Incentives for Pharmaceutical Companies to Develop Treatments for Rare Diseases: A Review of the Literature

Background: Numerous patients suffering from orphan diseases still lack a treatment. Pharmaceutical companies play a crucial role in the advancement of orphan drug development. This systematic literature review aims to identify and categorise current incentivising factors for pharmaceutical companies to develop orphan drugs. Methods: EMBASE and MEDLINE databases were systematically searched for terms related to orphan drug development incentives for pharmaceutical companies. Research findings were qualitatively evaluated and categorised into themes. Results: 752 publications were found in the initial search. Full-text review was performed on 64 publications and 14 publications were included in the final systematic review. Incentivising factors were split into four categories: Regulatory factors in the USA, regulatory factors in the EU, clinical trials and patient voice. Conclusion: Patient voice is a crucial factor in engaging and incentivising pharmaceutical companies in orphan drug development. Numerous policies are currently in place across both USA and EU targeting different stages of orphan drug research. Further qualitative data is required to evaluate stakeholder views on policy effectiveness.