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

向缺乏代表性随机试验数据的老年人开药的伦理问题

基本信息

批准号：
10366434
负责人：
Shirley Wang
金额：
$ 17.84万
依托单位：
BRIGHAM AND WOMEN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2018
资助国家：
美国
起止时间：
2018-08-01 至 2023-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10366434
关键词：
Address Anticoagulants Attitude Awareness Beneficence Benefits and Risks Bioethics Clinical Clinical Trials Complement Complex Computer software Data Decision Making Detection Disclosure Drug Approval Drug Labeling Drug Prescriptions Drug usage Effectiveness Elderly Ethical Issues Ethics FDA approved Focus Groups Foundations Goals Grant Healthcare Informed Consent Interview Justice Knowledge Label Modification New Drug Approvals Oral Outcome Parents Patients Persons Pharmaceutical Preparations Physicians Population Population Sizes Procedures Process Provider Randomized Controlled Trials Recommendation Regulation Research Risk Safety Signal Transduction Structure Time Uncertainty age group base clinical practice cost improved insight interest novel novel therapeutics older patient patient population patient registry randomized trial recruit response routine care shared decision making side effect study population treatment research

项目摘要

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.

老年人通常在随机对照试验中的代表性不足甚至不包括在内（RCT）导致FDA批准新药，但对于许多药物，它们代表了大多数患者批准后接受常规护理中的药物。证明在试验中具有某些结果的药物年轻的人群在老年人中可能没有相似的效力，这种使用也可能使患者进入代表性不足的老年人组增加了意外副作用的风险。当前的R01赠款（“在市场进入后不久了解新药在老年人中的有效性”）评估一个框架用于开发有关新旧药物在较旧的FDA批准药物的有效性的更及时，更强大的见解成年人。此补充应用程序通过生物伦理学研究增加了R01赠款，研究了当导致FDA批准的RCT不包括时，针对老年患者的药物问题那些患者。我们将探讨在种群中使用药物的生物伦理意义招募到其关键RCT中，包括作为知情同意书的一部分进行增强披露的需求程序和监管变更将使批准以实现的证据为条件世界患者人群。这些目标将通过与老年人患者进行焦点小组来实现以及针对处方者的半结构化访谈。根据这些研究工作的结果，我们将正式的监管和临床实践建议，以促进患者自主权并告知患者决策。

项目成果

期刊论文数量（13）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

A Framework for Visualizing Study Designs and Data Observability in Electronic Health Record Data.

DOI：
10.2147/clep.s358583
发表时间：
2022
期刊：
Clinical epidemiology
影响因子：
3.9
作者：
通讯作者：

DESIGN DIFFERENCES EXPLAIN VARIATION IN RESULTS BETWEEN RANDOMIZED TRIALS AND THEIR NON-RANDOMIZED EMULATIONS.

设计差异解释了随机试验与其非随机模拟之间结果的差异。

DOI：
10.1101/2023.07.13.23292601
发表时间：
2023
期刊：
medRxiv : the preprint server for health sciences
影响因子：
0
作者：
Heyard,Rachel;Held,Leonhard;Schneeweiss,Sebastian;Wang,ShirleyV
通讯作者：
Wang,ShirleyV

HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force.

DOI：
10.1002/pds.5507
发表时间：
2023-01
期刊：
Pharmacoepidemiology and drug safety
影响因子：
2.6
作者：
通讯作者：

Simulation for Predicting Effectiveness and Safety of New Cardiovascular Drugs in Routine Care Populations.

预测新心血管药物在常规护理人群中的有效性和安全性的模拟。

DOI：
10.1002/cpt.1045
发表时间：
2018
期刊：
Clinical pharmacology and therapeutics
影响因子：
6.7
作者：
Najafzadeh,Mehdi;Schneeweiss,Sebastian;Choudhry,NiteeshK;Wang,ShirleyV;Gagne,JoshuaJ
通讯作者：
Gagne,JoshuaJ

Reproducibility of real-world evidence studies using clinical practice data to inform regulatory and coverage decisions.