National Center for Health Research, February 22, 2018
Dear Members of the House Energy and Commerce Committee,
As a research center that advocates for the best medical treatments for all Americans, we strongly believe that terminally ill patients should have access to potentially life-saving medical treatments. Some terminally ill patients are willing to take big risks to have a chance to live longer, and if they want access to experimental treatments that are undergoing clinical trials, they should be able to do so as long as they are well informed of the risks as well as the possible benefits.
Unfortunately, many of us know desperate patients whose efforts to “try anything” made their remaining days miserable and left their families even more devastated. What can and should Congress do to make sure that desperate patients won’t be exploited, or suffer even more painful deaths as a result of legislation? That is the key question as you consider Right to Try legislation.
A key issue for Congress to consider is whether legislation should provide access to experimental treatments that have been in only one or two preliminary clinical trials. The earliest clinical trials (known as Phase I) often don’t include even one patient. Instead Phase I trials can include “healthy volunteers,” such as college students, who are much less likely to be harmed by an experimental drug or device than a terminally ill patient would.
In addition, these first (Phase I) clinical trials study very small numbers of people, and do not study whether or not a medical product works. They are designed to determine the immediate risks on just a few healthy volunteers or patients. Since so few people are studied, even if a treatment is immediately and painfully fatal to 5-10% of patients, for example, these first clinical trials probably would not be able to provide that crucial information.
There are several Right to Try bills under consideration. For example, the Right to Try bill introduced by Representatives Griffith and Brat (HR 1020) does not even require that a first (Phase 1) clinical trial be completed – it can have just started. In other words, the experimental treatment could be fatal to many patients and it would be impossible to know that, or to warn patients about it. That bill also completely restricts any oversight by FDA, and prohibits the agency from requiring the collection or disclosure of any information generated by the experimental drug therapy. This lack of disclosure is especially problematic since the bill would protect manufacturers and suppliers of such drugs from liability except in cases of “gross negligence or willful misconduct.”
The Johnson Right to Try bill (S 204) requires that a Phase I clinical trial be completed. That is an improvement over the Griffith and Brat bill, but 85% of drugs that successfully complete Phase I clinical trials are later found to be unsafe or ineffective and are therefore never sold in the U.S. or other countries. So neither of these Right to Try bills would help most patients, and could potentially be fatal to many patients. The Senate bill also restricts liability to any participant, except instances of “reckless or willful misconduct, gross negligence, or an intentional tort under any applicable State law.” The bill directs that an annual summary of the use of any such experimental drug be provided to the HSS Secretary’s Office for later posting on the FDA web site.
In contrast, the FDA’s current Expanded Access program requires at least some evidence that an experimental treatment might possibly be helpful. That’s not a very restrictive safeguard, but it helps protect many patients. The FDA routinely utilizes what the agency terms “compassionate waivers” for very ill patients when doctors request them, and FDA grants such requests 99% of the time.
Another important issue for Congress to consider is whether these bills would exploit patients financially. The experimental drugs provided through the current FDA Expanded Access program are provided for free most of the time, or “at cost.” Clinical trials also usually provide experimental treatments for free. The Johnson Senate bill also protects patients from financial exploitation by limiting what experimental treatments can cost.
The Griffith and Brat bill does not allow restrictions on the cost of experimental drugs, thus by such omission allowing companies to charge whatever they want to dying patients desperate for access to any experimental drug or device – even one that has absolutely no evidence that it is either safe or effective. That means that desperate patients could be required to pay exorbitant fees for the “Right to Try” to be treated like guinea pigs. That is unfair to families that do pay for these experimental treatments and it is also likely to induce tremendous guilt for families that could not afford to do so.
FDA’s Compassionate Use program could be improved, and improvements are already underway thanks to the Navigator program that the FDA has recently initiated with the Congressionally- created Reagan Udall Foundation. However, with or without Right to Try legislation, access to experimental drugs will also be limited if patients want drugs that are not yet being manufactured in large numbers, or if reputable pharmaceutical companies are reluctant to provide drugs that they fear will be harmful to patients who are too ill to benefit.
The GAO’s July 2017 report on the FDA’s current Compassionate Use/Expanded Access program was generally supportive, with a few recommendations for improvement. Importantly, GAO pointed out that most experimental drugs that pharmaceutical companies allow to be distributed under FDA’s Expanded Access eventually obtain FDA approval. In other words, the program is doing what was intended – giving patients earlier (usually free) access to experimental drugs that will eventually be proven safe and effective.
In addition to harming individual patients, Right to Try legislation that makes unsafe treatments available for sale harms our entire drug development enterprise, by eliminating the incentive for patients to participate in clinical trials that would help millions of patients in the future. If HR 1020 was to become law, then it is likely that the richest patients will buy access to experimental treatments and only the middle-class and low-income patients will participate in clinical trials.
Reputable companies would continue to study new drugs and devices in clinical trials, but progress would be slowed because of difficulty attracting enough patients to participate in clinical trials. Meanwhile, scam artists and fly-by-night companies would be motivated to make as much money as possible on dangerous or worthless experimental drugs for as long as they are available, and HR 1020 and would make it impossible to gather information about how dangerous or ineffective the experimental drugs might be unless the sponsoring company volunteers that information.
It is well documented that unproven treatments have been sold to dying patients at outrageously high prices in Mexico and elsewhere, and many patients have been irreparably harmed or killed by unproven treatments that were marketed dishonestly. Indeed, FDA was created to avoid the tragedies arising from the “right to try” unregulated medical sales of the 19th Century and early 20th Century.
To improve Right to Try legislation, Congress should:
- Ensure that experimental treatments cannot be sold at a profit by companies or medical professionals;
- Ensure that all experimental treatments have been proven safe in completed Phase I or Phase II trials conducted on a reasonable number of patients (not healthy volunteers);
- All experimental drugs and devices available through RTT should also be studied in clinical trials as part of FDA’s regulatory process;
- Information about harmful side effects and adverse events should be required to be reported to the FDA by the physicians.
We’d be glad to provide additional information upon request. The National Center for Health Research is a non-profit organization which analyzes medical and scientific data and produces original health-related research to inform patients, the general public, and policymakers. We advocate for patients and consumers to have access to safe, effective, and affordable drugs and medical devices. We accept no funding from the pharmaceutical or medical device industries.
Diana Zuckerman, Ph.D.