EURORDIS has published a position paper on patient access to rare disease therapies entitled “Breaking the Access Deadlock to Leave No One Behind”.
Due to major scientific advances and a favourable legislative environment, there has been a dramatic increase in the number of rare disease therapies developed in the last decade, however, there is a need to improve and widen access for patients to orphan medicines. In order to tackle the challenges that rare diseases patients’ currently face in order to have access to care and medicines, a four-pillar approach is proposed:
1. A new blueprint to cut costs and fast-track R&D;
2. Early dialogue and cooperation between healthcare systems on the determination of value of a medicine and on patient access;
3. A transparent European cooperation framework between national healthcare systems for the determination of fair prices and of sustainable healthcare budget impacts; and
4. A continuum approach to evidence generation linked to healthcare budget spending.
Through the recommendations in this access paper, and the collaboration of all stakeholders involved, EURORDIS and its members hope to acheive their ambition of having 3 to 5 times more new rare disease therapies approved per year by 2025, 3 to 5 times cheaper than today.
To read the full position paper click here