Rare diseases: Regulatory and reimbursement submissions, the challenges

Presented by

Dominic Tyer

About this talk

Companies developing new rare disease treatments face several unique challenges when it comes to their regulatory and reimbursement submissions. One of the most pressing of these is the paucity of information on patient populations, disease progression and appropriate Clinical Outcome Assessments (COAs), which form an important part of assessing signs and symptoms of rare diseases and treatment response.

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