Are Physicians Morally Obligated to Prescribe Experimental Therapies?

A doctor reassuring a patient.

(© missty/Fotolia)

The federal 'Right to Try' bill in the United States recently passed the House and requires Senate approval before it becomes law. The bill would provide patients access to experimental drugs and other products that have not received approval from the Food and Drug Administration (FDA), including stem cell treatments.

It's not enough to act on a hunch that it might work.

Most folks think this is a good thing, but several express concern over whether the law would truly help patients. Even if a company allows patients to access an experimental drug, an important question remains: Should a doctor prescribe it?

Before such a drug can be prescribed, the federal bill states that a physician must "certify" that the patient has exhausted all available treatments or does not meet the criteria for standard treatment. Even after determining eligibility, a physician needs to consider a few points first. It's not enough to act on a hunch that it might work. The concept of medical innovation could help doctors figure out if prescribing an experimental treatment is the right thing to do.

Medical innovation falls within the doctor's scope of practice. Based on their experience and sound scientific rationale, physicians can "innovate" and offer treatment tailored to a patient with the goal of improving health. This differs from the goal of clinical research, which is to produce generalizable knowledge, not necessarily to benefit patients. In medical specialties like surgery, many of the standard procedures were developed through medical innovation, not clinical trials. Under the 'Right to Try,' a physician could ethically prescribe an experimental therapy as medical innovation if the following conditions are met.

Medical innovation should follow similar ethical and scientific oversight as clinical research.

First, there must be sound scientific rationale, and evidence of safety and efficacy of the innovative treatment from preclinical (animal and lab) research or clinical (human) research. The 'Right to Try' bill permits access to experimental products only after safety is demonstrated from a phase 1 clinical trial. This initial testing, called "first in human," aims to determine safety and dosing of an experimental product on typically around 20 to 100 people who are healthy volunteers or have a condition. This way, a physician can be assured that there is some evidence indicating the product is safe.

Efficacy must be demonstrated in animal and lab preclinical studies in order to gain permission from the FDA to do a phase 1 trial in the first place. This way, a doctor can also be assured that sound scientific rationale exists indicating a potential benefit to the patient. Only through further phase 2 and 3 clinical trials on hundreds or more people would a doctor know with greater certainty that the therapy works, but this might take many more years.

A doctor should not completely rely on what others in the scientific community think about the experimental treatment and should have appropriate expertise. This includes knowledge about the disease, familiarity with treating such patients, and an understanding of how the experimental treatment works, including administering it.

Second, medical innovation should follow similar ethical and scientific oversight as clinical research. Physicians should write a protocol for administering the experimental therapy and have it reviewed by clinical, scientific, and ethics experts at their institution. A protocol would include all the information on how the doctor would provide the therapy to patients, including dosages, monitoring, what happens if there are side effects, and much more. The experts would examine various components of the plan, look at informed consent, and ensure a favorable benefit-to-risk ratio, among other aspects.

When weighing whether to prescribe an experimental treatment, doctors need to base this decision on sound science and relevant clinical experience, not on hope or desperation.

Third, doctors should properly inform their patients about the risks (including if the risks are unknown), possible benefits, and the details of the procedure to be undertaken, and they must obtain the patient's consent.

Fourth, physicians should thoroughly monitor and diligently document all aspects of the outcomes of the procedure, various clinical indicators, and adverse events. During the course of providing an experimental therapy, if harm to a patient occurs, the physician is obligated to alter the course of the treatment or stop it. Similarly, if evidence from an ongoing clinical trial shows that the experimental treatment might help some but not all patients, the doctor needs to modify the plan accordingly.

Fifth, upon completing the experimental treatment, physicians should publish their findings to share the knowledge. Note that medical innovation is not meant to replace clinical trials. The two can be complementary, and medical innovation can lead to the design of clinical trials to demonstrate safety and efficacy.

Other experts may not agree that it can be ethical for a physician to prescribe an unapproved drug. Such dissenters would claim that physicians should only prescribe medications when there is substantial scientific and clinical certainty that a product is safe and effective for patients. They are also likely to oppose most forms of medical innovation. Yet even after undergoing rigorous clinical trials, some approved products have been shown to be unsafe or ineffective and are removed from the market.

While it seems that more evidence is better, doctors need to be mindful that patients are suffering and some may never receive access to drugs still in the pipeline. Bound by the Hippocratic Oath – the main tenet being "do no harm" – doctors are obligated to prescribe therapies that will help their patients. When weighing whether to prescribe an experimental treatment, doctors need to base this decision on sound science and relevant clinical experience, not on hope or desperation. Given that patients who want to participate in the 'Right to Try' movement have exhausted all other options and their condition may be worsening, it would seem ethically appropriate for a physician to treat them with an experimental drug, as long as the criteria listed above are satisfied.

