OBJECTIVES : Patient access to orphan drugs is complex, given their high cost. The number of orphan drugs achieving European market authorisation has increased in the last decade, which, combined with rising drug prices, has led to greater payer scrutiny. Here, we review market authorisation and HTA decisions for orphan drugs in England and consider strategies to improve patient access.

METHODS : EMA orphan designated drugs granted European market authorisation between 2014-2018 were identified from the Orphanet lists of medicinal products for rare diseases in Europe (April 2019). The National Institute for Health and Care Excellence (NICE) website was used to extract HTA status and decisions.

RESULTS : Seventy-six orphan drugs with EMA orphan designation received European market authorisation from 2014-2018. Of these, 5 received market authorisation for more than one indication, totalling 81 orphan indications for the period. NICE appraised 50% of these orphan drugs. Positive reimbursement decisions were made for 30/81 indications. Of these, 10 were recommended without restriction, 9 were recommended with restrictions, 4 were recommended within the Cancer Drug Fund (CDF), and 7 were recommended within the CDF with restrictions. HTA appraisals are proposed for mexiletine HCl and pitosilant which received market authorisation in 2016 and 2018, respectively. Five drugs which received market authorisation between April 2017 and August 2018 are currently undergoing HTA appraisal, with expected publication dates between June 2019 and August 2020.

CONCLUSIONS : Market authorisation for orphan drugs is often received considerably in advance of HTA submission (approximately 2 years for NICE). Earlier HTA submissions for orphan drugs could reduce the time from market authorisation to HTA decisions and accelerate patient access. This process could be facilitated by HTA agencies implementing or increasing conditional access agreements pending company submission of further trial data.