Date of Award

January 2022

Document Type


Degree Name

Medical Doctor (MD)



First Advisor

Joseph S. Ross


US Congress authorized the US Food and Drug Administration (FDA) Breakthrough Devices Program (BDP) in December 2016 in an effort to expedite patient access to innovative devices indicated for the diagnosis and treatment of serious illnesses1. Since program inception, the BDP has grown at a rapid and increasing pace—as of January 1st, 2020, the FDA had granted breakthrough designation to 222 devices, representing nearly 200% program growth over the past year. To shorten device development and review times, the FDA has approved breakthrough devices based on less rigorous premarket evidence with the intention of collecting complementary data on safety and effectiveness postmarket, as the device becomes more routinely used. For example, the FDA has approved breakthrough devices without supporting effectiveness data (e.g., an insulin pump) or with significant safety risks (e.g., pneumothorax rates > 30%).

Even after FDA approval, because these devices tend to be priced more expensively than comparable diagnostic or therapeutic products, adoption tends to lag either because insurance plans do not cover outpatient use of the breakthrough device or because their costs exceed the DRG payments to hospitals to reimburse inpatient care. In August 2019, the US Centers for Medicare and Medicaid Services (CMS) finalized changes to a rule easing eligibility requirements for breakthrough devices to qualify for new technology add-on payments (NTAPs) in order to increase hospital reimbursement and thereby promote their clinical adoption.2 In August 2020, CMS proposed a novel rule establishing a pathway, the Medicare Coverage of Innovative Technology (MCIT) pathway, that would grant breakthrough devices four years of national Medicare coverage upon their approval by the FDA.3 Although the rule was finalized in January 2021, its implementation was delayed until March 2021 and the rule was ultimately rescinded in November 2021. Members of US Congress from both political parties have pushed to revive the MCIT, either as a new rule from CMS or through new legislation.

As BDP devices have begun to enter the market, patients, physicians, and policymakers should understand key features of the BDP and their implications for clinical decision-making. Herein, we detail early FDA experience with the BDP, including research characterizing premarket evidentiary standards and postmarket requirements, and offer strategies to guide policy and practice moving forward. Furthermore, we will examine different payment mechanisms to allow for patient access to breakthrough devices while still ensuring evidence generation and patient protection. To strengthen regulation of breakthrough devices, we argue that the FDA should collaborate with regulatory counterparts—particularly those in the European Union where devices may be commercially available before FDA approval—to leverage real-world postmarket experience. Additionally, the FDA and CMS should coordinate clinical study requirements for breakthrough devices to facilitate Medicare coverage determinations and support clinical decision-making. Finally, CMS should consider leveraging existing coverage pathways, such as the Coverage with Evidence Development pathway, to increase access to novel devices while still generating important data on device safety and effectiveness. Such measures may help ensure that the BDP helps deliver safe and effective devices to patients with unmet clinical needs.


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