The FDA vs. the flash mob: Hard questions as parents demand unproven drugs

Last week the parents of 7-year-old Josh Hardy managed to whip up a media tempest big enough to get the biotech Chimerix ($CMRX) and the FDA to work out a pilot study so that their child could be treated after their compassionate use request was denied, alongside many others. Now the parents of another young boy afflicted by a terrible disease are using some interesting PR tactics in an attempt to achieve a much larger goal: Push the FDA to approve experimental drugs for Duchenne muscular dystrophy (DMD) ahead of pivotal studies in the hope that it could let their young son, Ryan Dunne, live a "healthy life."

The local CBS affiliate in Colorado turned out for a flash mob demonstration yesterday, with protesters chanting Ryan's name and giving his parents a chance to highlight their campaign to get 100,000 signatures together to push the FDA to make unapproved DMD drugs available to a young patient population with no therapies approved for the ultimately lethal muscle-wasting disease. Other parents have been turning out in large groups to give FDA officials an earful on the same point. And the "Race to Yes" campaign today added heat with their own statement backing the petition, which is specifically designed to get an accelerated approval for Sarepta's ($SRPT) eteplirsen. 

"We don't have time to wait for the FDA," says Jennifer Dunne in her appeal at "We need to do it now," says Ryan's father, adding that his son can't wait years for the FDA to act.

"Every day that the FDA stalls, Duchenne takes children's lives," said Tracy Seckler, the parent of a child with DMD, in a statement. "The FDA claims this is a priority, but they've been sending us the same bureaucratic form letter for several months. If they grant accelerated approval, our children could be the first generation of Duchenne patients to survive."

The campaign raises compelling questions. Does the FDA have a responsibility to allow children access to unproven drugs, provided there's evidence of their safety and the possibility of a positive outcome--of any kind? A group of companies, in particular Sarepta, Prosensa ($RNA) and PTC Therapeutics ($PTCT), have all provided some signals from the data that could indicate a benefit. But Sarepta only has data from a tiny study the agency has already questioned, looking to a pivotal study to decide whether the drug really works. Prosensa's drug failed a Phase III study conducted by GlaxoSmithKline ($GSK). PTC's earlier study for DMD also failed. And all three biotechs insist that there are compelling signs that the drugs may work for some of these boys.

If the FDA eventually decides to capitulate on this point, the biotechs would have a chance to market drugs to children. That could set up another showdown with restive private payers which may well reject covering drugs that are green-lighted without solid evidence of efficacy, approved under traditional guidelines. And that might leave parents to pay a high cost for unproven medications which may have no more effect on the disease than a placebo.

Parents and children don't want to hear about traditional guidelines when they reflect on what's happening to their child. The public will side with the victims, and that leaves the FDA caught between the high R&D standards it's mandated to uphold and the passions aroused by parents, the victims of disease and protesters.

There are no easy answers here. Just hard questions. -- John Carroll, editor-in-chief (email | Twitter)