fighting kids' cancer, one voucher at a time · adults, according to the american cancer...
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1/26/15, 4:15 PMFighting kids' cancer, one voucher at a time
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Fighting kids' cancer, onevoucher at a timeMeg Tirrell | @megtirrellFriday, 21 Nov 2014 | 10:03 AM ET
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Nancy Goodman's son Jacob was 8½ when he started gettingdebilitating headaches.
"Jacob ended up being diagnosed on a Sunday afternoon, and Mondaywe went in to see the neurologist," Goodman said in an interview at herhome in Washington, D.C. "Tuesday morning, he had emergency brainsurgery."
Jacob had medulloblastoma, a rare brain cancer. After his surgery,Jacob's doctors prescribed a course of chemotherapy drugs.
"He didn't respond, and his doctors knew this chemotherapy regime
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was not going to work," Goodman said. "So they met and we decidedto continue the same regime for the rest of his treatment. Why wouldhis medical team recommend that we do that? It was because therewere no other treatments available for this kind of brain cancer."
The protocol they suggested, Goodman said, was 40 years old.
Nancy Goodman
Jacob with his parents Mike Froman and Nancy Goodman, and brother Ben (Spring 2008)
Jacob's situation is not uncommon among kids with cancer, a groupthat's dwarfed by the number of adults with the disease. Each year inthe U.S., about 14,000 kids are diagnosed, compared with 1.6 millionadults, according to the American Cancer Society.
Read More › Kids with cancer need access to drug trials
Though they seem rare, kids' cancers have a huge impact; cancer is thesecond-leading cause of death among children, after accidents. About1,350 kids under age 15 are expected to die from cancer in 2014, thesociety says.
Yet very few new drugs are developed specifically for kids' cancers;most drugs used for childhood cancer aren't approved for use in kids,Dr. Ronald DePinho, president of MD Anderson Cancer Center, said at aSeptember meeting on childhood cancer in Washington. As a result,doctors often use drugs that have been approved in adults "off label,"meaning in populations other than for whom they've been cleared byregulators, to treat kids.
Lack of access
"We're always opening up the newspaper and we're learning about allof these wonderful advances in science and treatments for cancer,"Goodman said. "I was shocked to learn they don't apply to kids. Kidsdon't get access to these drugs."
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Kids' cancers are different from those in adults, which are frequentlythe result of wear and tear over years of life, said Dr. Stephen Sallan, apediatric oncologist at Dana-Farber/Boston Children's Cancer andBlood Disorders Center. Yet because of the disparity in market sizes,pediatric cancers are often neglected by drugmakers, he said.
"That 14,000 versus 1.6 million determines a lot of what happens, notbecause it's right or wrong, but because that's the playing field. It's notlevel," Sallan said in a telephone interview. "In cancer medicine,virtually all new drugs in the 21st century come to children after they'vebeen tested in adults."
That leads to delays or lack of treatment options that can mean life anddeath for patients like Jacob. And it brings many patients into thecomplicated system of applying for compassionate use—access toexperimental drugs outside of clinical trials. It's often a move of lastresort—and many times, those requests are denied by drugmakers.
Read More › When unapproved drugs are the only hope
Goodman went through that with Jacob, and after trying several moretherapies, he died almost six years ago. He was 10.
Nancy Goodman
Jacob Froman before he contracted cancer.
The next day, Goodman founded a pediatric cancer advocacy group,Kids v. Cancer, with the goal of changing a system that can leave kidswaiting years to get access to new drugs—if at all. In 2012, legislationshe helped design, called the Creating Hope Act, was signed into law. Itawards a voucher to drugmakers that receive approval for a medicinefor a rare pediatric disease.
The voucher entitles the company to accelerated review, usually a six-month process, of another medicine by the Food and DrugAdministration. That compares with a standard timeline of 10 months.
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The vouchers are transferable, and in July, drugmakers Regeneron andSanofi bought the first from BioMarin Pharmaceuticals for $67.5 million,putting a price tag on the incentive Goodman helped create.
An incentive for drugmakers
BioMarin received the voucher when it got approval of the drugVimizim, for a rare pediatric disease called Morquio A Syndrome.Regeneron and Sanofi said they'll apply it to get a faster review for acholesterol drug in development, in a class in which they're in a tightrace with rival Amgen.
Though Morquio A isn't cancer, the sale showed the program's working,Goodman said. Still, there's more work to be done. Dana-Farber's Sallanpoints out that most drugs are tested in children only after themedication has been proven to be safe and effective in adults.
