Is Right to Try Legislation Really Necessary?

Update (May 24, 2018): The US House of Representatives has now passed the amended Right to Try legislation. The bill must now be signed by President Donald Trump for it to be written into law. Despite FDA Commissioner Scott Gottlieb’s former reservations about the Right to Try Act’s consequences, he tweeted that he will be “ready to implement it in a way that achieves Congress’ intent to promote access and protect patients.”

Originally published on October 27, 2017:

Improving patient access to potentially life-saving treatments is a goal that nearly everyone can agree upon, particularly when those patients are terminally-ill and have few other options. So when Wisconsin Senator Ron Johnson introduced the Trickett Wendler Right to Try Act in May of 2016 – a piece of legislation designed to help patients try investigational new drugs before they’ve been approved by the US Food and Drug Administration (FDA) – it received overwhelming bipartisan support.

“Right to Try is all about freedom – the freedom for terminally ill patients to have access to potentially life-saving treatment, and more importantly, the freedom to hope,” said Sen. Johnson in a February press release about the legislation.

The legislation was passed in the Senate in August, and is set to be voted on by the House of Representatives in the coming weeks. If approved, the bill would effectively remove the FDA from the equation and allow seriously ill patients to access medications that have only undergone safety testing in early Phase I clinical trials.

Federal Right to Try Legislation Would Override FDA

But as Health News Review points out, the FDA already has a program in place to address this patient access issue. Its expanded access program – also known as compassionate use – grants eligible patients the chance to try an experimental drug, provided their doctor and the hospital where they’ll be treated agree to administer the treatment.

Proponents of the Right to Try Act – namely the public policy group, the Goldwater Institute – say the legislation could help patients get immediate access to potentially life-saving medical treatments that would be considerably faster than the FDA’s current system. They argue that many terminally ill patients fail to meet the stringent enrollment requirements for clinical trials, and will often run out of time before being granted a compassionate use exemption, making Right to Try laws important in ensuring they have access to new treatments.

But while some say the FDA’s compassionate use program is slow, cumbersome and does not grant exemptions to enough dying patients, the regulator is telling a different story. In a recent blog post, FDA Commissioner Scott Gottlieb said that the regulator authorizes 99 percent of the over 1,000 compassionate use applications it receives each year. Moreover, he says that these authorizations are granted expeditiously, with most exemptions being approved in just a few days. In some cases, he says the FDA will authorize compassionate use of an investigation therapy in a matter of hours, if the circumstances surrounding the application are deemed to be urgent enough.

A study published in 2016 found that out of almost 9,000 applications for expanded access, the FDA recommended changes to key variables in patient care – such as dosing and safety monitoring – in 11 percent of cases. However, these amendments were made in an attempt to improve the outcomes of treatment, as opposed to preventing these patients from accessing the drugs altogether. Less than one percent of applications were denied, often because of the availability of alternative therapies, the proposed dosing regimen was deemed unsafe, or simply because an application was incomplete.

“Often the agency possesses safety information about investigational medical products that is unknown to the treating physician,” wrote Alison Bateman-House, a medical ethicist at New York University’s Langone Medical Center, in an article for Forbes.

No doubt in response to the introduction of the Right to Try Act by Senator Johnson, the FDA has also introduced new measures to simplify the process of applying for expanded access. While the regulator previously required physicians looking to administer an experimental therapy to a patient to first gain approval from their institutional review board (IRB), they now only require a single member of the committee to give the go-ahead.

“I believe the simplified IRB process will facilitate access while still protecting patients,” said Gottlieb of the change. “More simplifications and clarifications are in the pipeline.”

According to Bateman-House, pharmaceutical companies, and not the FDA, are most often the barrier in preventing terminally ill patients from accessing new, unapproved treatments. “The idea the FDA is the stumbling block is completely wrong. We don’t know how many times requests to the drug companies are approved or denied. But if the drug company says ‘no,’ that’s the end of it.”

Since even the FDA can’t compel pharmaceutical companies to provide their investigational drug to patients outside of a clinical trial, no federal Right to Try law would be able to address this barrier to patient access. In fact, the FDA has already started to address this problem by releasing updated guidance for drugmakers who may be wary about providing their products through the expanded access program for fear that any adverse event seen in these patients could jeopardize further clinical development of their drug.

States Have Already Implemented Right to Try Laws

So far, 37 states have introduced Right to Try laws, with Pennsylvania Governor Tom Wolf being the most recent politician to sign legislation in October.

“I have supported and will continue to support legislation that gives patients power in their health care decisions,” Governor Wolf said. “At no time is that more important than when faced with a terminal illness. Having the chance to try all options can offer hope and better quality of life for many”

So, with over one-third of US states enacting Right to Try laws, what’s the need for federal legislation? Since drugs and medical devices are regulated by the FDA – a federal body – supporters of Right to Try say that without a federal law, the regulator could trump any state attempts to enforce the legislation.

But removing the FDA from the picture has worrying implications. Patients may not be fully educated on the potential negatives of taking an experimental therapy, including side effects that haven’t yet manifested themselves during clinical trials of the drug.

With all the controversy and concern surrounding Right to Try, some might wonder how the legislation has garnered so much support from both sides of the aisle; after making an amendment, the Trickett Wendler Right to Try Act was unanimously passed in the Senate.

According to Beth E. Roxland, senior adviser on law, ethics, and policy at NYU Langone Medical School, it comes down to not wanting to be on the ‘wrong’ side of an emotional argument. She told STATNews, “It’s hard to argue against when it’s framed as the terminally ill having the right to save their lives,” she said. “How do you argue against that?”

At the heart of the issue, Right to Try is about providing patients who are out of options with the chance to test cutting-edge therapies. Most terminally-ill patients don’t have a decade to wait for an investigational drug to be given the go-ahead by the FDA, and most will never be approved.

It can take up to 12 years for a drug to be approved, and while that’s not an inconsequential amount of time, it’s necessary to assess whether a drug does what it’s designed to do without presenting intolerable risks to patients. Though President Trump and others have promised to deregulate the FDA for a faster approval process for new drugs, this would come at the expense of regulations put in place to protect patients from drugs that may turn out to be neither efficacious nor safe.

Bateman-House recently gave testimony to Congress on her research regarding compassionate use and some of the common misconceptions surrounding the FDA’s program. Her conclusions regarding Right to Try mirror those of others in the industry who are familiar with the FDA’s expanded access program, and are working towards making sure doctors and pharmaceutical developers understand it too.

“Instead of promoting Right to Try, we must focus on making expanded access even better by making sure people, particularly those in medical and healthcare settings, know what it is, how to use it and so on,” wrote Bateman-House. “And finally, since the ultimate decision to grant access to experimental medical products is up to individual companies, we need to work with these companies to find out why they deny requests and what, if any, policies or incentives would make them more likely to say yes.”

Do you think Right to Try laws are necessary to improve patient access to investigational treatments? Share your thoughts in the comments section below!