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by Andrew Blumstein

Beginning in May 2014 with Colorado, more and more states have been enacting “Right to Try” laws.  The purpose of these laws is to give the terminally ill access to potentially life saving investigational drugs, yet there are small differences between what the 24 states that have these laws require of patients and their doctors to become eligible.  However, each state sets the same threshold of what drugs are permissible by requiring an investigational drug to pass Phase I of the Food and Drug Administration’s (FDA) three Phase clinical trial program.

Proponents of investigational drug use argue that they have a right to pursue treatment in a timely manner; often patients or their doctors know of a promising drug, yet the drug has not completed the FDA approval process, and patients are forced to painstakingly wait as the drug passes through the clinical trial process.  On average, it takes anywhere from eight to twelve years for a drug to become approved by the FDA, so waiting is not a viable option. Opponents argue that patients who use investigational drugs could further harm themselves, as Phase I testing of drugs only tests for a drug’s basic safety for human use, and that there is an existing system for compassionate access to investigational drugs with the FDA.

The larger point missed throughout the whole debate is the role that drug manufacturers play.  The FDA takes the brunt of the criticism regarding access to investigational drugs, but their compassionate use program grants well over 90% of applicants access to investigational drugs.  Even if a terminally ill patient is granted the right to use an investigational drug, it is the drug manufacturer who must supply the drug, and they have the right to refuse any request.

Drug manufacturers have much more incentive to deny rather than grant access to an investigational drug: any negative results could derail the drug’s clinical trial process and ultimately render their drug, which requires hundreds of millions of dollars in investment for research, useless.  In addition, a significant percentage of drug manufacturers are small research companies working on a single drug; they often do not have the resources to donate their drug for compassionate use.  Finally, drug manufacturers would likely refuse to honor a patient’s request for a drug if it is based on a state’s Right to Try law, as the state Right to Try laws are preempted by existing federal regulations that govern access to investigational drugs, and there is no reason for a manufacturer to risk breaking federal law.

It is important to note that while there are numerous stories of the terminally ill using investigational drugs and improving in condition, there are no statistics available as to the success rate of the terminally ill being helped or cured by use of investigational drugs. Drug manufacturers can help improve access to investigational drugs by having clear policies regarding the use of investigational drugs, by advocating for a streamlining of the clinical trial process, and by predicting the potential demand of their drugs.  To make meaningful, effective change for the terminally ill will require cooperation and understanding between the three parties involved in this debate: the terminally ill, the FDA, and drug manufacturers.


Andrew Blumstein is an Articles/Comments Editor at the FIU Law Review. Mr. Blumstein can be reached at ablum010@fiu.edu.