Right for Patients?
“Right to Try” legislation may have compassion at its heart, but it does not do enough to ensure patient protection.
Ellen Sigal |
“Right to Try” is a newly passed piece of US legislation that allows terminally ill patients to request access outside of clinical trials to experimental therapies that have completed Phase I testing, but have not been approved by the FDA. In the US, the main advocate for the law was the Goldwater Institute – a libertarian think tank that created the legal template used by many states.
Prior to this bill being passed, hundreds of patient advocacy organizations pushed back against it – and although the bill was signed into law in May 2018, we along with others remain concerned about the impact on patients (1). You may be thinking: but surely, if there is no hope for a patient, is an experimental treatment not worth trying? I think we can all agree that terminally ill patients should have access to promising experimental therapies when they have exhausted all other options. The concerns, however, centered around the need to ensure that patients were not harmed by potentially lethal side effects, as well as the importance of maintaining FDA oversight.
First and foremost – as with any legislation that impacts patients – my organization, Friends of Cancer Research, evaluated how Right to Try may actually affect patients. I admit that personal experience motivated me further: my sister, Gale, died after trying an experimental therapy to treat her breast cancer when she had exhausted all other options. I understand firsthand why people with terminal illness want to try whatever they can to help treat their disease, but prior to Right to Try there were already processes in place that helped achieve this. In my view, this legislation does not protect patients and does not provide any further guarantees for them to gain access.
The new law does not stipulate that drug developers have to provide the experimental therapy to patients. In fact, there is no evidence that Right to Try increases access to new drugs because there is no data on the subject, which is alarming as many states have already passed bills relating to this subject. The former process, which was overseen by the FDA, allowed patients to apply for an experimental therapy (the application process took one hour). The FDA then reviewed the request within 24 hours and the agency approved more than 99 percent of the compassionate use requests they received. However, the drug developers then decided if they would provide the drug. Right to Try does not change that process, but instead aims to remove the FDA’s role – in my view, a terrible and dangerous idea. Without FDA input, will we see a scenario where “snake oil salesman” try to take advantage of dying patients by peddling ineffective or even harmful therapies without scientific merit?
We are urging Congress to ensure experimental therapies are developed, made available under appropriate expanded access, and approved rapidly when they are proved to be safe and effective. And that includes increasing research support for the basic, translational, and clinical research conducted by the National Institutes of Health (NIH) and fully funding the FDA.
In the healthcare and pharma industries, we all share the same goal of helping patients. It is imperative that we keep safeguards in place for patients, which includes FDA maintaining its oversight over the compassionate use process. In any case, the new federal legislation only provides the illusion of helping patients, as it does not stipulate that drug developers have to comply with compassionate use requests and provide experimental therapies to patients. The legislation also does not address the real obstacle to expanded access: very small supplies of experimental therapies, a lack of staff and resources, and safety concerns. All of us in the patient advocacy community will be watching this issue closely, and hope that future changes to the law can be made to ensure patients are protected.
- Friends of Cancer Research, Letter to Mitch McConnell and Chuck Schumer (2018). Available at bit.ly/2BHZjYb. Last accessed August 24, 2018.