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Advocates of the controversial Right to Try bill argue that stifling FDA regulations make it harder for terminally ill patients to receive potentially lifesaving treatment. Photo credit: FDA/Wikimedia

Will Controversial Bill Cure or Kill Patients?

Libertarians Want to Sideline FDA on Experimental Drugs

09/07/17

“Right to Try” legislation would allow pharmaceutical companies to give experimental drugs to desperate patients -- with no chance of being liable if things go wrong. Supporters of the measure cheer this approach but critics see dangers.

In 2016, 10-year-old Joshua Hardy lost his long battle with cancer, in part because of a viral infection that resulted from a bone marrow transplant. Hardy’s family tried to get access to a new experimental antiviral drug from Chimerix, the company developing it. The company refused, not wanting to divert time and resources from its efforts to gain FDA approval for the drug. It agreed to make the drug available only after a social media campaign.

Hardy responded well to the drug, but it was too late.

Hardy’s story is one of those featured in the lobbying campaign for a federal Right to Try (RTT) bill, which passed the Senate in August and may soon get a vote in the House.

But the Senate-passed bill would not require drug companies to provide dying patients access to their experimental drugs. Instead, it largely goes after the Food and Drug Administration, and would remove FDA oversight from the process, while insulating the pharmaceutical industry and doctors from virtually any liability if patients are harmed.

RTT pits some desperate patients, along with libertarians and conservative and anti-regulation Republicans, including President Donald Trump and Vice President Mike Pence, against public health advocates, medical ethicists, and some national patient-advocacy groups.

The bill’s supporters frame the legislation as striking a blow for the rights of patients to try any drug without government interference. They predict that more pharmaceutical companies would give patients access to their unproven drugs if they weren’t worried about the FDA looking over their shoulders.

The bill’s opponents contend that any possible expansion of access comes at the cost of eliminating crucial protections for the most vulnerable patients, potentially hastening their deaths and making their end more painful.

Opponents are circulating a letter to the House this week charging that RTT should actually be called the “False Hope” bill. Indeed, given the philosophical bent of its strongest supporters, and their connection to free-market crusaders Charles and David Koch, RTT may be more about advancing a conservative, anti-government ideology than helping the terminally ill.

As Dr. Michael Carome, director of the Public Citizen’s Health Research Group told WhoWhatWhy: “The bill reflects a libertarian philosophy that people can take whatever risk they want without any regulatory safety hurdles.” He added that RTT will “immunize just about everyone who might be involved” — the sponsor, the drug manufacturer, the dispenser, the doctor — from virtually any lawsuit. “It reflects a wider push to limit liability for consumers and patients who are injured.”

The Senate-passed bill would permit patients with life-threatening illnesses, certified by a physician to have exhausted all treatments and to be ineligible for a clinical trial, to ask companies for access to experimental drugs that have received only preliminary screening by the FDA. Companies cannot require patients to pay more than the cost of manufacturing the drug. But patients would have few rights to sue should the drugs harm or kill them.

RTT would essentially offer patients an alternative pipeline for experimental drugs than the one the FDA runs through its expanded access program.

The FDA program contains safeguards that RTT does not include. For example, the FDA, in conjunction with an independent panel of experts who are tasked with ensuring the ethical treatment of patients taking experimental drugs, evaluates the drug and its effect on the specific patient: Will the experimental drug make a difference? Are the risks of taking the new drug no greater than the risks posed by their disease?

Physicians must report any adverse events that patients experience to the sponsor and manufacturers, and they in turn must report these events to the FDA. Patients must give their consent, after being fully informed about the drug’s potential side effects.

That’s not the case for RTT. The bill removes all FDA involvement in the process, and greatly limits what the agency knows about the drug after it is administered.

That’s because RTT would not require physicians to report any adverse events to either the drug companies or the manufacturer, potentially shielding companies from their obligation to report such events to the FDA. You can’t report what you don’t know.

Even if the agency learned about a drug’s harmful side effects, the FDA could not use that information to “delay or adversely affect the review or approval of such drug,” except in instances when the head of the agency insisted, in writing, that the information was crucial to the drug approval process.  

RTT also expands the number of patients who could ask for access to experimental drugs, including not only terminally ill patients, but also patients with life-threatening diseases. That could mean, Carome said, that patients with a wide range of illnesses, including asthma, heart disease and diabetes, could be covered by RTT, provided that “they’ve exhausted” other treatment options.

