WASHINGTON -- Terminally ill patients want access to experimental drugs and devices that they believe are safe (or safe enough) but which are currently off-limits under FDA regulations. A new federal "Right to Try" bill would increase availability of such treatments to desperate patients, but critics of the legislation say it puts patients at risk of exploitation, or worse.
The bill would allow companies to sell, without restriction, any drug or device intended to treat terminally ill patients, as long it has completed a phase I trial and is currently in another FDA-approved trial, and a doctor certifies that the patient has exhausted other options.
"I've heard the argument that we'll risk side effects, but as you can tell, I'm struggling here," , a 32-year-old former naval officer, told the Senate Committee on Homeland Security and Governmental Affairs. Bellina was diagnosed with amyotrophic lateral sclerosis, (ALS) in April of 2014.
"At some point in the near future, I'm going to suffocate under the weight of my own chest. So what difference at this point does it make for me to have a side effect?"
Bellina has been excluded from clinical trials because he has been sick for too long.
"This is a situation where we're asking for a very specific carve out, for a very small exception, that doesn't cost us any money," he told the committee. One drug that he's interested in trying, , has already been approved in Japan, but not in the U.S.
"Right to Try" legislation has been enacted in 31 states. wants to put his bill, the " to a vote next week. Johnson has argued that the impact of the state laws will remain uncertain without a federal law.
Other patients, physicians, and lawmakers, each with their own stories of family members or constituents with a terminal illness, testified alongside Bellina in support of Johnson's bill.
But the legislation "doesn't really get at the heart of the problem," said , the committee's ranking member, who stressed that not only can drug companies reject requests for a treatment, but even with access many patients cannot afford the treatments.
The FDA has approved for expanded access to treatment from patients with life-threatening diseases, said , the agency's associate commissioner for public health strategy. Lurie noted that in one 6-month period a single pharmaceutical company, Johnson & Johnson, rejected more requests than the agency had in 6 years.
While the FDA has not taken an official position on the federal bill, Lurie highlighted three concerns over right-to-try legislation:
- Threat of safety problems
- Risk of "unscrupulous actors" preying on "vulnerable patients"
- Need to retain the integrity of the clinical trial process
Johnson's bill places no limits on what companies could charge patients for such treatments, and it shields companies and physicians providing experimental treatments covered under the bill against any type of liability. Furthermore, it specifically bars the FDA from using patients' outcomes in subsequent regulatory decisions about the treatments, no matter what those outcomes might be.
Lurie also noted that despite some patients' concerns that the agency would not allow them to take a drug because of the severity of their illness, medical officers have the expertise to distinguish between patients in clinical trials and patients given a drug because they are terminally ill, and will weigh any potential adverse events appropriately.
At one point in the hearing, the conversation between Johnson and Lurie got heated. Johnson bridled at the suggestion that the FDA helps patients by withholding access to treatments, when many of them are experts in their diseases.
"Who is the FDA to make that decision for them?" Johnson asked.
"We've been asked to look after patients and we feel that's exactly the responsibility that we're exercising," Lurie replied.
He later told reporters, "What we would not want is for some company with a drug that had no special evidence of effectiveness to be charging vulnerable patients an arm and a leg, and the next thing they've got a whole separate revenue stream which would then diminish the incentive for them to go though the clinical trials process."
Asked for examples, Lurie noted that the agency cannot discuss individual applications, but that he sees the problem "with some frequency."