Securing approval and reimbursement for new rare disease treatment is often one of the most challenging and frustrating parts of treatment development. It is also part of the process where many patient organisations are asked to provide their expert voice, opinion, and data to support the assessment process. With a potential therapy tantalisingly close, this can be a daunting prospect for many patient advocates, and the pressure on their shoulders can feel overwhelming.
In this RareChat we would like to explore your experience of engaging in the regulatory and reimbursement processes for new rare disease therapies.
This RareChat will be a great space to share ideas and get inspiration for the future from your fellow patient group leaders!