The cost of compassion

FDA reconsiders rules for letting patients who are gravely ill get unapproved drugs

April 15, 2007|By Tricia Bishop | Tricia Bishop,Sun reporter

Before he was even a year old, Luis Fernando Rojo was near death, suffering from a fatal disease that was causing his organs to fail and blisters to break out all over his small body.

Commercialized therapies weren't helping the Florida infant, and his doctors at Holtz Children's Hospital in Miami were desperate. So they called Baltimore's Osiris Therapeutics and asked a costly favor: They wanted free access to one of the company's experimental, unapproved and expensive stem cell treatments, allowable under a provision within federal regulations. Osiris said OK.

"After he was given the drug, he improved 100 percent," the boy's father, Hilario Rojo, said last week through a Spanish-speaking interpreter. Today, the toddler is 28 months old and faring well.

Biotechnology companies often deal with deadly diseases, developing drugs for the world's sickest patients, many of whom have few treatment options and often are willing to try anything to see if it will help save them. But relatively few eligible businesses want to make developing drugs available in circumstances like Luis', in part because such efforts do little to move the product to the mass market.

The U.S. Food and Drug Administration allows people to try therapies that haven't been approved for marketing on a "compassionate use" basis, as long as certain criteria are met. But the agency, which is considering changes to its regulations meant to expand access to such drugs, can't compel companies to offer up their investigational products.

And many say there are few reasons a business would want to make the offer, given the financial pressures they're already facing in drug development. Others worry that these experimental drugs can offer false hope to patients and could be dangerous given the lack of information known about how, or if, they work.

Such treatments offer little in the way of useful data, can be detrimental to clinical trials and often cost thousands of dollars to provide. Insurance companies typically won't reimburse patients for such therapies. And those who need them often can't afford them, leading sympathetic companies to forgo charging.

"I can't see any consistent benefits or any good business reasons why you might want to do this," said Christopher-Paul Milne, assistant director for the Tufts Center for the Study of Drug Development. "It's probably just for the sake of compassion."

The country has a long history of allowing access to unapproved drugs in serious or life-threatening situations, with more than 100,000 people being treated this way over the years, according to the FDA. Between 1997 and 2005, the agency said it received an average of about 660 requests for individual emergency treatments each year.

But it wasn't until 1987 that the FDA outlined rules for such drug dispensation, requiring that the therapies be: designed to treat grave illnesses, have no like alternatives, be in - or done with - clinical testing and on their way toward marketing approval.

Concerns about inequitable access to such drugs and inconsistent policies led the agency to revamp its rules in 1997 and look at doing it again this year.

People have complained that cancer and HIV get preferential treatment when it comes to gaining access to compassionate-use drugs, few patients know the option exists, and administrative and cost-recovery policies are unclear. The FDA is hoping that clarifying its rules, raising awareness and minimizing red tape will widen the practice.

Others are worried that access to such drugs could be harmful, however.

Most drugs under development never make it to market because they don't work or aren't safe. Dispensing experimental drugs before there's sufficient data to gauge their effect can be dangerous.

"FDA must do its utmost to prevent a `therapeutic misperception' that experimental drugs will definitely help desperate patients," Abbey S. Meyers, president of the National Organization for Rare Disorders, wrote in a letter to the agency, commenting on proposed changes to its rules.

In another comment letter, Dr. Bret Yarczower, medical director of Geisinger Health Plan, said he fears that making the drugs available will be detrimental to clinical trial enrollment.

In such trials, patients are often given placebos instead of the experimental treatments so effects can be measured, and the very ill often don't want to run the risk of getting a fake drug. If they have the option to ask for a compassionate-use drug, even if they have to pay, they might do that rather than participate in clinical trials, which are needed to gather data and eventually bring a drug to market.

Osiris made a decision to stop offering its adult stem cell drug Prochymal to adults on a compassionate-use basis once late-stage trials had begun.

"We treat children because it's the right thing to do," said chief executive C. Randal Mills, adding that the company needs "to cap it somewhere because it's not an inexpensive thing."

Mills declined to provide the exact cost.

Baltimore Sun Articles
Please note the green-lined linked article text has been applied commercially without any involvement from our newsroom editors, reporters or any other editorial staff.