An analysis of compassionate access programmes for novel oncology drugs

• DOI: 10.1007/s11845-025-03930-7
• Publication Date: 2025-03-22T20:47:45Z
• Authors: Sarah A. Kelly; Karine E. Ronan; Mohammed Zameer; Jennifer Brown; Grainne Johnston; Ruth Adams; Dearbhla Murphy; Deirdre Kelly; Waseem Darwish; John McCaffery; Geraldine O’Sulli...; Emily Harrold; Shahid Iqbal; Darren Cowzer; Austin G. Duffy
• Keywords: Compassionate Use; Reimbursement

Abstract Background Despite the rise in number of approved novel oncology drugs, just over half all new cancer medicines by EMA between 2017 and 2021 were granted reimbursement Ireland HSE. Compassionate access programmes (CAPs) are a means providing managed to drugs which proven benefit but have not yet received full approval for state, or where requested indication has been authorised/licensed. Methods A retrospective review was performed patients attending The Mater hospital treatment advanced malignancy who availed CAP August 2012 July 2022. Clinical data collected included disease type, received, duration best response treatment. To categorize outcome “Clinical Benefit” defined as radiological complete, partial, stable Results One hundred thirteen study. Ninety-three at least one dose Treatment ranged from 0 112 months, with 12 on ≥ 2 years. N = 47 (42%) experienced Benefit. Of these, 7 complete [CR]. Thirty (27%) did receive planned died within 3 months Conclusions In this decade CAPs our institution we observed that significant proportion derived clinical Unfortunately, however, due progression suggesting availability came too late. While can provide meaningful benefit, they substitute timely agents.