Drug Companies Have Complex, Sometimes Fatal Relationship With Cancer Patients

The drug, BMN 673, is what's known as a PARP inhibitor, which targets cancerous cells while minimizing damage to normal tissue. It isdesigned specifically to treat women like Sloan who have a particular type of geneticovarian and breast cancer that presents a genetic mutation in the BRCA1 gene.Sloans doctors said it was her best and perhaps only chance to survive. The drug has not yet been approved by the Food and Drug Administration, but agency officials said they would provide a waiver to her for "compassionate use" of the treatment if the drug maker gave her permission to use it. But BioMarin Pharmaceutical Inc., the company that manufactures the potentially life-saving treatment, refused to let her take it. On New Years Day, Sloan, a former corporate lawyer who quit her high-paying job several years ago to run an organization that provides free legal services for low-income victims of domestic violence, died of complications from pneumonia. She was 45. Andrea very much wanted to live, but she also wanted to make a difference for other patients out there who may not have the resources she had to put up such a public fight, said Michelle Wittenburg, Sloans closest friend, who's an attorney and a freelance Texas legislature lobbyist. Andrea was morally and ethically offended that a drug company could just summarily say no to a dying patient without even asking to look at her medical records or seriously considering her situation.

Andrea Sloan and her father, John Sloan, during one of numerous hospitalizations. Courtesy family of Andrea Sloan

Compassionate use, or expanded access, refers to situations in which the FDA allows drug companies to provide individual patients access to their experimental drugs outside of clinical trials. Between October 2009 and September 2012, the FDA reportedly signed off on 3,149 out of 3,170 compassionate use applications, which doesnt count the requests rejected by drug companies that never made it to the FDA.

Sloans doctors and the FDA both concluded that Sloan met the criteria for compassionate use: Hers was a life-threatening situation, both she and her doctor were on board, and her condition had not been improved by conventional treatments. But BioMarin, a Northern Calif.-based company whose revenue in 2012 was $500 million, turned her down, saying that while it supportscompassionate use policies for seriously ill cancer patients, it does not have such a policy for this particular drug.

BioMarin also told the International Business Times repeatedly that it could not guarantee the safety or efficacy of this drug, despite having touted its safety and efficacy to its investors last September, when it said the company was encouraged by the data presented at the European Cancer Congress, including the safety profile, and significant anti-tumor activity.

After being turned down, Sloan, with a little help from her friends, created an Andis Army campaignto urge the company to change its mind, and it started a petition at Change.org that received hundreds of thousands of signatures. In September, Sloan made a direct and impassioned plea to BioMarin CEO Jean-Jaques Bienaime in a video that is still available online.

I do have to tell you that Im a little frustrated at our inability to have an open dialog about how we might be able to get to a solution that both advances your goals of making sure that this treatment is available to everyone and advances my goal of saving my life, Sloan said in the video.

In a letter to BioMarin chief medical officer Henry Fuchs dated Sept. 3, 2013, which has never before made public, Karen Sloan, Andreas Mother, wrote, I am reduced to begging a corporation that has closed the door to meaningful communication I realize corporations must make tough business decisions to keep themselves healthy. Your compassion is my only hope If this were your child, would you find a way to grant her last, best hope?

BioMarin still wouldnt budge. In an interview with IBTimes, Karen Sloan said, Our daughter valiantly fought and beat cancer for over seven years but was denied life-saving and timely treatment by BioMarin with no meaningful explanation. They never even requested or reviewed her medical records to make an educated determination.

BioMarins spokeswoman, Debra Charlesworth, told IBTimes, The FDA has not approved the drug for compassionate use. She declined to say whether the company actually applied for compassionate use of the drug.

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Drug Companies Have Complex, Sometimes Fatal Relationship With Cancer Patients