Young boy’s struggle to survive sparked widespread interest DRUGS FOR TERMINALLY ILL

By Liz Szabo

Kaiser Health News (TNS)

WASHINGTON

At age 7, Josh Hardy became the poster child for a movement to help desperately ill or dying patients access experimental therapies which, they hope, will save their lives.

People around the world rallied 21/2 years ago when a small biotech company refused to provide the Fredericksburg, Va., boy, who had battled cancer since infancy, with an experimental drug that doctors believed could save Josh from a life-threatening infection. Within days, a social media campaign called SaveJosh, sparked by his mother’s anguished Facebook post and propelled by well-connected supporters, spurred thousands of people to contact the company, Chimerix Inc. of North Carolina, prompting it to release the drug.

For a time, Josh recovered. But his body remained weakened by disease, and Josh died last month at age 10.

Yet the SaveJosh campaign has had an enduring effect on efforts to expand “compassionate use” of experimental therapies.

Josh’s story – and the plight of thousands of others facing life-threatening diseases with no government-approved therapies – has inspired multiple, sometimes conflicting efforts by government, industry and activists to make experimental medications more widely available.

The Food and Drug Administration, whose staff initiated the clinical trial that provided Josh his medication, has reduced the time that doctors spend requesting an unapproved drug to just 45 minutes. The agency says it approves 99 percent of such applications, granting most requests in four days and emergency requests within hours. The agency is even considering hiring “navigators” to walk doctors through the process.

Nonetheless, some patient advocates are leading a drive to provide access to experimental drugs without the FDA’s help.

Thirty-two states have passed laws designed to give patients the “right to try” an unapproved medication, by protecting doctors who provide such therapies from losing their state medical licenses. Republicans have included the right to try unapproved drugs in their national presidential platform.

Christina Sandefur, executive vice president at the libertarian Goldwater Institute, which has coordinated right-to-try efforts, said terminally ill patients shouldn’t have to ask for a “permission slip” from the FDA. “If you are dying and you’ve exhausted all other options and this medication has passed basic safety testing, you should be able to work directly with the drug companies and your doctor without having to go through the FDA.”

Yet even some supporters of the right to try acknowledge the state laws have helped relatively few patients.

The renewed interest in compassionate use – which first emerged as a national issue during the AIDS crisis of the 1980s – also has spurred pharmaceutical companies to ramp up “expanded access” programs.

Right-to-try laws wouldn’t have helped Josh Hardy, because the laws don’t require drug developers to actually make their medications available, Bateman-House said. The laws also don’t require that insurance companies pay for the treatments. The movement’s main intent is to undermine the FDA, Bateman-House said.

“The cruel reality with the right-to-try legislation is that it will not grant patients the immediate access to treatments they desperately need,” said Andrew McFadyen, executive director of the Isaac Foundation, a nonprofit based in Ontario, Canada, that funds research to fight MPS, the fatal genetic disease that afflicts his 12-year-old son.

The first doctor to openly acknowledge using a state right-to-try law, Houston cancer specialist Ebrahim Delpassand, came forward in September at Johnson’s hearing, where he testified via video. Delpassand said that he has given an unapproved drug, LU-177, to 78 patients with advanced neuroendocrine cancers under Texas’s right-to-try law.

Those numbers are dwarfed by the number granted expanded access by the FDA, which last year approved 1,262 requests for all drugs. The actual number of patients included in these FDA requests is much larger, because some requests were made on behalf of trials that include hundreds or even thousands of patients.

For example, about 35,000 patients received the lung cancer drug Iressa through expanded access before that drug was approved, said Klein.

At the Senate hearing, Johnson acknowledged that state right-to-try laws haven’t produced as many big wins as supporters had hoped.

Although state right-to-try laws protect doctors from losing their licenses if they prescribe unapproved drugs, physicians may be reluctant to use the laws, for fear of violating federal regulations, Johnson said.

Even in a right-to-try state, doctors such as Delpassand “are taking a huge risk” in providing unapproved treatments, Johnson said. “We need the federal law for the state laws to kick in. Until the federal government acts, the state laws are not effective.”