Enabling, not rewarding: How to design a high unmet medical needs category to drive, not deter, innovation in orphan medicinal products
Enabling, not rewarding: How to design a high unmet medical needs category to drive, not deter, innovation in orphan medicinal products
The Draghi report’s warnings and the EU’s response
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 incentivise 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 increases overall knowledge in the area and allows transformative treatment to be developed. When incentives for incremental treatments are reduced, the pathway to transformative treatments weaken. 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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Related work
European Expert Group on Orphan Drug Incentives
Related links
AUTHORS
Yann Le Cam, EURORDIS
Simone Boselli, EURORDIS
Christian Jervelund, Copenhagen Economics
Malwina Mejer, Copenhagen Economics
Colin O’Donnell, Alexion, AstraZeneca Rare Disease
Matthias Heck, Alexion, AstraZeneca Rare Disease
Luana Banu, Takeda
Toon Digneffe, Takeda
This research was conducted by CE, as the knowledge partner, EURORDIS, Takeda, and Alexion, AstraZeneca Rare Disease in collaboration with Orphanet.