Friends of Cancer Research Cautions on ‘Right-to-Tryֹ’
Pending right-to-try legislation in Congress, which would give terminally ill patients access to investigational drugs, is an ill-advised political stance that would “circumvent” FDA’s authority and offer patients false hope, according to an online post from Friends of Cancer Research chair and founder Ellen V. Sigal. “We must ensure that patient safety is still a top priority,” Sigal wrote in her post. “The right-to-try legislation proposed by Congress seeks to grant all terminally ill patients access to experimental therapies once approved alternatives have failed and is a way to remove the FDA’s oversight authority by bypassing the expanded compassionate use access process.”
Sigal said that while FDA’s current compassionate use program authorizes more than 99.4% of requests it receives, proponents of the legislation claim the program is too slow. “It is important to note the current process has been streamlined, so that the FDA is reviewing most requests within 24 hours, and it takes patients less than an hour to fill out the application,” she said. “The problem is not the FDA, and it is imperative that the agency not be removed from the review process of patients receiving any type of therapy, even an experimental one. The unfortunate truth of the proposed legislation is that it effectively provides zero protections for patients and will not actually result in greater access to experimental therapies.”
Sigal said that any legislation should include informed consent provisions about the risks involved. “Current right-to-try laws allow patients to request therapies from companies that have passed a Phase I trial with the FDA,” her post said. “However, it needs to be clear that during a Phase I trial, only a small group of patients receive the therapy; the treatment is tested in carefully controlled conditions designed to find and detail obvious toxicities; and the trial is meant to identify a tolerable range of potentially effective doses before the drug advances to a larger Phase II trial. The extent that a patient can give informed consent based upon such little clinical information is limited.”
Additionally, Sigal said patients should be aware that almost 90% of new drugs never make it to market because they are eventually found to be too dangerous for patients or shown to be ineffective. “Not to mention that during the Phase I trial, there are no reliable data whether a therapy works, and toxicities can be discovered in later phases of trials,” she said. “By including an informed consent process that is well defined and supported by adequate clinical data in the final piece of legislation, there could be a stronger guarantee to patients that they can sufficiently judge the experimental therapy and weigh the potential risks and benefits as well as the magnitude of their decision.”