Right to Know: Why the FDA Should Not Be Cut Out of Expanded Access Requests

Over the past three years, the libertarian Goldwater Institute–led right to try (RTT) movement has had wind in its sails, propelling the passage of RTT laws in 38 states and counting. The movement, which aims to cut the FDA out of the process by which patients with serious or immediately life-threatening diseases without available therapies access investigational drugs and biologics, hit some choppier waters at the hearing held October 3rd by the Health Subcommittee of the House Energy & Commerce Committee. The House is considering passage of a federal RTT bill, and two potential options were presented at the hearing. S. 204, sponsored by Sen. Ron Johnson (R-Wis.), was passed by unanimous consent in the Senate on August 3. Another RTT bill, H.R. 1020, introduced by Representatives Morgan Griffith (R-Va.) and Dave Brat (R-Va.) in February, was also under consideration. Rep. Andy Biggs (R-Arizona), who in February introduced a third version of a federal RTT bill, H.R. 878, testified at the hearing. Senators Joe Donnelly (D-Ill.) and Johnson have urged the House to pass S.204 “as soon as possible” and “without amendment.” Making any changes to S.204 would require reconsideration of the new version by the Senate.

Notably, FDA Commissioner Scott Gottlieb, appointed by President Trump, did recommend revisions to S.204. In his testimony to the committee, Dr. Gottlieb expressed concerns about the definition of “eligible patient” in S.204. Written testimony he submitted included a recommendation to narrow the bill’s eligibility definition. Instead of making the RTT pathway applicable to patients with a life-threatening disease or condition, which includes patients with chronic, manageable diseases with FDA-approved treatment options, Dr. Gottlieb suggested eligibility be limited to patients with a terminal illness, defined as “a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months.”

We support Dr. Gottlieb’s recommendation. Additionally, we applaud his defense of the FDA’s own “expanded access” pathway, which already allows patients with serious or life-threatening diseases to access investigational drugs when they cannot enroll in a clinical trial and have no approved treatment options. The FDA clearly plays a valuable role in this process, and has made significant efforts to respond to criticism from patient advocates. The agency has simplified the request process and improved its expanded access website. In fact, Dr. Gottlieb announced additional measures to streamline the FDA pathway at the October 3rd hearing. Although the agency is often portrayed by the RTT movement as an obstacle to access to investigational medicines by desperately ill patients, Dr. Gottlieb confirmed findings that the FDA allows more than 99 percent of the requests it receives to go forward, usually within days and, in emergency cases, within hours.

Importantly, Dr. Gottlieb also emphasized that patients receive “key protections” by accessing investigational medicines through the FDA’s expanded access pathway. Because the FDA is likely to have information on investigational products that is not available to sick patients or their physicians, the involvement of the FDA ensures that treating physicians and patients alike are adequately informed and protected. Dr. Gottlieb noted that the FDA “makes meaningful changes in approximately 10 percent of these cases to enhance patient safety.” These changes include adjustments to dosing or duration of treatment, recommendations for additional safety monitoring, and additional information to include in informed consent documents. When sick patients and their physicians are weighing options in these desperate situations, don’t they have a right to know as much information as possible about these experimental therapies?

The FDA reviews single patient expanded access applications only when the manufacturer is able and willing to provide the desired investigational product. Dr. Gottlieb noted that in most cases, it is the companies, not the FDA, that deny patient requests for investigational therapies. Limited supply is one reason for denial, but there are numerous others. Because companies have no obligation to provide the experimental therapies, many have argued that “right to try” is a misnomer, giving the most vulnerable patients a misguided belief that the help they’ve been waiting for has arrived. In actuality, S.204 states that it “does not establish a new entitlement or modify an existing entitlement, or otherwise establish a positive right to any party or individual.” Therefore, the short title of the bill, which includes the words “Right to Try,” is sadly misleading.

Proposed RTT legislation opens the door to a considerably less controlled framework for providing access to unproven products. Rather than create an additional pathway that has the potential to erode the authority of the FDA over investigational medicines and put patient safety at risk, the House and Senate should continue to work with the FDA to improve its existing expanded access pathway. One idea might be to offer the liability protections included in S.204 to sponsors, manufacturers, prescribers, and dispensers that use the FDA’s expanded access pathways, although legislators should take heed of Dr. Gottlieb’s additional recommendations to clarify the language of these clauses. The bottom line is this: We should have one uniform, national pathway to access investigational medicines when appropriate, and we should keep the FDA involved. The agency’s mission is, after all, to protect patients.

Carolyn Riley Chapman, PhD MS is a Postdoctoral Fellow at NYU School of Medicine, in the Department of Population Health’s Division of Medical Ethics.

Richard Klein and Andrew McFadyen are members of the NYU Langone Health Working Group on Compassionate Use and Pre-approval Access (CUPA).

Richard Klein recently left FDA. For many years, in his official capacity at the agency, he assisted patients seeking expanded access. He also participated in the writing of the current FDA expanded access regulations, development of a new, streamlined request form, helped develop the FDA expanded access web site and guidance documents related to expanded access, and the new policy designed to facilitate IRB review of single patient access requests.

Andrew McFadyen is the executive director of The Isaac Foundation, a non-profit organization that provides support for patients and families impacted by rare diseases, including gaining access to pre-approved and potentially life-saving treatments.

 

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