OP19 Are Compassionate Use Programmes Good Predictors of Clinical Benefit?

Abstract

Introduction:In cases of high unmet clinical need, patients can access drugs prior to marketing authorization (MA) and Health Technology Assessment (HTA) through compassionate use programmes (CUP) or special access pathways (SAP). In theory, accelerated access is beneficial for patients with few therapeutic alternatives. In practice, it remains unclear if early access products actually deliver meaningful clinical benefit.Methods:Seventy-five drug-indication pairs were identified that have proceeded through a CUP or SAP in one or more countries including Canada, Australia, France, Sweden, England, and Scotland. Data was collected from regulatory and HTA websites on length of CUP or SAP, time prior to MA, time prior to HTA decision, time between MA and HTA decision, French Transparency Commission added clinical benefit (ASMR), and HTA decision. Cohen kappa scores were calculated in order to assess inter-agency agreement.Results:Across the 75 drug-indication pairs, average time between CUP and marketing authorization was 243 days, and average time between MA and HTA decision was 252 days. No products were deemed to be of major added clinical benefit (ASMR I), only 2.7 percent of products had important added clinical benefit (ASMR II), 26.7 percent of products had moderate added clinical benefit (ASMR III), 40.0 percent of products had minor added clinical benefit (ASMR IV), and 22.7 percent of products had no added clinical benefit (ASMR V). There is little inter-agency agreement in HTA recommendations for products that have proceeded through a CUP. The highest amount of agreement was seen between Canada and Scotland (k = 0.24).Conclusions:Preliminary results suggest that CUP and SAP products accelerate access, but often only provide only moderate or minor improvements in clinical benefit. Further, there is very little agreement across HTA agencies on the value of these products.