OBJECTIVES: 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.

METHODS: Our scope is France, Germany, the United Kingdom (UK), and the United States (US). We consider drugs launched from 2014 to 2021. We select 15 ODs and match to them 15 NODs. Matching variables include launch year, trial endpoints, effect sizes, and competitive context. We divide the markets into those with formal OD provisions in drug value assessment (i.e., Germany and the UK), and those without (i.e., France and the US), and compare prices of ODs and NODs in competitive context.

RESULTS: We reject the hypothesis that OD provisions in value assessment confer manufacturer benefits in terms of access, reimbursement, or evidence expectations.

CONCLUSIONS: Regulatory, evidence evaluation and tax incentives may encourage OD development where it would be neglected otherwise. However, OD provisions in value assessment confer no additional benefits in the access and pricing process compared to non-OD treatment evaluations. This is relevant to current policy debates in countries such as Germany, where there is increasing discussion about eliminating special OD provisions in HTA.