(Bloomberg) -- U.S. President Donald Trump has signed into law a national “right to try” measure, intended to make it easier for terminally ill patients to get access to experimental drugs. Both Republicans and Democrats in Congress voted in favor of the bill, making it a rare political issue with bipartisan support. Forty states have enacted their own versions of right-to-try. But some physicians and medical ethicists -- as well as the drug industry and even patient-advocacy groups -- oppose such a right as unnecessary and not in the public interest.
1. What’s the idea behind a 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 push for the national law was 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. What does the national law do?
Certain patients with life-threatening diseases who have exhausted all approved treatment options and can’t participate in a trial of an experimental drug will 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 must agree.
3. What options have these patients had until now?
They could apply, through their physicians, to receive experimental drugs with FDA permission through a system called compassionate use or expanded access. Right-to-try advocates consider that process overly 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.
4. 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.
5. How will the right-to-try law affect drug companies?
The federal legislation will 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 will be shorter, involving just the doctor and the manufacturer, without regulatory review. However, pharmaceutical companies can’t be compelled to provide anyone an unproven treatment.
6. What are the arguments against a 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.
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