Trump’s push for ‘right to try’ is harmful
In his State of the Union address, President Trump asked for legislation to grant access to experimental treatments for patients with terminal conditions, saying, “It is time for the Congress to give these wonderful Americans the ‘right to try.’ ”
Sounds like a no-brainer, but as an ethicist with several years’ experience on an Institutional Review Board evaluating and monitoring medical research, I have serious concerns about such a law.
The implication of Trump’s advocacy of “right to try” is that patients are somehow being denied access to lifesaving treatments. In fact, the careful, scientifically sound and adequately overseen practice of clinical research is the best way to protect patients and the pathways to new, effective drugs. The president clearly misunderstands how access to experimental drugs works and how “right to try” would undo that.
First, know that the overwhelming majority of experimental drugs that pass Stage 1 testing do not make it to clinical use. Either they are ineffective compared with standard treatment, or their side effects outweigh their clinical benefits.
Then, consider Jane Doe. She was diagnosed with aggressive breast cancer that has spread to her lungs and recently to her liver as well. She has been through several rounds of chemotherapy, changing drugs whenever her cancer progressed or spread. She is now showing early signs of liver and kidney failure.
Meanwhile, a drug giant I’ll call WonderPharmaCo is studying its new drug Phixitalla. The drug is now in Stage 2 trials, where more patients are trying the drug and researchers learn more about its adverse effects. While patients for whom previous chemo is no longer helpful are eligible to join some of these studies, patients as sick as Jane are not.
Under Food and Drug Administration “expanded access” (also called “compassionate use”) regulations, a patient like Jane with no other treatment options is permitted to have her doctor request an unapproved drug for her. If WonderPharmaCo agrees to provide the drug, the FDA must also sign off on the request.
The FDA considers the likely risks
and benefits in light of other information it may have about the drug and Jane’s medical status, and checks for other studies Jane might be eligible to join. Applications are reviewed within 30 days; in case of emergency, they can be reviewed immediately by phone. The FDA has approved more than 99 percent of these applications.
Jane’s doctor also submits information to the hospital’s Institutional Review Board, which evaluates the protocol, making sure Jane’s rights and safety — and her access to a drug that might help her — are protected. These review boards comprise scientists and nonscientists, ethicists and community members. The review board makes sure Jane has been adequately informed about Phixitalla. With FDA and review board approval, Jane has become a “research study of one.”
A federal “right to try” legislation would not grant any new rights and would take away FDA review. Paid for and pushed principally by libertarian groups such as the Goldwater Institute, such a law would undercut some aspects of the FDA patient-protection process but do nothing to give any patient a legal right to a drug that its manufacturer does not wish to share, nor does it eliminate potentially crushing costs for such drugs.
Bypassing the FDA is problematic. And it seems unclear whether Institutional Review Board oversight will remain part of any final federal legislation. Already, under the terms of a bill passed unanimously by the Senate last year and now under review by the House, no data or information from Jane’s experience may be used to evaluate Phixitalla unless the secretary of Health and Human Services deems it “critical to determining the safety of the eligible investigational drug.” Adverse events must be reported to the HHS and posted on the FDA website — but whether the FDA could make use of them for approval purposes is questionable.
Under FDA regulations, the physician’s qualifications for administering the medication are evaluated as part of the approval process. Nothing in the Senate version of “right to try” suggests that one’s physician need be particularly qualified to treat the disease in question. Under “right to try,” Jane’s physician is immune from malpractice prosecution barring recklessness, gross negligence or intentional harm. WonderPharmaCo is immune from prosecution, too. No one would be legally responsible for bad effects for Jane except Jane herself.
In California, Gov. Jerry Brown signed a “right to try” bill in 2016. Medical groups have spoken out against “right to try” on grounds that it takes advantage of a vulnerable population. Even the drug industry lobby, the Pharmaceutical Research and Manufacturers of America, while it has taken no final stance on this legislation, has been widely cited in favor of retaining FDA oversight.
Undermining important patient safety mechanisms might undermine public confidence in drug safety overall, and cause people to be reluctant to enter clinical trials. “Expanded access” allows a small group of patients to access a drug before the approval process is complete. “Right to try,” conversely, makes everyone a “research trial of one” and uses the understandable anxiety of terminally ill patients as an excuse for subjecting them to inadequately vetted processes.
No one wants to keep dying patients from drugs that might help them. And no one is.
Lisa Fullam is a professor of moral theolog y at the Jesuit School of Theolog y of Santa Clara University and has been a member of a California Institutional Review Board since 2010. To comment, submit your letter to the editor at SFChronicle.com/letters.