up. Applications to the MHRA increased by 9% between January and November 2025 compared with 2024, with notable growth in early and innovative research. Trials in healthy volunteers rose by 16%, first-in-human studies by 5% and trials newly run in the UK by 7%. The MHRA scientific advice meetings increased by 75%, reflecting earlier developer engagement.
From April 2026, new regulations will introduce a fast-track route for around 20% of lower-risk studies and a 14-day assessment pathway for phase 1 trials. The framework will also enable use of overseas safety data and assessment of in-silico simulations. Over 450,000 participants took part in studies across England last year. Research in the British Journal of Clinical Pharmacology reported that 99% of MHRA applications are reviewed on time.
The UK government announced an end to the cancer postcode lottery for patients
People in rural and coastal communities will gain faster access to cancer specialists under new government plans to end the postcode lottery in care across England. Targeted training places will address workforce gaps in underserved areas, encouraging more doctors to specialise in oncology through collaboration with the royal colleges.
The forthcoming National Cancer Plan will introduce national standards across the full cancer pathway and expand access to cutting-edge diagnostics and artificial intelligence. From April 2027, new tests and digital tools will undergo the same rigorous approval process as medicines, ensuring nationwide rollout once approved. National Institute for Health and Care Excellence will begin assessing the first technologies this year.
Over 450,000 additional timely diagnoses have been delivered since July last year, supported by 170 community diagnostic centres and £70 million invested in advanced radiotherapy machines, helping reduce inequalities and improve survival outcomes.
Publications that caught our eye
- One Pivotal Trial, the New Default Option for FDA Approval – Ending the Two-Trial Dogma. The New England Journal of Medicine
