Compassionate use changes: ‘A matter of life and death’

WASHINGTON, D.C. (KXAN) – On a beautiful autumn Sunday, Andrea Sloan was exactly where she loves to be: on the ranch riding her horse.

“I just feel energized and loved and grateful and at peace,” Sloan said. “It is impossible to have a bad day on a horse.”

But deep in the background of that tranquil scene is the bitter battle Sloan is waging for her life. Seven years ago, she was diagnosed with ovarian cancer. For a while, it appeared that she had beaten the disease. Two months after, her cancer returned – her body was getting tired, but not her resolve.

“I’m not dying of ovarian cancer,” she said. “I don’t know how I know it.  I just know it the way you know things.”

Sloan is not only battling cancer, she’s also battling to change the way patients like her can gain access to  experimental drugs that might be able to save lives that would otherwise be lost.

Over the summer, Sloan learned that her approved medications were no longer effective for treating her advancing cancer. She also learned that drug manufacturer BioMarin has a drug her doctors say could provide the best chance for prolonging her life.

The drug, BMN 673, has shown to be effective in clinical trials but it hasn’t been approved by the FDA. Sloan could receive it under the FDA’s compassionate use rules. However, BioMarin has denied her request for access to the drug under the compassionate use exception that is allowed in cases like hers. Sloan was too late for BioMarin’s clinical trial and the company cites concerns about her safety and the drug’s effectiveness if she were allowed in at a later stage.

IN-DEPTH // The Patient Choice Act

Here are some key points of the HR 2090:

  • Treats provisional approval of a drug in the same manner as approval of a drug, except that provisional approval is subject to requirements related to informed consent and continued pursuit of safety and efficacy data for purposes of gaining approval for the drug.
  • Considers a drug safe if the risk of death caused directly by an adverse effect of the drug is unlikely to be greater than the combined direct and secondary risks of death or morbidity of the disease and existing therapies.
  • Considers a drug safe if the drug has had a valid marketing authorization for at least four years in specified countries and data adequate for the approval of such marketing authorization has been submitted to proper federal authorities.
  • Allows a drug to be administered only for treatments for which it was being tested for which the drug is demonstrated to be adequately safety.
  • Prescribes requirements for termination of provisional approval, withdrawal of such approval, and application of market exclusivity to fast- track approval products.

Source: Library of Congress/Bill Summary

Sloan said the company’s fears are unfounded.

“There are just some choices that should be between a patient and his or her physician,” said Sloan.  “I think there is a commitment that is starting to take hold in Washington to look at this issue more completely.”

She has at least one ally in the nation’s capital.

“I find too many patients today in terminally ill conditions who cannot get the life-saving medications they need,” U.S. Rep. Michael McCaul, an Austin Republican, told KXAN in an interview at his office in Washington, D.C. “And, of course, if you’re terminally ill, you’re running out of time.”

A push to change policy

McCaul, who is co-sponsoring a bill called the Patient Choice Act, said passage of the bill is vital.

“It’s a matter of life and death for them, literally,” he said. “And if they don’t get the life-saving medication they’ll die and this bill is designed to get that medication to them.”

The Patient Choice Act would allow drug companies to apply for provisional approval after a drug has gone through the initial clinical trials. It would also give patients with terminal illnesses the option of buying those drugs before they’re approved by the FDA.

“Essentially, it incentivizes the pharmaceutical companies to provide these medications to terminally ill patients and get these medications much sooner in a shorter period of time, which I think at the end of the day will end up saving lives,” said McCaul.

Sloan is not the only person to run up against a drug company roadblock.

Chloe Drury, a cancer patient in Britain, was denied compassionate use of a drug from BioMarin in September 2012 because she was three months short of her 18th birthday. BioMarin’s minimum age for entering a clinical trial is 18.

BioMarin said her participation could jeopardize her safety and the integrity of the trial data.  Chloe was allowed in when she turned 18, but it was too late. She died a month later.

“We’re just sitting there watching our beautiful 17-year-old daughter get weaker and weaker knowing that there is something out there that she could have had and it just seems totally wrong to me,” said Chloe’s mother, Debbie Binner.

And although BioMarin officials told KXAN they support compassionate use programs, the company’s CEO, Jean Jacques Bienaime said this to shareholders in September:

“We believe that everybody, all the patients that we try to serve should have access to the drug in an absolutely equal way. No one has priority over anybody else.”

Dr. Mike Douglas, executive director of the Texas Life Sciences Collaboration Center in Georgetown, said  compassionate use “is a very common problem.” He supervises the center’s research labs in Georgetown where drug manufacturers develop new drugs. Douglas has worked through the compassionate use process and doesn’t believe the drug companies should have the final say.

“The pharma company can be a roadblock before it gets to the FDA or even after it goes through the FDA,”  said Douglas.  “I think the FDA is the ultimate decision-maker.  They’re here to protect the safety of man.”

A life-saving step or a government overreach

Richard Klein, director of the FDA’s Patient Liaison Program, said Douglas’ proposal is an overreach.

“The agency doesn’t have the authority to require that they make that drug available,” he said.

But should they?

“There are lots of reasons why companies can’t or aren’t willing to make those drugs available,” said Klein.  “One of them is the cost of manufacturing. They may not have the facilities or the ability to manufacture enough.

“The uncertainty about how much demand there’s going to be. If you start giving it to somebody, especially with the internet, everybody finds out– and a lot of people want access to it.”

Klein said the FDA supports compassionate use programs and encourages drug companies to have them. But he also said giving incentives for companies to make experimental drugs available before all of the research is complete could create other problems as companies would start to make money from these drugs before they’re FDA approved.

Sloan, the focus of an online campaign to persuade BioMarin to allow her the drug, said there ought to be more middle ground in the debate.

“I’m not trying to get people access to shiny new things before the time has come, but I think there is an ethical and moral imperative on drug companies who are in the business of saving lives to develop meaningful compassionate use policies,” she said.

When we asked what she thought her future looks like, Sloan replied: “I don’t know exactly. I know it will be full of beautiful days and the gift of cancer is nobody knows how many days we have but there’s something precious about feeling those days speed up because you don’t take any of them for granted.”

A month after that September day another company granted Sloan compassionate use of an experimental drug.  But, the cancer had already been growing for two months and Andrea was growing weaker.  Back in the hospital, just this week,she was still fighting and still spreading her message.

“No one should should have to beg for a drug that potentially extends his or her life,” Andrea said in a video she sent to KXAN.  “I probably needed my drug two months before I was able to get it and that two months would have made the process a lot easier for me.

“So for all these reasons I hope that we can all work together and try and come up with something meaningful that works for everyone.”

Andrea Sloan’s update, Part I

Andrea Sloan’s update, Part II provides commenting to allow for constructive discussion on the stories we cover. In order to comment here, you acknowledge you have read and agreed to our Terms of Service. Users who violate these terms, including use of vulgar language or racial slurs, will be banned. If you see an inappropriate comment, please flag it for our moderators to review.

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