The legislation has been strongly pushed by the libertarian Goldwater Institute, which successfully fought to pass RTT in 34 states.  

Goldwater Institute president Victor Riches praised Senate passage, declaring that “[T]here’s no more fundamental freedom than the right to save your own life. Right to Try guarantees that freedom by ensuring that patients, along with their doctors, are in control of the treatments they receive when facing a terminal diagnosis.”

Sen. Ron Johnson (R-WI), one of the Senate’s most conservative members, and an opponent of agency regulations, drove the push for passage. Before the Senate recessed in early August, Johnson threatened to block a must-pass bill reauthorizing FDA user-fees from drug companies, unless his proposal got a vote.

Johnson claimed that his bill “stood up for terminally ill patients who just want to reclaim their freedom — who want the right to hope.”

Ron Johnson

Sen. Ron Johnson (R-WI) was one of the proponents of the Right to Try bill, which would increase access to experimental drugs while decreasing liability of drug companies. Photo credit: US Customs and Border Protection/Flickr

Some groups, particularly those including patients with terminal illnesses and their families, have lobbied for RTT, framing the bill as their last chance to find a cure. But not all patient advocates agree.

National groups that have opposed RTT include Breast Cancer Action, the National Women’s Health Network, and the Isaac Foundation, which lobbies for new treatments for rare diseases. They share the concerns of public health advocates and medical ethicists about patient protections and the lack of FDA oversight. They also doubt that RTT will work.

Even some AIDS groups oppose this approach, despite their past efforts to prod the FDA to approve anti-AIDS drugs much faster. Those efforts bore fruit, and since the 1990s, the FDA has accelerated its drug approval process. Some AIDS activists now are concerned that libertarian efforts like Right to Try could weaken the agency and its authority to protect the public.

In truth, it appears that no one can quite predict the real-world impact of the bill. Up until now, drug companies have been reluctant to provide drugs to terminally ill patients, preferring to test their drugs on healthier subjects through formal clinical trials.

Despite RTT’s relaxed liability, lack of FDA oversight, and a potentially larger patient population, the pharmaceutical industry has not supported the bill. Indeed, some drug companies reportedly lobbied quietly against it.  

That does not surprise public health advocates, who spoke to WhoWhatWhy. Diana Zuckerman, president of the National Center for Health Research, points out that drug companies rely on a three-phase clinical trial process to gain FDA approval for their products. If RTT siphoned off patients with life-threatening illnesses from clinical trials, particularly for trials of medications serving limited populations with rare diseases, drug companies might find it even harder to enroll enough patients to complete them. And health insurers will not pay for drugs that lack FDA approval.   

And while RTT is designed to get FDA off patients’ backs, it does not appear that patients are being denied access because of FDA intransigence.

In congressional testimony last year, FDA official Dr. Peter Lurie said that the agency approves 99 percent of all requests from terminally ill patients to use these experimental drugs. Requests are usually processed within days, and emergency requests may be granted immediately.

Between 2010 and 2015, the agency refused just 66 patients out of the thousands of requests it has received. Drug companies are far more likely to refuse patient requests than the FDA, Lurie added. He also said that the FDA recently streamlined its forms and made the process more user-friendly for doctors. Over the past two years, Congress also passed two laws to improve the expanded-access program, and to require that drug companies disclose their policies on access to experimental drugs.

RTT’s true beneficiaries may be new drug startups that are neither “patient-oriented nor compassionate, trying to create buzz for their product,” Zuckerman said. A biotech startup, she speculated, might be struggling with the high costs of making its product, and be happy to find wealthy, desperate patients willing to pay the freight. This might be particularly attractive since RTT gives patients only limited rights to sue, even if they are seriously harmed by defective products.

Unscrupulous doctors also might benefit, she added, since the law does not place any restrictions on the fees they may charge patients to administer the drug and monitor their progress.

Charles and David Koch, too, may be taking a victory lap if RTT becomes law. The Goldwater Institute reportedly has received millions of dollars in donations from organizations connected to the Koch brothers. The Kochs are aggressive supporters of limited government and deregulation. They also have supported so-called “tort reform” efforts to protect businesses from consumer lawsuits.

Neither Koch has said much about the FDA. But in a wide-ranging interview last year with the Washington Post, Charles Koch did complain that the agency made it “almost impossible to develop a new drug.”


Related front page panorama photo credit: Adapted by WhoWhatWhy from painkillers by Vnukko/Pixabay

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