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.

基于基因的疗法正在改变罕见病患者的医学格局。这些一次性的、潜在的治疗方法对美国的医疗保健模式提出了挑战，在这种模式下，高昂的价格和保险范围的变化可能会阻止患者接受改变生活的疗法。当患者、支付者和政策制定者对治疗的价值和不确定性持有不同看法时，就会出现对高价的质疑。对患者来说，关键是高价需要合理化，公司需要与患者真诚合作。公司不应自动假设“基因治疗”标签和少量患者人群的结合证明了高价是合理的。为了加快开发过程并潜在地降低成本，患者希望行业通过共享数据来提高临床试验效率，包括自然史研究和失败的试验结果。从患者的角度来看，当前的价值评估框架对残疾人不利，可能无法准确反映患者和社会的观点，并忽略了对护理人员的影响、生产力损失和创新的未来价值等必要因素。价值确定方法需要为行业、付款人、监管机构，尤其是患者——需要公平和可持续地获得改变生活的基因疗法的勇敢先驱提供公平的激励和结果。以及创新的未来价值。价值确定方法需要为行业、付款人、监管机构，尤其是患者——需要公平和可持续地获得改变生活的基因疗法的勇敢先驱提供公平的激励和结果。以及创新的未来价值。价值确定方法需要为行业、付款人、监管机构，尤其是患者——需要公平和可持续地获得改变生活的基因疗法的勇敢先驱提供公平的激励和结果。