'Right to Try' When Hope Dims, the Case for Unproven Drugs

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Imagine this scenario: The clock is ticking and it s a desperate race against time to save your life or that of a loved one. There may be a life-saving therapy that has not undergone the rigorous approval process by the Food and Drug Administration, and by the time it is obtained it may already be too late. That begs the question: In this situation, should an unapproved drug be used in the chance that it may prove effective, or life saving?

And there are other questions to consider: If the process of trying an unapproved drug is made easier, will more people be harmed than helped? Will we all be headed down a slippery slope?

There is never going to be such a thing as an absolute right or wrong answer in philosophical or ethical debates. The best that one can hope for is to be reasonably sure that the benefits outweigh the risks/harm.

The FDA has had legislation in place since 1987 that enables individuals with terminal diseases to obtain experimental medications/treatments from pharmaceutical companies through their expanded access system, also known as compassionate use.

Now, some states have crafted and adopted their own legislation called Right to Try, that bypasses the federal government and places more of the decision-making power in the hands of patients and their physicians. Some may disagree with this move for many different reasons, one of them summed up in this question: Why would states create legislation that is redundant?

However, RTT laws are not redundant at all, as they generally aim to permit patient access to an investigational treatment if: (1) the patient is terminally ill; (2) a physician recommends use of the treatment; (3) the patient provides informed consent; and (4) the treatment has completed a Phase I clinical safety/dose limitation trial. Both manufacturers and physicians would receive liability protection against claims arising from adverse events caused by the experimental treatments.

The answer to that is obvious time. Having to apply for compassionate use means dealing with the federal government which is a lengthy and time consuming process. Although the FDA is required to review compassionate use requests within 30 days (sometimes it takes even less time) the average time required on the part of the treating physician is 100 hours to complete the requisite paperwork. While there are some doctors who certainly go above and beyond to help their patients, in reality, how many will avoid that burden?

The FDA has claimed that it will reduce the application time down to 45 minutes, however this hasn't yet been implemented. Even in the absence of additional state RTT laws, it seems clear that the FDA has the ability to move faster. Perhaps this is the nudge that the agency needs.

FDA s application process requires also that the treating physician start the application process, not the patient. This takes away the autonomy of the patient to advocate for him or herself. Under the given circumstances, this presents another grievous hurdle for the patient to jump over, especially given that they are completely reliant on their doctor to dedicate that quantity of time.

In bypassing the FDA s approval process, the patient, out of desperation, could be subjecting him or herself to more pain and suffering. It is important to keep in mind that the individual asking for experimental therapies has already done their homework and exhausted all treatment options. There is no fear of potential adverse side effects because the patient is already facing imminent death. That is the sad truth. What they have to lose is minimal in comparison to what they could gain extending their life, or best case scenario, saving it.

RTT laws require that experimental treatments have to have at least gone through Phase I clinical trials. This is the phase of drug development where the trials are performed on a small population of human subjects (after it has undergone extensive animal testing) to evaluate its safety, set dosage range and check for side effects. The treatment in question is not born in a vacuum. It has shown promise and has made it as far as the first phase of clinical trials that is an acceptable alternative to someone who has no options left.

Additionally, how many people subject themselves to quackery under such desperate circumstances. Everyone has heard horror stories and watched documentaries of patients with incurable diseases, i.e. ALS, who have trekked down to Mexico for experimental quack treatments. That is a far more insidious and dangerous scenario. With RTT, there is still some accountability and transparency.

Pharmaceutical companies may be reluctant to participate in the compassionate use program, given that all adverse side effects would have to be documented on these drugs that haven t hit the market yet. These treatments are being given to the sickest members of the population and their reactions may be much more pronounced than a study population. Limiting that liability would make manufacturers more willing to participate in RTT (this is a limiting factor with FDA s expanded access).

There is also argument that RTT could negatively impact clinical trials. Considering most of the persons applying for an experimental treatment are individuals or small groups there is no reason to think that the manufacturer could not proceed with a clinical trial.

The point that is most difficult to reconcile is equity. How is one person able to have access to potential treatment simply on the basis of their socioeconomic status? In reality, only those with the most resources will be able to advocate for themselves and their families and this creates a major disparity in terms of access. This may be irreconcilable but it is not reason enough by itself to deny anyone potentially life-saving treatments.

Is RTT better than the FDA's compassionate use clause? Maybe. Is this a better solution to a rather awful situation probably. The last thing anyone wants is to open up Pandora s Box and have everyone clamoring for experimental drugs. In reality, that will likely never happen, since we are talking about individuals and small groups of people.

If you strip away everything else, the real issue is the potential to save a life, a life that is tragically close to a premature end.