Leading healthcare industry consulting firm Health Advances today published a position paper in collaboration with the Partnership to Improve Patient Care and the EveryLife Foundation for Rare Diseases. The paper, entitled “Value to Whom? Incorporating Patient Perspectives into Value Assessment for Novel Cell and Gene Therapies”, examines the critical importance of value assessors incorporating patient experience data into healthcare decision making.
The paper recognizes that as the science of patient input has transformed the clinical trial and regulatory landscape, the access environment remains a ‘next frontier’. Since patients do not yet have a proverbial seat at the access partner table, decisions are made to inform payer decision-making first – patients and providers secondarily.
According to the paper, “These shortcomings have the potential to lead to very real consequences for patients in the form of restricted and delayed access to novel therapies. It is therefore essential that value assessors incorporate patient experience data more fully into their analyses, a movement known as patient focused drug development (PFDD) that has already gained traction in other medical research communities.”
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About the EveryLife Foundation for Rare Diseases
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.
A disease is defined as rare when it affects fewer than 200,000 people in the United States. On average, rare disease patients must wait an average of six years after symptoms first present before receiving a proper diagnosis. Ninety-three percent of the 7,000 known rare diseases have no U.S. Food and Drug Administration-approved therapies. Fifty percent of rare disease patients are children, thirty percent of whom will not live to see their fifth birthdays.
