The Right To Try

By

Leonard Zwelling

Let’s start with the obvious. There is no right for a sick person to have access to experimental therapy. There just isn’t. Nor should there be.

Clinical trials are an experimental endeavor in which the federal government exerts its regulatory authority to protect the public from unscrupulous people who might take advantage of a specific vulnerable population—the sick. This aspect of the regulatory system is overseen by the individual Institutional Review Boards under the auspices of the federal Office of Human Research Protections. The IRBs are there to assure everyone that the highest ethical standards as well as local standards are upheld in the performance of human subjects research and that human subjects are always protected from those who might take advantage of them for nefarious purposes.

There is another regulatory aspect of this, too. That is the one asserted by the U.S. Food and Drug Administration that assures that all such research has as its goal the commercialization of useful, safe and effective drugs or devices.

So there are two important parts of the system. One part oversees the ethics of the research. The other oversees the science that is being used to identify useful new treatments for otherwise resistant diseases. OK. That’s all good.

http://www.ascopost.com/issues/march-25-2018/why-right-to-try-laws-are-dangerous/

In the March 25 issue of The ASCO Post, Friends of Cancer Research leader and cancer patient advocate Ellen Sigal, a tireless champion of the clinical research system, wrote a piece she called “Why Right-to-Try Laws Are Dangerous.” To sum it up, these ideas that people with serious disease have “rights” to access to new experimental agents is just not true.

First, all clinical trials have strict eligibility criteria that are there to protect the integrity of the data and the safety of all participants. People with end stage liver disease cannot be entered on phase 1 trials seeking to elucidate the toxicity of a new anticancer agent.

Second, this is science. The interpretability of the results of any trial will depend upon the tightness and the integrity of the data. A trial in breast cancer cannot encompass patients with liver cancer and expect to generate interpretable results.

Third, who is going to pay for the right to try? Insurers? I don’t think they are in the clinical trial business? Drug companies? For them a trial is an investment in which they must exert some control over who gets access to free drug the cost of which runs into the thousands. The company’s goal is to have a marketable useful product approved by the FDA under the most rigorous of standards.

Fourth, what about the false hope this engenders? Just because the patient has “nothing to lose” doesn’t mean others don’t have anything to lose. They do—especially if there is an adverse drug reaction when a new therapy is used outside of the scope of a formal trial. Then people who could benefit may lose access as the cause of the adverse event halts progress of the trial.

Fifth, the FDA has a compassionate use program that is both effective and quick. The FDA is not the problem and neither are the IRBs.

Organizations like ASCO and the Friends of Cancer Research are doing everything they can to open the clinical trials process to as many patients as could benefit from participation. In the mean time, there is simply no call for a Right-to-Try. There is no right-to-try.

Cancer and other fatal diseases need new innovation so that the patients with the diseases have a better chance at normal lives. Breaking down the system that has been built successfully over the past 40 years since the Belmont Report tried to end abuses in the clinical trial system is a terrible idea and that is what the right-to-try movement is trying to do. Resist it. Sure the current system could be improved. Subverting it does not improve it.

Leonard Zwelling