An analysis of compassionate access programmes for novel oncology drugs.

Background: Despite the rise in the number of approved novel oncology drugs, just over half of all new cancer medicines approved by the EMA between 2017 and 2021 were granted reimbursement in Ireland by the HSE. Compassionate access programmes (CAPs) are a means of providing managed access to drugs which are of proven benefit but have not yet received full approval for reimbursement by the state, or where the requested indication has not been yet been authorised/licensed.

Methods: A retrospective review was performed of patients attending The Mater hospital for treatment of advanced malignancy who availed of a CAP between August 2012 and July 2022. Clinical data collected included disease type, treatment received, duration of treatment received, and best response to treatment. To categorize outcome "Clinical Benefit" was defined as a radiological complete, partial, or stable disease response to treatment.

Results: One hundred and thirteen patients were included in the study. Ninety-three received at least one dose of CAP treatment. Treatment duration ranged from 0 to 112 months, with 12 patients on treatment for ≥ 2 years. N = 47 (42%) experienced a Clinical Benefit. Of these, N = 7 experienced a complete response [CR]. Thirty patients (27%) did not receive a planned treatment or died within 3 months of treatment.

Conclusions: In this review of a decade of CAPs at our institution we observed that a significant proportion of patients derived a clinical benefit from CAP treatment. Unfortunately, however, a significant proportion of patients did not receive a planned treatment due to disease progression or died within 3 months of treatment suggesting availability came too late. While CAPs can provide meaningful benefit, they are not a substitute for timely approval of novel agents.