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

White, W.; Gene Ther (2019) doi:10.1038/s41434-019-0110-7

Gene-based therapies are changing the landscape of medicine for patients with rare diseases. Companies should not automatically assume that the combination of the “gene therapy” label and a small patient population justifies high prices. 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.