The Critical Role of Medical Institutions in Expanding Access to Investigational Interventions

Abstract

The U.S. federal government provides two tracks for eligible patients to obtain access outside clinical trials to investigational interventions currently under study for potential clinical benefits: the Food and Drug Administration’s expanded access pathway and the pathway created by the more recent Right to Try Act. In this issue of the Hastings Center Report, with a critical focus on patients, industry, and the research enterprise, Kelly Folkers and colleagues frame the inherent challenges that these pathways are meant to solve and have also inadvertently created. But an additional key focus is how the relevant situations should be managed at the bedside and how the system risks both inefficient and inequitable access to options at the institutional level. Although either pathway could be helpful to patients, the challenges of having the pathways coexist are greater than the sum of their parts. Individual clinicians represent the front line of the regulatory and eligibility challenges of expanded access and right to try, making clinical education a critical component of a comprehensive approach to using them well. But it is medical institutions that must take the lead on supporting access to investigational options in the most equitable and effective manner possible.

Navigating the Informed Consent Process When Using Innovative Surgery

“…Thus, research subjects are inherently more vulnerable, as their well‐being, while still a major consideration, is not the only or in fact the primary outcome of interest. As a result, how this information is shared and navigated with patients versus subjects as part of the informed consent process reflects these differences, as discussed below, with regard to framing around the medical decision itself vs a decision to participate in research 6 . In all aspects of these discussions, it behooves professionals to employ strategies to improve information transmission and facilitate mutual understanding and communication, such as including using simple verbal and written language, and the teach‐back method.…”

Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs

“…[5][6][7][8][9][10][11][12][13][14][15][16][17][18] However, there are also concerns. [7][8][9]11,12,[14][15][16][19][20][21][22][23][24][25][26][27][28][29][30][31] Novel interventions may not work and can pose serious risks. Nontrial access may be inequitably distributed and might impede companies' ability to gather safety and efficacy data.…”

“… 5 , 6 , 7 , 8 , 9 , 10 , 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 However, there are also concerns. 7 , 8 , 9 , 11 , 12 , 14 , 15 , 16 , 19 , 20 , 21 , 22 , 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 Novel interventions may not work and can pose serious risks. Nontrial access may be inequitably distributed and might impede companies’ ability to gather safety and efficacy data.…”