Ethical Issues in Prescribing Drugs to Older Adults for Whom Representative Randomized Trial Data Is Lacking

老年人在随机对照试验中往往代表性不足，甚至被排除在外 （RCT）导致FDA批准新药，但对于许多药物，他们代表了大多数患者 在批准后接受常规护理的药物。药物在试验中显示出一定的结果， 年轻人群可能对老年人没有类似的效果，这种使用也可能使患者 未被充分代表的老年组出现意外副作用的风险增加。当前R 01补助金 （“了解新药在进入市场后不久对老年人的有效性”）评估了一个框架， 对于FDA批准的新药在老年人中的有效性， 成年人了这一补充申请增加了R 01赠款与生物伦理学研究审查， 当导致FDA批准的RCT不包括 这些病人。 我们将探讨在不同人群中使用药物的生物伦理学意义， 招募到他们的关键RCT，包括需要加强披露作为知情同意的一部分 程序和监管变化，使批准的条件是在真实的- 世界患者群体。这些目标将通过与老年患者进行焦点小组来实现 和开处方者的半结构化访谈。根据这些研究工作的结果，我们将 制定法规和临床实践建议，以促进患者自主权和知情权 决策。

Older adults are often underrepresented in or even excluded from the randomized controlled trials (RCTs) leading to FDA approval of new drugs, but for many drugs they then represent the majority of patients receiving the drug in routine care after approval. Drugs demonstrated to have certain outcomes in trials of younger populations may not have similar effectiveness in older adults, and such use may also put patients in underrepresented older age groups at increased risk for unexpected side effects. The current R01 grant (“Understanding effectiveness of new drugs in older adults shortly after market entry”) evaluates a framework for developing more timely and robust insight regarding the effectiveness of new FDA-approved drugs in older adults. This supplemental application augments the R01 grant with bioethics research examining the issues in prescribing drugs for older patients when RCTs leading to FDA approval did not include those patients. We will explore the bioethical implications of the use of drugs in populations different from those recruited into their pivotal RCTs, including the need for enhanced disclosure as part of informed consent procedures and regulatory changes that would make approvals conditional on evidence generated in real- world patient populations. These goals will be pursued by conducting focus groups with older adult patients and semi-structured interviews with prescribers. Based on the results of these research efforts, we will formulate regulatory and clinical practice recommendations to promote patient autonomy and informed decision making.