Last May, US Senator Ron Johnson introduced a bill that seemed relatively uncontroversial: the so-called Right to Try bill, which sought to ease federal government restrictions on the access terminally ill people have to experimental drugs. In an era when bipartisanship is almost unheard of, the proposed legislation attracted 41 cosponsors from both parties.
On Wednesday, the bill is set to be voted on by the Senate. In order to fast-track the process, Johnson, a Wisconsin Republican, is seeking unanimous consent for the legislation — in other words, if just one senator votes against the proposal, it will fail. Nevertheless, Johnson is optimistic it will succeed, his office said this week.
"Terminal patients don't have a decade to wait for the FDA to judge whether a drug is effective — the cost of delay might be measured in the unnecessary loss of human lives," Johnson wrote in a Wall Street Journal opinion piece on September 22. "Right-to-try laws simply establish the freedom for patients and their doctors to try safe therapies where the potential benefits far outweigh the risks terminal patients are eager to take."
If signed into law, the legislation will allow doctors and pharmaceutical companies to bypass the Food and Drug Administration as they negotiate whether a terminally ill patient can obtain treatment options that are still in clinical trials. The bill also removes all liability from those who create, dispense, or use the experimental drugs through right to try.
Those in favor of the bill, including the terminally ill themselves, say it will cut down on a regulatory process that delays or even prevents people from gaining special access to these drugs.
But the bill does have opponents — among them bioethicists, healthcare policy experts, and doctors who argue there are two problems with Johnson's legislation. One, that it seeks to solve a problem that doesn't actually exist. And two, that its true aim is to weaken the FDA.
The rise of the Right to Try movement
Colorado lawmakers were the first in the country to approve a Right to Try bill in 2014, unanimously passing an act that identified access to drugs still in the clinical trial process as a "fundamental right."
Under current FDA policy, known as expanded access or compassionate use, patients seeking experimental drugs work with their doctors to petition a drug company. If the company agrees to allow access to its trial treatment, the physician then submits a request to the FDA. The Colorado bill cut the government out of the process by making the final approval step unnecessary.
After Colorado passed its law, 30 more states have enacted right to try laws largely based on recommendations from the Goldwater Institute, a libertarian think tank based in Arizona. Goldwater started working on the right to try issue because the institute didn't think terminally ill patients should have to "fight the government" when searching for lifesaving options, according to Goldwater spokesperson Starlee Coleman.
"We don't believe that the government should have the power to determine which terminally ill people will have access to promising treatments and which won't," she said.
Why Right to Try is going national
Despite their rapid spread at the state level, Right to Try laws have yet to make a dramatic impact.
In the last nine months, doctors in several states have reportedly used Right to Try laws to allow patients in clinical trials to continue using experimental treatment after the trials concluded. Typically, after a clinical research process concludes, use of the drug is put on hold until the FDA completes a final review, a process that can take up to 10 months. The Goldwater Institute said 78 patients in Texas, all of whom were stuck in limbo waiting for final review to be completed, benefitted from the right to try law in 2016. The year before, however, there were no known cases of experimental drug access granted under the laws.
Drug regulation falls under the purview of the federal government, meaning doctors and pharmaceutical companies currently risk legal action if they approve experimental drug use without FDA approval — even if they're in a state with a Right to Try law. It's a situation not unlike that of legalized marijuana; technically, federal drug laws outlawing pot trump any state legalization laws.
"The need for the federal legislation is to ensure that drug companies and doctors who work with a patient under a right to try law are not prosecuted or punished in any way by the FDA," Coleman said.
If passed, the federal legislation will apply only to states where right to try laws are on the books. Patients living in places without these laws would not qualify.
The right to try debate
One question at the heart of the debate over right to try is whether the legislation is even necessary.
In 2015, the FDA approved 1,416 compassionate use requests and rejected 14 — an acceptance rate of more than 99 percent. Speaking at a federal hearing earlier this month, FDA associate commissioner Peter Lurie said "emergency requests are usually granted immediately over the phone and non-emergencies are processed in a median of four days."
Many of the right to try guidelines are no different than compassionate use guidelines. Both policies require a drug to have completed Phase 1 trials to be eligible, consent from at least one doctor (some states require two), and company approval. The main difference is that Johnson's bill would eliminate the government's role in decision making.
While right to try laws erase one step from the process, they don't require doctors or pharmaceutical companies to make the drugs available to anyone not already participating in clinical trials. To date, none of the major drug makers have thrown their support behind the policy, according to Goldwater.
The pharmaceutical industry trade group PhRMA remains neutral on the Senate bill, but the organization emphasized the important role of the FDA in ensuring prescription drug safety and effectiveness.
"Any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients," PhRMA said in a statement.
Critics like Alison Bateman-House, a compassionate use expert at New York University Langone Medical Center's division of medical ethics, worry the current legislative focus on right to try is creating a "slippery slope" that diminishes the FDA's regulatory control altogether without actually making access to experimental drugs any easier.
In fact, Bateman-House, who co-chairs NYU's Compassionate Use Advisory Committee, believes weakening the FDA is one of the aims of the right to try legislative movement.
"I do not see these bills as helping any patients, or at least not any significant number of patients," she said. "I see it intending to be an opening to take power away from the FDA to regulate drugs."
Bateman-House has watched the right to try laws develop in recent years, and says they appear to be a reflection of the libertarian agenda of the Goldwater Institute instead of a genuine attempt to help the terminally ill.
Goldwater made a point to emphasize that the organization is not attacking the FDA.
"We are not challenging the role of the FDA in determining that drugs are safe," Coleman said. "We're simply saying that when you're dying, your risk-benefit equation is different, and that decision belongs with patients and their families and their doctors, not the federal government."
Johnson's office was unable to respond to a request for comment on these claims in time for publication.
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