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ICYMI: Gene therapy provides hope for Huntington’s Disease patients

Lauren Holder, Huntington’s Disease Patient Advocate and member of the Institute for Gene Therapy Patient Advocacy Advisory Council, recently penned an op-ed in The Hill on the importance of patient access to gene therapy.

As Lauren says, "There are however significant questions about how accessible these treatments will be, especially given current Centers for Medicare & Medicaid Services CMS coverage restrictions for Alzheimer’s treatments, and even the recent prior authorization decision by United Healthcare for colonoscopies. Gene therapies require enormous investments in research and, because they are life-saving treatments that may only require one dose, they present unique challenges in valuation and reimbursement. These challenges not only impact patient access to treatments once they’re FDA approved but also the research and development environment for additional new treatments, especially treatments that aim to address an unmet medical need."

"Fortunately, there are many stakeholders in this effort, and that’s where Congress comes in. Bipartisan legislation called the Medicaid VBPs for Patients Act, or the MVP Act (H.R. 2666) has been introduced in the U.S. House of Representatives. This legislation would position value-based purchasing approaches as one solution to put these therapies within the reach of many patients. Under a value-based purchasing structure, payment for a new gene therapy would be tied to the effectiveness of the treatment and would help mitigate the considerable cost involved for health care payers, creating greater access for patients."

To read the full piece, click here.


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