Terminally Ill People Want More Access to Experimental Treatments. Here’s Why Doctors Are Pushing Back

Posted on Feb 7, 2018 in Uncategorized

Doctors and medical ethicists are criticizing a bill supported by President Donald Trump that would allow people with fatal illnesses to have access to drugs not yet approved by federal authorities.

“Right to Try” is a policy that Trump mentioned during his State of the Union Addresson 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. 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—which has been introduced in the House—will ultimately make life better for patients. The bill for the federal law was passed by Senate in August by unanimous consent.

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 U.S. Food and Drug Administration (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 that the FDA would be cut out of the loop. The bill would allow patients and their doctors to bypass the FDA process and work solely with drug companies for access to unapproved drugs that have undergone at least basic safety testing. The federal bill would also prevent doctors and drug companies from being held liable should something go wrong, and bar the FDA from using data on negative outcomes from right-to-try cases to impact the approval of a drug unless that data is critical to assessing the drug’s safety.

“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, which currently has more than 300 signatures from ethicists and physicians, argues 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.

The House Committee on Energy and Commerce is currently considering the federal right-to-try bill, and held a hearing about the policy in 2017. “Chairman [Greg] Walden is dedicated to ensuring patients have access to potentially lifesaving treatment while also preserving FDA’s vital oversight authority,” said a committee spokesperson in an email to TIME. “Having held a hearing last year, the committee continues to engage with patients, members, and advocates to advance fair policy that helps patients understand the full benefit of access to investigational drugs and therapies. While we don’t have a scheduling announcement at this time, Chairman Walden looks forward to continuing to work with the administration to get these reforms over the finish line for patients.”

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.”

Source:  Time Magazine By ALEXANDRA SIFFERLIN  February 5, 2018