Author:
Aartsma-Rus Annemieke,Dooms Marc,Le Cam Yann
Abstract
Today policy makers face the challenge to devise a policy framework that improves orphan medicinal product (OMP) development by creating incentives to deliver treatments where there are none and to authorize innovative and transformative treatments where treatments already exist. The European Expert Group on Orphan Drug Incentives (hereafter, OD Expert Group) came together in 2020 to develop policy proposals to facilitate EU policy makers to meet this challenge. The group brings together representatives of the broad rare disease community, including researchers, academia, patient representatives, members of the investor community, rare disease companies and trade associations. The group’s work builds on the recognition that only an ambitious policy agenda developed in a multi-stakeholder setting can bring about the quantum leap needed to address unmet needs of rare disease patients today. Along the OMP development path, the OD Expert Group has identified four main needs that a policy revision should address: 1) Need to improve the R&D ecosystem for basic research and company take-up of development. 2) Need to improve the system of financial incentives and rewards. 3) Need to improve the flexibility, predictability and speed of the regulatory pathway. 4) Need to improve the coherence and predictability of demand and pricing for OMPs. This article presents the results of the OD Expert Group work as a set of guiding principles that the revision of the policy framework should follow and a set of 14 policy proposals that address the main needs of OMP development in Europe today.
Subject
Pharmacology (medical),Pharmacology
Reference39 articles.
1. Pharmaceutical Probability of Success 2018
Alacrita
2018
2. Estimated Impact of EU Orphan Regulation on Incentives for Innovation
Dolon
2020
3. Compounded Medication for Patients with Rare Diseases. Orphanet;Dooms;J. Rare Dis.,2018
4. Declaration on Good Off-Label Use Practice
DoomsM.
GoodwinG.
van der ZandenT. M.
de WildtS. N.
2018
5. EU Commits $1bn to Global Fund;Dorozynski;BMJ,2003
Cited by
16 articles.
订阅此论文施引文献
订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献