House Energy & Commerce Committee Republicans’ attempts to craft a Right to Try bill that patient advocates can support appears to have failed, with major groups refusing to back anything that cuts FDA out of the process. However the new bill does appear to answer many of the concerns laid out by FDA Commissioner Scott Gottlieb by narrowing the patient population covered and revising the informed consent process.

E&C Chairman Greg Walden (R-OR) and Rep. Michael Burgess (R-TX) unveiled a new version of the contentious bill over the weekend. The bill will be sent directly to the House floor Tuesday (March 13). While the path forward in the Senate remains unclear as of press time, prior to this weekend’s bill drop, Right to Try watchers told Inside Health Policy that any changes to the Senate-passed bill would necessitate another vote in the upper chamber.

The push for a federal Right to Try law, which would let terminally ill patients buy drugs that aren’t FDA approved and cut FDA entirely out of the process, was invigorated following President Donald Trump’s calls for the policy during both his State of the Union Address and a speech the next day at the GOP retreat. The Senate passed a Right to Try bill via unanimous consent last August but the House bill has been stuck in the Energy & Commerce Committee.

Walden told IHP in February that his committee was revising the bill to answer concerns raised by patient advocates. “We want to get a Right to Try bill that will work for patients. We think we are very close on language. I’m dedicated to this cause as well. It’s just that we think if you listen to the patient groups, they tell us that there are problems with the way the language is drafted that came over from the Senate. And so they’re asking us — the people representing patients are asking us — to make some improvements so that this will be an effective piece of legislation. I think we are very close on that piece,” Walden told IHP.

Nearly 40 patient groups wrote to House leadership in February opposing the Senate-passed bill, and many of those groups tell IHP they also oppose the new House bill.

“They have made significant safety improvements compared to prior versions of the legislation (informed consent, additional reporting to FDA, more public reporting by companies), but the legislation still cuts FDA out of the approval process, and removes their ability to amend the dosing, route of administration, dosing schedule, etc. to make the therapy safer. For these reasons, we still don’t support the bill,” Paul Melmeyer, director of federal policy at the National Organization for Rare Disorders, told IHP.

The American Cancer Society Cancer Action Network likewise told IHP that it cannot support the House bill because of concerns “that the bill would remove FDA from the important role that it plays in ensuring the safe use of experimental drugs.”

“The FDA would not have the opportunity, as they currently do, to provide consultation on dosing, route of administration, dosing schedule, and other important safety measures which they are uniquely positioned to understand,” ACS CAN said.

The National Health Council told IHP they “are pleased to see new patient safety provisions added,” but “remain opposed to this bill.” “It is still based on a flawed framework that eliminates FDA’s longstanding role in safeguarding the public’s health. We support increased patient access to investigational products but feel there are better ways to meet this goal by continually improving clinical trial criteria and the existing expanded access process,” Eric Gascho, vice president of policy and government affairs at National Health Council, told IHP.

Friends of Cancer Research also does not support the new bill. “We will not be supporting the new version. While we certainly appreciate what Chairman Walden and his team have tried to do in terms of addressing some of the existing concerns with the Senate proposal, at the end of the day we just can’t support something that excises FDA out of the process. … If they’re not part of any expanded access proposal were not going to be able to support it moving forward and hope that Congress won’t either,” Jeff Allen, CEO of Friends, told IHP.

Public Citizen, which also opposes the new bill, sent a letter Monday (March 12) urging House lawmakers to oppose the legislation unless three changes are made: adding a provision that would require FDA and Institutional Review Board review and approval of each request for use of an investigational drug; limiting the drugs eligible to those whom have completed at least one Phase II trial; and removing sections of the bill shielding manufacturers and physicians from liability.

In contrast, the Goldwater Institute, the libertarian think tank behind the push for Right to Try, praised the new bill calling it “a terrific development for patients.” The group argued the new bill should answer any questions from doctors and drug companies about who qualifies for Right to Try and how the FDA can use patient data.

