美国参议员对FDA渐进式器械批准提案感到“失望”

11月4日，美国参议员Elizabeth Warren （D-MA）和Patty Murray （D-WA）在一封致FDA官员的信中表示，他们对FDA重新针对某些医疗器械实行渐进式审批制度感到担忧。

这封信是写给FDA代理局长Brett Giroir和医疗器械与放射健康中心（CDRH）主任Jeffrey Shuren的。鉴于此前FDA前局长Scott Gottlieb于2018年告诉参议员，有条件的批准途径不适用于人用医疗产品，参议员在信中对FDA在器械渐进式批准方面的态度大转变提出质疑。

虽然器械的渐进式批准已经讨论了多年，但FDA首次正式提出这一途径是在其2020财年预算说明中。根据该提议，某些器械在证明其安全性和性能以及采取其他风险缓解措施的情况下，可获得临时批准上市销售，但在规定的时间期限后，申请人需根据上市后数据证明产品能够合理保证其安全性和有效性，然后才能继续在市场上销售这类器械产品。

针对这一提议，Warren和Murray于6月份致函Shuren和时任FDA代理局长的Ned Sharpless，该提案“令人担忧”，同时指出，“从书面上看，该项"临时批准"与前局长Gottlieb向国会和公众保证FDA不会寻求的“有条件批准”几乎没有区别。

负责立法事务的FDA副局长Karas Gross在8月份向参议员们做出回应，解释说：“FDA对医疗器械的看法在不断演变。”

Gross写道:“这一途径旨在帮助人们获得能够治疗或诊断威胁生命或不可逆转的衰弱性疾病和状况的医疗器械，并帮助解决尚未满足的医疗需求，特别是对儿童和其他长期缺医少药的少数人群。”

对此，Warren和Murray表示，他们对FDA的有关说法感到失望，同时还对FDA设想的渐进式批准资格标准以及用于完全批准该途径下器械的上市后数据的“质量和完整性”感到担忧。

此外，参议员们还质疑，如果在临时批准期限到期后，FDA无法证明临时批准的器械能够合理保证其安全性和有效性，那么该机构能够在多大程度上将这些器械从市场上撤出。

In a letter to top US Food and Drug Administration (FDA) officials on Monday, Sens. Elizabeth Warren (D-MA) and Patty Murray (D-WA) say they are concerned about the agency’s renewed interest in a progressive approval system for certain medical devices.

The letter, addressed to Acting FDA Commissioner Brett Giroir and Centerfor Devices and Radiological Health (CDRH) Director Jeffrey Shuren, questions the agency’s about-face on progressive approvals for devices after former FDA Commissioner Scott Gottlieb told senators in 2018 that a conditional approval pathway would not be suitable for human medical products.

While progressive approvals for devices have been discussed for years, FDA first officially proposed the pathway in its FY2020 budget justification. Under the proposal, certain devices could be marketed under a provisional approval based on a demonstration of safety and performance in addition to other risk mitigations for a three-year period. Afterward, the sponsor would be required to demonstrate a reasonable assurance of safety and effectiveness based on postmarket data to continue marketing the device.

In response to the proposal, Warren and Murray sent a letter to Shuren and then-Acting FDA Commissioner Ned Sharpless in June calling the proposal "alarming" and noting that "as written, this 'provisional approval' seems hardly distinguishable from the 'conditional approval' that former-Commissioner Gottlieb had assured Congress and the public that the FDA would not pursue."

FDA Associate Commissioner for Legislative Affairs Karas Gross responded to the senators in August, explaining that, "The agency's views with respect to medical devices have continued to evolve."

"This pathway is intended to help enable access to medical devices which treat or diagnose life-threatening or irreversibly debilitating diseases and conditions and address unmet medical needs, particularly for children and other small and rare populations that remain perpetually underserved," Gross wrote.

In response, Warren and Murray say they are "disappointed by FDA's clarification" and continue to have concerns about the agency’s envisioned eligibility criteria for progressive approval and the "quality and completeness" of the postmarket data that would be used to fully approve devices under the pathway.

The senators also question how well the agency would be able to remove provisionally approved devices from the market if they are unable to demonstrate a reasonable assurance of safety and effectiveness after the provisional approval period expires.