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Paul Melmeyer: Value Assessments Need to Take Into Account Unique Characteristics of a Disease

It is incredibly important for these assessments to consider the context of the therapy, rather than just the clinical benefit that it brings, explained Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders.
 


It is incredibly important for these assessments to consider the context of the therapy, rather than just the clinical benefit that it brings, explained Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders.

Transcript

How important is it for value assessments to take into account the unique factors of a disease state?

It is incredibly important for these assessments to consider the context of the therapy, rather than just the clinical benefit that it brings. Of course the clinical benefit is incredibly important and it is still going to form the core of the actual value assessment. But, the context for which that therapy is actually being delivered is important because if a patient is perhaps receiving the very first therapy for their disease that they’ve ever been able to actually obtain, that is so much more important than perhaps a patient receiving a therapy for the first time but perhaps it’s the eight therapy or the ninth therapy for that disease. That first therapy is monumental. It’s revolutionary for the patient, as well as their families, their caregivers, really everyone who cares about them. So, that context is incredibly important to consider as health technology bodies are assessing the value of these therapies.

 
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