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Sickle Cell Gene Therapy: Medicaid Needs Solutions For High Upfront Costs Soon

Executive Summary

With gene therapies for sickle cell disease looming and two new expensive therapies of to a slow start, access challenges could prevent a large portion of patients from receiving treatment, a report from the US National Academies of Sciences, Engineering and Medicine concludes. The report urges development of new payment models, offering five ideas.

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Sickle Cell Gene Therapy May Need New US-Backed Payment Program, Grogan Suggests

Former senior advisor to President Trump warns that failure to reform drug pricing practices has seriously handicapped prospects for reimbursing cell and gene therapy under existing US payer programs.

Value-Based Contracts Are Evolving

Value-based contracting in the US is moving toward high-cost drugs for rare disease and away from lower cost chronic treatments as payers plan for the coming pipeline of cell and gene therapies. Marketed gene therapies all have some kind of risk sharing deal in place but further progress on the most innovative payment models is hampered by regulatory and operational challenges. The Centers for Medicare and Medicaid Services has taken a first step toward addressing regulatory obstacles but there is a long way to go.

Medicaid’s Multiple Best Price Proposal Needs More Clarity Before Advancing, PhRMA Argues

Medicaid should go back to the drawing board on a key part of its proposal aimed at circumventing best price reporting obstacles to value-based purchasing.

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