"That brings one of these new drugs to a child usually three to fiveyears after it's started downstream in adults, which is justfundamentally, I think, not necessary and probably not right," Sallansaid.
Drugmakers may be concerned that a side effect seen in a small studyin pediatric cancer could derail an entire program for a much broaderadult market, Sallan said. But, he noted, "children are more resilient tothe vast majority of new drugs than are adults."
Read More › Where we stand in the fight against cancer
He suggested companies should test drugs in children alongside adults,establishing safety first in older patients and then extending themedicines sooner to kids. He said regulation could help, by protectingcompanies from the risk of a child having an adverse event that couldaffect a drug's entire development program.
Such potential changes are especially important as cancer changesfrom being defined by its location in the body—lung, breast, prostate—to one based on its underlying genetic characteristics.
Drugmakers say they're looking at the issue. Genentech, owned bySwiss drugmaker Roche, created a pediatric oncology drugdevelopment team in 2012, focused on addressing kids' cancers. Thegroup, of 25 people, looks at the science driving cancers anddetermines whether tumors affecting kids could potentially be targetedby its drugs in development, said Raphael Rousseau, who heads theunit.
"Historically, people have been reluctant (to expose) children to drugs,trying to protect them from research when really the right thing is toprotect them through research," Rousseau said in a telephone
1/26/15, 4:15 PMFighting kids' cancer, one voucher at a time
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interview.
Read More › Why there's no end in sight for higher drug costs
That's a sentiment echoed by Amgen's Dr. Lisa Bollinger, a generalpediatrician who spent 12 years overseeing pediatric drug developmentat the FDA. In a phone interview, she said she joined Amgen inSeptember 2012 to "really look at their entire pipeline and look atopportunities to study their products in the pediatric population."
Developing drugs for kids requires special considerations—about theirstage of development, different size and weight, and metabolisms, butalso about the concern for longer-term side effects. Rousseau saidGenentech has designed a program to follow up with kids for 20 yearsto monitor longer lasting effects of treatment.
And though progress—through the Creating Hope Act and effortsamong industry and government—is being made, Goodman said herwork is far from done. She wants to spur a focus on children's cancersnot just among existing drugs, but to give drugmakers incentives topursue drugs for kids even if they don't work for adults.
"When Jacob was diagnosed with cancer, of course I was devastated.But it never occurred to me that there are no drugs available to treat it,"Goodman said. "For me it was just a matter of walking down Broadwayin New York City, where I lived, and saying, 'How could I possibly behere on this Earth and Jacob isn't?' If the world set up the way it is can'taccept Jacob, then I need to change it."
Meg TirrellReporter
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• Reply •
mikeandle • 2 months ago
Here we find disgraced philanderer Bill Clinton lecturing Obama abouthonesty: http://politi.co/1nSIl5J
△ ▽
• Reply •
Al Amo • 2 months ago
With the criminal marxist regime of Obama, the lies just never cease.Yet another example: http://bit.ly/1B3xZtU
△ ▽
SonOfOlaf • 2 months ago
Want to make a good return on your money and help children withcancer?
St Judes Childrens Research Hospital has a charitableannuity.....check it out, it could be a part of your retirement or estateplanning and help cure childhood cancers
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AROUND THE WEB
• Reply •planning and help cure childhood cancers
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• Reply •
TKIRCHGESSNER • 2 months ago
I don't know whether to feel sad or angry for the doctors who offerwhat it is clear has no chance of helping. My wife was diagnosed withPSP which involves scaring at the base of the brain. There was notreatment for the condition or the symptoms. The neurologist said wecould try what was used for Parkinsons. When I asked why we woulddo that since the causes were not even related. He admitted that wastrue but said we had nothing else. Even the name of my wife'scondition was a reflection of the lack of knowledge. At least what wewere told was a blessing in that we then knew what to expect. Godholds us close and then he may well choose to bring us home soonerthan we had expected.
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• Reply •
YoonYoungJo • 2 months ago
That is one area of philanthropy that is sorely lacking in America. Notresearch or assisting with medical payments, although I'm surecharitable works for those types of things are always necessary.
But what about single parents who have a child with cancer. Thatwould one area that could go a long ways in helping someone. Asingle parent who can dedicate their life, temporarily to their cancerstricken child.
I always hear a lot about the good philanthropy billionaires do, butthere are a lot of areas, many on a more personal level, that couldreally help people.
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• Reply •
aamericannovice • 2 months ago
There has to be a demand for a drug for maximum profits. Kid'scancer is not one of them.
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