Presentation (CTI)

OBJECTIVES: To examine availability and market access timelines for medicines approved under expedited regulatory pathways. Regulatory pathways exist to accelerate the approval of new medicines. Conditional marketing authorization (CMA) and Exceptional circumstances in Europe, and expedited review pathways in the US allow for earlier approval despite evidence limitations. The relationship between early regulatory approval paths and reimbursed access is unclear.
METHODS: Nineteen orphan drugs approved by the European Commission under CMA and Exceptional Circumstances from 2020-2023 were identified. Fourteen of these were US approved under expedited review pathways. Availability and time to standard reimbursed access was compared to all orphan drugs approved in the same period using the EFPIA W.A.I.T. indicator.
RESULTS: • US time to access was similar regardless of regulatory pathway (i.e., ~2 weeks via expedited vs. ~5 weeks via standard pathway) • Exceptional Circumstances (n=8): o While all drugs approved under Exceptional Circumstances had completed P&R negotiations in Germany and Italy, only 50% (n=4) were available in France and Spain and 25% (n=2) in the UK (terminated NICE appraisals) o For 2 in the UK, access was substantially quicker (23 vs 60 weeks), while it was longer in Italy (89 vs 67 weeks) • CMA (n=11): o All drugs approved under CMA completed P&R negotiations in Germany, 91% in Italy, 73% in Spain, 64% in the UK, and only 36% in France o Time to access was longer in most markets (DE, IT, SP, UK) by ~14 weeks
CONCLUSIONS: In the US, access does not differ significantly based on regulatory approval pathway. In contrast, in Europe, medicines approved under Exceptional Circumstances face market availability challenges, and those with CMA experienced longer time to access. This highlights the need for early regulatory approval with to result in timely patient access, particularly in Europe.