The Central and Unacknowledged Role of the U.S. Food and Drug Administration in the Design and Execution of Medical Device Pivotal Trials

Aaron V. Kaplan; Ariel Dora Stern

Article | JAMA Cardiology | January 2018

The Central and Unacknowledged Role of the U.S. Food and Drug Administration in the Design and Execution of Medical Device Pivotal Trials

Abstract

The introduction of new medical devices has transformed cardiovascular care in recent decades. Devices, such as heart valves, pacemakers, stents, ventricular assist devices, and implantable defibrillators, have prolonged and improved the quality of life for millions of patients worldwide. Medical device innovation requires a robust ecosystem that involves medical technology innovators, often at start-ups, large medical device manufacturers, and clinical investigators. Central to this process is strong regulatory oversight, which in the United States is provided by the U.S. Food and Drug Administration’s Center for Devices and Radiologic Health (FDA/CDRH). This viewpoint discusses the effect of the regulatory approval process and the role that FDA/CDRH plays in the design and execution of the pivotal trials (clinical studies) that are used to support the regulatory approval of high-risk devices. We also review norms in the publication of pivotal trials in peer-reviewed medical journals; these publications often fail to acknowledge the role of the FDA/CRH or discuss results within the context of the device approval process.

Keywords: Health Testing and Trials; Business and Government Relations; Governing Rules, Regulations, and Reforms; Information Publishing; Medical Devices and Supplies Industry; United States;

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Citation:

Kaplan, Aaron V., and Ariel Dora Stern. "The Central and Unacknowledged Role of the U.S. Food and Drug Administration in the Design and Execution of Medical Device Pivotal Trials." JAMA Cardiology 3, no. 1 (January 2018): 5–6.

Remote patient monitoring is a subset of telehealth that involves the collection, transmission, evaluation, and communication of patient health data from electronic devices. These devices include wearable sensors, implanted equipment, and handheld instruments. During the pandemic, such monitoring programs have proven valuable. But special measures and conditions made that possible. By encouraging regulators to make permanent the temporary measures introduced during the pandemic and by following six guidelines to integrate these programs into health care, providers realize their tremendous promise.

Article | Health Affairs | July 2020

Lessons From The Impact Of Price Regulation On The Pricing Of Anticancer Drugs In Germany

Victoria D. Lauenroth, Aaron S. Kesselheim, Ameet Sarpatwari and Ariel Dora Stern

Worldwide spending on prescription drugs has increased dramatically in recent years. Although this increase has been particularly pronounced in the US, it remains largely unaddressed there. In Europe, however, different approaches to regulating drug prices have been implemented. Under the 2011 German Pharmaceutical Market Restructuring Act (Arzneimittelmarktneuordnungsgesetz, or AMNOG), for example, manufacturers freely set the prices of newly authorized drugs during their first year on the market. Benefit assessments are carried out during this year and then used in price negotiations between manufacturers and representatives of the country’s statutory health insurers. Using data on fifty-seven anticancer drugs launched in Germany from 2002 to 2017, we found that implementation of AMNOG was associated with drug prices being more closely aligned with clinical benefit. Introducing price negotiations led to a 24.5 percent decrease in negotiated prices relative to launch prices. We did not find evidence that manufacturers responded by setting higher launch prices. AMNOG is an example of how government price negotiation can be designed to better align prices with clinical benefit without delaying patient access.

Keywords: Pharmaceuticals; prescription drug costs; drug pricing; Access to care; cost reduction; Health Care and Treatment; Price; Governing Rules, Regulations, and Reforms; Cost Management; Germany;

Citation:

Lauenroth, Victoria D., Aaron S. Kesselheim, Ameet Sarpatwari, and Ariel Dora Stern. "Lessons From The Impact Of Price Regulation On The Pricing Of Anticancer Drugs In Germany." Health Affairs 39, no. 7 (July 2020): 1185–1193. View Details

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Over recent years, the adoption of connected technologies has grown dramatically, with potential for improving health care delivery, research, and patient experience. Yet, little has been documented about the prevalence and use of connected digital products (e.g., products that capture physiological and behavioral metrics) in formal clinical research. Using 18 years of data from ClinicalTrials.gov, we document substantial growth in the use of connected digital products in clinical trials (~34% CAGR) and show that these products have been used across all phases of research and by a diverse group of trial sponsors. We identify four distinct use cases for how such connected products have been integrated within clinical trial design and suggest implications for various stakeholders engaging in clinical research.

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The Central and Unacknowledged Role of the U.S. Food and Drug Administration in the Design and Execution of Medical Device Pivotal Trials

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