Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.
JAMA Intern Med. 2023 Sep 1;183(9):999-1004. doi: 10.1001/jamainternmed.2023.3961.
When the US Food and Drug Administration (FDA) approves a drug or medical device on the basis of limited clinical evidence, the Centers for Medicare & Medicaid Services (CMS) must decide whether the therapy is "reasonable and necessary" for coverage among Medicare beneficiaries. However, the legal underpinnings of CMS's authority to shape coverage of FDA-regulated products under Medicare Part B are controversial. To clarify this area, we reviewed relevant legal precedents on CMS's approaches to limit coverage and recent decisions Medicare has issued affecting coverage for FDA-regulated products.
The CMS continues to exercise considerable legal discretion to limit coverage of FDA-authorized products to only uses it determines are reasonable and necessary for patients with Medicare. Courts have upheld this discretion repeatedly, emphasizing the difference between Medicare's coverage criteria and the FDA's review standards. As more new drugs and devices come to market without solid evidence of efficacy on clinical outcomes, or have narrow benefit-risk considerations, CMS may increasingly rely on forms of limited or conditional coverage, including coverage with evidence development (CED), which provides reimbursement only in the context of a clinical trial or registry.
The ability of CMS to condition or limit coverage of FDA-approved products is a commonsense necessity for this crucial taxpayer-funded program. Although courts have thus far deferred to the authority of CMS to make such decisions on the basis of its clear statutory discretion and public health expertise, Congress may want to act to reaffirm statutory language giving CMS sufficient flexibility to craft coverage determinations that reflect the evidence for a product's use.
当美国食品和药物管理局 (FDA) 根据有限的临床证据批准一种药物或医疗器械时，医疗保险和医疗补助服务中心 (CMS) 必须决定该疗法是否“合理且必要”，以便 Medicare 受益人获得覆盖。然而，CMS 有权根据 Medicare 第 B 部分对 FDA 监管产品进行覆盖的法律依据存在争议。为了澄清这一领域，我们审查了 CMS 限制覆盖范围的相关法律先例以及 Medicare 最近发布的影响 FDA 监管产品覆盖范围的决定。
CMS 继续行使相当大的法律酌处权，将 FDA 授权产品的覆盖范围限制在 CMS 确定对 Medicare 患者合理且必要的用途上。法院一再支持这种酌处权，强调了 Medicare 的覆盖标准与 FDA 的审查标准之间的区别。随着越来越多的新药和设备在没有临床结果疗效确凿证据或受益风险考虑狭窄的情况下投放市场，CMS 可能会越来越多地依赖有限或有条件的覆盖形式，包括有证据开发的覆盖 (CED)，仅在临床试验或注册的背景下提供报销。
CMS 有条件或限制 FDA 批准产品覆盖范围的能力是这个关键的纳税人资助计划的必要条件。尽管迄今为止，法院一直尊重 CMS 根据其明确的法定酌处权和公共卫生专业知识做出此类决定的权力，但国会可能希望采取行动，重申赋予 CMS 足够灵活性的法定语言，以制定反映产品使用证据的覆盖范围决定。
