The "Right to Try" Act: What Cancer Patients Need To Know
- Gives “eligible” patients access to “eligible” experimental therapies, not yet approved by the FDA—but guidelines remain in place.
- It’s similar to the “Expanded Access Program” which is FDA-supervised.
- Patients assume all risks for treatments that have not yet been approved for use by the FDA.
- Critics say the Act offers false hope, while some patients, like Natalie Harp, say that it saved their lives.
Harp says she was in a wheelchair for years with pain that was “out of this world” when she ran out of options. “When I failed the chemotherapies that were on the market, no one wanted me in their clinical trials. I’d make them look bad. They didn’t give me the right to try experimental treatments,” she said, “I’d have died waiting for them to be approved.” Her standing there, talking to us, was nothing short of amazing.Read More
Naturally, Harp, who serves on the advisory board of the President’s re-election campaign, is grateful for Right to Try, which was signed into law in May 2018, and was designed to lift regulatory roadblocks that prevent a patient with “little to lose” from trying an un-approved experimental therapy as a last-ditch effort.
Let’s hope none of us find ourselves making the kind of difficult decision Harp had to make. But what if we—or a loved one—does? What are the options?
Clinical Trials, First, Doctors Say
Clinical trials are the first choice for patients seeking experimental therapies, Dr. Michael Sekeres, Director of the Leukemia Program and Vice-Chair of Clinical Research at Cleveland Clinic Cancer Center told SurvivorNet.
“Clinical trials are something I prefer because they are rigorously monitored including for whether or not a drug works — and for the toxicities that a patient may experience on that clinical trial.”
Dr. Mikkael Sekeres, Director of the Leukemia Program at Cleveland Clinic Cancer Center, points out that, compared to clinical trials, the Right to Try approach makes it difficult to track patient response and side effects of new cancer therapies.
In fact, the Right to Try Act specifies that patients must have exhausted treatment and clinical trial options before they’ll be eligible. So, before exploring untested therapies, always seek clinical trials first.
Another option for obtaining drugs that aren’t yet FDA-approved are called “Single Patient Investigational New Drug Protocols” (or Single Patient INDs).
“We actually submit a very brief protocol through our institutional review board. And a patient signs a consent to receive an investigational drug,” says Dr. Sekeres, who adds that the “FDA approves 99 percent of the applications submitted for single patients INDs.”
“We monitor that patient closely, for side effects and for whether or not that drug works,” he notes.
Right to Try: What Is It?
The Right to Try Act, which was signed into law in May of 2018, offers certain patients access to certain unapproved treatments, according to the Food and Drug Administration (FDA).
It was designed for patients who have been diagnosed with life-threatening diseases or conditions, who have exhausted all treatment options, and who are unable to participate in a clinical trial.
While some have described the Right to Try Act as the medical equivalent of a Hail-Mary pass, the FDA outlines specific criteria for the patients who opt-in—as well as for the treatments they hope to try.
For instance, the Act defines “eligible investigational drugs” as those for which a Phase 1 clinical trial has been completed. “We’ve seen so many times, drugs that have failed our patients after early phase studies and that’s when drugs would be available through Right to Try,” says Dr. Sekeres.
“After early phase studies, my preference would be to enroll patients onto clinical trials of investigational drugs—or to obtain single patient IND’s—where we can monitor those patients much more closely and educate other patients about the efficacy of these drugs than we would through a Right to Try approach,” he notes.
Criteria For Right to Try Drugs
According to the FDA, drugs and other therapies eligible under the Right to Try Act include:
- Those that have not been approved or licensed by the FDA for any use
- Those for which an application has been filed with the FDA or is under investigation in a clinical trial in support of FDA approval
- Those whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by the FDA
Dr. Jim Allison, Chair of the Department of Immunology at MD Anderson Cancer Center, and the winner of the 2018 Nobel Prize for Physiology or Medicine tells SurvivorNet that the next big wave of research in cancer treatment involves combining immunotherapy with targeted drugs.
In other words, the Right to Try Act does not grant patients the right to be treated with oddball magic elixirs or snake oil — and it does protect them from doctors who may have a financial incentive to try a new drug or medical device on an unsuspecting patient.
To become eligible, the FDA says, patients must provide their own consent along with certification by a licensed physician who is not being directly compensated by the treatment manufacturer—like a drug company.
Expanded Access Program
The Right To Try Act is similar to the FDA’s “Expanded Access Program” which grants access to investigational medical products when no other options are available.
Under this program, treatment is supervised by the FDAs Institutional Review Board, which reviews and approves all requests.
What’s The Role Of Drug Companies?
Just because a drug exists, doesn’t mean it will be available to a patient invoking their Right to Try—or that a drug company will release it for the requested use.
Companies who develop and make drugs and biologics —known under the Act as “sponsors”—determine if they are able to provide a particular drug under the Act’s requirements, listed above. Ultimately, the FDA says, sponsors developing drugs for life-threatening diseases or conditions determine whether to make their products available to patients who qualify for access under the Right to Try Act.