Sanket S Dhruva1,2, Jonathan J Darrow3,4, Aaron S Kesselheim3, Rita F Redberg5,6. 1. Philip R. Lee Institute for Health Policy Studies, University of California, San Francisco, San Francisco, CA, USA. sanket.dhruva@ucsf.edu. 2. Section of Cardiology, Department of Medicine, San Francisco Veterans Affairs Medical Center, San Francisco, CA, USA. sanket.dhruva@ucsf.edu. 3. Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA. 4. Department of Law and Taxation, Bentley University, Waltham, MA, USA. 5. Philip R. Lee Institute for Health Policy Studies, University of California, San Francisco, San Francisco, CA, USA. 6. Division of Cardiology, Department of Medicine, University of California, San Francisco School of Medicine, San Francisco, CA, USA.
Abstract
BACKGROUND: Drugs and high-risk medical devices are increasingly likely to receive Food and Drug Administration (FDA) approval through expedited pathways, which has implications for informed treatment consent (i.e., consent in clinical practice). OBJECTIVE: To obtain expert opinion about the clinical and ethical implications of the increasing availability of new drugs and devices approved through expedited development and regulatory review pathways. DESIGN: Qualitative study using individual semi-structured videoconference interviews. PARTICIPANTS: National leaders in medicine, ethics, and law (n=12) with expertise in medical product regulation, payor policymaking, bioethics, physician practice, patient advocacy, public health expertise/advocacy, clinical trials, the pharmaceutical and device industry, institutional review board oversight, and real-world evidence. MAIN MEASURES: Principal themes in 3 domains: expedited regulatory pathways, physician and patient understanding of and reliance on FDA approval, and informed treatment consent. KEY RESULTS: Respondents pointed out that more common use of expedited pathways translates to increased reliance on surrogate measures, some with uncertain clinical significance. While expedited development and review can have advantages, participants expressed worry that physicians were unaware when medical products were expedited and did not communicate about uncertainties in knowledge about new drug or device approvals effectively with patients. Many participants felt that informed treatment consent discussions about new drugs or devices should include some explanations of expedited pathways and use of surrogate measures. CONCLUSIONS: Experts identified advantages of expediting development and of FDA flexibility in applying its standards to new drugs and medical devices, but highlighted concerns that patients may not be adequately informed about the risks of shorter review times or about uncertainties in the evidence that result. There is a need to identify approaches to ensure effective clinical use of drugs and devices when approved through expedited pathways.
BACKGROUND: Drugs and high-risk medical devices are increasingly likely to receive Food and Drug Administration (FDA) approval through expedited pathways, which has implications for informed treatment consent (i.e., consent in clinical practice). OBJECTIVE: To obtain expert opinion about the clinical and ethical implications of the increasing availability of new drugs and devices approved through expedited development and regulatory review pathways. DESIGN: Qualitative study using individual semi-structured videoconference interviews. PARTICIPANTS: National leaders in medicine, ethics, and law (n=12) with expertise in medical product regulation, payor policymaking, bioethics, physician practice, patient advocacy, public health expertise/advocacy, clinical trials, the pharmaceutical and device industry, institutional review board oversight, and real-world evidence. MAIN MEASURES: Principal themes in 3 domains: expedited regulatory pathways, physician and patient understanding of and reliance on FDA approval, and informed treatment consent. KEY RESULTS: Respondents pointed out that more common use of expedited pathways translates to increased reliance on surrogate measures, some with uncertain clinical significance. While expedited development and review can have advantages, participants expressed worry that physicians were unaware when medical products were expedited and did not communicate about uncertainties in knowledge about new drug or device approvals effectively with patients. Many participants felt that informed treatment consent discussions about new drugs or devices should include some explanations of expedited pathways and use of surrogate measures. CONCLUSIONS: Experts identified advantages of expediting development and of FDA flexibility in applying its standards to new drugs and medical devices, but highlighted concerns that patients may not be adequately informed about the risks of shorter review times or about uncertainties in the evidence that result. There is a need to identify approaches to ensure effective clinical use of drugs and devices when approved through expedited pathways.
Authors: Aaron S Kesselheim; Steven Woloshin; Zhigang Lu; Frazer A Tessema; Kathryn M Ross; Lisa M Schwartz Journal: JAMA Intern Med Date: 2019-05-01 Impact factor: 21.873