Why Can’t Dying Patients Bypass FDA Drug Approval?

Parallax News presents big issues broken down into multiple perspectives. This piece looks at 3 perspectives on FDA regulations for the terminally ill.

This month, the terminally ill, their families, friends, and members of Congress rallied at the Capitol for the “Right to Try.” Right to Try laws were created to help terminally ill patients access experimental drugs that have not been fully approved by the FDA — which is often criticized for bureaucratic slowness. The laws have passed in 30 states and counting, but the rally championed a federal bill introduced in May — the Trickett Wendler Right to Try Act of 2016, sponsored by Sen. Ron Johnson (R-Wis.) and co-sponsored by 24 other senators, including John McCain and Ted Cruz — who spoke at the rally. The act, also backed by the Goldwater Institute, would remove the FDA as middle man, barring it from restricting the production of drugs intended for dying patients.

I. Governor Jerry Brown

Governor Brown says the FDA has a viable process for getting terminally ill patients experimental drugs.

Right to Try bills have passed in 30 states, but California Governor Jerry Brown vetoed the bill last October, instead pointing to the FDA’s “Expanded Access” program as a viable means for getting investigational drugs to terminally ill patients. Expanded Access is often referred to as “compassionate use.”

The FDA’s Expanded Access program “provides a pathway for patients” that are not eligible for clinical trials to gain access to unapproved drugs for “serious” diseases. Patients can apply for Expanded Access through the FDA, making sure to remember the risk these drugs hold — that they may be or may not be effective — and that “unexpected serious side effects” may occur.

Brown prefers the Expanded Access approach because it requires institutional review boards made up of “scientists, doctors, and lay people” … “to ensure that appropriate steps are taken to protect the rights and welfare of participants as subjects of research.” If the risk is found to be too great, the review board will not approve an application.

This regulated approach spans back to the Thalidomide Tragedy in 1962, in which patients in 46 countries were allowed to try the newly developed drug and their children were born with “shortened, absent, or flipper-like limbs” as a side effect. The FDA inspector at the time, Frances Kelsey, became a “heroine,” because she did not approve the drug in the U.S. due to “incomplete and insufficient data on its safety and effectiveness.”

Yet patients have complained about months spent completing the necessary paperwork for Expanded Access. FDA commissioner Robert Califf, however, promised that the FDA has “streamlined” its process — providing new documentation that took physicians a total of 45 minutes to fill out. Brown wants California to give this newly “streamlined” process a chance to work.

Additionally, the FDA cites that it approves 99% of the expanded access applications it receives, and points to success stories such as that of 10-year-old Will Goodale, who had a cancerous brain tumor and got his expanded access request approved in just 10 days.

II. Angelina Fanous

ALS victim Angelina Fanous says patients have no time to wait for the FDA’s approval on drugs that could save lives.

Two years ago, journalist Angelina Fanous was diagnosed with ALS, also known as Lou Gehrig’s Disease, a disease in which the motor neurons die slowly, eliminating one’s ability to move, to speak, and ultimately — to breath on one’s own. Around 30,000 people in the U.S. live with ALS, there is no known cure, and the average life expectancy upon diagnosis is anywhere from two to five years.

Fanous has spent the past year reporting on ALS around the country. She concluded her VICE episode on HBO, Die Trying, with the assertion that “the FDA’s one-size-fits-all system doesn’t work for [fast-moving] neurodegenerative diseases like ALS.”

When speaking to Parallax, Fanous said that she didn’t “lose her ability to assess risk when she got ALS,” and that if a drug was showing promise, she didn’t want the FDA to slow down her means of trying it.

“We should have better, [more widely accessible] expanded access programs,” Fanous told Parallax, explaining that even though there are experimental trials underway, the FDA’s rigid screening process makes them nearly impossible to get into. She points to an Expanded Access trial for a drug called NurOwn that has slowed down progression of the disease in 92% of the patients that received the full treatment — but of the 2000 patients who applied for the trial, only 16 were accepted.

Last June, Fanous was approached to apply for an Expanded Access trial for a promising drug called GM6 — fully aware that she was singled out because she was a member of the press. She explained that, “what the FDA doesn’t tell you” is that their institutional review boards take a ton of time to review a case — stretching out the “streamlined” 45-minute application process to what took Fanous, a team of HBO researchers, and her physician a total of three months to complete. For patients with ALS, a few months can mean losing “their ability to walk, speak, or breath on their own.”

When Fanous did start receiving GM6, it stopped the progression of her ALS for 6 months, until she moved to the West Coast for work and couldn’t find a doctor in Los Angeles who was willing to take on the liability of administering an unapproved drug — giving her no choice but to stop the treatment.

Additionally, Fanous explains that it’s illegal for drug companies to charge for unapproved drugs — thus de-incentivizing the companies from selling directly to American patients and forcing many patients to raise money to go abroad for treatment. “You should have the right to try a drug on American soil,” says Fanous, and without the FDA’s approval, you can’t do that.

III. Arthur Caplan

Bioethicist Arthur Caplan says that blaming the FDA is “ideological” because the drug companies are the ones that decide who gets access to their products.

Arthur Caplan is the Director of Medical Ethics at the New York University School of Medicine and a founding member of the interdisciplinary NYU Langone Medical Center’s Working Group on Compassionate Use and Pre-Approval Access.

Caplan told Parallax that the problem with Right to Try legislation is that it “puts the focus on the FDA as a roadblock,” but the truth is that “it’s all up to a drug company, whether it’s going to honor a request for a drug or not.”

Caplan says that the Right to Try bill has no provision for reimbursing drug companies for the drugs they are being asked to provide. Because there is no money in the bill, Caplan says that there is nothing incentivizing drug companies — who ultimately make the decision which drugs they will or will not give away — to provide drugs to willing patients, as well as nothing in the bill helping patients pay for the drugs themselves.

He explains the step-by-step process that goes into getting an experimental drug: before the FDA enters the equation, a doctor goes to the drug company, asks them whether they want to give away the drug to the patient in need, and then the CEOs and investors of the company debate whether it’s profitable to provide it to the patient, which it isn’t, because there’s no money backing the Right to Try bill.

He says “what the proponents of Right to Try keep missing is that we’ve got a free market system, and that because drug companies have no means of being reimbursed for their drugs, they are basically being asked to give drugs away for free — and that’s never going to work because these are small companies that don’t have the means to do that.”

Dr. Caplan’s team is still working on a “big picture” proposal for those seeking expanded access, but he told Parallax that his current solution would be to “put money into the bill.” If the drug companies aren’t being promised money to subsidize the production of their drugs, the bill is purely ideological — “feel good legislation.”

Caplan thinks the U.S. needs to give the companies incentive to invest in the drugs, suggesting that Obamacare be amended to include a “Compassionate Use Fund” that supports continued drug research, funds online research-exchange registries, and helps patients fund the purchase of experimental drugs.

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This piece was written by Nastasya Popov.

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