FDA期望使伴随诊断、实验室检测变得更可靠

美国食品药品管理局（FDA）7月份宣布了推进个体化医疗的最新步骤。

首先，FDA最终确定了伴随诊断产品开发指南。其次，FDA释放出了旨在对某些实验室自建检测项目（IDT）开始监管的信号。所谓LDT，是指由单一实验室设计、生产和使用的检测项目。

该指南作为草案最初于2011年7月发布，表明了FDA对搭配诊断产品开发前景方面的观点。FDA官员称，最终指南与草案没有实质性改变。

FDA局长Margaret Hamburg博士在电话新闻发布会上说道：“当制药企业开发仅针对特殊少数人群的药品时，这些药品与FDA确认准确可靠、患者和医生可以信赖的诊断检测方法同时获批非常重要。”

FDA还在致国会函件中透露了如何监管LDT的初步想法。FDA需要在公布有关草案和最终指南前60天向国会提交有关议案。即将面世的草案体现了FDA在如何监管LDT方面的改变。

“FDA在过去对LDT实行自由裁量，即意味着FDA通常不对这些检测实行监管要求，因为通常认为这些检测风险较低，且仅在特定机构有限范围内使用，”Hamburg博士说道。“目前，LDT或许上市，应用更为广泛，与FDA批准的检测方法竞争但缺少支持其应用的临床研究。更深层次的担心是，医生和患者在做出关键医疗决策时所依据的诊断检测通常未经FDA审评。未经FDA上市前审评，患者、医生或FDA本身不能保证这些检测方法的准确性和可靠性。”

Hamburg博士指出，FDA计划对高风险LDT实施上市前审评要求，“包括用于确定药物治疗的LDT和目前已上市的具有同样用途的FDA批准和FDA许可的伴随诊断产品。”

FDA的行动获得了癌症研究人员的赞赏。

“FDA最新公告的目的是，使患者和医生相信这些诊断检测所提供的非常复杂的分子和基因信息具有较高水平的可信性和确定性，”美国癌症研究协会（AACR）主席Carlos L. Arteaga博士在声明中说道。“作为代表整个研究环节的组织，从实验室到临床，包括临床研究人员和从事癌症患者治疗的医生，AACR期望与FDA继续合作，以确保医生和患者使用的分子与基因诊断检测建立在坚实科学证据支持的基础上。”

许多医学界，特别是肿瘤学界人士正在考虑如何最好地利用个体化治疗作为实现最佳治疗效果和控制医疗费用的手段。

爱思唯尔版权所有未经授权请勿转载

By: GREGORY TWACHTMAN, Cardiology News Digital Network

Aug 14, 2014

Last month, the Food and Drug Administration announced the latest steps it is taking to advance the use of personalized medicine.

First, the agency finalized guidance on the development of companion diagnostics. Second, it signaled its intent to begin oversight of certain laboratory developed tests (LDTs), tests that are designed, manufactured, and used within a single laboratory.

The guidance, initially issued as a draft in July 2011, offers the agency’s thinking on its expectations on the development of companion diagnostics. No substantive changes were made to the final guidance document from its draft version, agency officials said.

"When drug companies develop therapies that will only work on specific subpopulations, it’s important those therapies are approved with a diagnostic test that the FDA knows is accurate and reliable and patients and their physicians can count on," FDA Commissioner Dr. Margaret Hamburg said during a press teleconference.

The agency also revealed its initial thoughts on how it will regulate LDTs in a letter to Congress. The FDA is required to let Congress know 60 days prior to the issuance of draft and final guidance. The forthcoming draft represents a change in how the agency is approaching LDTs.

"The FDA has historically exercised enforcement discretion over LDTs, meaning the agency generally did not enforce applicable regulatory requirements on the tests because they were generally considered lower risk and used on a limited basis within a given institution," Dr. Hamburg said. "Today, they may be marketed and used more broadly and compete with FDA-approved tests without clinical studies to support their use. The deeper concern is about physicians and patients making critical health decisions based on diagnostic tests that have generally not been reviewed by the FDA. Without premarket review by the agency, patients, their health care providers, or the FDA itself cannot be assured those tests are accurate and reliable."

Dr. Hamburg said the agency plans to enforce premarket review requirements for higher-risk LDTs, "including those used to determine medical treatment and that have the same intended use of FDA-approved and FDA-cleared companion diagnostics currently on the market."

The agency’s actions received praise from cancer researchers.

"The recent announcements by the FDA are aimed at providing patients and their physicians with an important level of confidence and certainty with regard to the highly complex molecular and genetic information that these diagnostic tests are determining," American Association for Cancer Research President Dr. Carlos L. Arteaga said in a statement. "As an organization that represents the entire continuum of research, from the laboratory to the clinic, including the clinical researchers and physician-scientists engaged in cancer patient care, the AACR looks forward to continuing to engage with the FDA to ensure that the molecular and genetic diagnostic tests that are being utilized by physicians and patients are based on solidly supported scientific evidence."

Many in the health care community, particularly those in the oncology community, are looking at how best to use personalized medicine as a means of achieving better treatment outcomes and controlling costs of health care.