The right-to-try legislation guaranteeing that the FDA cannot deny patients the right to try experimental drugs was both championed and criticized by many groups. The conservative/libertarian think tank, Goldwater Institute, has been in the forefront of those promoting the legislation. The institute drafted model legislation upon which 40 state right-to-try laws and the federal law is based.
Critics have pointed out that the legislation is unnecessary. The FDA already has a compassionate use program and grants 99% of the requests. The legislation is really about weakening the FDA some critics say.
Senator Ron Johnson (R-Wisc), who sponsored the bill, appeared to give credence to those who said the law's real intent was to weaken the FDA when we wrote to the FDA Commissioner that, “This law intends to diminish the FDA’s power over people’s lives, not increase it. ”
We thought it would be useful to examine the issues involved and get input from both sides. An obvious place to start was with the Goldwater Institute and Senator Ron Johnson. We sent them both several similar sets of questions inquiring about their views on the FDA.
Though Senator Johnson has yet to get back to us, Christina Sandefur of the Goldwater Institute took the time to provide answers to our questions laying out the Institute's stance on the relevant issues. Christina is an executive vice president with the Goldwater Institute and co-drafter of the right-to-try model legislation.
The questions and her answers are below. In a number of cases it seemed to us that the issues she discussed were more complex than the way they were presented. So in those cases, we have included additional information to help provide a more complete picture.
CP Q1: The Right to Try legislation (both state and federal) has been said by some to be a way to weaken the FDA's control over pharmaceutical development and patients' lives. Do you believe that is an important aspect of the legislation.
CS: Right to Try does not weaken the FDA’s control over pharmaceutical development at all. Right to Try does not change the drug approval process – treatments must still receive the same FDA approval before they can be offered to patients on the market. Right to Try doesn’t threaten the clinical trial process, either. Only patients who are unable to participate in a clinical trial are eligible for treatment under Right to Try. Right to Try simply extends to all patients with life-threatening illnesses the same option of trying investigational treatments that the FDA already allows to the fortunate few who qualify for clinical trials or who are granted Expanded Access, or those who have the means to travel overseas to get treatments that aren't approved in the US.
The right to try to save your own life is the most personal right we have. The Right to Try law simply says that the federal government doesn’t get a veto stamp over that decision when the patient, under a doctor's care, and the manufacturer all agree that a treatment might help.
CP additional information on this issue: It has been noted that the FDA does have a compassionate use program for patients that are not able to participate in clinical trials. The FDA approves 99% of the requests it receives via its compassionate use program. Usually it is companies that deny patients access to drugs in development, not the FDA. Critics of right-to-try legislation point out that right-to-try legislation does nothing to compel companies to provide experimental drugs to patients outside of clinical trials.
One complaint that was apparently previously valid about the FDA's compassionate use program was the substantial time and paperwork required to make a request. However, in 2016, The FDA introduced a streamlined form to reduce the procedural burdens on healthcare providers.
The form for streamlined submission is FDA form 3926. It can be downloaded from a link on this page.
The form itself is not too onerous and is two pages long. However it does require that an institutional review board (IRB) be responsible for initial and continuing review and approval of the treatment use, consistent with applicable FDA requirements. Requiring IRB approval is waived in cases of emergency request.
CP Q2: Can you say how you believe the FDA has too much power (if you do believe the agency has too much power)?
CS: At the Goldwater Institute, we believe that it was improper for the FDA to prohibit terminal patients and their doctors from making potentially lifesaving treatment decisions, especially when those same treatments are being offered to other patients in clinical trials.
Thousands of Americans every year suffer and die while treatments that could help them are tied up in red tape being tested not just for safety, but for efficacy. The system doesn’t provide fair or viable options for many terminal patients. Very few terminal patients are able to get enrolled in clinical trials – many are too sick (and some, ironically enough, are too healthy!). The so-called “compassionate use” exception is cumbersome and doctors say that the paperwork can be insurmountable. Dying patients don’t have that time to wait.
CP additional information on this issue: As previously noted, the FDA rarely prohibits patients from trying experimental drugs (99% of requests approved). Also trial sponsors, usually pharmaceutical companies, normally write patient eligibility requirements – not the FDA. The requirements can be narrow. There are justifiable reasons for this (such as protecting very sick patients from harm, making sure that the trial accurately determines the cause of adverse events, etc.). However, a number of doctors and researchers have protested that the requirements are too narrow and too often exclude patients with terminal diseases from trials that are their only hope. Again, companies are usually the ones creating these requirements.