The views expressed are the author's personal views, and do not necessarily reflect the policy or position of Mayo Clinic.

Zubin Master
Zubin Master, PhD is an Associate Consultant II in the Biomedical Ethics Research Program at Mayo Clinic. Previously, he was an Associate Professor at the Alden March Bioethics Institute of Albany Medical College. He has also held appointments at the University of Alberta, University of Montreal, the National Institute of Environmental Health Sciences, and the Ottawa Hospital Research Institute. Dr. Master also worked in public service as a Senior Policy Advisor at Health Canada in areas of assisted reproduction and scientific integrity. He holds an undergraduate degree in genetics from York University, a PhD in cellular and molecular biology from the University of Toronto, and completed post-doctoral fellowships in bioethics and health policy at Dalhousie University and the University of British Columbia. His research interests focus on the ethics and policy of research specializing in stem cell research, genetics, and research integrity. Dr. Master serves on several committees and journal editorial boards and has published over 70 articles. Dr. Master has not received any remuneration for writing for leapsmag.
Get our top stories twice a month
Follow us on

David Kurtz making DNA sequencing libraries in his lab.

Photo credit: Florian Scherer

When David M. Kurtz was doing his clinical fellowship at Stanford University Medical Center in 2009, specializing in lymphoma treatments, he found himself grappling with a question no one could answer. A typical regimen for these blood cancers prescribed six cycles of chemotherapy, but no one knew why. "The number seemed to be drawn out of a hat," Kurtz says. Some patients felt much better after just two doses, but had to endure the toxic effects of the entire course. For some elderly patients, the side effects of chemo are so harsh, they alone can kill. Others appeared to be cancer-free on the CT scans after the requisite six but then succumbed to it months later.

"Anecdotally, one patient decided to stop therapy after one dose because he felt it was so toxic that he opted for hospice instead," says Kurtz, now an oncologist at the center. "Five years down the road, he was alive and well. For him, just one dose was enough." Others would return for their one-year check up and find that their tumors grew back. Kurtz felt that while CT scans and MRIs were powerful tools, they weren't perfect ones. They couldn't tell him if there were any cancer cells left, stealthily waiting to germinate again. The scans only showed the tumor once it was back.

Blood cancers claim about 68,000 people a year, with a new diagnosis made about every three minutes, according to the Leukemia Research Foundation. For patients with B-cell lymphoma, which Kurtz focuses on, the survival chances are better than for some others. About 60 percent are cured, but the remaining 40 percent will relapse—possibly because they will have a negative CT scan, but still harbor malignant cells. "You can't see this on imaging," says Michael Green, who also treats blood cancers at University of Texas MD Anderson Medical Center.

Keep Reading Keep Reading
Lina Zeldovich
Lina Zeldovich has written about science, medicine and technology for Scientific American, Reader’s Digest, Mosaic Science and other publications. She’s an alumna of Columbia University School of Journalism and the author of the upcoming book, The Other Dark Matter: The Science and Business of Turning Waste into Wealth, from Chicago University Press. You can find her on and @linazeldovich.

Reporter Michaela Haas takes Aptera's Sol car out for a test drive in San Diego, Calif.

Courtesy Haas

The white two-seater car that rolls down the street in the Sorrento Valley of San Diego looks like a futuristic batmobile, with its long aerodynamic tail and curved underbelly. Called 'Sol' (Spanish for "sun"), it runs solely on solar and could be the future of green cars. Its maker, the California startup Aptera, has announced the production of Sol, the world's first mass-produced solar vehicle, by the end of this year. Aptera co-founder Chris Anthony points to the sky as he says, "On this sunny California day, there is ample fuel. You never need to charge the car."

If you live in a sunny state like California or Florida, you might never need to plug in the streamlined Sol because the solar panels recharge while driving and parked. Its 60-mile range is more than the average commuter needs. For cloudy weather, battery packs can be recharged electronically for a range of up to 1,000 miles. The ultra-aerodynamic shape made of lightweight materials such as carbon, Kevlar, and hemp makes the Sol four times more energy-efficient than a Tesla, according to Aptera. "The material is seven times stronger than steel and even survives hail or an angry ex-girlfriend," Anthony promises.

Keep Reading Keep Reading
Michaela Haas
Michaela Haas, PhD, is an award-winning reporter and author, most recently of Bouncing Forward: The Art and Science of Cultivating Resilience (Atria). Her work has been published in the New York Times, Mother Jones, the Huffington Post, and numerous other media. Find her at and Twitter @MichaelaHaas!