WASHINGTON -- By a vote of 250-169, the House of Representatives passed a law Tuesday aimed at expanding access by critically ill patients to therapies not yet approved by the FDA.
The so-called was approved by the Senate last August. It would allow individuals with life-threatening illnesses to obtain experimental drugs prior to FDA approval. However, the law does not require drug companies to make their products available, and the FDA already has a pathway to allow such access. Industry in general has not sought changes to that pathway. Nevertheless, some in Congress are unsatisfied with the FDA's policies and implementation and are seeking to loosen the reins.
"I still hear from patients with serious, life-threatening conditions who remain frustrated with current regulatory processes that prevent them from experimenting with new therapies when everything else has failed them," said Rep. Michael Burgess, MD (R-Texas), chairman of the House Energy & Commerce Health Subcommittee, during Tuesday's debate on the House floor.
"As a physician, I understand that access to investigational drugs and therapies is a deeply personal priority for those seeking treatment for their loved ones," he continued, asking House Democrats, "Why do you not want to allow these patients to exercise their right to fight for their future? These patients are our constituents; let's take this opportunity to improve access to experimental treatments for them and give them new hope."
Burgess stressed that the law would apply only to "certain investigational drugs that have successfully completed a Phase 1 clinical trial and have an active application at FDA. They also must be under active development or production by the manufacturer. It's important to note that these patients have exhausted all FDA-approved treatment options and are unable to participate in clinical trials involving these investigational drugs."
But Democrats did not agree. "If [this bill] is signed into law, patients would be taken advantage of, and would be harmed," said Rep. Frank Pallone (D-N.J.). "It's an unprecedented attempt to roll back FDA's oversight of investigational treatments.... The problem that I have with all this is once you take out the FDA, how do I know, if I'm the patient and I hear from some doctor that there's something that might help me, and I'm desperate, how do I know this is actually not a bad actor?"
The bill also garnered criticism from 100 patient advocacy groups, including the National Organization for Rare Disorders and Friends of Cancer Research. "We reiterate our concern with creating a secondary pathway for accessing investigational therapies outside of clinical trials," the groups to House Speaker Paul Ryan (R-Wis.) and House Minority Leader Nancy Pelosi (D-Calif.). "This pathway removes FDA approval and consultation and would not increase access to promising therapies for our patients because it does not address the primary barriers to access."
Rep. Jan Schakowsky (D-Ill.) tried to get her House colleagues to support an alternative measure that would "offer a more targeted approach to improve FDA's expanded access program that allows patients access to experimental drugs that could save their lives."
Schakowsky said that under her version of the bill, "The FDA would provide manufacturers guidance to clarify how the FDA will consider clinical outcomes associated with treatments under expanded access ... It also provides transparency as to how many patients are making expanded access requests and how often requests are granted or denied by FDA or manufacturers. It also offers manufacturers or sponsors liability protection if they comply with the expanded access program." Her bill was voted down 231-187.
The Trump administration applauded the law's final passage. "This legislation increases access to investigational treatments by amending the Federal Food, Drug and Cosmetic Act to create a new, alternative pathway for patients with life-threatening illnesses," the White House press secretary said in a statement. "The President expressed his support for the need for 'right to try' legislation in his ... State of the Union Address earlier this year, and is happy to deliver on that promise."
"This administration believes that treatment decisions for those facing life-threatening illnesses are best made by the patients with the support and guidance of their treating physicians," the statement continued. "This legislation advances these principles. Almost 40 states have passed their own versions of this important legislation, and we look forward to addressing this at the federal level."