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
News: The EMA tracked progress towards 2030 clinical trial targets
The EMA, the European Commission and the Heads of Medicines Agencies, have published the first progress report on the European Union’s (EU’s) new clinical trial targets, introduced in 2025 to strengthen Europe’s position in clinical research and improve patient access to innovative medicines.
The report, covering January to March 2026, shows early progress, including 19 additional multinational clinical trials above the historical average, supporting the EU’s goal of 500 extra multinational trials by 2030. Recruitment timelines are also improving, with 40.5% of trials recruiting participants within 200 days of submission, compared with the 2030 target of 66%.
The targets align with the proposed EU Biotech Act and initiatives such as FAST-EU, which aims to accelerate multinational trial evaluations. The quarterly reporting framework is intended to improve transparency, accountability and ongoing assessment of the EU’s clinical trial performance and competitiveness.
News: The EMA welcomed political agreement on the Critical Medicines Act
The EMA welcomed the provisional agreement on the European Union’s (EU’s) proposed Critical Medicines Act (CMA), which aims to strengthen the resilience, security and sustainability of critical medicine supplies across the EU. The legislation responds to ongoing medicine shortages and supply chain vulnerabilities exposed by events such as the COVID-19 pandemic and geopolitical tensions.
The CMA combines regulatory measures with industrial policy initiatives to improve preparedness, reinforce manufacturing capacity and strengthen supply chains for critical medicines, including treatments for rare diseases. It builds on existing EU pharmaceutical reforms and the EMA’s expanded regulatory mandate.
The EMA is already supporting implementation through supply chain vulnerability assessments, expansion of the European Shortages Monitoring Platform and evaluation of medicines listed on the EU’s critical medicines list. The Act also supports innovation, joint procurement and state aid measures aimed at improving medicine availability and access across the EU.
News: The EMA launched a new advisory group on vaccine confidence
The EMA established a new advisory group on vaccine confidence to provide expert guidance on vaccine hesitancy and to support efforts to strengthen public trust in vaccines. The group, comprising more than 20 European and international experts from academia, healthcare, patient organisations and public health bodies, held its first meeting on 29 April 2026 and will meet quarterly.
The advisory group will help assess trends in vaccine hesitancy, advise on initiatives to improve vaccine confidence, enhance public communication about vaccine benefits and risks, and support EMA’s science outreach activities.
The initiative forms part of EMA’s broader Vaccine Outreach Strategy, which aims to improve understanding of vaccine safety, quality and effectiveness. It will also support projects such as Vaccine Essentials, a vaccine literacy resource developed with healthcare partners, and contribute to public information campaigns addressing vaccine-preventable diseases and misinformation.
News: The FDA expanded its AI capabilities
The FDA announced a major artificial intelligence (AI) initiative, including the launch of Elsa 4.0, an upgraded internal AI tool now available agency-wide. It also introduced HALO (Harmonised AI & Lifecycle Operations for Data), consolidating more than 40 FDA data systems and portals into a single platform. HALO is being integrated with Elsa to allow staff to query data and automate workflows without manually uploading documents.
Since Elsa 1.0 launched in June 2025, the FDA has expanded AI-driven efficiencies across operations. New Elsa 4.0 features include custom agents, document generation, data analysis and visualisation, secure web search, voice-to-text dictation, optical character recognition for scanned files, enhanced chat functions, and improved search across large repositories.
Elsa operates within a FedRAMP High secure Google Cloud environment, does not train on sensitive FDA or industry data and is not connected directly to the internet.
News: The FDA issued guidance to improve the collection of pregnancy safety data for drugs and biologics
The FDA issued final guidance on Post-approval Pregnancy Safety Studies, outlining recommended methods for assessing the safety of drugs and biological products used during pregnancy after they reach the market. Because limited human pregnancy data are often available at approval, post-approval studies are critical for understanding potential risks to pregnant patients and developing foetuses.
The guidance highlights several approaches for collecting safety data, including pregnancy registries, real-world data studies, descriptive studies and case report analyses. It also emphasises the importance of multidisciplinary expertise from fields such as obstetrics, paediatrics, genetics and statistics.
The FDA recommends combining these methods with established scientific standards and best practices for observational research, as many pregnancy studies are non-randomised. The goal is to generate evidence that can inform drug labelling and support healthcare providers and patients in making informed treatment decisions.
News: The FDA has advanced drug repurposing to address unmet medical needs
The FDA is seeking public input on drug repurposing efforts to help address unmet medical needs by identifying new uses or patient populations for existing FDA-approved drugs. Repurposing can accelerate treatment availability by building on established safety and efficacy data.
