OBJECTIVES: To assess the affect of Orphan designation on reimbursement

Currently 1,696 orphan drugs registered in the EU, only a very small percentage have been granted EMA marketing authorisation and subsequent reimbursement. Orphan drug status in the EU is intended to provide an incentive to bring drugs to market for rare diseases that would otherwise not justify investment. Is it failing to meet this objective?

METHODS: A retrospective study of data from EMA Orphan designation, EMA Marketing authorisation data, NICE and NHS reimbursement information (June 2020) was compared.

RESULTS: Of the 1,696 currently designated orphan drugs, less than 8% have been granted MA (132) and only 4.8% (82) have been reimbursed by NICE or funded for use by NHS England. There is a lengthy lag between designation, MA and reimbursement. Typically, of those that achieve MA in the EU it takes on average of just over 5 years from designation to approval (quickest being 12 weeks and the longest 17.5 years). The 82 reimbursed orphan drugs in England are made up primarily of treatments for rare cancers (36.6%), a selection of rare hereditary disease (31.7%) and treatments for ocular conditions (4.9%), the remaining 26.8% covering a range of rare conditions. England reimburse 62% of orphan drugs that get EMA MA status on average within 2 years but have taken as up to 7 years for some indications.

CONCLUSIONS: A paucity of patient data provides a challenge in demonstrating the efficacy, safety and value of products to achieve MA and reimbursement. The paradox of a framework to encourage therapies for rare diseases verses ever constrained NHS budgets and difficulties faced in demonstrating orphan drugs value has yet to be solved, ultimately meaning patients suffering with these conditions are potentially not receiving life changing therapies in a timely fashion or in many cases at all.