Andrea Sloan had no idea that volunteering to be a guinea pig for a new cancer drug would be so hard.
Her doctor had signed off on it. The Food and Drug Administration was on board. Sloan was willing to risk the unknown for a chance to stave off her progressing ovarian cancer.
But what the Austin woman discovered was a complicated, arbitrary process that puts the future of desperately ill people into the hands of pharmaceutical companies. Since mid-August, Sloan has been waging a social media campaign to persuade BioMarin Pharmaceutical to give her access to BMN 673, an investigational medication with an early success rate the company recently touted to investors.
But BioMarin won’t give it to her. Company officials say that it has been tested on fewer than 30 people and that to provide it to everyone who wants it without additional research would be “unethical and reckless.”
“There have been previous circumstances where early access to large groups has resulted in adverse consequences that were worse than the course of the disease,” BioMarin said in a statement.
An untold number of terminally ill patients get the same sort of message every year. At the core of the conflict is the FDA’s“compassionate use” exemption, which allows pharmaceutical companies to provide unapproved medications to seriously ill people who are not in clinical trials and have exhausted their other treatment options.
But getting that exemption can be difficult. There is no single policy or process followed by the drug companies, no single list of drugs is available through compassionate use, and there’s no way to force the drug company or FDA to approve the desired treatment. Doctors say many patients don’t even know the exemption exists.
Between October 2009 and September 2012, the FDA signed off on 3,149 out of 3,170 compassionate use applications. But that does not count the number of requests turned down by drug companies that never made it to the FDA.
Pharmaceutical companies offer lots of reasons to deny compassionate use requests, said Bob Erwin, co-founder of the Marti L. Nelson Cancer Foundation, a small, California-based organization that works to make experimental cancer drugs available on a compassionate access basis. Erwin says drug companies often claim the medications need more research, or that the company doesn’t have a policy allowing it, or that the company believes a poor outcome could affect FDA approval.
None of those reasons holds water, Erwin said.
“There is an ethical obligation to humanity to make these drugs available as early as reasonably possible,” he said.
No harm in asking
Sloan says she wants the BMN 673 because she believes it’s her best chance to live. But she also wants to advocate changes to what she calls a broken and unnavigable system that hurts the very people it should be helping.
“To say there are wrinkles here is the understatement of the century,” said Sloan, 45.
Such advocacy comes naturally to Sloan. She is executive director of the Texas Advocacy Project, which provides free legal services to victims of domestic violence, sexual assault and stalking. She has volunteered with Austin’s HIV Planning Council, the United Way and other organizations. She also was a policy aide in the Texas Legislature.
So when friends learned that BioMarin wouldn’t give Sloan BMN 673, they took their fight online. Since late August, more than 125,000 people have signed an online petition urging BioMarin to change its position. More than 80 Texas legislators signed a letter asking the California State Assembly to put pressure on the San Rafael-based company. Sloan’s supporters have flooded BioMarin’s Facebook page with pleas on Sloan’s behalf.
“She shouldn’t have to fight this hard to get life-saving treatment,” said Sloan’s friend Michelle Wittenburg.
Sloan was first diagnosed with ovarian cancer seven years ago after she began feeling intermittent pain in her side. Since then she’s gone through chemotherapy, a stem cell transplant and multiple surgeries. But this year, Sloan’s body could no longer handle traditional chemotherapy. Her doctors at the MD Anderson Cancer Center suggested she pursue a compassionate use exemption for BMN 673, which early research shows has had a positive effect on 44 percent of the ovarian cancer patients who took it.
Sloan is a good candidate for the drug because she is still in relatively good health, her tumor has been receptive to other treatments, she is highly informed, and she can give consent, said Dr. Charles Levenback, Sloan’s gynecologic oncologist. But there are no ongoing clinical trials in which she can participate.
The application process for a compassionate use exemption requires patients to get approval from their doctors, the pharmaceutical company and the FDA. The process involves lots of effort on everyone’s part, such as paperwork, insurance claims and coordination with medical providers, Levenback said.
“Nobody even wants to get involved because it’s so difficult, so time-consuming and so painful,” he said.
Her doctors signed off on it. And the FDA, which has to approve such exemptions, said it would give her the thumbs-up if BioMarin agreed to give Sloan the drugs.
“I figured, no biggie,” she said. “I’ll just call the company and ask for it.”
A plea for transparency
Sloan said she found nothing on the BioMarin website about compassionate use. She called people on the company phone tree, left messages and ultimately received a form letter referring her to clinical trials. She emailed the company’s chief medical officer, Henry Fuchs.
In a series of polite emails between the pair, Fuchs repeatedly told Sloan she could only get the drug through a clinical trial.
In its statement to the newspaper, BioMarin said that it supports such exemptions in the later stages of drug development, but in the case of BMN 673, it is “too early to know if the experimental therapy is safe or effective or will even prolong life.”
The company also said in its statement that it needs to “preserve the integrity of the clinical trial process.”
Erwin disputed those arguments. Giving such drugs to as many qualified patients as possible benefits the company because it provides more information about the product, he said. It also generates interest in the medical community because, if it works well, it creates a growing demand for the drug before it is even officially approved, he said.
“If you have no confidence in your product, why bother?” Erwin said.
Companies also worry that any bad outcomes in a compassionate use patient could ultimately affect the FDA’s willingness to approve the drug. FDA spokeswoman Stephanie Yao said that has never happened. The patients receiving drugs through through compassionate use exemptions are already terminally ill, she said. The approval process evaluates the overall performance of the medication.
“When we look at a drug approval application, we take into consideration the benefits versus the risk,” Yao said.
Erwin said he agrees that experimental drugs aren’t right for every patient and should not be given to people in the early stages of testing. But companies need to be clear and set firm policies on compassionate use, he said. If they don’t have policies, they need to write them, he said. Otherwise, they can end up with an ugly public battle on their hands.
Sloan said she never intended for her story to spread the way it has. But it went viral. She’s been on national TV shows such as “CBS This Morning,” and her story has been printed in newspapers across the country. Last week, the Texas Democratic and Republican parties sent letters asking BioMarin to reconsider.
Sloan isn’t the only one to publicly take on a company over experimental drugs. Earlier this year, a Minnesota family successfully lobbied Pfizer to give a 7-year-old boy drugs for his tumor. The company did so, and the boy improved.
Last year, more than 200,000 people signed an online petition pushing Bristol-Myers Squib to give an experimental drug to a Canadian man suffering from cancer. The man, who has since died, was denied the medication.
Company executives might not worry about that sort of bad publicity, but their boards of directors do, Erwin said.
“When their company is being trashed for being the Great Satan, they don’t like it,” he said.
So far, BioMarin has showed no signs of changing its position. But the more resistance she gets, the more Sloan and her supporters publicly push for the medication. Last week, she visited legislators on Capitol Hill, asking them to help reform the compassionate use system. And despite the obstacles still between her and BMN 673, she is confident BioMarin will eventually give her the drug.
“I have the most blessed life on the planet,” Sloan said. “I’d like to keep it going.”