Genetic-based therapies: looking ahead to ensuring access to a cure for cystic fibrosis

Recent scientific advancements have accelerated research for a cure for cystic fibrosis (CF). Research is underway for genetic-based therapies such as gene therapy, gene editing, and RNA therapy. However, great optimism is countered by concerns about how and if patients will have access to a future cure. As health care decisionmakers look to solutions for paying for and ensuring access to curative therapies, they should: integrate patient preferences into new payment mechanisms for a cure; design insurance benefits to incentivize highly effective therapies; and ensure equity.

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