Experimental Drugs for Terminally Ill Are at Center of Growing Debate Over States’ “Right to Try” Laws

Monday, May 19, 2014
Josh Hardy (photo: Save Josh)

Imagine you were terminally ill and had exhausted all conventional treatments for your sickness. Then you hear about a promising drug that had the potential to cure you, but it hadn’t been approved for use. You might be willing to give that drug a try, figuring you had nothing to lose.

That’s the reasoning behind three states’ recent approval of “right to try” laws. In Colorado, Missouri and Louisiana, physicians will be allowed to recommend that their patients be able to access a potentially life-saving drug before the Federal Drug Administration (FDA) has given its approval.


“For people who are facing death and have one last hope, they should have a choice to try every possible drug,” Colorado state Rep. Joann Ginal (D) told The Washington Post.


Frank Burroughs, founder of the Virginia-based Abigail Alliance for Better Access to Experimental Drugs, told the Post that the FDA doesn’t move quickly enough for patients who otherwise have no hope. “The risk-benefit is much different than someone who’s waiting for a new allergy medication or a new toe fungus cream,” he said.


The FDA can and sometimes does grant “compassionate use” exemptions for patients in life-threatening circumstances to use investigational drugs. But the right-to-try laws can make it easier for patients to access those medications. Manufacturers, however, are not obligated to provide the drugs.


One case that drew nationwide attention was that of 8-year-old Joshua Hardy. After four bouts of cancer and the treatment to fight it off, Joshua’s immune system was damaged and a virus was about to kill him.


Chimerix, a small drug company, was working on a promising treatment for Joshua’s condition. However, it had halted its compassionate use program to focus on testing its drug to get it to market. A social media campaign was begun to get the company to provide the drug to Joshua, but Chimerix said it was unfair to provide it to one person while denying it to many others. Eventually, the FDA approved a trial for the drug’s use by children. Joshua was put into the trial and so far the drug is working for him.


The social media campaign took a toll, however. Chimerix chief operating officer Kenneth I. Moch received death threats over his company’s initial stance and he has since been replaced.


At least one company is embracing the right-to-try laws. Neuralstem is looking for Colorado doctors willing to try its treatment for Lou Gehrig’s disease. The treatment involves surgically transplanting neural stem cells in the patient’s spinal cord.


Right-to-try laws are coming too late for Amy Auden, of Lone Tree, Colo. Her husband Nick died of melanoma last year. The Audens had tried—unsuccessfully—to persuade Bristol-Myers-Squibb and Merck to allow Nick to try promising treatments the companies were developing. Nick died in November. The drug the Audens attempted to access works in 52% of patients.


“Not a day goes by where it doesn’t haunt me,” Auden said. “Those with serious illnesses should not have to fight the illness as well as fight for the right to gain access to lifesaving treatments.”

-Steve Straehley


To Learn More:

‘Right to Try’ Laws Spur Debate Over Dying Patients’ Access To Experimental Drugs (by Brady Dennis and Ariana Eunjung Cha, Washington Post)

Crowdsourcing Medical Decisions: Ethicists Worry Josh Hardy Case May Set Bad Precedent (by Ariana Eunjung Cha, Washington Post)

Chimerix CEO Out In Wake Of Josh Hardy Compassionate Use Media Frenzy (by David Kroll, Forbes)

Why is Obama Administration Spending $433 Million on Experimental Smallpox Drug When Last Case was in 1949? (by Noel Brinkerhoff, AllGov)


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