Doctors Support the Right to Try for Terminal Patients

As a former panel consultant to the FDA Ophthalmic Device Panel, Dr. Ming Wang, is intimately familiar with the FDA approval process for medical devices and drugs. Half a million cancer patients and thousands with other terminal diseases die each year waiting for access to already developed drugs that have proven effective at treating or reversing their conditions in clinical trials. Currently, the ‘compassionate use’ provision is burdensome and forces patients to endure ongoing delays. That’s often time they simply don’t have. The worst conversation to have with a patient who has a terminal disease is that there’s an experimental drug that might help but it’s unavailable to them because of arcane regulations. A Right to Try law injects a needed dose of humility and humaneness into a bureaucratic process that means well but at times acts arrogantly and inhumanely. Such a law, if applied intelligently, would not only help millions of terminal patients now, but those in the future who would beneft from the science and learnings from those treatments.

– Murray S. Feldstein, MD, a former Mayo Clinic Assistant Professor of Urology



Bureaucratic red tape should not stand between my terminally ill patients and their right to try medications and treatments already determined as safe by the FDA, that might offer them some hope for quality survival. Right to Try legislation allows patients, in consultation with their doctors, decide what risks they are willing to take to save their own lives–not the government. And with proper monitoring by clinicians, this may lead to new knowledge and advances in the treatment of lethal diseases.

– Jeffrey A. Singer, MD, FACS General Surgeon



Prior to the Right to Try law passage in Texas, Dr. Ehrahim Deplassand, Medical Director & Chairman, Excel Diagnostics & Nuclear Oncology Center in Houston would have to wait 8 – 10 months, and sometimes 2- 3 years, to treat patients with drugs that were proven safe and effective but still waiting for FDA approval.


The Right to Try Law can really help us in the medical profession by bringing life saving or life extending medications to our patients when it’s not yet commercially available or approved by the FDA. Right to Try is a cry for help in every sense. It’s about time that patients’ pleas are heard and real compassion shows. These laws are beautiful in that they are grounded on the fundamental sanctity of life, yearning to live, and the desire to fght. They can work effectively; we only need to put the patients first in our thinking.

– Joseph Gulfo, MD, Executive Director of the Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University



Right to Try is a scaffold on which an industry wide national infrastructure can be built, state by state if that’s what it takes to help patients across the spectrum of fatal diseases participate actively in trying to find a cure for their disease; and become active partners in the process rather than passive victims. RTT will provide hope and comfort to many patients and it will accelerate the effort to fnd cures for currently incurable diseases.

– Richard Garr, former CEO of Neuralstem, a biotech company developing a treatment for ALS



Leighton Read, MD, a venture partner at Alloy Ventures who has previously held appointments at the Harvard Medical School and School of Public Health, supports the use of state Right to Try laws to provide access to compounds after a Phase I clinical trail, in some cases. He says states are “laboratories of democracy,” and elected state governments should be free to experiment with the provision of investigational drugs outside FDA oversight for patients with terminal illnesses who have run out of options.


Every day Americans want to have faster and less cumbersome access to potentially life-saving medications. While the medical community and academic centers embrace the culture of clinical trials, there are simply not enough trials that can provide opportunities to all patients. Sometimes these new medications have early indicators that they will be effective for the treatment of very serious diseases such as cancer. The Right to Try laws are a very positive step forward toward easier and less cumbersome access to life-saving options. These laws remove bureaucratic and administrative barriers and give the decision back to physicians and patients.

– Rafael Fonseca, MD, a Mayo Clinic hematologist whose research has been funded by the National Cancer Institute, the Leukemia and Lymphoma Society, the Multiple Myeloma Research Fund



Dr. Razelle Kurzrock set up and ran early-stage clinical trials at the MD Anderson Cancer Center in Houston from 2004 until 2012, eventually building it into the largest such program in the country before she left. About 1,300 patients went through the trials in her department every year. Dr. Kurzrock said her unit only tried to get compassionate use for about one patient annually because of the time and runaround involved in preparing the application. Even before starting the process of assembling the data and flling out the form, Kurzrock spent hours on the phone calling the FDA and drug companies to fnd out if there was even a chance that the request would be approved. In most cases, the answer was no. “So you never get to the point where you put in an application,” she says. It’s almost a self-fulflling prophecy for the FDA to say they approve everything, because you don’t even put in the application before you sort of get a verbal approval from the FDA that it’s worth doing. The fact that we did maybe one a year in our department, which was the largest of its type, probably in the world, I think says it all. There’s only two possibilities: that there was only one patient per year that needed compassionate use, and that’s really laughable. Or that there were so many barriers that even at one of the best places in the world and one of the largest departments that did this as their day in and day out job, it was still very challenging. The number of patients that we would give compassionate use drugs to would probably be much, much higher if the bar for compassionate use was not so high.