WASHINGTON -- New "Right to Try" legislation will not remove the primary roadblock facing terminally ill patients who want to test unapproved, investigational therapies, FDA Commissioner Scott Gottlieb, MD, told a House subcommittee Tuesday.
While Gottlieb told the health subcommittee for the House Committee on Energy and Commerce that he does not favor a federal Right to Try law, he said he would commit to implementing it if one passes.
But he also noted in that the FDA already approves 99% of the 1,000 requests for expanded access, or "compassionate use" it receives each year. Still, Gottlieb stressed that he'd like to keep a tight reign on these untested drugs through the FDA's expanded access pathway, because 70% of those drugs ultimately do not gain FDA approval for marketing.
"The vast majority of people who will use a drug through expanded access are using a drug that doesn't work," he said.
Even so, through an administrative action announced Tuesday, the FDA eased some of its requirements for physicians and companies wishing to make investigational drugs available through the expanded-access program.
Expanded access represents a similar pathway to Right to Try laws, which serves the same terminally ill patients with the very same goals as Right to Try laws. To date, 37 states have passed laws similar to Right to Try laws.
While members of the committee noted that critics of expanded access see it as "too slow" and "burdensome," Gottlieb disagreed, adding that, "I think that the biggest obstacle to providing drugs through expanded access is the supply constraints."
When companies conduct clinical trials, they don't continuously manufacture a product. Instead, they produce "discontinuous batches," he noted, and neither the House or the Senate bills address this problem.
Different incentives or a different clinical trial framework would be needed to resolve such challenges, he explained.
Even with approval from the FDA, the expanded access pathway does not compel drug companies to make their products available, he added.
Right to Try Act
In August, the Senate unanimously passed The Trickett Wendler Right To Try Act, which would allow patients to access investigational drugs that haven't yet been approved by the FDA, as long as all other treatment options have been exhausted.
Representatives Brian Fitzpatrick (R-Pa.) and Andy Biggs (R-Ariz.) in the House earlier this year.
"I am sure that Commissioner Gottlieb works tirelessly to help as many terminal patients as he can, but that program is simply not enough," said Biggs in his witness statement.
If passed, a federal Right to Try law would run parallel to the FDA's expanded access pathway, but could mean patients circumvent the FDA entirely.
Some Republican members of the committee were critical of the FDA's compassionate use approach.
"It sure does seem to me that even with the best of intentions, the FDA still thinks they know better than the doctor whose treating the patient," said Rep. Joe Barton (R-Tex.), alluding to the fact that physicians are already involved in helping certify that a patient has tried other alternatives before requesting an untested therapy under the Right to Try framework.
Gottlieb responded that the FDA is not blocking access for patients. "In 10,000 encounters, requests for expanded access in a non-emergency setting, we denied about 25 of them," he pointed out.
Moreover, roughly half of the rejected requests were denied because a drug wasn't available. In other cases, the FDA was aware that "a clinical hold" had been put on a drug, but could not reveal that publicly because that information is confidential, he noted.
"The bottom line is in the vast, vast majority of cases, we do approve [requests]," he said. "The issue is, is [a drug] always available? And do patients always know about it?"
Matt Bellina, a retired lieutenant commander with the U.S. Navy who has amyotrophic lateral sclerosis (ALS), agreed with Gottlieb that the supply issue is a problem. However, he noted that in other countries, such as France, the demand in the market drives the supply.
Bellina had spoken previously of the challenges he faced trying to access the neuroprotective agent edaravone (Radicut and Radicava), which received in 2015, but at that time had not been approved for use in ALS in the U.S. (The FDA approved Radicava in May 2017.)
However, Bellina said the idea that people don't request expanded access because they don't know it exists was "deeply offensive," adding that those in the ALS community know as much as their doctors about their conditions, and are informed of their options.
Positive Steps, But More Needed
Gottlieb also announced recent changes to expanded access at the hearing.
In July, the FDA partnered with the Reagan-Udall Foundation, a network of patients advocates, to launch the , an online "one-stop portal for access to information about expanded use programs," he said.
A year ago, the FDA streamlined its application process for expanded access requests, which now take roughly 45 minutes to complete, according to his written testimony.
And today the agency announced it would simplify the institutional review board (IRB) requirements for physicians requesting expanded access options for an individual patient. would allow physicans to waive the process of a convened IRB meeting "if the physician obtains concurrence by the IRB chairperson (or designated IRB member) before the treatment use begins."
Gottlieb also told subcommittee members that the language in current Right to Try bill is either "too expansive" or unclear. For instance, the current bill may not provide the same patient protections to those who request drugs from individual sponsors, or small clinics, as it would for large drug manufacturers, he noted.
Gottlieb said he was also concerned that some of the Right to Try measures introduced by Congress include terminally ill patients and those with life-threatening illnesses.
"As a clinician, I can certainly contemplate diseases that are life-threatening, but not immediately life-threatening," he said, citing advanced diabetes as one example.
Broadening the definition to include patients who aren't terminally ill risks "undermining the central purpose of this legislation," he said.
Adverse Events
One reason drug companies may be reluctant to offer treatments to patients using the FDA's expanded access pathway is concern that reports of adverse events would negatively impact the overall drug approval process.
In an FDA , Gottlieb wrote that "suspected adverse reactions must be reported 'only if there is evidence to suggest a causal relationship between the drug and the adverse event.'"
He noted only two cases where such adverse event reports related to expanded access affected the products' review process. In one case, the adverse event helped the drug win approval, and in the other it led to a label change, he said.