Sapien经导管瓣膜说明书不再推荐入路

美国食品与药品管理局(FDA)宣布，Sapien经导管心脏瓣膜的说明书中将不再提及特定入路，从而扩大了可接受这种设备治疗的患者群体。

经皮瓣膜于2011年通过核准，用于治疗被认为无法手术的重度主动脉狭窄，使其成为首个无需开放式心脏手术即可用于置换受损瓣膜的人工心脏瓣膜。2012年10月，进一步扩大核准适应证的范围，纳入无法手术的高危患者。

根据FDA于9月23日发布的声明，已核准将这一设备用于经股动脉或经心尖的方法。但目前已核准对这一设备的说明书修改，删除了对特定入路的参考。FDA和瓣膜生产商Edwards Lifesciences的发言人确认，对于无法手术和高危患者，已取消特定入路参考。

根据声明中的观点，对这一变化的核准是基于来自美国和欧洲的患者注册以及其他来源的数据。Edwards向FDA提交了在美国的经导管瓣膜治疗注册(TVTR)、在欧洲的经导管心脏瓣膜(THV)设备注册以及FDA核准的临床研究和同行评审医学杂志中获得的数据，支持修改说明书。自2012年以来，TVTR收集了在美国进行的所有经导管主动脉瓣膜置换的数据。生产商将使用这些数据，在通过不同入路接受Sapien瓣膜手术的患者中评估短期和长期结局，FDA声明中说。由美国心脏病学会(ACC)和胸外科医师协会管理的TVTR数据来自“在使用不同入路的患者中进行的数千次操作，结果显示，未见在不同入路设备性能和效益-风险对比存在差异的证据”。

“在THV进入市场用于特定患者人群后仅2年，来自TVTR的数据已被用于支持FDA核准扩大这一挽救生命的治疗的适用人群，”FDA设备与放射卫生中心主管Jeffrey Shuren博士在声明中说。“TVTR等医疗设备注册不仅在FDA的上市后监督系统中发挥重要作用，并且可以及时有力地收集数据，用于确定可以从治疗中获益的其他患者人群，”他补充道。“使用一种设备注册框架中的杠杆临床研究扩大治疗的适用患者人群，这是FDA、研究者、注册申办者和医疗设备企业工作的一种新典范，”Shuren博士指出。ACC主席John G. Harold博士在声明中说，“FDA的决策是对严格的临床注册效率的一种真实验证，这使得FDA可以迅速做出正确决策，惠及数以千计的之前无法接受这一操作的患者。”

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By: ELIZABETH MECHCATIE, Cardiology News Digital Network

The labeling for the Sapien transcatheter heart valve no longer includes references to specific access points, which will expand the group of patients who can be treated with the device, the Food and Drug Administration announced. 

Approval has been for use via a transfemoral or transapical approach. But revised labeling for the device has been approved, which removes references to specific access points, according to the FDA statement issued on Sept. 23. Spokespersons for the FDA and for Edwards Lifesciences, the manufacturer of the valve, confirmed that the references to specific access points have been dropped for both inoperable and high-risk patients.

The approval for this change was based on data from patient registries in the United States and Europe and other sources, according to the announcement.

The percutaneous valve was approved in 2011 for treating patients with severe aortic stenosis who are considered inoperable, making it the first artificial heart valve that could be used to replace a damaged valve without open heart surgery. That approval was expanded in October 2012 to include patients who are operable but at high risk.

Edwards submitted data to the FDA for the labeling change from the Transcatheter Valve Therapy Registry (TVTR) in the United States, transcatheter heart valve (THV) device registries in Europe, as well as FDA-approved clinical studies and peer-reviewed medical journals. Data from the TVTR, which is managed by the American College of Cardiology (ACC) and the Society of Thoracic Surgeons, came from "several thousand procedures performed on patients using an alternative access point, and showed no evidence that the device performs differently or has a different benefit-risk profile based on the access point," the statement said.

"Just 2 years after the THV entered the market for a specific patient population, data from the TVTR was used to support FDA approval that expands patient access to a lifesaving therapy," Dr. Jeffrey Shuren, director of the FDA’s Center for Devices and Radiological Health, said in the statement. "Medical device registries like the TVTR not only play an important role in the FDA’s postmarket surveillance system, they also collect robust and timely data that can be used to identify additional patient populations that benefit from the therapy," he added.

"Leveraging clinical research inside the framework of a device registry to expand access to therapy for more patients is a new paradigm for the FDA, researchers, registry sponsors, and the medical device industry," Dr. Shuren noted.

Since 2012, the TVTR has collected data on all transcatheter aortic valve replacements performed in the United States. The manufacturer will use these data to evaluate short- and long-term outcomes in patients who receive the Sapien valve through an alternative access site, the FDA statement said.

ACC President John G. Harold, M.D., said in a statement that "the FDA’s decision is a true testament to the efficiency of rigorous clinical registries, which allowed FDA to make a prompt decision that will impact thousands of patients who previously would have not had access to this procedure."