Dolon sponsored World EPA Congress 2023 and had the opportunity to present at the conference. In this infographic, we provide a summary of key discussion points from the roundtable ‘Paid early access in Europe – Is it needed, and what... View More
In recognition of Rare Disease Day 2023, we explore four key reasons why rare diseases warrant special treatment. These reasons are summarised below, building on published work by Dolon team members.
Dolon is pleased to announce the launch of the Dolon Institute, which aspires to contribute to the policy discussion that surrounds the development and availability of innovative medicines for rare and severe diseases globally. We hope to do our part... View More
This paper brings together a multi-disciplinary panel of expert co-authors who share perspectives on the current challenges, implications and potential solutions for absence of comparative survival data in health technology assessment (HTA) of oncology therapies.
Gene therapies are revolutionising the treatment of severe conditions, offering previously unattainable advances in health gains. Simultaneously, this therapeutic potential is matched by concerns around price and affordability. Are these fears reflective of reality? To address this question requires an... View More
Although the Orphan and Paediatric Regulations have been successful at stimulating innovation in these previously underserved areas, significant unmet medical needs remain. EFPIA held in May 2021 a workshop, which gathered Senior staff from member company R&D, Clinical, Strategy, and... View More
The divergence between the rapid pace of scientific progress in rare diseases and declining sentiment towards orphan medicines reflects a paradox that is at the heart of the European orphan drug debate. While the last twenty years have witnessed a... View More
The European Commission is currently reviewing the 2000 Regulation on orphan medicinal products (OMP). It is critical that any consideration of reform of the Regulation should be informed by a robust understanding of the relationship between incentives, investment, innovation and... View More
Gene and cell therapies represent some of the most advanced treatments being employed in biomedical research to improve the length and quality of life of patients with rare and complex diseases. Patients’ access to these therapies has been hampered by... View More