[CNBC] wo years ago, Nathalie Traller spent her days like any seventh-grader might. She played soccer and swam and studied for classes. Then Nathalie started getting bad headaches, the kind that made it hard for her to concentrate.
A trip to the doctor revealed the unthinkable: a mass the size of a baseball in her chest. Cancer. It had already spread to her brain.
Nathalie was diagnosed with ASPS, alveolar soft part sarcoma, a cancer so rare that her oncologist, a sarcoma specialist, said Nathalie is her only patient with the disease. It makes up less than 1 percent of all sarcomas, which themselves make up just 1 percent of all cancers.
Nathalie, now 15, has since been through 10 surgeries, four medicines and several bouts of radiation to shrink the tumors in her brain and elsewhere. Still the cancer persists. Her doctors at the Knight Cancer Institute at Oregon Health and Science University in Portland have identified a new class of drugs they think could help her, but Nathalie hasn’t been able to access them. They’re still experimental, and Nathalie doesn’t qualify for the clinical trials: they start at age 18.
“She met the criteria in every way for a number of trials except for her age,” Nathan Traller, Nathalie’s dad, said in an interview in Portland. “So we were hoping maybe there could be an age exception made or some sort of provision so that she could try some of medicine’s best ideas. And we just kept finding that the door was closed.”
The U.S. has a system meant to help: it’s called compassionate use and it’s a way for terminally ill or otherwise optionless patients to access experimental drugs outside of clinical trials. For a patient to receive a medicine on that basis, she must have the support of her physician, the consent of the Food and Drug Administration and a pharmaceutical company willing to supply the drug. That’s where the Trallers, and many others, have gotten stuck.
The Trallers have asked Genentech, Bristol-Myers Squibb and Merck for access on a compassionate use basis to their experimental medicines. They’re in a new class called immunotherapies—drugs that harness the immune system to fight cancer. So far, the Trallers say, all of the companies have said no.
Drugmakers are navigating the issue in an increasingly public arena, fueled by the rise in viral social media campaigns that grab the nation’s attention. Nathalie’s case has been taken up by people across the globe.
Last year, BioMarin Pharmaceutical had a public tussle with Andrea Sloan, a woman with ovarian cancer, after it denied her request to access one of its experimental drugs. Earlier this year, drugmaker Chimerix was the target of an onslaught of negative media when it initially refused an experimental antiviral medicine to Josh Hardy, an ailing 7-year-old boy, before finding a way to enroll him in a new trial.
“People like to distill this down to: ‘I want you to save this child now,’” Kenneth Moch, chief executive of Chimerix at the time, said in an interview. “But it was not only Josh (we had to consider); it was the many Joshes. For example, if the drug were made available to Josh, would we say no to the next child? Where and how do you set these limits?”
“The current process is incredibly cumbersome for sick patients,” Darcy Olsen, CEO of the Goldwater Institute, a conservative policy organization that designed the legislation, said in an interview. “The Right to Try laws make it so that as soon as a doctor tells a patient there is a promising drug for you, the patient can then go out right away to the company and seek that approval, and that’s going to cut down on the time that patients have to wait enormously.”
As Goldwater and other supporters lobby for Right to Try, Olsen said she expects that those laws will be in at least half the United States by this time next year.
‘Right to beg’
Critics, though, say the laws are misguided by focusing on the FDA. The agency says it can approve urgent requests in as quickly as 24 hours. And of 977 applications for compassionate use made to the FDA in fiscal 2013, all but three were granted.
“The major roadblock isn’t a bunch of pointy-headed bureaucrats in Washington saying, ‘No, no, no,’ to the dying; it’s that there’s nothing that mandates that a company, big or small, give this drug that they might have to a terminally ill person,” said Arthur Caplan, director of the division of medical ethics at NYU Langone Medical Center. “And the laws don’t do anything to guarantee that the drug company, large or small, is going to give you something.”
Caplan said he thinks of the laws as “Right to Beg,” rather than Right to Try. And that what’s needed instead is funding to help patients pay for treatment and travel as well as legislation to protect companies if they give their drugs on an emergency basis and something goes wrong.
“We need to institute a two-track system where if you took a drug as a terminally ill person, sure, we want to know what happened, but we don’t want to count it against the approval process that the drug company’s trying to push forward,” Caplan said.
