Work

Stimulating development of innovative medicines in the European Union: does a new definition for unmet medical need add value?

Year: 2024

Type: review

Abstract: • The 2023 EU pharmaceutical legislation proposal redefines ’unmet medical need’ • Flexibility, feasibility, fairness, and sensitivity to risk are not fully addressed. • A more high-level definition would be preferred while allowing further specification. • Redefining unmet medical need alone may not resolve the root causes of barriers to innovation. • Narrowing the regulatory–HTA/payer gap is essential to improve access to innovative medicines. The European Commission (EC) has proposed to redefine ‘unmet medical need’ to steer pharmaceutical innovation and link the definition to incentives and regulatory tools and procedures. A multistakeholder meeting of the Regulatory Science Network Netherlands (RSNN) discussed the impact of this proposal. Four principles were highlighted (flexibility, feasibility, fairness, and sensitivity to risk) that were not considered sufficiently addressed. Concerns were raised about whether innovation would be incentivised, given the diverse and complex reasons why areas of unmet medical need may remain unaddressed. A more high-level definition of unmet medical need that gives guidance but allows for specification for specific uses throughout the medicine lifecycle was generally preferred. As the legislative process unfolds, stakeholder engagement will be essential to refine the definition to achieve the intended policy goals.

Source: Drug Discovery Today

DOI: 10.1016/j.drudis.2024.104251