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A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies


Gene-based therapies are changing the landscape of medicine for patients with rare diseases. These one-time, potentially curative treatments pose a challenge in the US healthcare model, where high prices and insurance coverage variation may prevent patients from receiving life-altering therapies. Questioning of high prices occurs when patients, payers, and policy makers hold divergent views on the value and uncertainty of therapies. The key for patients is that high prices need to be justified, and companies need to partner authentically with patients. Companies should not automatically assume that the combination of the “gene therapy” label and a small patient population justifies high prices. To speed up the development process and potentially reduce costs, patients want the industry to improve clinical trial efficiency by sharing data, including natural history studies and failed trial results. From the patient perspective, current value assessment frameworks disadvantage people with disabilities, may not accurately reflect patients’ and societal views, and omit necessary factors such as impact on caregivers, lost productivity, and the future value of innovation. Value determination methods need to provide fair incentives and outcomes to industry, payers, regulators, and especially patients—the courageous pioneers who need equitable and sustainable access to life-changing gene-based therapies.

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The author thanks Carl Erickson, Pat Furlong, Jayne Gershkowitz, Eric Grinstead, Marie Lamont, Doug Paul, Mary Pittman, Ted Slocomb, Beth White, and Alex Wyke for their invaluable contributions, support, and review of this article and Karin Horler for editing an early version of the paper.

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Correspondence to Wendy White.

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Wendy White has received compensation as a consultant and employee of Vitrisa Therapeutics. She is also a member of TREND community advisory board. She owns small amounts (<1%) of stock in Amicus Therapeutics, Rocket Therapeutics, Abeona Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics. She is currently chair of the board of Global Genes and was previously director-at-large of National Organization for Rare Disorders (NORD). She is also the parent of a child with a rare connective tissue disorder with no available therapy.

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White, W. A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies. Gene Ther 27, 474–481 (2020).

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