PHP275 - ORPHAN MEDICINAL PRODUCTS GIVEN AN INITIAL NEGATIVE RECOMMENDATION BY NICE'S STA PROCESS BUT APPROVED FOLLOWING RE-EVALUATION IN ENGLAND

Abstract

OMPs present a challenge to HTA bodies in that there may be high levels of uncertainty in their clinical and economic data due to the inherent limitations in their trial size. This study assessed the factors that influence overturning initial negative recommendations for OMPs assessed through STA. Products for evaluation through STA between 2011 and 2017 were identified through STA topics published on NICE’s website, and filtered for topics that had not been recommended. The list of topics that had received a negative recommendation was then cross referenced with products and indications that were granted Orphan status by the EMA. The published guidance for each OMP granted a positive recommendation following a re-review were analysed to assess the changes to the submission that may have influenced the change in recommendation, either submission of new evidence, provision of a Patient Access Scheme, or definition of a new patient population. Of the 254 technologies considered between 2011 and 2017, 38 topics were granted an initial negative recommendation, as the technology was not considered to be an appropriate use of NHS resources based on the data available. Of the 38 topics 6 were OMPs, and 4 were recommended following re-review and updated guidance. In two of the re-evaluations additional data was provided by the company, Patient Access Schemes were applied in all 4 cases, and narrowing of the patient population was applied in all 4 cases. This study provides evidence of the measures that have been taken to overturn a negative STA recommendation for OMPs reviewed through STA process in England. Where clinical evidence is scarce due to the nature of the therapy, companies must accept discounts and subgroups in the patient population in order to be given a positive recommendation in the event of a re-review.