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03/11/2015

“Right to Try” Laws, Compassionate Use, and Adverse Events: A Second Cautionary Tale

by Alison, Bateman-House, Ph.D., MPH

In 2014, Colorado became the first state to debate and then pass a so-called “Right to Try” law. In early 2015, there are now five states that have such laws: Colorado, Michigan, Louisiana, Arizona, and Missouri. The Utah and Virginia state legislatures have passed their own right to try laws, both of which await signature by their respective governors, and over twenty other states are actively debating right to try legislation.

These right to try state laws are based on a model bill that was circulated by the Goldwater Institute, a libertarian think tank based in Arizona. According to the Goldwater Institute, the U.S. Food and Drug Agency’s regulations for new drugs take a toll on terminally ill Americans, a number of whom die before they can get access to potentially lifesaving experimental treatments. Thus, the model law cuts the FDA out of decisions about whether patients may be granted access to experimental treatments. Under right to try laws, a patient may ask the company developing a drug or device to make that experimental medical product available to her. The company does not have to provide it, and if the company does decide to provide the patient with the experimental drug or device, they may charge her for it (and her insurance company has no obligation to pay for it). But, if the patient and the company come to a mutually agreeable decision, there is no need to seek FDA approval of their plan. This is a controversial change in that such decisions were previously understood to be regulated by the FDA under its federal legal mandate. Right to try laws thus entail an individual state claiming for itself authority over compassionate use.

Supporters of right to try legislation often invoke emotionally charged stories, real or apocryphal, to explain why the laws are needed. In these stories, a person – a child, a parent, a newlywed – is dying and learns of an experimental drug that might offer salvation. The patient invokes her state’s right to try law and obtains access to the drug. Hopefully the drug works, rescuing the patient from otherwise certain death. However, whether the drug works or not, the patient was given the oh-so American right to try to preserve her own life.

Right to try laws can be criticized on numerous grounds. Citing the fact that states are claiming for themselves authority that was already granted to the FDA, one could claim that the laws are unconstitutional. Citing the fact that the new laws offer not a right to try but rather a right to ask for access in order to try, one could claim that the laws are misleading and will generate false hope in patients.

Furthermore, citing Thomas Duncan’s case, one can argue that there is no need for the laws. Duncan’s physicians did not need a right to try law in order to ask Chimerix to let them try Brincidofovir. Rather, they used the existing, federal rules that permit “compassionate use” of investigational medical products. Contrary to the narrative put forth by the Goldwater Institute, this request did not sit in regulatory purgatory: rather, both the company and the FDA acted expeditiously, and Duncan got the experimental drug quickly.

Despite such objections, the new laws are popular, and no state legislature that has considered a right to try bill has rejected it. Americans believe in self-determination, and we support the idea that a patient ought be able to choose what level of risk to take in the final chapter of life. Some terminally ill patients are willing to risk the long odds of taking an experimental drug, and this decision is theirs to make. What has not been articulated, however, in the right to try debate is that individual patient’s outcomes have very real implications for other patients. When a pharmaceutical company declines to make an experimental drug available to a patient, it may be acting on the belief that it is not safe for that patient to use the drug. Equally likely, however, the company is seeking to protect the drug in development from negative repercussions should the individual’s case turn out poorly. Certainly such a decision protects the company’s multiyear financial investment in the drug. This decision also benefits the unidentified, faceless population of patients who will one day use the drug, should it be approved for use.

That drug developers would choose to prioritize populations over individuals is not surprising. After all, drug development is a business of populations: in the quest to develop treatments, humankind is carved into groups along the lines of age, sex, genetic markers, hormone receptors, etc. Pharmaceutical companies do not make individual treatment decisions: rather, they say on their product labels what populations and what indications the drugs or devices are intended to treat. More cynically, pharmaceutical companies understand that their profit is derived from populations, not individual patients.

Given this context, it is surprising that many pharmaceutical companies choose to make their investigational medical products available to patients on the grounds of compassionate use. It would have been completely reasonable for Chimerix to decline to make Brincidofovir available to Thomas Duncan. It seems likely that Chimerix executives decided to make the drug available thinking that if Duncan recovered the company would benefit and that if Duncan’s case went poorly, the company would not be much affected: after all, it was not testing the drug as an Ebola treatment but rather as a treatment for other viruses such as cytomegalovirus and adenovirus. This assessment proved overly optimistic: as described previously, Duncan’s death precipitated a steep drop in the company’s share price, an event that could have been fatal for a different company. (It would be interesting to know whether Chimerix’s stock price drop was viewed in board rooms around the world as evidence that compassionate use was potentially more trouble than could be tolerated: the dramatic dip was certainly covered by the pharmaceutical and business press.)

Americans love the idea of rescue, whether it is for a child who fell down a well, a kitten trapped in a walled-up building, or miners caught in a mine collapse. Granting patients access to investigational drugs or devices offers a chance that they can be “rescued” from their medical problems. Advocates of right to try legislation hit upon this rescue theme, focusing on one patient and her no-stone-left-unturned quest to stay alive. In contrast, the drug development story told here focuses on how the experience of one patient has ramifications for other patients. The political environment in which right to try laws are being debated and enacted focuses on the needs of specific, identified patients while ignoring the claims of a larger number of unidentified patients. This makes sense in that stories of individual patients seeking rescue from illness or disease tug on the heartstrings the way that speaking of a population of future patients never will. But if you ask patients whose best hope for an effective treatment was dashed when the development of an apparently-promising drug was halted, I am certain that they would say that no individual’s right to try should come at the expense of another’s.

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