Health Freedom, Right to Try, and Informed Consent

Starting with the Terrible Twos, when toddlers hear the word “No” on a regular basis, people don’t like to be told they can’t do something. They often believe government regulations interfere with their rights. The government requires vaccinations for school attendance; parents complain that they should be able to make their own decisions about whether to vaccinate their children. The government requires that drugs be approved by the FDA before marketing; desperate patients dying of cancer complain that regulations are preventing them from getting the one new treatment that might just save their life.

Health Freedom

The “health freedom” argument is that everyone has the right to use whatever treatments they want, to control what goes into their bodies; and it’s none of the government’s business. But as Stephen Barrett and William Jarvis explained on Quackwatch:[i]

“Quacks use the concept of “health freedom” to divert attention away from themselves and toward victims of disease with whom we are naturally sympathetic. “These poor folks should have the freedom to choose whatever treatments they want,” cry the quacks—with crocodile tears. They want us to overlook two things. First, no one wants to be cheated, especially in matters of life and health. Victims of disease do not demand quack treatments because they want to exercise their “rights,” but because they have been deceived into thinking that they offer hope. Second, the laws against worthless nostrums are not directed against the victims of disease but at the promoters who attempt to exploit them.”

Vaccine refusers don’t recognize that the government has a duty to protect the welfare of children and to protect the population from vaccine-preventable diseases. They tend to think parental rights and personal preference should trump everything else.

People go to other countries to get stem-cell treatments that have not been tested, and cancer treatments like Laetrile and the Gonzalez protocol that have been tested and shown not to work. In a trial of pancreatic cancer, patients treated with the Gonzalez regime survived on average for 4.3 months; those using standard chemotherapy survived on average for 14 months and reported a better quality of life. But desperate patients still want to believe in these treatments and they insist it was wrong of the government to prohibit them in the US.

Right-to-try legislation

38 states already had right-to-try laws, and as of May 30, 2018 there is now a federal right-to-try law. It gives terminally ill patients the right to get experimental drug treatments that have not been approved by the FDA, that are still in clinical trials, as long as they have passed a Phase 1 trial, the first step in the Food and Drug Administration’s approval process. At first glance, it sounds like a good idea; but the devil is in the details.

The FDA already has an Expanded Use or Compassionate Use policy.[ii]It can be used when: 

  • Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
  • There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
  • Patient enrollment in a clinical trial is not possible.
  • Potential patient benefit justifies the potential risks of treatment.
  • Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication 

Over a ten-year period from 2005 to 2014, there were 9000 applications to use investigational drugs; 99% of these were approved. Emergency requests are usually granted immediately over the phone, and non-emergency requests are generally processed within a few days.

My colleagues on the Science-Based Medicine blog, cancer researcher/surgeon David Gorski and lawyer Jann Bellamy, have covered this legislation extensively and have pointed out a number of problems:

  • It cuts the FDA out of the loop, so it doesn’t have the opportunity to offer other options that might be safer or to give the patient what information it has about possible side effects.
  • It removes the right to sue: doctors and drug companies are not liable for any harm that might occur to patients under this program.
  • There is no oversight by an Institutional Review Board (IRB).
  • It assumes that it is safe to try drugs that have passed phase I trials; these trials only involve a few patients and can’t establish drug safety. After drugs pass phase I testing, there are still high odds that they don’t work and aren’t safe.
  • It forbids the FDA from using any information about deaths or other harms to patients under this program from being considered in its deliberations on approving the drug. 
  • It exposes patients to exploitation.
  • It is a foot in the door to furthering undermining the authority of the FDA.
  • The drug companies are not obligated to provide the drug, but they have no incentive not to. They can charge the patient whatever they want, they incur no liability, and if the drug kills the patient, the FDA can’t use that information to delay or stop approval of the drug.
  • Insurance companies don’t pay for experimental treatments; drug manufacturers can charge patients whatever they choose. Less well-to-do patients will either forgo treatment or deplete their finances, depriving their survivorsof an inheritance. 
  • Terminal patients who have “nothing to lose” actually do have something to lose: money, quality of life, time with loved ones. 

