Regulatory Focus™ > News Articles > 3 > House to Vote Tuesday on ‘Right-to-Try’ Bill

House to Vote Tuesday on ‘Right-to-Try’ Bill

Posted 12 March 2018 | By Zachary Brennan 

House to Vote Tuesday on ‘Right-to-Try’ Bill

The libertarian Goldwater Institute’s attempt to undermine the US Food and Drug Administration (FDA) gained new steam over the weekend as House Republicans quietly unveiled their “Right-to-Try” bill just before midnight on Friday.

The bill claims that it will improve access to experimental treatments for people with terminal illnesses, but based on what has been seen with similar legislation enacted in almost 40 states, the “Right-to-Try” movement has done little more than offer politicians a chance to claim that they are helping the country’s most vulnerable.

When asked last August which patients have been helped by the enactment of 38 states’ “Right-to-Try” legislation, the Goldwater Institute could only point to a single physician treating six patients in Texas, and questions have previously been raised by his intentions in running such a trial.

House Bill

The House bill, meanwhile, is similar to the Senate-passed bill, though different enough that the Senate would have to reconsider the legislation if the House passes its bill.

Still, both bills would remove FDA from the process of signing off on certain patients accessing experimental treatments and instead allow institutional review boards to essentially make such decisions. Both bills also do not mandate or encourage companies to provide such treatments to those with terminal illnesses, which is usually the rate-limiting step as FDA typically signs off on more than 99% of all expanded access requests (previously known as “compassionate use” requests).

A spokesperson for House Energy & Commerce Chairman Greg Walden (R-OR) told Focus that it is challenging to place a specific number on how many with terminal illnesses will be impacted by this legislation, but FDA has previously told him that the two largest hurdles to the existing expanded access program are uncertainty on liability and the use of outcomes.

Alison Bateman-House, assistant professor of medical ethics at New York University, who testified before the House E&C committee in October on expanded access, explained to Focus that the bill’s attention to liability concerns and use of outcomes are based on myths.

For instance, in the last 10 years, no one has been sued because of an adverse event in an expanded access situation, she said, and in that same time period, there have been just two trials (here’s one example of a partial clinical hold from 2014) because of a patient death and both of those holds were short-lived.

The bill's section on the use of outcomes also would not stop FDA from putting a clinical hold on a trial because of an adverse event. The bill makes clear that companies will still have to report adverse events. The only change is that companies will have to publicly report “any known or suspected serious adverse events.”

The House bill also adds public reporting requirements for biopharma companies on: The number of right-to-try requests received, the number of requests granted, the number of patients treated and the therapeutic area of the drug made available.

Walden’s spokesperson also said clarifications on liability and use of outcomes could mean more manufactures and sponsors will increase access to unapproved drugs. But it is unclear how that will play out or how that will be tracked.

FDA Commissioner Scott Gottlieb said in the October House hearing on a previous version of the bill: "The perception is that this legislation will pressure companies to supply more expanded access drugs but that remains to be seen."

Bateman-House added that there are more pragmatic ways to help smaller companies in responding to and providing access to their experimental treatments although this bill does not include such provisions.



© 2021 Regulatory Affairs Professionals Society.

Regulatory Focus newsletters

All the biggest regulatory news and happenings.