Appraisal of NICE’s cost-effectiveness thresholds for assessing orphan drugs

Abstract

A rare disease is one that affects less than 1 in 2,000 in general population, and they are often chronic and life-threatening. Due to the high cost to develop and small market for orphan drugs, historically manufacturers are reluctantly to explore the rare disease therapeutic areas. There is highly unmet need from patients with rare diseases. As the pharmaceutical expenditure rises, governments around the world are increasingly under pressure to contain the costs on drug reimbursement. Meanwhile patients with rare diseases need to be ensured for access to orphan drugs, and the industry needs to be incentivized to innovate. Health technology assessment and reimbursement bodies set out measures and policies for orphan drugs to be given market access. In this literature review, the landscape of health technology appraisals for orphan drugs in England UK is studied. The process for an orphan drug to gain marketing authorisation in the European Union (EU), the methods and criteria that used by National Institute for Health and Care Excellence (NICE) are reviewed. All orphan drugs with marketing authorisations from European Medicine Agency (EMA) are searched from the agency’s online database, whereas each of the authorised orphan drug is searched in NICE’s database for any technology appraisals and recommendations made for the orphan drug. The search results are then analysed to find factors that may influence NICE’s decisions on whether the drug should be considered cost-effective. Based on the analyses, suggestions are made for NICE to potentially improve its way of assessing orphan drugs.