A Guide to the ‘Right to Try’ Debate

by Dramelin 7:30 AM 0 comments

During his first State of the Union Address, U.
President Donald Trump endorsed a proposed national "Right to Try" law, which aims to make it easier for terminally ill patients to get access to experimental drugs.
The idea has widespread and bipartisan support: A bill passed unanimously in the Senate last August, and 38 states have enacted their own versions.
However, some physicians and medical ethicists, as well as the drug industry, oppose such legislation, arguing that it’s unnecessary and not in the best interests of patients or the general public.
What’s the idea behind ‘Right to Try’? The notion of giving terminally ill Americans access to a broader universe of experimental therapies has been around for decades.
In the 1970s, many states legalized an apricot pit extract that was heralded as a breakthrough for some people with terminal cancer but had been banned by the U.
government from importation and interstate commerce.
(The drug, Laetrile, has never been shown to be a safe or effective treatment and isn’t approved by the U.
Food and Drug Administration.
) Advocates of greater experimentation have gone to court, unsuccessfully, to show that a "right to try" is guaranteed by the Constitution.
The latest push is aided by the Goldwater Institute, a libertarian think tank based in Phoenix that, among other things, runs a pro-Right to Try website.
The group worked with lawmakers in Colorado to get the first state-level law passed, in 2014.
What’s the status of a national version of the law? Under the bill passed by the Senate, a patient with a life-threatening disease who’s exhausted all approved treatment options and can’t participate in a trial of an experimental drug would be allowed to take an unproven medicine without FDA permission.
Just the patient, his or her physician, and the drug manufacturer would need to agree.
A House committee held a hearing on a version of the bill last October, but it has yet to advance.
In his State of the Union address Jan.
30, Trump gave it a dramatic endorsement, describing terminally ill patients going from country to country to seek cures.
“It is time for Congress to give these wonderful Americans the right to try,” he said.
What options do these patients have now? They can apply, through their physicians, to receive experimental drugs through an FDA-run system called compassionate use or expanded access.
"Right to Try" advocates say the FDA process is time-consuming.
While acknowledging that was a problem in the past, the FDA says its current application takes about 45 minutes to complete.
The agency approves more than 99 percent of compassionate-use requests, usually within four days, and emergency requests are usually granted immediately, over the phone.
So what’s the problem with access? It’s not so much the FDA but rather drug companies that limit patient access to unproven treatments.
The reasons are varied.
Biotechnology startups, the center of some of the most daring medical innovation, usually don’t have the resources to handle compassionate-use requests while trying to bring a product to the market.
Many companies make limited batches of experimental drugs and may not have quantities available beyond what they need for clinical trials.
They may fear that adverse effects of experimental drugs given to very sick patients will compromise efforts to win regulatory approval of those treatments.
Adverse reactions could also spark lawsuits and attract immediate FDA scrutiny.
How would a ‘Right to Try’ law help? The Senate bill would protect manufacturers from any liability or increased regulatory review should a critically ill patient be harmed -- or even killed -- by an unapproved treatment.
However, pharmaceutical companies could not be compelled to provide anyone an unproven treatment -- just as they aren’t today.
(Under a law enacted in 2016, drugmakers are required to give patients information about compassionate-use requests on their website.
What are the arguments against ‘Right to Try’? Critics say that taking the FDA out of the process removes protections for patients.
Regulators, they say, are likely to be more aware than a patient’s physician of little-known risks with similar medications and can give guidance on how best to use drugs.
Opponents also argue that tending to the urgent needs of one person, no matter how compelling, can slow the development of medicines for everyone else.
Patients who are terminally ill may suffer complications or die after taking experimental medicines, potentially jeopardizing clinical studies for drugs that could benefit others.
For instance, trial volunteers might reconsider their participation if there are reports of a devastating side effect with another patient.
Similarly, investors may pull their support from a company if one of its medicines is tied to an unexpected complication, hindering future development.
The Reference Shelf — With assistance by Anna Edney.