PNS96 Orphan Drug Compassionate Use Programmes: Scale of Use and Association with Regulatory and Reimbursement Decisions in France, Germany and the UK

Abstract

To understand the scale of use of compassionate use programmes (CUPs) for orphan products in France, Germany and the UK, and whether these programmes affect regulatory and reimbursement decisions. Websites of regulatory authorities from France (Agence Nationale de Sécurité du Médicament et des Produits de Santé), Germany (Bundesinstitut für Arzneimittel und Medizinprodukte, and the Paul-Ehrlich-Institut) and the UK (Medicines and Healthcare products Regulatory Agency) were searched for cohort CUPs in June 2020. Drugs with European Medicines Agency (EMA) Orphan Drugs Designation (ODD) in the indication of the CUP were identified using the Community Register of orphan medicinal products. Searches of the EMA website confirmed the marketing authorisation (MA) status of products. Between January 2010 and June 2020, 1,348 EMA ODDs were awarded; 17, 5 and 4 CUPs for orphan products were approved in France, Germany and the UK, respectively. In total, 19/26 (73%) of the identified CUPs have so far preceded successful MA. In France, 14/17 products obtained MA, 2/17 products had not yet received a MA decision and 1/17 products had applications withdrawn prior to a decision. In Germany, 1/5 products obtained MA, and 4/5 products had not yet received a decision. All products obtained MA in the UK. The average length of time between CUP start date and MA was 310 days (range -4 to 1,326 days). Further research is ongoing to assess the association of CUPs with reimbursement decisions. Only a small proportion of products with ODDs had been awarded CUPs, with notably more in France than in Germany and the UK, suggesting greater obstacles to establishing CUPs may exist in Germany and the UK. The majority of orphan products with a CUP have been successful in gaining MA, suggesting they may offer a benefit with respect to regulatory decision making.