Enabling, not rewarding: How to design a high unmet medical needs category to drive, not deter, innovation in orphan medicinal products

In light of the European Commission’s recent proposal to revamp the incentive system for orphan medicinal product (OMP) development, we delve into the implications of the new High Unmet Medical Need (HUMN) category, which aims to bring transformative treatments for rare diseases that currently lack effective therapies. The introduction of the HUMN category represents a paradigm shift in how we incentivize OMP development. From today’s system, where incentives make the investment case for innovation in OMPs possible, to a reward-based system that introduces a separate category for HUMN treatments reducing incentives anywhere else.

While the reward-based approach may seem attractive, we show that transformative treatments tend to emerge as the results of many incrementally innovative treatments that serve to build up the knowledge base that allows transformative treatment to be developed. When incentives for incremental treatments are reduced, the pathway to transformative treatments weakens. This may in fact lead to fewer transformative treatments being developed, thereby working against the ambitions of the Commission.

Click on the link to read our publication, co-authored by EURORDIS, Copenhagen Economics, Alexion AstraZeneca Rare Disease, Takeda, and in collaboration with Orphanet.

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European Expert Group on Orphan Drug Incentives Orphan medicine incentives