COMMENTARY

Why 'Right-to-Try' Laws Won't Help Desperately Ill Patients

Arthur L. Caplan, PhD

Disclosures

June 19, 2014

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Hi. I am Art Caplan, from the Division of Medical Ethics at New York University's Langone Medical Center in New York City.

Should people who are terminally ill have the right to use whatever drug they may want in order to have a shot at saving their lives? This comes up again and again for people dying of cancer. It also can come up for people in late stages of diseases like Parkinson's and Alzheimer's, and even for people who are becoming blind from macular degeneration. There are many, many circumstances and conditions where people run out of what contemporary medicine can give them and want a chance at something that may be showing promise in animal studies or in early human trials. Should they have the right to get these treatments, regardless of what the US Food and Drug Administration (FDA) or anyone else thinks?

Some states are saying yes. Listening to the pleas of people on social media saying that they cannot get a drug, or that their child cannot get a drug, some states have responded by enacting laws that say every person should have a right to try experimental drugs, as long as they know what they are doing and as long as these drugs have undergone phase 1 trials, meaning that the drugs have been shown to be safe in a small group of people. Essentially, these states are saying that the FDA should get out of the way -- that there should not be restrictions on this kind of drug access. I suspect that by the end of this year, we may see as many as a dozen states with laws like this.

But Do the Laws Make Sense?

Do the laws make sense? I do not think they do. They are well intended. Their purpose is not bad, but I do not believe that these laws will achieve what the American people (and many treating physicians) may want to happen for people in desperate straits.

The FDA can be an obstacle; there is no doubt about that. But during the past decade or so, the FDA has instituted a number of pathways to speed up access to drugs for people who are terminally ill. The FDA has fast-track pathways and other streamlined processes by which drugs can get to market or into the hands of patients more quickly. Some of these processes take time. Some are not well known in terms of what patients, patient groups, or doctors need to do to get the paperwork to the FDA, although the Websites are out there and are clear about what to do. But there can be delays, and people who are dying often do not have that kind of time. Sadly, if you cannot get the process moving within 2 or 3 weeks, it may be too late.

The Real Source of the Problem

I do not believe that the FDA is the major source of the problem. The major problem is fairly straightforward: It is money. The problem is that even with these states passing laws, they have moved from a right-to-try to what I would call a right-to-beg. A small drug company with very little drug in its possession and few resources, perhaps only 30 or 40 employees, does not have the money to run an emergency program to provide a given drug to terminally ill people. Moreover, a small company may not even have a supply of drug that would allow the company to give it away without ruining attempts to get it through the clinical trials process. And once in a while, a company is making a drug that costs too much to give it away, unless someone pays for it. None of these state laws put any money behind the effort to give people a right to try.

Even the big companies may say that if hundreds of people are going to ask for a new cancer drug before it is approved, "It is going to cost too much; we will not do it." Or the veritable cost of giving the drug away is more than their investors or the stock market will support.

If we give people a right to try, then we have to move past worrying about what the FDA does to worrying about what the manufacturer does. Will the company make the drug available, and make it available for free? Can the company afford to run a program and keep tabs on who is applying and what happens to them? That takes money. Without money being budgeted, I do not believe that we will see drugs getting into the hands of those who are dying or becoming disabled very quickly. If that is our goal, it should be part of the health reform debate.

I am Art Caplan, at the Division of Medical Ethics at the NYU Langone Medical Center. Thanks for watching.

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