Right-to-try experimental drug bill fails to pass in House vote

The "right to try" approach allowing terminally ill patients to access experimental, unapproved drugs while bypassing federal regulators' pathway to such medications has failed to pass by two-thirds majority in a House vote held Tuesday evening.

In a vote taken under suspension of the rules, House members fell short with its vote of 259 to 140 in favor of the Right to Try Act. The bill would allow patients with only months to live and no other treatment options to ask drug makers for access to their experimental treatments outside of the clinical trial process and out of the purview of the Food and Drug Administration.

A version of the bill passed in the Senate last August with huge bipartisan support. President Donald Trump has indicated his support for the measure and has said he plans to sign the bill once it gets to his desk.

A majority of House Democrats stood in opposition to the measure on the grounds that it took away the safeguards granted under the FDA's program to ensure patient safety. They also criticized the manner in which the bill had been introduced. Under a suspension of the rules, a two-thirds majority is needed to pass legislation that avoids normal procedures in order to be fast tracked without the ability to include amendments.

Critics have argued the legislation is unnecessary since the FDA already approves by one study's count 98% of requests from patients to access yet unapproved drugs through what is called the Expanded Access program. But that hasn't stopped a movement to bypass the federal agency from growing in recent years. A total of 38 states have passed their own right-to-try laws since 2014, usually with large bipartisan support.

On Monday, more than 70 patient advocacy groups signed a letter to the leaders of both parties in the House expressing their concerns that the bill would not improve access to experimental therapies, while making it riskier for patients by eliminating federal oversight from the process.

"The FDA has never been the problem in terms of the time for approval nor whether or not to approve unless there is evidence that expanded access would be problematic," said Dr. Barbara Bierer, professor of pediatric medicine at Harvard Medical School and program director of the Regulatory Foundations, Ethics and Law Program at the Harvard Clinical and Translational Science Center.

Risks aside, there are broader concerns about whether the rise of the right-to-try movement has increased therapeutic misconceptions around experimental therapies, and in turn made it more difficult for doctors to engage patients in discussions over how they should approach the end of their lives.

Healthcare providers have urged greater focus in recent years on providing hospice and palliative-care options for patients with serious conditions as a means of improving patient satisfaction and reducing healthcare costs by avoiding unnecessary procedures that would have minimal health benefit.

Bierer could not comment on whether she felt the attention given to right-to-try has altered those types of doctor-patient discussions. But she did acknowledge a key factor involved in those kinds of discussions was knowing the potential effectiveness of an experimental drug and trying to limit expectations, which she said was one of the benefits of having a process overseen by the FDA.

"The pivotal point is what do we know about the drug?" Bierer said.

There have also been questions over whether such laws are any more effective at providing patients greater access to experimental treatments than the current process overseen by the FDA. Under both right-to-try and Expanded Access, it's up to drugmakers whether or not to provide their products.

More than 5,000 requests for expanded access were approved by the FDA between 2010 and 2014, according to a September study published in the Journal of Clinical Pharmacology, for an approval rate of about 98%. So far there have been reports of only a few patients who have even used much less benefited after a right-to-try law was enacted in their state.

Critics of the agency argued that the process to apply for expanded access was too cumbersome and led to delays in getting dying patients the drugs they need. But the agency in 2015 made changes to address those concerns, streamlining the application process to where it takes up to 45 minutes instead of hours, with most emergency requests receiving a reply in about 24 hours.

Other questions arise as to what impact using an experimental drug under a right-to-try law would have on a patient's health insurance coverage. In 19 states that have passed right-to-try laws, insurers are allowed to deny patients hospice care if needed after the use of experimental drugs. Such laws to do not require payers to cover the cost of subsequent care that may be needed as a result of side effects from an experimental drug.

If the bill is passed and signed, the campaign that made it law may not stop there. Some believe there is a chance the program's scope could be expanded beyond terminally-ill patients to one day include those with severe chronic conditions. Such a possibility was one of the concerns around the bill that passed the Senate. That version defined qualified patients as those with "life-threatening" diseases, which some including the FDA feared was too broad and could include people with long-term chronic conditions. The House version narrowed the definition to only allow patients who had months to live could apply.

"I think it kind of depends on how widely used this mechanism becomes and how people feel it can be better used," said Nielsen Hobbs, a pharmaceutical industry analyst and executive editor of Informa Pharma Intelligence.

Updated 6:05 CT