Executive Summary

US House rejects tempered legislation as many stakeholders remain opposed – but vote allows members to signal to the Trump administration that they tried.

Right to try efforts on Capitol Hill are left searching for answers and maybe another legislative pathway after the defeat of House legislation on March 13.

The House bill, which had been introduced over the weekend and rushed to a vote on Tuesday night, failed to meet the high threshold required under the suspension of the rules mechanism that was used to bring it to the floor. It needed 290 affirmative votes to pass, but only got 259.

Extensive patient and stakeholder opposition and FDA concerns may have triggered the reticence from lawmakers about the legislation, which is intended to allow terminally ill patients the ability to receive experimental treatments without FDA approval. Opponents argued that the bill would only create false hope and does not assure access, while those in favor argued the bill would help patients overcome hurdles to access that exist, such as liability concerns and the potential for adverse events to be used against approval. Both are addressed in the bill. (See chart at the end of the story.)

In retrospect, the haste to get the bill to the House floor seems ill advised, since the Senate wouldn’t have moved quickly even if it had passed and proponents now have to go back through the committee process. But there may have been brilliance in the move as well: since the opposition had firmed up, the vote allowed many House members to say “yes” on an issue that is top priority of the Trump administration but not worry that their vote would actually enact a policy that is viewed with trepidation by many pharmaceutical stakeholders – all while painting Democrats as over-regulators.

The House bill is a modified version of Senate legislation that passed unanimously in August, with changes that attempt to address concerns raised about that bill by FDA Commissioner Scott Gottlieb and others. (See sidebar). 

But the new provisions do not appear to have calmed those fears among stakeholders. Jeff Allen, CEO of the Friends of Cancer Research, said in a statement before the House vote that while it appreciates the Energy and Commerce Committee’s effort to add patient protections, “by removing FDA from the process, this bill remains one which will cause more harm than good.”

“We would urge congress not to pass this bill, and instead, continue a dialogue on this important issue with all stakeholders,” Allen said.

The Pharmaceutical Research and Manufacturers of America also said before the vote that as with previous proposals “PhRMA believes any legislation must benefit patients and not disrupt the future of clinical trials and the research and approval of new medicines.” The trade association added that it is “engaged in a broad array of conversations with House, Senate and administration officials and are committed to driving towards an outcome that continues to enhance patient access to innovative treatments.”

Better Than Previous Versions, But…

The House bill retains the existing compassionate use program, where FDA must sign off on the unapproved drug’s use. The agency approves nearly all the requests it receives annually through the program. But the bill also creates an alternative pathway where patients can access an unapproved drug for compassionate use purposes without FDA approval. The agency still would have to be notified within seven days and adverse events reported immediately.

Momentum for a federal right-to-try law has been building for some time as now 38 states have passed similar bills. Proponents say patients should be able access to an unapproved treatment without first gaining government approval.

The Goldwater Institute, which has been pushing state and federal right-to-try laws, said the House bill should resolve uncertainties about the process.

“The clarifications in this bill should answer any questions from doctors and pharmaceutical and biotech companies about exactly who qualifies and how the FDA can use patient data collected and reported from right to try patients,” Starlee Coleman, Goldwater Institute senior policy advisor, said before the vote.

FOCR Chair and Founder Ellen Sigal and Treatment Action Group Executive Director Mark Harrington argued in an op-ed published in STAT that the bill would create more uncertainty, rather than address issues such as supplies of experimental therapies and safety concerns.

“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,” they wrote. “The absence of data here is both alarming and telling.”

The National Organization for Rare Disorders also remains opposed to the bill. Paul Melmeyer, NORD director of federal policy, said that the House version “makes substantial patient safety improvements,” but ultimately “still creates an unnecessary, less-safe alternative pathway that will not increase access to investigational therapies for our patients.”

Alison Bateman-House, assistant professor in the Department of Population Health at the NYU School of Medicine, also an opponent of the bill, said the alternative pathway now may only be used by a group of patients “so small to be virtually non-existent,” but could be enlarged later. She said there already are efforts to expand a similarly worded state law in Texas to include chronically ill patients.

“This bill is better than earlier versions,” Bateman-House said told the Pink Sheet. “But it does not give patients anything they currently lack and it opens the door for abuses.”

Gottlieb’s concerns about the Senate bill focused on eligibility; he said that only terminal patients should be allowed to use the pathway.  (Also see “Right-To-Try Legislation Must Change Language To Narrow Spectrum Of Patients, Gottlieb Says” – Pink Sheet, 4 Oct, 2017.)

FDA Still Working on Patient Protections

FDA provided technical assistance for the design of the House bill. The agency, which generally does not comment on pending legislation, said it would continue working with Congress “to help ensure patients are also protected.”

“We at FDA share the strong desire to help patients facing terminal illnesses access promising experimental treatments in their fight,” FDA said in a statement before the vote. “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.”

Current and former FDA officials already have said they are opposed to the bill.  (Also see “Regenerative Medicines Provisions Of Cures Act A Top Priority For CBER” – Pink Sheet, 9 Jun, 2017.)

The broad opposition to the bill may have created an awkward position for Commissioner Gottlieb, who was tasked by President Trump with a key role in ensuring enactment of the legislation.  (Also see “Trump On Right-to-Try: Gottlieb’s ‘Heading It Up’?” – Pink Sheet, 2 Feb, 2018.)

https://pink.pharmaintelligence.informa.com/PS122707/New-Right-To-Try-B…