Also previously mentioned, the FDA has streamlined the process for requesting compassionate use requests. Of course it is always possible that the process could be made even more efficient.
CP Q3: How would you like to see the FDA's powers reduced or changed? Can you give us any specific examples?
CS: When it was created over a century ago, the FDA focused on ensuring that products marketed to the public at large were safe and correctly labeled, so that patients had truthful information to make informed decisions about treatments. Over time, federal law shifted from a focus on empowering patients, to a more paternalistic approach – replacing the patient’s decision with that of the FDA’s. The ultimate question of what risks are acceptable to a terminally ill patient should be answered by that person, not the FDA. The decision should belong to the person whose life it is. That’s what Right to Try seeks to accomplish.
Sadly, federal regulations not only block patients from making treatment choices, they have taken away the tools patients and doctors need to make informed choices. Off-label treatment (when doctors use medicines for purposes other than what the FDA approved) is legal—Medicaid will even pay for it—but the government routinely censors the communication of valuable and truthful information about off-label uses that could help doctors and patients make informed decisions. Federal law strictly limits how pharmaceutical companies—which know the most about their drugs—can share information about legal off-label uses of their products. That censorship hurts patients, whose doctors may never be informed of the full range of treatment options.
Although the FDA is not authorized to regulate medical practice at all, its regulations that stop patients from making life-or-death decisions, or stop doctors from learning of important treatment options, imposes de facto regulation on the medical profession nationwide. That needs to stop.
CP additional information on this issue: Again it is rare that the FDA is preventing patients from trying experimental drugs – usually companies are not making the drugs available for experimental use. It is true that the FDA's role has expanded from its early days. In 1962 the Kefauver-Harris Amendment was signed into law. It established a framework that required drug manufacturers to prove scientifically that a medication was not only safe, but effective.
Some argue that the FDA should not be in the business of determining efficacy – only safety and proper labeling. However this seems to be a difficult position to support. If there were no efficacy requirements, would companies have incentive to exaggerate drugs' efficacy? Would any companies ever do this? Given that the FDA employs highly educated experts in medicine, statistics, bioinformatics, etc. and even they sometimes still make mistakes in both safety and efficacy decisions, are most patients equipped to evaluate drugs' effectiveness?
The discussion about off-label marketing and allowing companies to provide information about uses of drugs outside of labeling is an important one. Doctors are able to prescribe drugs to treat conditions other than those for which the drug is labeled. This is something everyone should want to continue. And of course doctors have to learn about off-label use from some source.
However, the question of what companies should be allowed to share is tricky. Should reps be allowed to share that, “Dr. Jones down the hall uses our drug off-label for X” be allowed? If studies are required for off-label use promotion, how many patients should be the minimum included in the study?
Companies do have a lot of monetary incentive to promote drugs off-label. Important issues of safety and expense are at stake in this discussion. After all, we will all be patients at some point, and we all pay for off-label use in one way or another. It makes sense that we allow information to be shared about off-label use but it also makes sense that the FDA (or some governing body) develop reasonable requirements for sharing off-label information and that they enforce the requirements. To assume that companies will be responsible in off-label marketing activities is to have an overly optimistic view of corporate morality.
CP Q4: What do you see as the FDA's proper role - if any?
CS: The FDA should teach, aid, and empower patients—not dictate to them the terms on which they will live, or not live, their lives.
CP Q5: Are you aware of any specific examples where the FDA's current requirements and oversight functions have resulted in harm to a patient or patients?
The clinical trial system can have unintended but horrific consequences. For example, Jenn McNary’s sons Austin and Max were diagnosed with Duchenne’s Muscular Dystrophy—an incurable, fatal, degenerative muscle disorder. Jenn tried to enroll both boys in a clinical trial for a promising treatment, but Austin’s disease had progressed too far for him to qualify. Jenn was forced to watch while one son’s condition improved significantly under treatment, and her other son’s condition worsened until he could no longer dress or use the restroom without help. 13-year-old Max became 16-year-old Austin’s caregiver.
