OBJECTIVES

: The European Medicines Agency (EMA) published an overview of the EU’s orphan designation program in 2017, implying its success in encouraging the development of therapies for the 30 million people in the EU that suffer from one of over 6,000 rare diseases (defined as a prevalence of <5 in 10,000 people). Since 2000, over 1,900 medicines have received an EMA orphan designation, with over 140 having received marketing authorization in the EU. However, for patients to access these therapies also need to be publically reimbursed, typically requiring a positive recommendation from a national Health Technology Assessment (HTA) body. This research investigates HTA appraisal outcomes of orphan drugs.

METHODS

: Publically-available data from EMA, National Institute of Health and Care Excellence (NICE), Scottish Medicines Consortium (SMC), Haute Autorité de Santé (HAS), and Gemeinsame-Bundesausschuss (G-BA) were screened up to 30 September 2017.

RESULTS

: There are 100 currently approved orphan drugs. In Germany, all orphan drugs receive automatic positive rating by the G-BA (i.e. additional benefit) qualifying them for the price negotiations and potential price premiums. In France, HAS has given positive recommendations (Amelioration du Service Medical Rendu I-III) to 37% (26/70) of orphan drugs appraised. In the UK, NICE and SMC have given positive recommendations (defined as ‘recommended’/’accepted’ or ‘conditional’/’optimized’) to 9% (3/35) and 12% (8/66) of orphan drugs appraised, respectively. Not only are fractions of approved medicines being reimbursed, there are substantial delays between regulatory approval and HTA decisions, average delay by HAS, NICE and SMC are 14, 24 and 15 months respectively.

CONCLUSIONS

: Orphan designation may appear to be a numerically successful scheme to incentivize drug development. However, only around 7% of orphan designations translate into market authorizations. Further, there are significant additional barriers in translating regulatory approval into successful reimbursement. Arguably, the substantial unmet treatment need for rare diseases remains.