National Center for Health Research, March 23, 2018
We are writing to urge you to vote against the Right to Try bill that recently passed the House of Representatives (HR 5247) despite strong opposition from the Democratic leadership. We agree with the idea that terminally ill patients should have access to potentially life-saving medical treatments, and understand that some terminally ill patients are willing to take big risks to have a chance to live longer. 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.
That is supposed to be the goal of the federal Right to Try bill, but it fails. That is why four previous FDA Commissioners as well as the American Cancer Society; American Lung Association; National Physicians Alliance; American Society of Clinical Oncology (ASCO); Cystic Fibrosis Foundation; International Society for Stem Cell Research; National Consumers League; National Health Council; National Organization for Rare Disorders (NORD); Vietnam Veterans of America; and dozens of other patient and public health organizations oppose the bill, as we do.
We have spoken to families whose efforts to “try anything” made their loved ones’ remaining days miserable and left their families even more devastated. The House bill has a very loose definition of which patients would be eligible (since many patients with diabetes and heart disease have conditions that can cause irreversible damage that will cause premature death). In addition, it provides access to any drug that has passed Phase I clinical trials, which 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 than a terminally ill patient.
Another problem is that these very preliminary (Phase I) clinical trials usually include very small numbers of people, and do not study whether or not a medical product has any benefit at all. They are designed to determine the immediate risks on just a few healthy volunteers or patients. That is why 85% of drugs that pass Phase I clinical trials are never proven safe and effective and never approved by the FDA.
Fortunately, the FDA’s current Expanded Access program requires at least some evidence that an experimental treatment could potentially be helpful. The FDA uses compassionate waivers when doctors request them for very ill patients, and FDA agrees to such requests 99% of the time.
The GAO’s July 2017 report on the FDA’s current Compassionate Use/Expanded Access program pointed out that most experimental drugs that pharmaceutical companies distribute under that program eventually obtain FDA approval. That shows that the program is working: It gives patients earlier (usually free) access to experimental drugs that will eventually be proven safe and effective.
Although HR 5247 includes some potentially useful requirements that the results of patients’ access to experimental drugs be made available to the FDA, so that they will be aware of serious harm that could be caused, there is no enforcement mechanism to make sure that information is made available. In other words, even if a drug was found to be extremely dangerous when used by patients through the Right to Try program, that information might not be available to FDA, patients, or physicians.
We have included our Center’s Right to Try Fact Sheet below, which we hope you will find useful.
Jack Mitchell, Director of Health Policy
The Right to Try bill creates a program that is not as good as the existing FDA “Expanded Access” program, which has approved 99% of requests they received.
- FDA’s Expanded Access program makes sure that there is some evidence that the experimental drug is safe and effective. Most of the drugs that go to patients through this program are eventually approved by the FDA.
- Lowering the standards to drugs that completed “Phase 1 clinical trials” means that 85% of the drugs will never be proven safe and effective.
- When standards are that low, desperate patients can die sooner and more painfully than they would have otherwise.
- FDA physicians are available 24 hours a day to approve any emergency Expanded Access requests that the agency receives. They usually grant emergency requests immediately over the phone and non-emergency requests in an average of 4 days
- Pharmaceutical companies may choose to deny patients access to experimental drugs if there is not enough of the drug available or they are concerned about dangerous side effects. When a patient is denied access to an experimental treatment, it is almost always because the company has said no, not the FDA.
- State “right to try” laws do not give patients a “right” to try and have done little to expand access to investigational treatments. There is no evidence that anyone has obtained an investigational treatment via these laws that couldn’t have been obtained through FDA’s expanded access program.
- Right to try laws do not require companies to provide patients access to an experimental treatment. They only give the right to request the treatment from the company. Patients already have that right.
- The bills would weaken FDA’s ability to oversee dangerous side effects from the use of an experimental drug while protecting companies from law suits if the drugs are more harmful than the patients were informed.