And of course, despite the FDA’s risk-averse nature, under the current system, “unsafe” drugs still get FDA approval. For example, in 1997, the FDA removed fenfluramine after 25 years on the market when reports associated it with heart conditions. The FDA itself recognizes that “there is never 100% certainty when determining reasonable assurance of safety and effectiveness.” But how many patients have suffered and died because of a system that rewards delay, reduces incentives to innovate, and takes life-and-death decisions away from patients?
CP additional information on this issue: As previously mentioned patient eligibility for clinical trials is usually determined by trial companies (usually pharmaceutical companies), and not the FDA. In the specific case of the McNarys, the oldest son, Austin, was determined to be too sick to be eligible for a clinical trial that the younger was in. The McNarys’ neurologist petitioned the company developing the experimental drug on a compassionate use basis. The company, not the FDA, denied the request.
Austin's mother even spoke directly with the company CEO and asked why her son could not get the drug. The CEO explained that the company was small and cash strapped. It barely had enough supply for the children in the trial. It wouldn't be fair to make an exception in one case and not in others. The Mom was not initially satisfied and considered, in her words, “raising a huge stink.” However when she discussed the issue with Austin, he told her that he knew two kids who were sicker than he was. Was she going to get the drug for them also?
This changed her thinking and her plans. She joined forces with others and pressured the FDA to speed up approval. The FDA did dramatically speed up approval and even approved the drug against the recommendation of an outside panel. So it was by no means a simple case of the FDA preventing a child from participating in a clinical trial and then denying compassionate use of a potential life saving drug.
The Boston Globe has a good article on the specific case and broader surrounding issues. The article can be found here.
CP Q6: Would you like to see the FDA completely eliminated?
CS: We would like to see the FDA return to the agency it is supposed to be – an agency that empowers informed patients and their doctors to make their own decisions, rather than an agency that becomes the decisionmaker. Important improvements have been made - many prompted by the Right to Try movement. But there is more work to be done.
CP Q7: Does the Goldwater Institute have any additional legislation in mind that would be intended to reign in the FDA's role and or increase innovation in drug development?
CS: Yes. The Institute has crafted model legislation called the Free Speech in Medicine Act, which seeks to address the off-label speech issue I raised previously by restoring the right to freely exchange truthful information about legal treatments, providing doctors and patients with the tools they need to make informed healthcare decisions. So far, the law has passed in Arizona and Tennessee, both times with unanimous, bipartisan support. The Free Speech in Medicine Act enhances patients’ medical autonomy and helps increase access to essential healthcare. Sharing truthful information about off-label uses contributes to better-informed providers and better-served patients.
CP additional information on this issue: As previously discussed in question 8, it does make sense to allow sharing of information about off-label use. Should there be no restrictions on what companies can share, however? The language in the Arizona bill cited by Ms. Sandefur grants broad leeway to companies to share information. The information need only be “truthful.” The relevant section reads, “NOTWITHSTANDING ANY OTHER LAW, A PHARMACEUTICAL MANUFACTURER OR ITS REPRESENTATIVE MAY ENGAGE IN TRUTHFUL PROMOTION OF AN OFF-LABEL USE OF A DRUG, BIOLOGICAL PRODUCT OR DEVICE.” We will have to decide as a nation if we want to rely solely on companies (with enormous profit motive) to determine the meaning of “truthful.”
Ultimately, helping patients with life threatening illnesses gain access to experimental drugs is much more complex than most right-to-try proponents suggest. The FDA is rarely an impediment to reasonable compassionate use of experimental drugs. It is usually the companies developing the drugs that stands in the way.
Another serious impediment can be cost. Experimental drugs are not often covered by insurance companies. The cost can be thousands, tens of thousands or even more for treatment with experimental therapies. If we, as a society, are serious about giving seriously ill patients the option of trying last chance, experimental drugs, perhaps the government should create a fund for financing these options.
The fund could help alleviate the burden on companies who may be devoting precious resources to getting drugs approved as quickly as possible. It could also help remove an insurmountable obstacle many patients face when attempting to gain access to possible life-saving therapies. Of course then decisions would still have to be made about which patients are eligible, which therapies, etc.
In future articles, we will examine the views of other proponents of right-to-try legislation, opponents of the legislation, and other suggestions for improving FDA oversight and processes.
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