HTA24 Orphan Drugs: What Is the Impact of Special Provisions in Value Assessment?

Abstract

Favourable regulatory, evidence evaluation and tax regulations for orphan drugs (ODs) in the US and Europe aim to encourage OD development to fill clinical unmet needs and facilitate access to ODs. Manufacturers have responded positively to these incentives, measured by the number of OD launches since 1982 – when the US Orphan Drug Act became the first OD law in the world. More recently, HTA agencies and US payers have begun to expect evidence as solid for ODs as they expect for non-ODs (NODs). In this research, we test the hypothesis that OD provisions in value assessment confer manufacturer benefits in HTA and pricing.