“Right-to-try,” the controversial plan to help the terminally ill that just failed in the House, explained – By Dylan Scott (vox.com) / March 13 2018
In a surprising twist, the House failed to pass a bill on Tuesday that would have given terminally ill patients more opportunities to try experimental treatments — but that experts worried would actually expose them to more risk.
The legislation is the brainchild of the so-called “right-to-try” movement. The gist of the movement’s mission is that terminally ill patients should be given wide latitude to try out unproven treatments if they have exhausted all their other options.
But it came up short in a House vote on Tuesday evening. Republicans tried to use a procedural shortcut to pass the legislation, which required two-thirds of lawmakers to support it. It failed, with 259 votes in favor and 140 votes against, almost all of them Democrats, plus two Republicans: Erik Paulsen of Minnesota and Patrick Meehan of Pennsylvania. (That’s 65 percent — just shy of the threshold.)
More than 30 states have passed a “right-to-try” bill, often favored by more libertarian-minded people. Supporters portray it as a humanitarian exercise for people with no other hope that removes the risk of a bureaucracy getting in their way. It has been a priority for the Trump White House: President Trump urged Congress to pass a bill during his State of the Union speech, and Vice President Mike Pence has been outspoken in favor of it.
“It’s about restoring hope and giving patients with life threatening diseases a fighting chance,” Pence tweeted earlier this year.
But skeptics have two big objections: First, that these bills are unnecessary because the Food and Drug Administration already does a good job letting terminally ill people access experimental treatments. And second, that the only thing these bills achieve is opening up patients to more risk. The bill is opposed by prominent patient groups, like the American Cancer Society and the National Organization for Rare Disorders.
Alison Bateman-House, who studies medical ethics at the New York University Langone Medical Center, walked me through the complexities last year.
“We have a functioning expanded access procedure through the FDA,” she told me in August when the Senate passed its version of “right-to-try.”
The status quo versus “right-to-try,” explained
It might help to understand how the FDA’s current program works and what it would look like under “right-to-try.”
Under the FDA’s current policy, patients who want to try a treatment that has cleared only the first round of clinical trials must:
- Find a doctor who will agree to try the therapy.
Contact the drug company to ask permission to test the treatment. - With that permission, fill out paperwork for the FDA and send it in.
- If the FDA doesn’t object, the patient can try the treatment.
- A board at the institution where the treatment is taking place must also be notified and grant approval.
- The patient must grant consent and may have to pay money for the treatment.
Under a “right-to-try” bill, the process is almost exactly the same, except the FDA is removed from the equation:
- Find a doctor who will agree to try the therapy.
Contact the drug company to ask permission to test the treatment. - With that permission, the patient can try the treatment.
- A board at the institution where the treatment is taking place must also be notified and grant approval.
- The patient must grant consent and may have to pay money for the treatment.
The House legislation includes a few other notable conditions for patients, doctors and pharmaceutical companies using “right-to-try”:
- Drug companies must notify the FDA when a patient is giving an unapproved treatment under “right-to-try.”
- Drugmakers must also report any adverse effects that a patient using a “right-to-try” drug experiences.
- The clinical outcomes from patients using a drug under “right-to-try” cannot be used for determining whether the drug is eventually approved for broader use by the FDA.
(Please note that pharmaceutical policy is hopelessly complex, so these are the broad strokes. Also, state laws differ, so how they would interact with any new federal law could differ as well.)
Why experts are dubious about “right-to-try” bills
The core issue with the legislation, from its critics’ point of view, is that there really isn’t a problem here to solve.
Based on the available data, the FDA already approves 90 percent or more of the requests it receives to try unproven therapies, and the agency usually responds in a matter of days, Bateman-House told me. There isn’t much evidence of a pandemic of terminally ill patients being blocked from testing treatments because of bureaucratic snags.
Second, in both cases, the real hurdle is getting approval from the drug company to test a treatment. That would still be true under a “right-to-try” regime.
“You have a right to ask a drug company if you can try,” Bateman-House said. “The only thing that disappears is that FDA step.”
And therein lies the concern of Bateman-House and others. The existing program, which would still be in place even if “right-to-try” becomes law, allows the FDA to be an independent arbiter.
Without that federal oversight, Bateman-House believes there is a greater risk of fraud and abuse at the expense of patients.
“By getting rid of that, what you’re doing is you’re relying on a doctor and a quote-unquote drug company,” she explained, “but there are no specifications about that relationship.”
“For all I know, the doctor works for the drug company, owns the drug company, owns stock in the drug company, or the drug company gives the doctor kickbacks,” she continued.
The House bill does differ slightly from the Senate version. A smaller number of patients would be eligible to utilize “right-to-try”: They must be terminally ill, have exhausted all their other treatment options, be ineligible for other clinical trials, and be expected to die within a matter of months.
But that doesn’t change the foundational issues that opponents like Bateman-House have.
“This version of the bill is an improvement over the last, but it doesn’t change the fact that the purpose of this bill is to sever access to investigational drugs from FDA review and approval,” she told me this week in an email.
She noted, too, that while the current population that’s eligible for “right-to-try” is so narrow as to be nonexistent, now that the procedure exists, it could be expanded to include more and more patients. In Texas, for example, “right-to-try” supporters are attempting to expand the program from terminally ill patients to chronically ill ones.
This is the bottom line, based on my conversations with Bateman-House about this issue over the past few months:
- The FDA already has a functioning procedure for experimental treatments.
- The “right-to-try” bill actually leaves that procedure in place.
- All the bill does is potentially create an alternative pathway for patients.
- We don’t even know if patients will take it.
- If they do, because the FDA is no longer a part of the process, there is a greater risk of fraud and abuse.
“This area is already replete with confusion, and we don’t need any more confusion,” Bateman-House told me. “I think patients are going to end up losing.”
https://www.vox.com/policy-and-politics/2018/3/13/17113690/right-to-try-laws-congress
