While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant... View More
This ISPOR Europe panel explored the evolving landscape of CAR T therapies, their high potential for lifelong benefits, and the economic and logistical hurdles they face. From complex manufacturing to unique pricing and reimbursement challenges, CAR T therapies require novel... View More
On March 12th 2024, Richard Sear and Emilie Neez explored alternative pricing approaches for orphan medicines in a presentation at the World Evidence Pricing and Access Congress in Amsterdam. Supporting slides are available here; this companion piece provides our (the... View More
While the revision of the General Pharmaceutical Legislation (GPL) targets EU-level frameworks and processes, proposed changes are sure to have immediate as well as downstream consequences at the country level. This paper explores these potential consequences, focusing on a selection... View More
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.
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