Healthcare Economist February 9, 2024
Jason Shafrin

That is the topic of a recent white paper from the Innovation and Value Initiative titled “Valuing Rare Disease Treatments in Healthcare: Real Experience, Real Impact“. The report notes a number of challenges in assessing treatment value in rare disease (see my white paper “Challenges in Preserving Access to Orphan Drugs Under an HTA Framework” for a more detailed discussion of this topic). For instance, there is often a lack of consensus on the on the outcomes that need to be assessed in clinical studies, in part due to the small size of the patient population. Further, because these diseases are rare, any real world data is likely to have–by definition–a very small sample size.

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