Gottlieb, who Trump has claimed is leading the charge on Right to Try, during an October 2017 E&C hearing on the policy suggested a number of tweaks to the Senate bill, many of which appear to be in the new version. Gottlieb suggested narrowing the patient population covered, further restricting promotion and commercialization of the experimental drugs, ensuring FDA’s authority to take certain enforcement actions and protecting FDA from federal tort liability.

Notable changes in the newly released bill include: narrowing the patient population from a patent “who has been diagnosed with a life-threatening disease or condition (as defined in section 312.81 of title 21, Code of Federal Regulations (or any successor regulations))” and who has exhausted other options, to a patient with “(A) a stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months; or (B) a disease or condition that would result in significant irreversible morbidity that is likely to lead to severely premature death,” and who has exhausted other options.

Nonetheless, some are still concerned about how the bill defines the covered patient population. Allen noted the ambiguity of “severely premature death.” “I don’t know if it is that narrow,” Allen said. “I think what is concerning to a number of other groups as well as the patient advocacy community is: is this language really clear enough in terms of being able to make a distinction of who should be able to have access through some of these new programs versus not. … There is some question around ‘severally premature death.’ What does that actually mean?”

One academic who has staunchly opposed Right to Try worries that while the new bill only affects a narrow subset of patients, it could set a dangerous precedent.

“As it is written here, the [number] of patients impacted is so small to be virtually non-existent. But it creates a precedent that can be enlarged. For example, in TX, where there is a similarly-narrow state right to try act, there have already been efforts to broaden it to chronically ill patients. This bill is better than earlier versions. But it does not give patients anything they currently lack and it opens the door for abuses. Therefore, I continue to oppose it,” Alison Bateman-House, assistant professor in the Department of Population Health at New York University, tells IHP.

Bateman-House spearheaded a February letter from physicians and ethicists opposing the Senate-passed Right to Try bill and she is now collecting signatures for another letter set to be delivered to House Speaker Paul Ryan (R-WI) and Democratic leader Nancy Pelosi (CA) Tuesday.

An E&C spokesperson also told IHP the new bill “improves patients protections with clearer informed consent.” The Senate bill appeared to require informed consent, but did not reference directly the relevant portion of the Code of Federal Regulations. The Senate language stated: that an eligible patient must have “provided to the treating physician written informed consent regarding the eligible investigational drug, or, as applicable, on whose behalf a legally authorized representative of the patient has provided such consent.” The House bill uses largely the same language but defines informed consent: “as described in part 50 of title 21, Code of Federal Regulations (or any successor regulations).”

The spokesperson also pointed to a new requirement for “real-time adverse event reporting” and “a standard to notify the FDA when a patient receives an unapproved drug through the new alternative pathway.” The House bill would require that the physician “immediately report to such sponsor or manufacturer any serious adverse events, as such term is defined in section 312.32 of title 21, Code of Federal Regulations (or any successor regulations), associated with the use of the eligible investigational drug by the eligible patient.”

The Senate-passed bill requires “the manufacturer or sponsor … submit to the Secretary an annual summary of any use of such drug under this section … [including] the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events.”

On notification, the House bill requires “the sponsor of such eligible investigational drug notifies the Secretary of the provision of such eligible investigational drug for use by an eligible patient pursuant to this section. Such notification shall be submitted within 7 business days of the provision of such eligible investigational drug as correspondence to the investigational new drug application described in subsection (a)(2).” The Senate-passed bill’s only comparable provision is the annual reporting requirement.

When asked whether the new bill answers the concerns raised by Gottlieb, an FDA spokesperson sidestepped answering. “We have been working closely with legislators to provide technical assistance as Congress works on this important issue to help ensure patients are also protected. FDA will continue to work closely with Congress as they advance Right to Try legislation that gives terminal patients the chance to more safely access experimental treatments as part of our commitment to protecting patients while promoting public health,” the spokesperson replied.

The plan in the Senate, should the bill pass the House, at this point remains largely unclear. “We are still sorting through exactly what this means and what the next steps will be. We don’t have any information on any deals that were cut–no one has indicated that to us, but I think we will know more in the next couple of days what the plan will be to go back through the Senate,” Coleman told IHP.

Senate bill sponsor Sen. Ron Johnson (R-WI)’s office did not respond to requests for comment.

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