The initiative supports broader FDA efforts to modernise and update drug labelling using current scientific evidence. The agency is inviting feedback from patients, clinicians, researchers, and other stakeholders on priority disease areas and promising drug candidates, particularly where limited commercial incentives may hinder further development. Key focus areas include metabolic diseases, neurodegenerative conditions, women’s and men’s health, substance use disorders, and rare diseases.
The FDA is requesting information on repurposing candidates supported by existing clinical evidence, preliminary clinical findings, or emerging preclinical data, including AI- and machine learning-based discoveries. It is also seeking input on barriers to repurposing and innovative strategies to advance these treatments.
News: The FDA issued draft guidance to cut unnecessary animal testing for cancer drugs
The FDA’s draft guidance, Oncology Pharmaceuticals: Streamlined Nonclinical Safety Studies for Biologics and Conjugated Products, aims to reduce unnecessary animal testing and accelerate drug development. It recommends that, in some cases, animal studies may not be needed when there is no binding or pharmacologic activity. Where testing is required, the guidance may support using rodent studies only or replacing three-month non-human primate studies with a weight-of-evidence risk assessment that can incorporate New Approach Methodologies (NAMs).
The guidance is part of the FDA’s broader effort to shorten the drug development timeline, which can take 10–12 years from discovery to patient access. It builds on FDA analyses of toxicology studies, lessons from COVID-19-era practices that reduced non-human primate use, and complements existing guidance from the International Council for Harmonisation (ICH) and FDA recommendations for oncology therapeutic radiopharmaceuticals.
News: The MHRA launched a public consultation on a framework for rare diseases
The MHRA launched a public consultation on a proposed new regulatory framework designed to accelerate the development and approval of treatments for rare diseases, which affect around 3.5 million people in the UK. The framework aims to make therapy development faster, less costly, and more accessible while maintaining patient safety.
The proposal introduces a flexible, risk-based approach for rare conditions affecting no more than 1 in 50,000 people, particularly where standard clinical trials are difficult to conduct. A key feature is a new Investigational Marketing Authorisation, which would combine clinical trial approval with a progressive pathway to market authorisation, potentially enabling earlier patient access to innovative therapies, subject to NICE approval for NHS use.
By streamlining the regulatory process, the MHRA hopes to reduce development costs and improve affordability. The framework was developed in collaboration with the Rare Disease Consortium, including patients, carers, researchers, industry, and healthcare partners.
News: The MHRA opened a consultation on redefining gene therapies
The MHRA together with the Department of Health in Northern Ireland, has launched a consultation on updating the UK’s legal definition of gene therapy medicinal products to reflect advances in gene therapy, synthetic biology and gene editing technologies.
The proposed changes would classify gene therapies based on their mechanism of action rather than their biological origin, providing greater regulatory clarity and ensuring consistent oversight of both biologically derived and synthetic products. The revisions would also explicitly include sequence-specific genome editing products while maintaining the exclusion of vaccines against infectious diseases.
The MHRA emphasises that the changes will not affect existing approval processes, licensed products, or current safety, quality and efficacy standards. The update aims to support innovation, provide greater certainty for developers and create a future-proof regulatory framework while maintaining robust patient protections. The consultation runs from 11 May to 22 June 2026.
News: The MHRA shared its targets to increase access to medicines and reinforce UK position as a global destination for life sciences
The MHRA reported that it met or exceeded all statutory performance targets in 2025/2026, supporting faster patient access to medicines and medical devices while strengthening the UK’s position as a leading life sciences hub.
According to its Results and Forecast report, the MHRA improved patient safety by tackling health misinformation, enhancing medical device surveillance, removing nearly 28 million unauthorised medicine doses and strengthening safety monitoring systems. The agency also met all key regulatory performance targets, ended the year with a financial surplus and advanced work in areas such as artificial intelligence regulation, clinical trial reform and rare disease therapies.
The MHRA further strengthened national and international partnerships, including collaborations with the National Institute for Health and Care Excellence, the FDA, and Singapore’s Health Sciences Authority, while reporting improved industry perceptions and higher staff engagement. Building on this performance, the agency plans to launch a new 5-year strategy later in 2026 to support innovation, patient safety and growth in the UK life sciences sector.
Publications that caught our eye
- Orphan Designation for Drugs Approved in the United States and European Union: A Comparative Analysis. Clinical Pharmacology & Therapeutics.
- Tumor-Agnostic Therapies: Translating Scientific Breakthroughs Into Global Implementation. Journal of Clinical Oncology.
- Trends in EU regulatory assessment of oncology medicines. ESMO Open.
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