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.
News
The FDA advances rare disease drug development with new evidence principles
The FDA has introduced the Rare Disease Evidence Principles (RDEP) to speed and clarify the review of therapies for very rare, genetically defined diseases with high unmet medical need. Jointly developed by CDER and CBER, the RDEP offers sponsors clear guidance on acceptable evidence to demonstrate effectiveness when traditional trials are not feasible. Under this framework, approval may be based on one well-controlled study supported by robust confirmatory evidence, such as mechanistic or biomarker data, non-clinical models, pharmacodynamic data or case reports. Eligible therapies must target a known genetic defect in a population of fewer than 1,000 US patients lacking effective treatments. Sponsors must apply before pivotal trials begin and collaborate closely with the FDA, patients and experts to define the evidence package. Post-marketing studies may be required. The RDEP process is independent of orphan-drug designation.
The EMA published a reflection paper on patients’ perspectives in medicine regulation
The EMA has released a draft reflection paper on patient experience data for public consultation. These data capture patients’ direct experiences, perspectives and treatment preferences without clinical interpretation. The EMA recognises the importance of such data in complementing traditional scientific outcomes, offering insights into what matters most to patient, for example, prioritising quality of life over survival in cancer treatment.
The paper provides general principles for generating, collecting and analysing patient experience data throughout a medicine’s lifecycle and encourages developers to integrate these insights in pre-authorisation, benefit-risk evaluation and post-authorisation phases. Patient experience data may come from Patient Reported Outcomes, Patient Preference Studies or real-world sources such as mobile health and social media.
Developed by a multi-disciplinary EU group, the paper complements ICH (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use) global harmonisation efforts and invites stakeholder feedback to refine the EU’s approach to patient-centred evidence generation.
The EMA and the World Health Organisation (WHO) mark 10 years of collaboration
The EMA and the WHO mark 10 years of formal collaboration, established through a confidentiality arrangement in 2015. Their partnership focuses on scientific evaluation, capacity building and improving global regulatory efficiency to advance public health. A key initiative, EU-M4All (EU Medicines for all), allows the EMA and the WHO to support global regulatory systems and access to essential medicines. A recent success was the EMA’s 2025 recommendation of Yeytuo (lenacapavir) for HIV-1 pre-exposure prophylaxis, evaluated with experts from several African and Asian countries—demonstrating inclusive, harmonised global review.
Together with the European Commission’s Directorate-General for Health and Food Safety (DG SANTE), the EMA and the WHO have collaborated to address public and animal health threats, accelerate access to medicines, promote reliance practices, and strengthen pharmacovigilance, inspections and regulatory harmonisation. Their joint efforts continue to build global capacity, ensuring equitable access to safe, effective medicines worldwide.
The MHRA’s CEO reflects on its future strategic choices in developing its 5-year strategy
The MHRA has begun developing a new multi-year strategy to 2030, reflecting on its evolving role within a rapidly changing healthcare landscape. CEO Lawrence Tallon highlights priorities including adapting to personalised medicine, artificial intelligence (AI)-driven technologies, and the consumerisation of healthcare, while maintaining patient safety as the agency’s core mission. The MHRA aims to foster a ‘preference zone’, where patients, guided by clinicians, can make informed choices about treatments that meet safety and efficacy standards.
The strategy focuses on risk-proportionate regulation, greater use of automation and AI, and international collaboration to balance innovation with safety. The MHRA, now larger and back on performance targets, will prioritise areas of specialisation while partnering with peer regulators globally. Developed through broad stakeholder engagement, the strategy seeks to strengthen the MHRA’s role as a trusted, agile and globally recognised regulator supporting UK health and life sciences innovation.
Publications that caught our eye
- Beyond traditional endpoints: A multi-state modelling approach to integrating morbidity and mortality in cancer treatment evaluation. European Journal of Cancer.
- Acceptance of external control arms by HTA agencies: a review of oncology submissions in France, England, Germany and Norway from 2021 to 2023. British Journal of Cancer
- Precision Oncology and Modernizing Evidence Standards: A Critical Commentary on the Cochrane Review. Journal of Clinical Oncology
- Global regulatory variability in small molecule inhibitor approvals: Differences in timelines, dosing, and pediatric indications across FDA, EMA and PMDA. Clinical & Translational Oncology
- EU clinical trials during the 3-year transition period. European Medicines Agency
