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Wake Forest University School of Law




This symposium article examines the use of investigational (un-approved) medical products in the United States, with particular focus on who pays for this use. In the United States, the question of who pays for the use of approved medical products for their intended indications is complicated enough, with some expenses borne by private payers, some by public payers, some covered as charity care, and some paid out of pocket by patients. A separate question is off-label use, in which an approved medical product is used for an unapproved indication. In this article, we focus on a narrower issue: what entities in the United States pay for access to unapproved medical products, e.g., investigational drugs, devices, or diagnostics that have not (yet) received Food and Drug Administration (“FDA”) approval.

We examine the various forms of preapproval access (“PAA”) to experimental medical products available in the United States—clinical trials and non-trial preapproval access via the Expanded Access (“EA”) and Right to Try (“RTT”) pathways. For each, this paper analyzes which entity—individual, insurer, sponsor, or other—bears the cost and what limitations or caps, if any, exist on these costs. This paper considers various proposed novel payment mechanisms that may permit more equitable use of investigational medical products.

Part I outlines payment-related disparities in access in- grained in the current United States healthcare system. Part II focuses on access in the context of clinical trials, which most payers have begun to cover, but where remaining uncovered expenses can disincentivize participation in clinical trials, even among those highly motivated to enroll. Part III discusses non-trial preapproval access pathways, specifically Expanded Access and Right to Try, where coverage is scant. Part IV briefly deals with investigational products (such as stem cell treatments) that are available via unregulated or underregulated direct-to-consumer sales. Part V then re- views the ethical considerations inherent in paying for investigational medical products.



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