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STAT – ‘Right-to-try’ law threatens patient safety and rational drug development

STAT – ‘Right-to-try’ law threatens patient safety and rational drug development

In President Trump’s first State of the Union address, he challenged Congress to give people who are terminally ill immediate access to experimental treatments without going through the FDA. A number of states have already passed such “right-to-try” laws. The Senate has passed a federal version, and a revised version is now under consideration in the House, with a vote likely on Tuesday.


Backers of the right-to-try law would have you believe that the FDA is barring the distribution of all sorts of miracle cures. But many experimental treatments turn out to be totally ineffective. And as it stands, the FDA approves more than 99 percent of the thousand-plus applications it gets each year — usually within days of receiving the application — for access to experimental therapies through an existing program called expanded access or compassionate use.


Everyone wants to help those suffering from terminal illness. But rather than save lives, a right-to-try law would give patients false and fleeting hope, threaten patient safety, and impede the approval of therapies that are safe and effective. The House Energy and Commerce Committee has shown its commitment to terminally ill patients by working to make some important changes to the Senate right-to-try bill. Yet even the House proposal retains key elements that will undoubtedly be harmful.


Right-to-try legislation would create a pathway to remove FDA oversight of experimental, unproven therapies. These are the treatments that most need rigorous regulatory oversight tempered by willingness to allow pre-approval access for patients who need them most: those who have run out of treatment options. Excluding the FDA from the development and delivery of these therapies will make it harder to gauge their safety and effectiveness.

Advocates like us have spent decades fighting to accelerate the pace of scientific discovery and the development of new treatments, with patient safety and effectiveness as the North Star. We have each seen firsthand the perils of disease and the anguish of loved ones who have run out of options to fight deadly diseases like cancer or AIDS. But we cannot allow this pain, and our unwavering commitment to end suffering, blind our judgment about legislation that would create a climate where the most vulnerable patients are taken advantage of, their safety endangered, and drug development driven off track.


During the peak of the AIDS crisis, with 50,000 Americans dying from the disease each year, brave and determined activists pushed the FDA to increase regulatory flexibility — creating a variety of mechanisms that brought patients faster access to lifesaving therapies, therapies that are now saving millions of lives around the world and preventing new HIV infections. Cancer advocates have also long worked side-by-side with the FDA and Congress and continue to successfully enact innovative policy changes and expedited approval mechanisms such as Accelerated Approval and the Breakthrough Therapy designation. These changes have made a significant and lasting difference for patients.


If the right-to-try legislation can’t speed expanded access by circumventing FDA review, then what would it do? One result is certain: It would create uncertainty. In the 38 states that have passed similar bills, there is no evidence so far showing if these policies have increased patient access to experimental therapies. The absence of data here is both alarming and telling.


The latest right-to-try legislation would not address the real obstacles to expanded access: small supplies of experimental therapies along with lack of staff and resources, as well as safety concerns. In addition, nothing in the proposed legislation would compel a company to provide access to an experimental treatment.


We share the same goal as some advocates for the federal right-to-try law — saving and lengthening lives. But their approach, no matter how well intentioned, will not remedy the challenge. Despite recent modifications by the House Energy and Commerce committee to add some additional protections, this bill still creates a dangerous precedent that would threaten both research and patient safety by removing the FDA from any pre-notification of the use of unapproved therapies.


We urge Congress to ensure that experimental therapies are developed, made available under appropriate expanded access, and approved rapidly when proved safe and effective. That means increasing research support for the basic, translational, and clinical research conducted by the National Institutes of Health. It also means fully funding the FDA, which is chronically understaffed and under-resourced. Only then can these vital organizations fulfill their mission to protect Americans from dangerous or ineffective treatments while speeding research, access, and approval.


This is the safe and sustainable path that gives patients not a right to try but a true chance to succeed.…