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 published a concept paper on developing a reflection paper on the external controls for evidence generation in regulatory decision-making
The EMA has published a concept paper outlining plans for a reflection paper on the use of external controls in regulatory decision-making. While randomised controlled trials remain the gold standard for establishing the benefits and risks of medicines, external controls may be appropriate in certain circumstances where control data are sourced from other clinical trials, real-world data or other datasets rather than a randomised arm within the same trial. The reflection paper will examine the key challenges, appropriate circumstances, and methodological constraints for using external controls to generate pivotal or supportive evidence for efficacy, safety and other regulatory objectives. The concept paper was finalised following public consultation and a workshop held in November 2025.
News: The EMA published its 2025 artificial intelligence (AI) observatory report
The 2025 AI Observatory report highlights the rapidly evolving role of AI across the medicines lifecycle. Following the entry into force of the European Union (EU) AI Act in 2024 and its transition to practical application in 2025, the report reflects efforts to support the trustworthy, transparent and ethical use of AI while encouraging innovation. Published annually by the Network Data Steering Group, the Observatory monitors AI activities, trends and emerging domains. The 2025 report provides an overview of AI-related guidance and policy, current applications, collaborations, stakeholder engagement, EU-funded initiatives and regulatory science research across both human and veterinary medicines.
News: The FDA issued draft guidance to help accelerate cell and gene therapies for patients
The FDA has issued draft guidance to help developers bring gene therapies to patients more efficiently by enabling greater use of existing scientific and regulatory knowledge. The guidance describes how sponsors can leverage publicly available data and established platform knowledge, including chemistry, manufacturing and controls, non-clinical and clinical data, to streamline regulatory submissions for genome editing therapies. It complements the FDA’s Plausible Mechanism Framework and related draft guidance on safety assessment using next-generation sequencing, providing a science-based approach to product development while maintaining rigorous patient safety standards. Sponsors must justify the relevance of any data used to their product and are encouraged to engage with the FDA early in development through programmes such as INTERACT (Initial Targeted Engagement for Regulatory Advice on CBER/CDER Products) and pre-investigational new product meetings.
News: The MHRA reapproves controversial PATHWAYS trial paused over safety concerns
The British Medical Journal has reported that the UK Pathways trial investigating puberty blockers for children with gender incongruence has been reapproved following revisions to its study protocol. The trial was established in response to the 2024 Hilary Cass review, which found limited evidence on the benefits and harms of puberty blockers and recommended further research. After being paused by the MHRA in February due to concerns about participant well-being, the protocol was updated to improve information provided to patients and guardians and to introduce a minimum age for participation. Led by King's College London, the trial plans to enrol 226 children into two study arms comparing immediate versus delayed access to puberty blockers.
News: The European Society of Medical Oncology (ESMO) reported that an artificial intelligence (AI)-driven multimodal strategy improves risk stratification in early breast cancer
An AI-driven multimodal approach integrating digital pathology with genomic and clinical data has shown improved risk stratification in early hormone receptor-positive (HR-positive), human epidermal growth factor receptor 2-negative breast cancer. Presented at the ESMO Breast Cancer 2026, the research demonstrated that combining AI-based digital pathology with the Oncotype DX 21-gene recurrence score and clinical tumour features improved the prediction of late distant recurrence compared with existing risk assessment tools. Analysis of over 6,300 patients from the TAILORx trial, supported by an independent validation cohort, showed the novel AI-PathClinRS biomarker provided more accurate risk prediction and identified approximately twice as many patients at high risk of recurrence, potentially improving selection for adjuvant treatment intensification.
News: The MHRA approved the first glucagon-like peptide-1 (GLP-1) tablet for weight loss
The MHRA has approved the UK’s first GLP-1 receptor agonist tablet for weight loss and weight management, oral semaglutide (Wegovy). The treatment is indicated, alongside diet and exercise, for adults with obesity (BMI ≥30) or those who are overweight (BMI 27–30) with at least one weight-related comorbidity. The once-daily tablet is introduced through dose escalation to a maintenance dose of 25 mg, and patients currently receiving weekly semaglutide injections may transition directly to the oral formulation. Semaglutide works by mimicking the GLP-1 hormone to reduce appetite and food intake. Although approved for use in the UK, the treatment is not yet available through the National Health Service (NHS) and will require evaluation by the National Institute for Health and Care Excellence before routine NHS adoption.
News: The MHRA is launching its artificial intelligence (AI) sandbox to accelerate medicines development and improve safety
The MHRA has announced the launch of a first-of-its-kind AI regulatory sandbox to evaluate how AI can improve medicine development and safety assessment. Supported by UK Government funding, the initiative will provide a controlled environment for companies and researchers to test AI tools that predict medicine behaviour, identify potential safety risks and reduce reliance on animal testing. The programme will also assess how AI and clinical data can improve understanding of treatment effects across diverse patient populations. Initially, up to five AI-driven approaches will be evaluated, with industry and academic collaboration beginning in summer 2026. The findings will help inform future regulatory decision-making and support the safe adoption of AI in medicines development while encouraging innovation in the UK.
Publications that caught our eye
- Major objections in EU medicines authorisation applications: an analysis of dossier submissions by small- and medium-sized enterprises. Drug Discovery Today.
- From complexity to approval in the European Union: Regulatory pathways and data challenges in the evaluation of non-oncology orphan medicines. Drug Discovery Today.
- Ivonescimab: The Next United States Food and Drug Administration Project Optimus Flashpoint? JTO Clinical and Research Reports.
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