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.

Chapter | Innovation Policy and the Economy, Volume 19 | 2018

The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century

Nicholas Bagley, Benjamin Berger, Amitabh Chandra, Craig Garthwaite and Ariel Dora Stern

On the 35th anniversary of the adoption of the Orphan Drug Act (ODA), we describe the enormous changes in the markets for therapies for rare diseases that have emerged over recent decades. The most prominent example is the fact that the profit-maximizing price of new orphan drugs appears to be greater today than it was in 1983. All else being equal, this should reduce the threshold for research and development (R&D) investment in an economically viable product. Further, the small size of patient populations for orphan drugs, together with the increasing prevalence of biologics among orphan drugs, have created a set of natural monopoly-like markets in which firms face little competition, even after the end of formal periods of patent protection and market exclusivity. Additionally, the evolving technologies of drug development—in particular, the increasingly common use of auxiliary endpoints in clinical trials and the use of biomarkers for patient selection for treatment—now allow manufacturers to target smaller populations. Taken together, these changes raise doubts about whether the ODA encourages the development of products that otherwise would not have been brought to market—or whether, instead, it simply rewards the producers of inframarginal products. After presenting empirical support for our claims of an evolving marketplace, we discuss the tradeoffs associated with reshaping the ODA for the 21st century.

Keywords: Health Care and Treatment; Laws and Statutes; Research and Development; Investment; Markets; Monopoly;

Citation:

Bagley, Nicholas, Benjamin Berger, Amitabh Chandra, Craig Garthwaite, and Ariel Dora Stern. "The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century." Chap. 4 in Innovation Policy and the Economy, Volume 19, edited by Josh Lerner and Scott Stern, 97–137. University of Chicago Press, 2018. View Details

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This study sheds new light on first- and early-mover advantages. Research on this classic topic often assumes that each firm participates in the entirety of the innovation process and that all firms aim to monetize their innovations on product markets. However, a division of labor between innovative new entrants and industry incumbents, endowed with complementary assets, is common in many industries. Such settings are distinct because new entrants have the additional option to sell their innovation in a “market for technology” and may, therefore, seek acquisition rather than shepherding an innovation through the entire commercialization process. We argue that this binary outcome—i.e., success via acquisition—creates different opportunities and threats for new entrants and has important and novel implications for the following question: is it advantageous to be early to market? Using data from the U.S. medical device industry, we find that pioneer (dis-)advantages in a market for technology setting are similar to those typically seen in product markets but different in some important respects. In particular, pioneers must pave the way for a new product type in order to reduce the technological and market risks, where reducing technological risk is of paramount importance. As a reward, pioneers ultimately realize a higher likelihood of acquisition, but among acquired firms, early entrants wait longer for acquisition to happen. To a certain extent, therefore, later movers can free ride on early-movers’ efforts: although they are less likely to experience acquisition, acquisitions of later entrants happen more quickly.

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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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