OBJECTIVES:

Orphan Medicinal Products (OMPs) for rare diseases undergo health technology appraisals (HTA) by the National Institute for Health and Care Excellence (NICE). Drugs for ‘very rare’ conditions undergo further extensive Highly Specialized Technology (HST) reviews, to establish the overall magnitude of their therapeutic benefits, cost effectiveness, and budget impact. On April 1, 2017, NICE published an incremental cost-effectiveness ratio (ICER) threshold for OMPs set at £100,000 per quality-adjusted-life-year (QALY) gained, in addition to a QALY weighting dependent on the QALY gain offered by the new treatment. The objective of this analysis was to investigate the OMPs previously recommended by NICE and assess to what extent they meet this new guidance.

METHODS:

Currently, 96 drugs possess European Medicines Agency (EMA) orphan designation and market authorization. This list has been reviewed to establish which OMPs have acquired NICE recommendations. All publicly available ICER data were collated from NICE consultation, appraisal and committee papers between January 2007 and June 2017.

RESULTS:

As of June 2017, a total of 33 EMA authorized OMPs have undergone a standard HTA appraisal, while 7 have been reviewed via the HST process. Of those appraised, 27 ICER’s have been published. NICE recommended 27 OMPs; twelve of these involved Patient Access Schemes (PAS) of which three (therapeutic areas: pulmonary and musculoskeletal/nervous system) had ICERs greater than the £100,000 threshold. The majority of the products approved before April 2017 involved a PAS, making it unclear to which level the ICER was reduced to allow for the product to receive a positive opinion from NICE.

CONCLUSIONS:

Considering the pubically available information, the majority of OMP drugs recommended before April 2017, fell within the threshold of £100,000. For the exceptions, applying the new guidelines on the QALY weighting resulted in ICERs unlikely to fall within the thresholds adjusted for therapeutic benefit.