U.S. Senate Passes 'False-Hope' Act

On August 3, the U.S. Senate unanimously passed the so-called Right to Try Act,[1] a bill that Public Citizen and many other consumer, patient and physician groups strongly opposed.[2] The deceptively named bill — which would more appropriately be titled the “False-Hope Act” — would put countless patients at risk by undermining important Food and Drug Administration (FDA) safety rules related to the use of unapproved, experimental medications.

Currently, the FDA oversees the use of experimental medications in the U.S. Use of such drugs most commonly occurs in clinical trials sponsored by pharmaceutical companies. But for patients who are not eligible to enroll in a clinical trial and for whom there is no satisfactory treatment available, access to experimental drugs can be obtained via the FDA’s long-standing expanded access program.[3]

To protect patients, the FDA and an institutional review board (IRB) must approve each use of an experimental drug under the expanded access program. As conditions of approval, there must be sufficient evidence of the safety and effectiveness of the experimental drug to support its use in a particular patient, and the probable risk to the patient from the drug must not be greater than the probable risk from the disease or condition.

The FDA has approved 99 percent of all expanded access program requests and, in urgent circumstances, can respond to such requests[4] within one to two days.

But the false-hope legislation passed by the Senate creates a dangerous, uncharted pathway for access to experimental drugs that essentially bypasses the protections of the FDA’s expanded access program for patients diagnosed with a life-threatening disease or condition. The bill would eliminate the requirements for review and approval by the FDA and an IRB, which have helped to ensure that the proposed use of an experimental drug does not pose unacceptable risk to patients and that the patients are fully informed of the risks and other key information when their consent is obtained.[5]

The bill also would undermine patients’ rights by barring lawsuits in a variety of situations in which state law might reasonably impose liability on a drug manufacturer or physician. For example, a manufacturer could not be sued for harm caused by contamination of an experimental drug.

This legislation would expose vulnerable patients to risks of serious harm, including dying earlier and more painfully than they otherwise would have, without appropriate safeguards. Many of the experimental drugs made available through the FDA’s expanded access program ultimately are not shown to be safe and effective in clinical testing and are not approved by the agency.

A much more prudent approach for Congress to take would be to find ways to improve rather than destroy the FDA’s expanded access program while maintaining the program’s patient safeguards.

References

[1] Congress.gov. S.204 - Trickett Wendler Right to Try Act of 2017. https://www.congress.gov/bill/115th-congress/senate-bill/204/actions. Accessed August 4, 2017.

[2] Public Citizen et al. Letter opposing approval of S. 204. https://www.citizen.org/sites/default/files/2383.pdf. Accessed August 4, 2017.

[3] Food and Drug Administration. Expanded access (compassionate use). https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm#Statutory_Background. Accessed August 4, 2017.

[4] Food and Drug Administration. Expanded access INDs and protocols 2009 – 2016. https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm443572.htm. Accessed August 4, 2017.

[5] Congress.gov. S.204 - Trickett Wendler Right to Try Act of 2017. https://www.congress.gov/bill/115th-congress/senate-bill/204/text. Accessed August 4, 2017.