Center for Medical Ethics and Health Policy

Helping Neuro-oncologists Navigate Expanded Access

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Content

Expanded Access Program: Evaluating perception and knowledge of EAP by patient and physician dyads to improve access and utilization.

Project Description

The federal Right to Try Act was signed into law May 18, 2018. RTT is designed to give patients with life-threatening illnesses access to unapproved drugs, bypassing the FDA’s Expanded  Access Program  (EAP; called  “compassionate  use ”for  individual  requests).RTT laws—also  enacted  in  41  states—are  popular  with  the  public  and  politicians  who  claim  they remove  regulatory  barriers  so  patients  can  obtain  lifesaving  drugs. For last-option patients and their physicians, there are now two competing processes for access to unapproved drugs: RTT and EAP. Publicity surrounding RTT may mean physicians receive more requests for unapproved drugs, causing conflict with patients and increased moral distress. Oncologists practicing in large research centers are especially vulnerable, where trials in cohorts of last-option patients are common and demand for experimental drugs is most acute. Patients may be confused about a law billed as patient centric legislation, which may lead to emotional and physical harm. RTT removes federal and local oversight, prevents the FDA from using adverse events during the drug approval process, and largely blinds the agency to safety data. As a result, bioethicists, policy scholars, and professional organizations are overwhelmingly critical of RTT law.

We know little about how physicians and patients navigate these requests and their understanding of EAP/RTT law and policy, or how they manage informed consent after a request has been approved. Only a handful of studies capture physicians’ experiences with EAP, yet these were performed in specific settings and none have focused on RTT. While research and surveys have captured the views of patients considering  experimental  drugs none   have specifically examined the ethical and professional obligations of physicians, and none have studied oncology  patients  who  request  drugs  under EAP/RTT. One  group  used  an imbedded ethics consultation model with a  manufacturer to adjudicate EAP requests for daratumumab, but there is no data on how many times a request is received by manufacturers and how many times they say yes or no. When individuals face life-threatening and life-limiting illnesses, our project seeks to understand the “good” uses of  health  care,  as  these values and responsibilities may be differently perceived and answered for families, patients and doctors.

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