The Dolon Institute aspires to contribute to the policy conversations around patient access and promoting innovation in rare and severe diseases. Our work is grounded in the fundamentals of orphan medicine economics and experience engaging with stakeholders around the launch of orphan medicines. We seek to develop analyses and evidence-led insights disseminated through peer-reviewed publications, white papers and external events.
Revision of the Orphan Regulation: Estimated impact on incentives for innovation of changes proposed by the European Commission
Emilie Neez, Adam Hutchings
September 2023
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…
Read more…
Amyotrophic Lateral Sclerosis, a rare neurodegenerative disease: European landscape assessment and policy recommendations for improved diagnosis, care, and treatment.
Kate Quigley, Gisela Rovira Tomas, Elena Nicod
September 2023
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…
Read more…
Innovative contracting for ATMPs in Europe: Recent learnings from the manufacturer experience
Adam Hutchings
August 2023
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…
Read more…Explore Dolon Institute publications here