There aren’t data counting how many requests are made to companies for compassionate use—the FDA’s tallies include only those that companies agree to. Genentech said it’s received more than 100 applications for its experimental immunotherapy drug, the one Nathalie Traller is seeking to try, known by its target, PD-L1. Thus far, Genentech doesn’t have a compassionate use program for it, though it does have a program for another therapy.
Bristol-Myers said it doesn’t comment on individual patients, but noted that it “recognizes that outcomes for pediatric patients with recurrent or metastatic tumors remain poor” and said it’s committed to studying its cancer drugs in those patients. The company said it does have programs offering the drug on expanded access, another term for compassionate use, to adults with melanoma.
“However, because there are no data that establish the benefit/risk profile of nivolumab in pediatric tumors, access through expanded access or compassionate use is not available,” Sarah Koenig, a spokeswoman for the New York-based drugmaker, said in an emailed statement, referring to its immunotherapy drug in testing. “We understand the urgency for this population and are working with regulatory agencies to begin development of carefully conducted clinical trials in pediatric patients.”
Merck also has a program to provide its drug, pembrolizumab, through expanded access. But it’s limited to patients with advanced melanoma “because that is the only patient group where we believe we have enough data on safety and efficacy,” the company says on its website.
Looking for support
“We’re just really determined. And we want to make it happen. So we’re going to try with everything we have to get that medicine.”
The Trallers, because of Nathalie’s age and the rareness of her cancer, say they feel they’re outside the system.
“We reached a point as we learned more that there might be some things that could help Nathalie,” her dad said. “But the way the system was built, she couldn’t access it. We realized it was a lot bigger than us. And that we might just need to tell her story and gain support.”
So they began a social media campaign, taking cues from myriad other patients and families who have been through the same thing.
“We implement expanded access programs when we have substantial scientific evidence to support both the safety and the efficacy of a product for an indication,” the company said in an emailed statement. BioMarin had such a program for its medicine Vimizim, for a rare disease, before it was approved earlier this year. The drug Sloan was seeking, BioMarin said, “is in the early stages of development,” and doesn’t yet meet that criteria.
Sloan’s family and friends took her story to Facebook and Twitter, and a supporter started a petition on Change.org, which has served as a platform for many similar campaigns since. It garnered more than 234,000 signatures. Former House Speaker Newt Gingrich supported her cause in a blog post.
“When the door’s slammed in your face and your life is on the line, you can’t just go, ‘OK, I lost this one, I’ll come back again,’” Sloan’s friend Michelle Wittenburg said in a telephone interview. “You have to keep persevering.”
Sloan ultimately got access to another medicine in the same class, known as PARP inhibitors, from a company that insisted on anonymity—Wittenburg calls it the “white knight pharmaceutical company.”
It took eight weeks, though, for Sloan to get the first course of that drug, and her health deteriorated rapidly, Wittenburg said. Yet about two months after her first dose, the medicine appeared to be working.
“The scans were clean,” Wittenburg said. “She had gained back 10 pounds.”
But pneumonia ultimately struck, and Sloan passed away on New Year’s Day.
Two months later, the issue of compassionate use was back in the headlines. A 7-year-old boy named Josh Hardy contracted a viral infection after receiving a bone marrow transplant to treat his cancer, which he’d battled since he was 9 months old. Chimerix’s antiviral medicine, brincidofovir, was still in clinical trials.
The company had received hundreds of applications for compassionate use of the medicine before the Hardy’s. Josh’s, though, gained widespread media attention. The pressure on the company became so severe that Moch, the CEO, had an armed security detail for a week in response to threats he and his wife received.
Eventually, Chimerix agreed on a plan with the FDA to start a new clinical trial, of which Josh would be the first patient. The medicine cleared his viral infection and Josh is recovering—though he still has a “tough road ahead of him,” according to his uncle, William Burns.
The issue of who gets a drug and who doesn’t, though, raises questions of inequity in the system. Patients whose stories are more appealing or who have more social media savvy may attract more attention than others with equal need.
“It isn’t a fair system,” NYU’s Caplan said. “It favors the people who know how to be noisy.”
A month after the company found a path forward for Josh Hardy, Moch—the public face of the controversy—was replaced at the helm by the company’s chief medical officer. Chimerix said he resigned to pursue other interests. Moch declined to comment on the reasons for his departure.