To mark Rare Disease Day 2024, this week we are exploring four critical elements to sustaining rare disease innovation in Europe. Today focuses on requirement #3: Supportive P&R.
Whether a medicine exists matters little for patients who cannot receive it because it is not reimbursed. Reimbursement is also key for manufacturers who rely on the sale of medicines to recover their considerable investment in development and to support their future pipeline.
Continued innovation and access in rare disease in Europe relies on country-level pricing and reimbursement (P&R) approaches that can recognise and reward promising orphan developments. But this is by no means simple since many extremely novel, transformative treatments fall foul of traditional P&R systems designed for chronic therapies in common conditions.
One example is cell and gene therapies. These unique treatments present immense value owing to their one-time, potentially curative profile. They also come with a significant price tag, complex delivery, and an inevitable degree of uncertainty around long-term benefit. In our recent paper for the Alliance for Regenerative Medicines (ARM), Dolon's Managing Director, Adam Hutchings, explores these challenges and their implications for P&R, patient access, and the sustainability of ATMP innovation in Europe. In particular, we focus on innovative contracting as an opportunity to mitigate these challenges as well as recent learnings from manufacturers’ experience in negotiating these.
P&R frameworks need to evolve with science if they are to fully reflect the value of emerging therapies to patients and health systems alike. Failure to do so will come at the expense of patients and health systems today, and Europe’s standing as a global centre for innovation in the longer term.
Read on to learn more: https://bit.ly/42UC0Uf
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