Advocates Fight for Legislation to Allow Early Access for Experimental Drugs

The Right to Try Act allows terminally ill patients to take investigational treatments that have passed basic safety testing.

The Right to Try Act has gained popularity among both citizens and legislators alike, who support legislation to allow terminally ill patients to gain access to investigational drugs. The law targets drugs that are not yet available to the public, but have advanced passed the phase 1 trial stage.

The Goldwater Institute, a libertarian think tank in Arizona, are a chief advocate of right-to-try laws. According to their website righttotry.org, more than 1 million Americans die from a terminal illness each year.

Although there are options for a select few patients, such as participating in clinical research trials or using the FDA’s expanded access program, this still does not provide enough access, according to the institute.

“Fewer than 3% of terminally ill patients gain access to investigational treatments through clinical trials,” righttotry.org reported. “Right to Try was designed to help the other 97%.”

As for the expanded access program, only about 1000 patients are granted compassionate use per year, with many of the patients dying before they can qualify for the exemption or complete the process. As of now, there are 31 states who have passed a Right to Try law, with Colorado being the first to enact the law in May 2014.

Although the laws have gained overwhelming support, there are still critics who believe the lesgislation is providing false hope.

“Patients with terminal illness are the perfect cases for being exploited and exposed to high levels of uncertain risks with low chance of benefit,” Ezekiel J. Emanuel, MD, PhD told HemOnc Today. “Some people argue that these people would die anyway. Yes, but we all know that there are better and worse ways of death. Part of what doctors are committed to is making it a better death and not preying on people’s false hopes.”

Other critics have described right-to-try laws as “feel good” legislation that do not yield positive results.

“The whole Right to Try movement is premised on the fact that these are life-saving, miracle drugs that are going to bring people back from the brink,” said Lisa Kearns, in an interview with New Hampshire Public Radio’s Morning Edition.

Most critics agree that patients believe the drugs will provide them with a cure, and that the FDA is only holding out on them. However, supporters of the law state that the description is inaccurate.

“Right-to-try laws give people with terminal illnesses the legal right to use investigational medications years before they might otherwise be available on the market,” wrote righttotry.org. “No one can guarantee that a particular treatment will be effective, but these laws return choice and control over treatment options to where it is most effective: with patients and doctors.”

The mindset for terminally ill patients who are in support of the law is that they have nothing to lose, so why can’t they just try?

In a YouTube video, ALS patient and veteran Matt Bellina said assisted suicide laws allow terminally ill individuals to end their life, yet he is unable to gain access to investigational drugs that could potentially help his disease.

“There are states in this country where I could go legally end my life, but I’m not allowed to go try a drug that I believe could save my life,” Bellina said in the video.

During a press conference held by US Sen. Ron Johnson (R-Wis) last month, he announced a Right to Try bill called the Trickett Wendler Right to Try Act of 2016. Several patients with ALS patients spoke at the conference, as reported by The Hill.

Currently, there are some experimental drugs for ALS in clinical trials, but the entry criteria are restrictive, requiring compassionate use requests for a majority of ALS patients who cannot be enrolled in the studies.

“The patients are smart enough to read into things, and as long as the company’s being honest about the potential, why won’t you just let us try it? I think it’s ridiculous that the FDA doesn’t let us,” Bellina told Specialty Pharmacy Times.