During his first State of the Union Address, U.S. President Donald Trump endorsed a national “Right to Try” law aimed at making it easier for terminally ill patients to get access to experimental drugs. An effort to pass such a law failed this week when the House of Representatives voted down a version of it that had passed unanimously in the Senate last August. Still, the debate over “Right to Try” will continue. The idea has widespread and bipartisan support: 38 states have enacted their own versions. Some physicians and medical ethicists as well as the drug industry and even patient-advocacy groups oppose such legislation, arguing that it’s unnecessary and not in the public interest.
1. What’s the idea behind ‘Right to Try’?
The notion of giving terminally ill Americans access to more experimental therapies has been around for decades. Advocates have argued unsuccessfully in court 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.
2. Is the effort to pass a national law dead?
No. Its supporters say they will continue to push for one, and their prospects are good. The bill in the House failed only because the vote required a two-thirds rather than the simple majority that voted for it. That’s because the bill was put to a vote without going through deliberation by a committee, using a maneuver usually reserved for noncontroversial measures.
3. What would a national version do?
A federal law would prevent the U.S. regulatory and legal systems from punishing those who participate. 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 the permission of the Food and Drug Administration. Only the patient, his or her physician, and the drug manufacturer would need to agree. The version considered by the House more narrowly defined which patients may have access to the experimental drugs. In his State of the Union address Jan. 30, Trump offered 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.
4. What options do these patients have now?
They can apply, through their physicians, to receive experimental drugs with FDA permission through a system called compassionate use or expanded access. “Right to Try” advocates say that 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. Under a law enacted in 2016, drugmakers are required to give patients contact information and other details about compassionate-use requests on their website.
5. Then why are there complaints about access to drugs?
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 patients will try to get access to experimental drugs rather than participate in clinical trials needed to win wider approval of the medicines. Adverse effects of experimental drugs given to very sick patients could compromise efforts to continue development of those treatments, spark lawsuits, spook investors and attract immediate FDA scrutiny.
6. How would a ‘Right to Try’ law help?
The federal legislation would protect manufacturers from any liability or increased regulatory review should a critically ill patient be harmed -- or even killed -- by an unapproved treatment. The path to access would be shorter, involving just the doctor and the manufacturer, without regulatory review. However, pharmaceutical companies could not be compelled to provide anyone an unproven treatment -- just as they aren’t today.
7. What are the arguments against ‘Right to Try’?
Critics say 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.
--With assistance from Anna Edney
To contact the reporter on this story: Michelle Fay Cortez in Minneapolis at firstname.lastname@example.org.
To contact the editors responsible for this story: Cecile Daurat at email@example.com, Lisa Beyer
©2018 Bloomberg L.P.