Comparison of health technology assessments and time to reimbursement for orphan drugs

Comparison of health technology assessments and time to reimbursement for orphan drugs

To encourage manufacturers to invest in the development of orphan drugs (ODs), various incentives have been introduced including research and development grants, tax incentives, accelerated regulatory processes and 10-year market exclusivity. However, ODs still need to overcome a series of challenges posed by health technology assessment (HTA) bodies, which are unique in each European market. ODs can often launch with highly uncertain evidence packages which may include immature Phase II single arm trial data with a small patient number.

In this article, Steven Kelly, Charlotte Poon, Nimisha Raj, and Ioanna Stefani review and compare the reimbursement recommendations for ODs issued by four of the most well-established European HTA bodies (France, Germany, England and Scotland) and assess the potential effect of HTA outcomes in relation to time to reimbursement (T2R). 

To read the article published in European Pharmaceutical Review, click here.