OBJECTIVES

: For potentially innovative devices and diagnostics, Breakthrough Device Designation (BDD) has been touted as a key facilitator for accelerating market access – broadly defined as U.S. Food and Drug Administration (FDA) approval and favorable coverage policy determinations by U.S. health plans. We assessed the association between BDD status and positive coverage by health plan payer stakeholders.

METHODS

: Study authors identified interventions with BDD status and U.S. regulatory approval as of December 2019 and confirmed accuracy through correspondence with FDA. Available clinical evidence for included interventions, including data submitted for FDA approval, was reviewed along with target patient distribution, coverage status, and commentary from Medicare and the top 20 commercial insurers (by number of covered lives).

RESULTS

: Eight FDA-approved interventions with BDD status were identified across 7 broad therapeutic indications – emphysema, diabetic retinopathy, iris defects, type 1 diabetes, and chronic heart failure therapies; and traumatic brain injury and solid tumor cancer diagnostics. Of these devices, 3 were widely non-covered, 4 had limited coverage, and 1 had insufficient data for analysis among commercial insurers; in contrast, Medicare and its regional contractors explicitly covered 1 of these devices, with no formal policy for the remaining devices. Non-coverage rationale stated by health plan policies was primarily insufficiently compelling evidence of clinical efficacy. A prospective analysis of these products, assuming retroactive coverage aligned with H.R. 5333 (116^th Congress; 2019-2020) or similar legislation, determined that during a 3-year effective period, market access would increase by 55% or more, driven primarily by the distribution of Medicare beneficiaries across target patient populations.

CONCLUSIONS

: Breakthrough Device Designation, while potentially valuable to expedite U.S. regulatory clearance, does not directly correlate with coverage by payer stakeholders. While pending legislative efforts may offer guaranteed, temporary Medicare coverage, innovators must pair these initiatives with additional, payer-resonant clinical evidence to secure widespread market access.