Posted on August 9, 2017 at 11:13 AM
by Craig Klugman, Ph.D.
Last week the U.S. Senate passed bill S. 204, the Trickett Wendler Right to Try Act of 2017. Trickett Wendler was a woman with ALS. The ALS association and her family lobbied Congress to support this bill to give all patients living with a terminal illness the “right” to purchase experimental drugs from pharmaceutical companies. Essentially, this bypasses the FDA’s compassionate use program. Instead of filing an application for FDA compassionate use (which the FDA approves 99% of the time), the patient asks the drug manufacturer directly. As I have written in this space before, such laws threaten public health efforts through the FDA to ensure safe and efficacious pharmaceuticals.
Currently, 37 states have such right-to-try laws and more have considered them. The push for these laws begins with the Barry Goldwater Institute, a libertarian think-tank, funded in part by the Koch brothers. Last month I had the surreal experience of debating two right-to-try supporters (one from a business school and one from the Goldwater Institute) about these laws which they base in a “right to self-medicate.” There were two of us against self-medication and two in favor of it. You can read the commentaries and responses of the debaters here.
In this essay, I do not plan to restate my arguments against right-to-try. You can read my thoughts on this policy here and here. Instead, this post will examine the tools of argumentation that my opponents employed in the debate. It is important to show their debate techniques so that we all have a better understanding of how to analyze claims to support right-to-try and to realize why better facts and better arguments are falling on deaf ears.
First, it is because I say it is. My debate opponents held that a “right to self-medicate” and “right to try” existed simply because they said it did. Philosophically, they pointed to John Stuart Mill who stated that the state should not control what drugs people take. They also elevated autonomy to the position of prime directive. In their expressed views, autonomy is not a guideline to self-deliberation or a prima facie principle, but rather is the moral law against which all action and policy must be measured. In their writing, the supporters stated that autonomy can rarely be abridged.
Second, dismiss any fact that support the other side. In my essay, I wrote that FDA review has saved lives and billions of dollars. My example is the classic public health case of thalidomide. Their response was not to disprove the facts or the argument but to say “They always do” use this example. What did the writer mean by saying “they?” Was she dividing “us” from “them” with them being Liberals? Scholars? Public health workers? Anyone who disagrees with her? The reference source for her rebuttal of this case is a piece on the website of the Reason Foundation, a libertarian think tank which is funded by the same sources as the Goldwater institution. This “article” is purely an opinion piece lacking scientific and scholarly rigor. When shown facts, my opponents threw personal attacks, dismissed the facts, and attempted to demonize the writer. However, facts trump opinion when it comes to argumentation and policy.
The problem with this dismissiveness is that thalidomide is not the only case of the FDA protecting the public.
- When TPA, a life-saving drug that breaks up stroke-causing clots was first brought to the FDA, the review panel initially rejected it after clinical trials showed that at presented dosing, the drug, often caused fatal bleeding in the brain and was more expensive than the current drug. As a result, the company re-tested the drug at a lower dose which was found to be effective and with fewer fatal side effects. At that point TPA was approved.
- In another example, the company and doctors who developed a new revolutionary diabetes drug credit the FDA with incentivizing them to create a unique drug that will change diabetes care.
- In a third example, the careful FDA review of a new Alzheimer’s drug is credited with saving Americans “as much as $100 billion” because trials showed the drug was ineffective. Without that testing, many people would have spent a lot of money to use a drug that did nothing.
Third, my opinions trump your data. After criticizing my use of the Thalidomide example, the opponents used the only example that has ever been pointed to as a right-to-try “success.” The problem, besides the irony, is that their example does not hold up to scrutiny. The only case ever used to support right-to-try is Dr. Delpassand’s treatment of cancer patients. However, this case has been disproven. Delpassand has published scientific articles on his clinical trials, but he has not published a single scientific article with his right-to-try results. In fact, discussions of his work only show 6 individuals stating that they found the treatment lifesaving. Where are the scientific articles? The peer-reviewed sources? In other words, where is the evidence? There is also the question of what is meant by “success?” Does that mean that these patients went into remission and survived for five years (the general measure of cancer treatment effectiveness)? Does it mean that patients lived for an extra month than was expected (even though doctors are notoriously poor at estimating life expectancy in people with cancer)? Did they suffer side effects? Was there any measured pain? Did the drugs offer a quality of life? No data is offered to support the claims.
Fourth, conflicts of interest are fine as long as it supports my perspective. Many of their quoted references are from other partisan websites funded by similar groups as their own. Even the golden example of Delpassand’s work is rife with conflicts of interest. In short, any situation where a doctor’s recommendations may be influenced by personal gain outside of the physician-patient relationship is considered unprofessional and unethical. Given that Delpassand’s work is the example trotted out as “proof” in right-to-try articles, I was surprised to learn that Dr. Delpassand, along with his wife, are the owners of the clinic where his clinical trials and right-to-try cases took place. He created his off-label treatment here and administered it to desperate patients. Thus, one must be suspicious about whether his recommendation to use these drugs was based on what was best for these patients—unapproved drugs—or what was best for himself. Even the suspicion of a conflict of interest is supposed to be avoided.
Fifth, history is to be rewritten or ignored. In one commentary, FDA responsibility for ensuring the efficacy of pharmaceuticals was dismissed because that responsibility was not part of the organization’s original charter. However, this “change” to the responsibilities of the agency took place 55 years ago. Federal agencies change names and missions all the time. The 1906 Pure Food and Drug Act that created what later became known as the FDA out of the Department of Chemistry has had three charges over its lifetime: accurate labeling, safety and efficacy. I hold that these are all related. One cannot have accurate labeling unless one knows that a drug is safe and efficacious. After all, how can there be a listing of dosing information and side effects without knowing what levels of use are safe and unsafe; without knowing what levels are effective. Basing an argument on a change in mission over a 110+ year history seems specious.
Sixth: When in doubt, make a personal attack. After I presented a philosophical argument that autonomy was not the only consideration in making policy and that sometimes the public good can limit or override individual choice, my opponents responded with, “who is the FDA, Klugman, or anyone else to deny these patients their choice?” Again, they show the high regard in which they hold autonomy but they also make a personal attack. Good argumentation would address the issues rather than attacking the individual.
Seventh: Policy should be based on my pure theory. One problem with right-to-try is that it assumes that every individual has access to a physician, access to medical journals, that no one is ever pressured into treatment or research, and that everyone should only get the care for which they can pay. Thus, when I pointed out that right-to-try violates notions of social justice, the response was that the individual has the right to do whatever he/she wants to her/himself. That would be true if everyone was equal and if everyone had access to care and money to pay for care. Perhaps the scariest thing in this whole debate was that for my opponents, our dialogue appeared not to be a philosophical game but rather that they were out to win: Their theory should be the only basis for policy.
The ideas behind good debate: logical arguments, reason, and good facts are important. These are the rules that allow for a civil discourse on issues upon which people may disagree. It is how as a society we can determine the best course of action among many. The problem is that there are people who believe these guidelines are irrelevant. Winning at all costs is the name of the new game and it does not matter how many pawns are sacrificed. In theory, right-to-try is interesting and could offer some ideas about altering our current system of pharmaceutical regulation. However, a pure market approach to pharmaceutical distribution sacrifices safety, accurate labeling, scientific approaches to treatment, and protecting the public from harm. In their winner-takes-all arena, there must be winners and losers. If right-to-try wins, then we all lose.