Early Access To Medicines Pathways – Results Of A Global Survey

Abstract

Objectives: Regulators and HTA bodies are increasingly promoting early access to medicines for the treatment of diseases with unmet clinical need. A variety of different early access pathways have emerged across settings. A global survey was undertaken to identify, map, and differentiate early access pathways. MethOds: Two survey tools were developed to map early access pathways across 25 countries. The first tool included 28 questions on different types of accelerated marketing authorisation, and was distributed to a selection of contacts from a number of regulatory bodies (n= 12) . The second tool included 15 questions on the use of medicines prior to marketing authorization (MA) and was distributed to HTA agencies from a number of countries (n= 25). Both survey tools were available in English via Qualtrics from May 2017. Results: Preliminary results from the survey (n= 17) suggest that early access schemes can be grouped in four different pathways: a) MA with faster review time (n= 3), b) MA with lower evidence thresholds (n= 2), c) MA based on approval in other settings (n= 2), and d) access to medicines prior to MA (n= 14). Five regulatory bodies (EMA, FDA, MEDSAFE, PMDA, and HSA) reported some type of accelerated MA procedure. Fourteen countries