In a bizarre twist for the newly signed “right-to-try” law, the FDA — cut out of the process of signing off on certain uses of experimental drugs for terminally ill patients by that law — now has to figure out how to implement it. And already, the bill’s main author is unhappy with commissioner Scott Gottlieb in the aftermath and has pointedly made his feelings known in a letter.
“This law intends to diminish the FDA’s power over people’s lives, not increase it,” Senator Ron Johnson, Republican from Wisconsin, wrote to Gottlieb yesterday, displeased over tweets and statements the commissioner has made recently around implementation of the law. The statement is remarkable because the issue has long been framed by “right-to-try” supporters as a win for terminally ill patients and not an end-around to weaken the FDA.
As we have reported, “right-to-try” is a landmark win for the libertarian Goldwater Institute and its political allies.
The FDA says it will review and directly reply to Senator Johnson’s letter.
Before the senator’s letter was published yesterday, the agency said in an emailed statement that it “is convening an internal group to assess how to effectively and efficiently implement the new law. We will report on our implementation steps regularly.”
In an email to FDA’s Center for Drug Evaluation and Research (CDER) staff yesterday, CDER Director Janet Woodcock said: “We are reviewing the legislation and will work to implement it in a manner consistent with Congressional intent and with FDA’s public health mission. We realize that you may receive questions about this process. We will be working expeditiously to develop further information on how to respond to such inquiries. However, we believe that sponsors are in the best position to provide information on the development status of their products (which is critical to determining whether a drug or biological product is eligible for use under Right to Try) and whether a sponsor intends to make an investigational product available under Right to Try.”
President Donald Trump signed into law the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (Right to Try Act) Wednesday to amend the Federal Food, Drug, and Cosmetic Act.
Woodcock said the law “is intended to increase access to certain unapproved, investigational treatments (drugs or biological products) for patients diagnosed with life-threatening diseases or conditions who have exhausted approved treatment options and who are unable to participate in a clinical trial involving the investigational drug, as certified by a physician.”
She also explained what eligible patients and treatments are for ‘right-to-try’:
An eligible patient is one with a life-threatening disease as defined in 21 CFR 312.81 and who meets certain other conditions set forth in the statute. An investigational drug or biological product can only be provided under Right to Try if it:
- Is not approved or licensed for any use;
- Has completed a Phase 1 trial;
- Either (1) is the subject of an NDA or BLA filed with FDA or (2) is the subject of an active IND, and is under investigation in a clinical trial that ‘is intended to form the primary basis of a claim of effectiveness’; and
- Is actively being developed (‘active development…is ongoing’), not discontinued, and not on clinical hold.
“In the near term,” Woodcock told staff, “if you receive inquiries about the legislation from patients or physicians about a specific product, please refer them to the sponsor of the investigational drug or biological product. If sponsors contact you regarding their obligations under this law, we suggest that you refer them to the statute. If you receive more detailed questions regarding Right to Try, please refer those inquiries to Druginfo@fda.hhs.gov or by phone: (855) 543-3784.”
Holly Fernandez Lynch of the Department of Medical Ethics and Health Policy at Perelman School of Medicine, University of Pennsylvania, told Focus: “I think there is lots of room for FDA to protect patients from potentially dangerous effects of the law, which I hope they will implement through rulemaking and guidance, although that will take time.”
She also offered a few possibilities for implementation:
- Clarify that it is not enough to have a life-threatening illness, but rather that the illness should be immediately life-threatening.
- Require adverse event reporting not via the right to try law, but rather under the terms of the overarching IND for the drug (this may be tricky, but not entirely implausible).
- Clarify what it means for a Phase 1 trial to be completed, i.e., what is success?
- Require that sponsors develop contracts with certifying physicians that in order to access the drug, the physician must collect and report safety data.
- Specify the sorts of adverse event data that needs to be collected in order to satisfy the requirements of the annual summary.
- Post as much information publicly as possible, to help patients and their physicians understand that the unlikelihood that right to try will be helpful.
- Encourage patients and companies to utilize the expanded access pathway rather than right to try.
- Encourage sponsors and investigators to allow for the broadest clinical trial inclusion consistent with participant safety and scientific integrity.
Arsalan Arif contributed reporting to this story.
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email firstname.lastname@example.org for more information.
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