The origin of right-to-try

David Gorski calls right-to-try laws “a cruel sham and scam.” He says they were concocted by “the quackery-friendly for-profit hospital chain the Cancer Treatment Centers of America and foisted on gullible legislators by the Goldwater Institute, a libertarian propaganda group disguised as a think tank.[iii]It was the Goldwater Institute that came up with the name “right-to-try,” wrote model legislation, and promoted it to state legislatures. 

The profiteering begins

The new law allows drug companies to make money by selling unproven therapies to desperate patients. Unscrupulous drug companies will be incentivized to skip the traditional clinical trial and FDA review process altogether.[iv]  BrainStorm Cell 

Therapeutics is already planning to take advantage, offering stem cell treatments to patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). It plans to charge $300,000 for each NurOwn treatment. In the company’s own phase 2 trial, NurOwn failed to slow the progression of ALS compared to a placebo. They nevertheless embarked on a Phase 3 trial. Without waiting for trial results, they decided to offer the treatment to patients under right-to-try because of “intense demand from patients who have no other options.” 

Informed consent

One of the major principles of medical ethics is autonomy, the right of the patient to self-determination. In earlier days, doctors were paternalistic. They decided what was best and ordered whatever treatment they chose. They were like parents saying, “You will eat your broccoli because I say so. Mommy knows best.” Those days are long gone. Today patients have the right to make decisions about their own health and to refuse treatment, even essential life-saving treatment, as long as they are of sound mind. Consent to treatment is meaningless unless it is informed consent.  Patients must be given adequate information and must be able to understand the diagnosis, what the proposed treatment will entail, the risks and benefits of the treatment, the consequences of not treating, and whether any other treatment options are available. 

Informed consent is mandated by both ethics and law. No one has the right to even touch, much less treat, another person without consent; such actions can be prosecuted as physical assault or battery. Obtaining consent is considered a must for anything other than a routine physical examination.[v]Entering the doctor’s office and expressing a problem can be considered implied consent for a physical examination. Intimate examination (especially in a female), photography, and any invasive test or risky procedure requires specific expressed consent, which can be either oral or written. Written consent is preferable. Doctors are expected to document in the patient’s chart that they have discussed all the pertinent facts with the patient; and for surgery, the patient is required to sign an informed consent form.  

In everyday practice, informed consent is not always honored. Doctors may not take the time to explain everything in detail. They may influence the patient’s decision by their language or by the way they present the facts or omit some information. Patients regularly sign surgical consent forms without reading them. 

I worry that under the right-to-try law, the patient may not fully understand the risks and the low probability of success, and drug companies may take advantage to sell their products to desperate people at inflated prices. I worry that since these patients weren’t able to qualify for clinical trials, they may be less healthy and more likely to suffer problems with the experimental treatments than those who enroll in the trials; they may have other medical conditions or factors that make them more susceptible to complications.

Conclusion

No reasonable person wants health freedom without information. Imagine a world without laws, where patients are free to use any treatment any quack or snake oil salesman might invent, where patients can’t give informed consent and have no way of knowing what they are getting into. We rely on laws to protect us from contaminated and adulterated foods. We rely on the FDA to protect us from dangerous drugs and disasters like Thalidomide. 

As Jann Bellamy summarized:

  “the claims that “potentially lifesaving” drugs will actually benefit terminal patients are overblown, the risks underappreciated, and the procedures for obtaining unapproved drugs inadequate to protect patients from adverse consequences, both medical and financial.”


[i]https://www.quackwatch.org/01QuackeryRelatedTopics/hfreedom.html

[ii]https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm

[iii]https://respectfulinsolence.com/2018/06/22/as-i-predicted-the-exploitation-of-patients-under-right-to-try-begins/

[iv]https://www.statnews.com/2018/06/20/right-to-try-opportunism/

[v]https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2840885/

This article was originally published as a SkepDoc column in Skeptic magazine.

Dr. Hall is a contributing editor to both Skeptic magazine and the Skeptical Inquirer. She is a weekly contributor to the Science-Based Medicine Blog and is one of its editors. She has also contributed to Quackwatch and to a number of other respected journals and publications. She is the author of Women Aren’t Supposed to Fly: The Memoirs of a Female Flight Surgeon and co-author of the textbook, Consumer Health: A Guide to Intelligent Decisions.