July 31, 2017
The time is at hand for a momentous decision in the Senate which could greatly affect how patients get access to experimental drugs. We are writing as members of the Patient, Consumer, and Public Health Coalition, representing millions of Americans, because we are very concerned about the controversial legislative measure that Senator Ron Johnson (R-WI) is trying to insert as an amendment to the Senate version of the FDA’s user fee legislation. The User Fee bill must pass soon to avoid mandatory layoffs at the agency.
This bill, S. 204, the Trickett Wendler Right To Try Act of 2017, purports to help dying patients gain access to experimental treatments that can save their lives. However, patients already have access to the FDA’s Expanded Access Program, which gives patients compassionate access to experimental drugs if their doctor believes it is appropriate and the company which makes the drug agrees to provide it for that use.
The Expanded Access program is not a restrictive program; FDA grants these requests more than 98% of the time. A recent GAO report affirmed the FDA’s appropriate handling of their Expanded Access Program and outlined agency efforts to improve it and streamline its requirements for eligibility. Also, please keep in mind that the experimental drugs provided through the FDA program are almost always free. In contrast, Sen, Johnson’s proposal would remove FDA from the process, allow companies to charge whatever they want, not allow FDA to consider any harm that the experimental drug causes when used under the Right To Try auspices, and thus eliminate the safeguards and monitoring that otherwise are done to determine whether the patient has been helped or harmed by the experimental drug.
We strongly support the need for terminally ill patients to receive the best medical treatments as quickly as possible. Unfortunately, the right to try approach of this legislation would have the following dangerous outcomes:
➢ Hinder patients from obtaining the experimental drugs that are more likely to help them.
➢ Enable companies to charge extremely high prices for experimental drugs, instead of providing them for free in clinical trials.
➢ Reduce or eliminate the incentive of pharmaceutical companies to perform scientific studies to prove that their experimental medications are safe and effective, and for whom. Why would drug companies agree to spend time and money researching their product when they can instead spend that money on marketing to desperate patients?
➢ The resulting lack of patients participating in clinical trials for these experimental drugs could make it difficult, if not impossible to determine if the medication will make the patient feel better or live longer, and what dosage the patient should take.
➢ Make it nearly impossible to prove if the risk outweigh the benefits for these drugs dispensed without any oversight.
A new study published last week in BioMed Central found that 76% of the drugs provided through FDA’s current compassionate use program eventually received FDA approval. This indicates that while 3 out of 4 experimental drugs being made available through the current program have benefits that outweigh their risks for many (though not all) patients, one out of every four is not. Since the loosening of safeguards in the proposed Right to Try law would lower those odds, probably substantially, study authors bioethicist Dr. Arthur Caplan and his colleagues concluded, “allow the FDA to retain its oversight and approval role for these programs, in order to help mitigate safety risks for patients.”
There is no scientific evidence that Right To Try programs already operating in a number of states are helping patients survive longer or have a better quality of life. Nobody has studied how many patients were harmed by these programs, and the proposed legislation would not allow such studies either. Desperate patients will be vulnerable to false or misleading promises. Drug companies which now agree to supply free experimental drugs under FDA’s Expanded Access Program may refuse to do so when there is no oversight.
The proposed law would potentially harm many patients and family members, including seriously ill patients who could be harmed by scam artists or unconventional medical practitioners who will take advantage of them. This legislation could open the floodgates to undermine the scientific and carefully-monitored medical requirements that made FDA approval the gold standard for the world. Please oppose this amendment or any other efforts to have this legislation become law.
National Center for Health Research
Breast Cancer Action
Breast Cancer Consortium
Jacobs Institute of Women’s Health
MRSA Survivors’ Network
National Consumers League (NCL)
National Organization for Women (NOW)
National Physicians Alliance (NPA)
National Women’s Health Network (NWHN)
Our Bodies Ourselves
Treatment Action Group (TAG)
Washington Advocates for Patient Safety (WAPS)
For more information, please contact Jack Mitchell at email@example.com.