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Rare diseases

Companies developing rare disease treatments face several unique challenges when it comes to regulatory and reimbursement submissions.

The most pressing of these include the paucity of information on patient populations, disease progression and appropriate Clinical Outcome Assessments (COAs) as well as the challenges associated with collecting appropriate evidence in rare diseases. Alongside this is determining the needs for HTA and payer bodies to ensure a positive value assessment and reimbursement.

As well as these challenges, there are fundamental issues for clinical trial design that are magnified in rare diseases such as determining appropriate sample sizes and comparators and generating evidence to support selected endpoints.

On 22 March this PMLiVE webinar, in association with Adelphi Values, will use real-world examples to highlight the importance of evidence such as appropriate endpoints, including COAs, and comparators in rare diseases to support regulatory and reimbursement submissions. 

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