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 Europe and the US.
Regulatory News
The FDA published filing checklists to prevent submission delays
The FDA has published the internal filing checklists used by the CDER to determine whether drug and biologic applications are complete and reviewable. This move aims to increase transparency, reduce filing deficiencies and accelerate access to new treatments. During the initial filing period, CDER reviewers use discipline-specific checklists to assess completeness; minor issues can be resolved quickly, but applications with significant deficiencies receive a Refuse-to-File (RTF) notice, requiring resubmission. Over the past decade, more than 200 applications have received RTF notifications, with new molecular entities facing an average 426-day delay before resubmission. The checklists are included in the latest update of CDER’s MAPP 6025.4 Good Review Practices: Refuse to File. While the checklists serve as a valuable guide for sponsors, the FDA retains full authority to determine filing eligibility. Similar checklists for Abbreviated New Drug Applications were previously published.
The FDA awarded the first national priority vouchers
The FDA has announced nine recipients of the new Commissioner’s National Priority Voucher (CNPV) pilot program, which recognises products with strong potential to address major national priorities such as unmet medical needs, public health crises, domestic manufacturing and drug affordability. Recipients benefit from accelerated reviews, receiving decisions within 1–2 months of submitting complete applications, along with enhanced communication throughout development. Reviews are conducted by multidisciplinary teams working concurrently, concluding with a one-day “tumour board–style” meeting. The FDA may extend timelines if applications are incomplete or manufacturing issues arise. Each review division nominated one product, and sponsors may also apply. The selected products are: Pergoveris (infertility), Teplizumab (Type I diabetes), Cytisinicline (nicotine vaping addiction), DB-OTO (deafness), Cenegermin-bkbj (blindness), RMC-6236 (pancreatic cancer), Bitopertin (porphyria), Ketamine (domestic anaesthesia drug manufacture), and Augmentin XR (domestic antibiotic production). Further recipients will be announced soon.
The MHRA announced that UK clinical trial approval times are twice as fast with AI and reforms
Clinical trial approval times in the UK have been cut from 91 to 41 days after major reforms backed by new digital platforms at the MHRA, allowing patients earlier access to life-saving treatments. Research published in the British Journal of Clinical Pharmacology confirms that reforms and new digital platforms at the MHRA have reduced average approval times by more than half, with 99% of applications reviewed within statutory timelines. Introduced in 2023, the risk-proportionate review model speeds up approvals while protecting patient safety, with some lower-risk studies authorised in just 14 days.
AI now supports assessors by reviewing complex data, improving consistency and verifying manufacturing documents in seconds, while decisions remain with experienced assessors. Digital dashboards provide real-time visibility of active applications, and the Combined Review process streamlines ethical and regulatory assessments.
The reforms support the government’s 10 Year Health Plan and Life Sciences Sector Plan, with new legislation in April 2026 requiring public registration and plain-language trial results to strengthen trust and participation in research.
The MHRA and NICE invite early adopters to trial the accelerated aligned pathway
The MHRA and the National Institute for Health and Care Excellence (NICE) announced that pharmaceutical companies developing qualifying medicines can now access the aligned approvals pathway earlier than expected, following the MHRA and NICE decision to open applications 6 months ahead of schedule. The pathway, part of the Regulatory Action Plan, 10-Year Health Plan for England, and Life Sciences Sector Plan, aims to streamline regulation, accelerate patient access and enhance the UK’s global competitiveness in life sciences.
By combining the MHRA’s licensing process with NICE’s value assessment, both organisations will now publish decisions simultaneously, closing the former 90-day gap between marketing authorisation and NICE guidance, and enabling faster access for patients and greater efficiency for industry.
A joint scientific advice service, launching by April 2026, will offer a single-entry point for coordinated guidance, improving clarity and reducing delays. Companies are encouraged to register products on UK PharmaScan 3 years before marketing authorisation and engage early with both agencies.
The MHRA announced the first investigational products through the Innovative Licensing and Access Pathway (ILAP)
Three investigational therapies have become the first to join the UK’s ILAP, a new scheme designed to help promising medicines reach National Health Service (NHS) patients faster. The products, which include potential treatments for neonatal onset ornithine transcarbamylase deficiency, Duchenne muscular dystrophy and a rare neurodegenerative condition, have each received an Innovation Passport from the MHRA NHS, and UK health technology assessment bodies (NICE, Scottish Medicines Consortium, All Wales Therapeutic and Toxicology Centre).
The Innovation Passport provides early, coordinated support on clinical development, safety and value, helping accelerate access where current treatments are limited. The updated ILAP, building on the 2021 version, focuses on transformative products addressing unmet clinical needs, offering bespoke support, predictable timelines, NHS and patient engagement, and a single point of contact.
This world-first, end-to-end pathway aligns with the Rare Diseases Action Plan, the 10 Year Health Plan for England and the Life Sciences Sector Plan, supporting faster, system-wide adoption of innovative therapies.
The MHRA's Airlock programme announced seven AI healthcare technologies that could detect diseases earlier
Seven emerging AI healthcare technologies have been selected for the second phase of the MHRA’s AI Airlock programme, which provides a controlled environment to test AI-powered medical tools safely and assess their effectiveness, limitations and regulatory pathways. The selected technologies include innovations that could reduce bowel cancer test times from weeks to minutes and help detect skin cancer and genetic eye diseases earlier. Phase two builds on the pilot programme, with insights from the first cohort – including reports from Philips, Newton’s Tree, OncoFlow and Automedica Ltd – highlighting opportunities to improve synthetic data validation, AI decision explainability and responses to emerging risks such as AI hallucinations.
Findings from the Airlock will inform future MHRA regulation of AI and contribute to recommendations for the National Commission into the Regulation of AI in Healthcare, supporting the UK Government’s vision for the NHS to become the world’s most AI-enabled healthcare system.
The MHRA released a paper outlining its intentions for regulatory considerations for rare therapies
This position paper outlines the approach by UK regulators and partners to create a forward-looking framework that meets the needs of patients with rare diseases while supporting robust evidence generation, system sustainability and international convergence.
The programme will address the full lifecycle of rare therapies, from early scientific engagement with developers, through a proportionate and adaptable licensing process, to post-market surveillance. Continued collaboration with HTA bodies and the NHS will ensure alignment across the system and promote uptake.
Consultation on the new framework will begin in early 2026, with the goal of establishing a refined and implementable model by late 2026.
We have recently written a white paper detailing the regulatory landscape for orphan medicines across the European Union, the US and the UK, comparing the regulatory requirements for rare diseases across regulatory authorities.
Publications that caught our eye
- Estimands in equivalence trials and non-inferiority trials: a cross-sectional study of EMA scientific advice to drug developers. Springer Nature
- Scientific and regulatory progress in advancing paediatric oncology drug development in the EU and in the US. Frontiers in Medicine
- Accelerating precision medicine oncology by converging pragmatic trials and real-world evidence. Nature Reviews Drug Discovery
- Flaws in the FDA’s New Priority Voucher Program. The new England Journal of Medicine
- Exercise as a new therapeutic modality in oncology: CHALLENGE trial refines survivorship care. Nature Reviews Clinical Oncology.
