Congress has passed the so-called “Right-to-Try” bill, which is intended to give people with fatal illnesses a way to access drugs that are still experimental and not yet approved for use. The bill will now be sent to President Donald Trump, who is expected to sign it into law.
“Right to Try” is a policy that Trump mentioned during his State of the Union Address on Jan. 30. “People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home,” he said. “It is time for the Congress to give these wonderful Americans the ‘right to try.'”
Right-to-try laws have passed in some 38 states, but have been criticized by doctors and medical ethicists. While no one wants to argue against giving potentially beneficial drugs to people who need them, not everyone in the medical community is convinced a national right-to-try policy will ultimately make life better for patients. Though Dr. Scott Gottlieb, the commissioner of the U.S. Food and Drug Administration (FDA) expressed his concerns about the bill in the past, he made clear that he would embrace the changes.
For a drug to be approved for use in the United States, it must undergo several rounds of clinical testing, a process that can take several years. The FDA—the agency that oversees that approval process—does already have a system in place, often referred to as compassionate use, for getting unapproved drugs to people who are out of other options. Physicians can submit an Emergency Investigational New Drug (EIND) application to the agency if their patient has no other treatment options. With FDA permission and drug company willingness, a patient can try that treatment.
One of the biggest concerns with the federal right-to-try bill is the weakened oversight of the FDA. The bill will allow patients and their doctors to work with drug companies directly to access unapproved drugs that have undergone at least basic safety testing. Men and women will be eligible if they have a life-threatening disease and have exhausted all other approved treatment options and do not qualify for a clinical trial. The patients will need to have informed consent in writing, and drugmakers will have to provide federal authorities with a yearly summary of the drug’s use, including any adverse events. Drug companies are not required to provide their drug for patients, but the bill will prevent doctors and drug companies from being held liable should something go wrong, unless the company or medical providers engaged in “reckless or willful misconduct” or “gross negligence.”
“The proposed legislation is a thinly veiled attempt to dismantle the FDA, which could take us back a hundred years or more,” says Dr. Steffanie Strathdee, the associate dean of global health science at the University of California, San Diego (UCSD) who requested an EIND for her husband in recent years. “Dying patients and their families are vulnerable; we could become victims to the likes of snake oil salesmen offering ‘treatments’ that could kill rather than cure. Treatments that work need to be monitored so their success can push forward randomized clinical trials that will decide if they work on a broader scale. And we need to know when treatments don’t work, so that the deaths of these patients are not in vain and that their failures aren’t repeated.”
A letter drafted and circulated by medical experts, with more than 300 signatures from ethicists and physicians was sent to the House Committee on Energy and Commerce in February. It argued that weakening the oversight of the FDA through the right-to-try law could ultimately be dangerous. The signed letter will be sent to the House Committee on Energy and Commerce on Monday. “Expanded access can be improved, but the right to try approach is misguided and would likely do more harm than good,” the letter reads. “This legislation sells vulnerable patients and families false hope at the expense of weakening the FDA’s critical role in making sure that all Americans can have confidence in the safety and effectiveness of our medical products.”
The FDA’s drug approval procedures were put into place in 1962 after women in Europe who were taking the drug thalidomide gave birth to babies with birth defects. The drug, which was marketed as a treatment for morning sickness and as a sleeping pill, was not approved for use in the U.S., but the tragedy helped spur Congress to pass a law that would require drug-makers to prove both safety and effectiveness.
As the letter authors point out, the FDA currently approves 99% of the patient requests for expanded access that it receives. “Because the FDA is not the obstacle to patient access to investigational drugs and plays a vital role in ensuring proper patient safeguards are in place, we implore the Committee to not pass legislation that would remove the FDA from the initial authorization process for accessing an investigational therapy outside of a clinical trial,” the letter reads.
The Goldwater Institute, a libertarian think tank, is one of the biggest supporters of right-to-try laws. The institute created a model bill that many states have used for their own right-to-try laws. “Ultimately the way we feel about all of this is it’s a matter of time. The genie is sort of out of the bottle,” said Starlee Coleman, the institute’s chief policy adviser, to Politico. “Patients have too much information now. They know when drugs in trials are working because they are connecting now more than they ever have been before. Maybe people who don’t want this will be able to stem the tide for a little while, but there will be something that comes later.” The federal policy has also received significant support from Freedom Partners, a network that supports the political initiatives of Charles and David Koch and the billionaire brothers’ political allies. (Time Inc., TIME’s parent company, was recently acquired by Meredith Corp. in a deal partially financed by Koch Equity Development, a subsidiary of Koch Industries Inc.) The group argues that even though the FDA grants the vast majority of compassionate use requests, it only received 1,757 requests in 2016, which the group says is small compared to the number of people who could potentially benefit.
Proponents of right-to-try legislation argue that it will cut down on the time it takes for a drug to go through the FDA for compassionate use. But UCSD’s Strathdee disagrees. In 2015 Strathdee’s husband contracted a drug-resistant infection that wasn’t responding to treatment. Strathdee and her husband’s medical team submitted an EIND to the FDA to request access to phage therapy, a treatment that is not approved for widespread use.
“In our case, the FDA official involved was far from obstructionist; she helped connect us to U.S. research teams we were unaware of, who agreed to make their experimental treatment available to us,” says Strathdee. “But the FDA was also careful. They required documentation to show that safety concerns had been considered and minimized to the extent possible.” This was vital, says Strathdee, since the proposed cure for her husband’s infection was derived from sewage. If improperly purified, the treatment could have caused septic shock and killed him.
As Politico reports, the Goldwater Institute says it does not want to get rid of the FDA, but it wants to call out the agency’s flaws, arguing that the review process for drug approval is expensive and long and that if it was easier, drugs would be less costly.
“In our view, the system is under-funded,” says Strathdee. “But it is not broken.”