Document Type

Article

Publication Date

2020

Abstract

In May 2018, Congress passed the controversial Right to Try (“RTT”) Act, creating a process for terminally ill patients to request access to investigational drugs. The federal RTT Act is not the first legal mechanism that fosters quicker access to investigational drugs. This new right to try is distinct from existing pathways created by law, regulation or federal administrative agency policy. Various mechanisms facilitated by the U.S. Food and Drug Administration (“FDA”) are significantly more substantial and important in the context of “faster” access to therapeutic products. These mechanisms lie along a spectrum of product development spanning investigational new drug status to postmarket studies and surveillance. I categorize these mechanisms into three areas: expansion, acceleration, and extension. The federal right to try can be characterized as an expansion, expanding patient access to investigational new drugs as an alternative mechanism to the FDA’s long-standing expanded access program. As the Senate notes, the RTT Act “does not establish a new entitlement or modify an existing entitlement, or otherwise establish a positive right” and “is consistent with and will act as an alternative pathway alongside existing expanded access policies of the Food and Drug Administration.”

This article positions the new RTT in proper context and explores additional FDA mechanisms that serve to speed up patient access. In Part I, this article will first discuss the content and scope of the RTT legislation, as well as the reasoning for the support and opposition for the legislation. The legislation is only four pages long, as enacted, and introduces a concise procedure for requests for access to investigational drugs. The article will next examine FDA mechanisms for expansion in Part II, comparing the RTT with the current expanded access program at the FDA. Part II will explore two mechanisms of expansion at the FDA: expanded access for patients to investigational drugs and devices (the “expanded access” program) and expanded input from patients about experiences with drugs and devices under review at the FDA ("patient experience" data). This expansion of patient input was initiated and implemented within the FDA but further directed by provisions within the 21st Century Cures Act.

Part III will analyze several mechanisms of accelerated review and approval at the FDA, including FastTrack status, priority review, accelerated approval, Orphan Drug status, and Breakthrough designation of promising therapeutics. The collection of hastened mechanisms of review and approval are poorly understood and have led to confusion and misunderstanding by physicians and patients alike. They also alter the foundational new drug approval framework originally set forth by Congress in the Kefauver-Harris Drug Amendments enacted in 1962, which strengthened the drug approval process to include not only premarket safety review but also substantial evidence of efficacy demonstrated through “adequate and well-controlled” clinical trials. Recent empirical scholarship informs this Part, revealing that the growing array of statutory mechanisms to speed up clinical trials, review, and approval are cutting away at long-standing protections afforded by robust measures of safety and efficacy. Part IV will then discuss extension at the FDA, particularly the extension of evidence gathering in post-market clinical trials to support showings of safety and efficacy. The article concludes with several reflections on the relationship of the federal RTT to these FDA mechanisms of "faster" access, as well as potential implications of expansion, acceleration, and extension on patient safety, patent protections, and drug costs.

Download

Share

COinS