What would you do if you were suffering from a terminal illness and you’d exhausted all medical options? If you answered “I’d try anything,” you’re not alone.
Enacted in 1987, the FDA’s Expanded Access program (also referred to as “compassionate use”) allows patients with life-threatening illnesses to apply for and use drugs that are in development but not yet fully approved.
In a move that has been causing some confusion, the Trump administration passed the Right to Try Act in 2018, which has the same goal as Expanded Access: Helping people with terminal illnesses access drugs not yet approved by the FDA. Even though it was passed years ago, you’ve likely been hearing about this federal law during election season because former President Trump has referenced this law to illustrate his achievements in office, and has noted he is open to expanding the law’s reach should he win reelection.
While there is much overlap between the two pathways, patients should be aware of key differences.
Expanded Access vs Right to Try
The FDA’s Expanded Access program allows a patient’s doctor to request access to a drug, biologic, or medical device that’s currently in clinical trials. There are concrete steps involved in the Expanded Access program, which include a physician filing paperwork that explains their treatment plan with the FDA and the Institutional Review Board (IRB), and both the IRB and FDA reviewing the paperwork to make sure the patient fully understands what they’re getting into, and that the treatment plan is solid.
Under the Right To Try Act, a physician can side-step the FDA and reach out to a pharmaceutical company directly, and the treatment plan physicians have to provide is not held to the same standards as Expanded Access. In fact, according to Alison Bateman-House, Ph.D., MPH, an assistant professor in the Department of Population Health at NYU Langone Health who is quoted in a piece from “ASH Clinical News,” a news magazine aimed at clinical practitioners in hematology and oncology:
“In the typical FDA procedures for informed consent, physicians are required to describe the potential risks and benefits, alternatives to the investigational drug, and more. All that is removed with ‘Right to Try’ [legislation]. There are no standards about what these documents need to say.”
Expanded Access Approval Process: Easier Than You May Have Heard
While proponents of the Right to Try Act say that sidestepping the curated FDA paperwork and approval process means patients will be able to access experimental treatments quicker, a 2018 article published in the open access journal JACC: Basic to Translational Science, notes that, typically, the FDA provides “emergency approval within hours, and non-emergency approval within an average of 4 days.” Additionally, an assertion on Righttotry.org’s own website that it takes “100 hours or more to complete the application process” for the Expanded Access program is not easily verified, as the assertion currently links to a broken page on the Regulatory Affairs Professionals Society website. As of publication, efforts to contact the think tank for clarity have not been answered.
The Expanded Access program also seems successful in approvals, with 99% of all single-patient applications approved. It also appears relatively popular. In 2022, the Expanded Access program approved 2,248 experimental drug applications.
Conversely, according to Politico, Right to Try was only used four times in the last year.
So why, if Expanded Access seems to be working well, are people still pushing for Right to Try and its expansion? According to the Goldwater Institute, a conservative, libertarian public policy think tank behind Righttotry.org, genetic sciences are now making it possible to create individualized treatment plans with emerging medicines. But the FDA’s clinical trial process is not set up to allow such an individualized lens. The expansion of Right To Try would, in theory, allow physicians to access drugs tailored to their patient’s needs without waiting for a clinical trial.
How Much Risk Is There In Unapproved Drugs?
Drugs go through three levels of human trials before FDA approval:
- Phase 1 trials, which focus only on the safety of the drug (side effects, dosage, etc.), typically have 20 to 100 volunteers. This process takes several months. According to the FDA, approximately 70 percent of new drugs move on to the next phase.
- Phase 2 trials, which continue to focus on side effects and on how effective the drug is, typically have several hundred volunteers and can run up to two years. About 33 percent of new drugs move on from this stage.
- Phase three trials, which can include 300 to 3,000 volunteers, typically run from 1 to 4 years, and are the best way to uncover rare and long-term side effects. Approximately 25 to 30 percent of new drugs move on from this phase to FDA approval.
With only several thousand people involved in these clinical trials, it can be difficult to know—even for drugs that have received full FDA approval— the full scope of side effects.
As a member of the FDA Advisory Committee on Drug Safety and Risk Management, Suzanne Robotti, MedShadow’s founder, notes that the risk of taking drugs not approved by the FDA is high.
“Right to Try allows drugs in Stage 2 trials to be released. Expanded Access requires that the drug has passed Stage 2 trials and is in Stage 3. Taking these drugs in either program is a very serious decision. You have no assurance that it will help and the side effects could ruin the quality of the last few months of a person’s life.”
If you or a loved one are interested in learning more about the Expanded Access program or Right to Try (including who is eligible and how the cost structure works), you can learn more about Expanded Access here, and Right to Try here.
