From time to time courts are called upon to determine whether or not desperate patients have a right to experimental treatments. In the US we are also currently in the middle of the spread of “right to try” laws from state to state. The issue has recently come up in Brazil over patient access to an experimental cancer drug, phosphoethanolamine.

This is an unfortunate issue, because over the last century experts have developed a thoughtful system that attempts to carefully balance patient autonomy, hope for very ill or terminally ill patients, patient safety, and the good of society. Now thoughtless legislators or misguided courts are attempting to bypass this system with predictably horrible results.

However, on a superficial emotional level it is much easier to persuade people to bypass the system than respect it.

Here are the issues:

First, most experimental drugs will eventually not work. They will either fail to produce a clinically relevant result, or they will cause more toxicity than benefit. Even drugs that are promising when studied in cell cultures or animals will often fail human trials. It has to be understood, then, that what we are talking about is giving patients access to drugs that probably won’t help them and may harm them.

Patients, however, want the hope that a new treatment provides and are often willing to take the risk. Terminally ill patients often feel they have nothing to lose, and that is a reasonable feeling.

Then again, if we are to make progress treating terminal diseases we need to do clinical research, and if we just give experimental drugs to ill patients, we’ll never figure out if they actually work. Anecdotes are simply not sufficient, even though they are frequently offered to argue that clinical trials are not necessary.

The current compromise, to meet all these needs as best as possible, is to offer experimental drugs in the context of clinical trials. These trials are carefully monitored, patients that are more likely to be harmed are often excluded, placebo arms are no larger than they have to be, and as soon as a statistically significant benefit emerges the trial can be stopped and the drug given to everyone.

Further, in a clinical trial, patients are treated for free, including all necessary treatment for complications of the experimental drug.

Right to try laws, as I stated, bypass this system. As is often the case, the laws are presented as favoring patients and patient freedom, but they are actually anti-patient. The laws currently spreading in the US, as David Gorski has thoroughly explain on Science-Based Medicine, allow patients to bypass clinical trials and gain access to experimental drugs. This means they bypass all of the protections.

What patients are being given the “right” to do, is pay for their experimental treatment and also be on the hook for any cost of treating complications. Insurance companies can refuse to pay for complications of an experimental drug obtained through such laws. Also, patients who often want to take advantage of such laws cannot get into clinical trials for some reason, likely because the treatment is too risk for them. Or if they can get into clinical trials, bypassing the trials makes it harder to do clinical research.

I also have concerns that such laws will be exploited by quacks to sell their false hope to desperate patients. The laws essentially create a right to sell for profit experimental drugs to desperate patients, without any of the safeguards provided by clinical trials. These are terrible anti-patient laws.

In Brazil these issues have played out with respect to an experimental drug for cancer. For some reason the chemist who was studying the drug thought it might be a treatment for cancer and started giving it to patients, without doing the requisite clinical trials. Once this happens, the genie is out of the bottle. Inevitably, no matter what the effect of the drug (even negative) those patients who survive will tout the effects of the treatment, and those patients who die will be strangely silent.

When the chemist retired, the university where he worked try to shut down the distribution of this drug, but then of course patients complained. The courts eventually forced the university to distribute the drug, against the testimony of medical experts.

Now a court in the Brazilian state of São Paulo reversed the previous court’s decision, saying the university can stop distribution. This only applies in São Paulo, but the university says that is about 80% of their orders.

Phosphoethanolamine does show promise in petri dish and animal studies, but so have thousands of other compounds. What this means is that the drug is appropriate for clinical trial in humans. That’s it.

Conclusion

The issue here I think is pretty clear – the system in place (prior to meddling by right-to-try-laws or misguided courts) makes a reasonable balance to patient hope, safety, proper treatment, and the needs of all future patients who would like to benefit from knowing whether the treatment actually works.

The system can always be tweaked. About a decade ago there was a debate about “compassionate” access to experimental drugs, which was considered a reasonable addition to the standard of care. Under compassionate use, a terminally ill patient with no other options can be given access to an experimental drug, under the supervision of a physician who can determine whether or not the treatment is at least reasonably safe for that patient.

The difference between “compassionate use” and “right-to-try” is all in the details. In the former patients are still protected and there is proper oversight. In the latter, all other concerns are subservient to alleged “freedom,” which in practice means that patient protections are thrown out, and patients can even be exploited by snake oil peddlers.

The bigger issue here is the willingness of politicians to interfere with the complex decisions of professionals, even over the advice of experts. Such actions are rarely in the public interest.