Evans Barbara J
Health Law & Policy Institute, USA.
Am J Law Med. 2012;38(4):577-606. doi: 10.1177/009885881203800401.
In 2007, Congress granted the Food and Drug Administration (FDA) new powers to order pharmaceutical companies to conduct drug safety studies and clinical trials in the postmarketing period after drugs are approved The methodologies include observational studies that examine patients' insurance claims data and clinical records to infer whether drugs are safe in actual clinical practice. Such studies offer a valuable tool for improving drug safety, but they raise ethical and privacy concerns because they would entail widespread use of patients' health information in commercial research by drug manufacturers. This is the first article to explore the ethics of these section 505(0)(3) observational studies, so named after the section of the Food, Drug, and Cosmetic Act that authorizes them. Data access problems threaten to make the FDA's section 505(0)(3) study requirements unenforceable. Under existing federal privacy regulations, it appears highly unlikely that pharmaceutical companies will have reliable access to crucial data resources, such as insurance claims data and healthcare records, to use in these studies. State privacy laws present another potential barrier to data access. If pharmaceutical companies do manage to gain access to the needed data, this will raise serious privacy concerns because section 505(0)(3) observational studies do not appear to be covered by any of the major federal regulations that afford ethical and privacy protections to persons whose data are used in research. If the FDA's program of section 505(o)(3) observational studies fails because of the above problems, this failure will have a number of bad consequences: the public will be exposed to avoidable drug safety risks; taxpayers may be forced to bear the costs of having the FDA conduct drug safety investigations that would have been funded by drug manufacturers if data had been available; and, perhaps most troubling, the FDA may be forced to order postmarketing clinical trials to answer questions that could have been answered using observational studies. Problems with access to data for section 505(0)(3) studies thus could directly imperil human research subjects by forcing a needless over-reliance on risky postmarketing drug safety trials. This Article concludes by describing a promising new legal pathway for resolving these problems. Congress has provided the FDA a new set of powers that if skillfully exercised will allow the agency: (1) to facilitate pharmaceutical companies' appropriate access to data for use in section 505(0)(3) observational studies, (2) to impose strict ethical and privacy protections for persons whose data are used in these studies, and (3) to mobilize private-sector funding to generate much-needed evidence of the safety of FDA-approved drugs.
2007年，美国国会授予食品药品监督管理局（FDA）新的权力，命令制药公司在药品获批后的上市后阶段开展药物安全性研究和临床试验。这些方法包括观察性研究，即通过审查患者的保险理赔数据和临床记录，来推断药物在实际临床实践中是否安全。此类研究为提高药物安全性提供了一种有价值的工具，但也引发了伦理和隐私方面的担忧，因为这将导致制药公司在商业研究中广泛使用患者的健康信息。本文是第一篇探讨第505(o)(3)条观察性研究伦理问题的文章，该研究以《食品、药品和化妆品法案》中授权此类研究的条款命名。数据获取问题可能会导致FDA的第505(o)(3)条研究要求无法执行。根据现行联邦隐私法规，制药公司极不可能可靠地获取关键数据资源，如保险理赔数据和医疗记录，用于这些研究。州隐私法是数据获取的另一个潜在障碍。如果制药公司确实设法获取了所需数据，这将引发严重的隐私问题，因为第505(o)(3)条观察性研究似乎未被任何主要联邦法规涵盖，而这些法规为研究中使用其数据的人员提供伦理和隐私保护。如果FDA的第505(o)(3)条观察性研究计划因上述问题而失败，这一失败将产生一系列不良后果：公众将面临可避免的药物安全风险；纳税人可能被迫承担FDA开展药物安全调查的费用，而如果有数据的话，这些费用本应由制药公司承担；也许最麻烦的是，FDA可能被迫下令开展上市后临床试验，以回答本可通过观察性研究回答的问题。因此，第505(o)(3)条研究的数据获取问题可能会直接危及人体研究受试者，因为这会导致不必要地过度依赖有风险的上市后药物安全性试验。本文最后描述了一条有望解决这些问题的新法律途径。国会已赋予FDA一系列新权力，如果巧妙行使这些权力，将使该机构能够：（1）促进制药公司适当获取用于第505(o)(3)条观察性研究的数据；（2）对研究中使用其数据的人员实施严格的伦理和隐私保护；（3）调动私营部门资金，以生成FDA批准药物安全性急需的证据。
