Jessica Marchionda was desperate for a drug that would help her fight GIST in November 2011. Her mother, Linda Martin, recalls how Jessica had tried and failed on a series of drugs since being diagnosed in 2007.

“It was such a frustrating time. It was awful,” Linda says, recalling how doctors had recommended she give Glaxo-SmithKline’s drug Votrient a shot but was unable to get Jessica’s insurance company to cover it despite appeals documented by GIST specialists.

“We even sent a scan showing how Votrient had shrunk the tumor,” she says.

The family’s decision had been to start the drug, which costs around $60,000 a year, and somehow pay for it on their own through patients-votrient-treatmentfundraising efforts, until coverage could be obtained. Jessica died in February 2012 at age 36 while an application for patient assistance with the drug was awaiting approval by Glaxo-SmithKline. Insurance approval never came through.

Susan Farmer, another GIST patient, also was denied coverage by her insurance company for Votrient. When appeals failed she opted to participate in a clinical trial of Votrient to gain access to the drug.

Still another patient, who asked not to be identified, says Votrient is what’s keeping her alive. After switching insurance companies she lost coverage only to win it back after lengthy appeals.

The obstacles Jessica and others faced in getting insurance coverage for Votrient are common, according to Dr. Jonathan Trent, a GIST specialist at Sylvester Cancer Center in Miami. Dr. Trent says he frequently prescribes Votrient to his GIST patients when the first- and second-line treatments of Gleevec and Sutent no longer work. And his patients frequently face denials from insurance companies.

This is partially due to the fact that the drug is being used off-label, not having been approved for treatment of GIST by the Food and Drug Administration (FDA). Even though clinical trials have not shown the efficacy of Votrient in GIST, Dr. Trent says as many as 20 percent of the patients he puts on the drug respond well. Dr. Trent uses information from his practice to help patients appeal denials by insurance companies.

And sometimes the insurance companies acquiesce. “If you can show the patient is benefitting, the insurance company doesn’t have a leg to stand on,” Dr. Trent says, saying as many as half of his patients who are denied Votrient coverage eventually win appeals. “You have to get past the first couple of levels and talk to the medical director,” he says.

Frank Burroughs, President of the Abigail Alliance for Better Access to Developmental Drugs, puts part of the blame on the FDA for what he considers an outdated approval process.

“There’s a need for earlier access, for provisional or early approval of drugs,” he says. “What [the FDA] calls the fast track is the slow line for people fighting for their lives.”

Burroughs says his organization has been pushing the FDA to follow a White House panel that in 2007 recommended a process for speeding approval of off-label drugs. “This would be a big help in getting insurance approval of these drugs,” he says.

A spokesperson for the FDA said the panel’s recommendation is under consideration, but didn’t comment further.

Burroughs suggested that the FDA take into consideration evidence from specialists like Dr. Trent rather than relying on just clinical trials for drug approvals.

“A big factor is modernizing clinical trials,” he says, particularly doing away with studies where cancer patients who are fighting to survive are given placebos.”

“I’d much rather have my patients on Votrient than taking a placebo in a clinical trial,” Dr. Trent says.

In the meantime, insurance companies have been cutting back on their coverage of off-label drugs for cancer treatment, according to some industry observers.

Susan Pisano, a spokesperson for America’s Health Insurance Plans, an industry association, says insurance companies take three factors into consideration in coverage of off-label drugs: FDA approval, support from compendiums such as the National Comprehensive Cancer Network (NCCN), and published studies on the efficacy of the drugs. Pisano says she doesn’t know of any trends limiting coverage of off-label cancer drugs. “I’m not sure if some kind of early approval of drugs by the FDA would make a difference,” she says, since the other factors are also taken into consideration. “It’s just one of the things they look at.”

Pisano says she’s not aware of any provisions in the Affordable Care Act (ACA) that might affect how off-label drugs are covered in the future, but some fear that insurance companies, facing a squeeze, will cut back more.

“The jury is still out on that,” the Abigail Alliance’s Burroughs says, noting that there are significant benefits from the ACA, such as coverage for pre-existing conditions, which will help cancer patients.

The issue of access to off-label drugs is only going to become more important as targeted treatments become more available and personalized medicine grows.

“As we get more data, we’ll figure out which patients respond better to which drug. We’re not quite there yet,” Dr. Trent says.

As for Linda Martin, she is continuing the fundraising she started to help pay for Jessica’s Votrient. Jessica loved learning, and completed her master’s degree in May 2011. In her honor, Linda has raised money to start a college fund for people with life-changing diagnoses.