Presentation (CTI)

OBJECTIVES: The regulatory and reimbursement landscape is rapidly changing, with increasing focus on closer collaboration to ensure faster access to medicines and the use of real-world data (RWD) to assess treatment effectiveness beyond safety considerations. This research aimed to understand the evolving landscape of regulatory filing routes and evidence requirements across two major regulators, the Food and Drug Administration (FDA) and European Medicines Agency (EMA), and describe the MHRA’s potential future position as an independent regulator in the post-Brexit environment.
METHODS: We conducted a pragmatic review of guidance documents, white papers and press releases from the FDA, EMA and MHRA. We considered the most recent version of each guidance document, and white papers and press releases from the past three years. A thematic analysis was conducted to critically consider challenges of filing routes across agencies and the potential impact of future MHRA directions on United Kingdom (UK) regulatory and access pathways.
RESULTS: Drug approval is based on distinct regulatory pathways, with the FDA using a single route for most treatments and both centralised and national options available in Europe. The MHRA increasingly prefers its own independent national route. Differences exist across agencies in the approach to using raw data versus company-shared analyses. While all agencies acknowledge the value of RWD in decision-making, the FDA is more flexible than EMA in considering such data to assess treatment effectiveness. The MHRA’s position on RWD is still evolving, but early trends suggest a focus on data quality, validity, and provenance.
CONCLUSIONS: The regulatory landscape is characterised by differing filing pathways and evolving evidence requirements. The MHRA is moving towards a national filing approach, following also global trends on wider integration of RWD in decision-making. These changes present both challenges and opportunities for pharmaceutical companies, impacting market access strategies and the alignment of UK regulatory and reimbursement submissions.