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Trump Fuels False Hope for Dying Patients

Health Letter, May 2017

By Sarah Sorscher, J.D., M.P.H.

Image: Oldmandshand/Shutterstock.com

In February, President Donald Trump and Vice President Mike Pence both signaled their support for efforts to sweep aside federal oversight for experimental medical treatments used by dying patients, fueling false hope that deregulation will lead to “more miracles.

Trump makes no secret of the fact that he loathes regulation. Yet most of his outlandish promises, including or trimming regulations from 9,000 to 100 pages or hiking industry user fees by nearly $1 billion annually, seem to come from nowhere with no clear path forward.

This outlandish promise is different. Several dangerous federal bills that could easily make it a reality are now gaining momentum in Congress, thanks in large part to an aggressive lobbying effort funded by the conservative Goldwater Institute.

A direct attack on the FDA

The Goldwater Institute has successfully helped spin the heartbreaking stories of countless dying patients into a national movement, seductively branded with the name “Right to Try.”

This movement seeks to gut an existing program run by the Food and Drug Administration (FDA) that currently allows patients who are not enrolled in a clinical trial to obtain experimental medical products.

This FDA Expanded Access Program does not itself provide medical products to patients. Instead, it gives the green light to manufacturers that wish to provide them after ensuring that minimal scientific, medical and ethical standards are being followed. It also prevents manufacturers from earning a profit before FDA approval and verifies that patients are not being diverted away from clinical trials, which helps to maintain incentives for timely product development.

The FDA grants over 99 percent of all expanded access requests. Yet some frustrated patients cannot find a doctor who agrees to help with the application or convince the manufacturer to offer the product.

The Goldwater Institute has helped channel these frustrations into a targeted attack on the FDA, resulting in the passage of laws in 33 states in less than three years that ostensibly allow dying patients to access experimental medical products.

Preempted by federal law, these state efforts have had only symbolic effects. Yet the state successes have emboldened proponents to pursue a more direct attack at the federal level that would open the floodgates, gutting virtually all FDA oversight in this area.

The passage of these bills would be the ultimate victory for the Goldwater Institute. In one sweeping move, they would do away with federal standards for dying patients, including requirements for safety and efficacy testing, research ethics and even quality control in manufacturing.

False hope and perpetual uncertainty

The movement that the Goldwater Institute has helped to build is based on a tragic underlying truth: in spite of medical progress, patients continue to die from diseases that lack an effective treatment.

Though these circumstances are undeniable, offering broad access to experimental drugs is a grab at false hope rather than a true solution. The vast majority of drugs fail during clinical testing — even those that initially show promise. Some even bring about a swifter and more painful death.

There is a deeper risk as well: Offering false hope through inadequately tested products has the potential to undermine drug development by diverting patients away from placebo-controlled trials. This is a challenge for rare diseases in particular because finding enough patients for testing can be difficult.

The charismatic seven-year-old child whose story currently graces the front page of the Goldwater Institute’s polished website illustrates the potential pitfalls of side-stepping rigorous scientific review. This boy, the face of a movement, has Duchenne muscular dystrophy (DMD), a devastating condition that often leaves children bound to wheelchairs before their teenage years. When he was diagnosed, there was no FDA-approved treatment for DMD.

His family fought to find an effective treatment, eventually enrolling him in a clinical trial for an experimental drug called eteplirsen. Yet his mother chafed at the thought that he might receive a placebo. As she told one reporter, “There is a possibility my son will be receiving saline in that IV for 96 weeks, but what else are you going to do?”

In the trial, eteplirsen did not prove more effective than placebo, although FDA staff later pointed out that the trial was probably too small and poorly designed for all but the most miraculous of treatments to have succeeded. FDA officials internally opposed approval, as did a panel of outside expert advisers.

But DMD patients’ families pressed hard, arguing that the drug allowed children to walk long after they would normally be in wheelchairs. And they won: Eteplirsen was approved in September 2016 by FDA center director Janet Woodcock, overruling rank-and-file staff who had evaluated the scientific evidence.

Opponents of regulation may have felt vindicated by Woodcock’s decision. Yet what it truly offered patients was a chance to engage in false hope and perpetual uncertainty. There will be no more placebo-controlled trials for eteplirsen, and it will likely be more difficult to persuade families to enroll their children in better-designed trials for potential new treatments for DMD, impeding progress for this rare disease.

In a sad twist, eteplirsen’s $300,000 annual price will not be covered by insurance for many patients because insurers remain unpersuaded by the clinical trial data.

The uncertainty that persists about eteplirsen would likewise plague any drug made available under the federal false-hope bills. A key difference? Evidence for any benefits will be even slimmer.

Controversial treatments

The most troubling aspect of the federal bills that are being considered is the boost they would give to more controversial treatments, for which any evidence is so poor that the prospect of FDA approval is wholly imaginary.

At least one controversial cancer doctor, Stanislaw Burzynski, has already used Texas state law to circumvent FDA requirements and treat a five-year-old patient with brain cancer (the child subsequently died). If a federal false-hope bill passed, it would become nearly impossible for the FDA to stop doctors like Burzynski from profiting from unproven treatments.

The recent federal false-hope bills inflame this risk even further by preventing the FDA from scrutinizing side effects, injuries or deaths in patients treated under the terms of the bills. The federal bills also block state laws allowing doctors and manufacturers to be sued if they fail to exercise reasonable care in providing medical treatment.

The provision that blocks FDA scrutiny of adverse events will help questionable drug makers conceal injuries caused by unapproved products. By contrast, legitimate drug makers have little to fear from FDA review, as FDA interprets adverse events carefully in light of all the available information. As one FDA official testified last fall, after almost 11,000 expanded access requests over a decade, only twice has the FDA ever paused drug development in response to an adverse event in a patient using the program, and in both cases development resumed after issues were addressed.

Likewise, the main beneficiaries of the shield against lawsuits will be fraudsters and medical quacks who currently are liable for medical malpractice under state law. Such lawsuits are essentially non-existent for careful doctors operating under the FDA’s Expanded Access Program.

Another path forward

Last year, the FDA took significant steps to streamline paperwork for its Expanded Access Program with a new application form that should take doctors only 45 minutes to fill out. Congress also passed a law last fall to address patients’ frustration with uncooperative drug manufacturers, requiring the manufacturers to clearly post their expanded access policies.

Another positive step Congress could consider would be to create a patient advocacy office within the FDA tasked with further assisting dying patients. Such an office could also identify potential problems with the program and propose additional streamlining measures where needed.

With the President on board and members of Congress beginning to fall in line to support it, false-hope legislation may soon become the law of the land. But there is still time for lawmakers to take a different path, one that strengthens the FDA’s Expanded Access Program, rather than taking a hatchet to the system that currently serves and protects patients.