In recognition of Rare Disease Day 2024, we explore four critical elements to sustaining rare disease innovation in Europe. These elements are outlined below, building on published work by Dolon team members.
Categories for Dolon Institute
Revision of the General Pharmaceutical Legislation: Impact Assessment of European Commission and EFPIA proposals
The European Commission has proposed revisions to the Pharmaceutical Legislation, with the view to tackle unmet medical needs, competitiveness, access and affordability. This update represents a once-in-a-generation opportunity to strengthen the European biopharmaceutical ecosystem, if fit-for-purpose policy options are implemented... View More
Addressing remaining unmet needs in rare diseases hinges on a positive and enduring policy environment which helps to achieve the necessary clinical and economic conditions for drug development in small, hard-to-treat populations. This white paper examines through historic case analysis... View More
Revision of the Orphan Regulation: Estimated impact on incentives for innovation of changes proposed by the European Commission
Despite the tremendous therapeutic advances delivered in rare diseases since the adoption of the Orphan Regulation in 2000, the European Commission has proposed a set of legislative revisions aimed at bolstering innovation whilst enhancing access and affordability. This study, based... View More
Amyotrophic Lateral Sclerosis, a rare neurodegenerative disease: European landscape assessment and policy recommendations for improved diagnosis, care, and treatment.
The European Amyotrophic Lateral Sclerosis (ALS) Coalition has published a paper that sets forth 10 policy recommendations aiming to improve the lives of people living with ALS and their caregivers. The paper sheds light on what it means to receive... View More
The past 5 years have been a period of great innovation for cell and gene therapy with a wave of very exciting and effective therapies approved in Europe. While the clinical potential of these products are great, there are recognised... View More
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.