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Why Don't Kids with Cancer Get the Drugs They Need?

Alexis Agin died of an inoperable brain tumor two weeks shy of her fifth birthday after being denied access to a trial drug. Her father says the Patient Choice Act could have saved her life.

Alexis Agin died of an inoperable brain tumor two weeks shy of her fifth birthday after being denied access to a trial drug.

A little over a week ago, 8-year-old Josh Hardy was discharged from the hospital after a deadly battle with adenovirus. A four-time cancer survivor, Josh still has a hard road ahead of him, but after a massive social media campaign helped him get last-minute access to an experimental drug, the turnaround in his health was nothing short of amazing.

That lifesaving drug, by the way, was an antiviral medication, not a cancer drug. Here’s a hard truth: If the Hardy family had needed a new pediatric cancer drug, they probably wouldn’t have found one.

In the last 25 years, just two drugs have been approved specifically to treat pediatric cancer in the United States, Vickie Buenger, president of the Coalition Against Childhood Cancer, told me.

The Hardy family and their supporters are advocating for more pediatric cancer research funding and they suggested that I talk to Buenger.

Buenger’s daughter Erin died of the cancer neuroblastoma before her 12th birthday. Unlike Josh, there was no brand-spanking new drug to save Erin’s life.

Instead, for years, she benefited from drugs approved for use in the 1950s and 60s.

“It’s really hard to believe your own child is taking drugs that your parents took in the ’70s to keep her alive,” Buenger said.

It thrills me to write “good news” stories about children like Josh Hardy and Lilly Bumpus, a toddler who triumphed over a rare cancer that was once considered unbeatable. But there are too many stories on the other end of the spectrum — stories like Buenger’s. Like Lacey Holsworth’s. Like Sam Sommer’s.

Why Aren’t More Pediatric Cancer Drugs on the Market?

Children with cancer keep dying in part because, for all the advances of modern medicine, few drugs are being developed to combat the disease killing more American children than any other. Funding for childhood cancer is sparse — less than 4 percent of federal cancer research funding is dedicated to childhood cancer research — and pharmaceutical companies don’t have a ton of motivation to develop the drugs on their own. The grim statistics notwithstanding, the market for childhood cancer drugs is relatively small compared to markets for potentially more profitable medications.

Efforts to improve the situation, so far, have yielded mixed results. Most recently, both parties celebrated the passage of Gabriella Miller Kids First Research Act, a bill inspired by a 10-year-old cancer awareness advocate who died of brain cancer last year. The bill would provide $126 million in funds for pediatric medical research over a decade.

But Buenger, whose day job is working as a business professor at Texas A&M University, notes that the bill covers a range of childhood illnesses, meaning far fewer funds than you might think will actually be dedicated to cancer research. It’s also unclear whether any of the money authorized by Congress will actually be spent.

The Gabriella Miller act “is authorization legislation, not appropriation legislation. There is no reason to believe that any money will ever actually be appropriated and added to the budget,” Buenger told me. She pointed to a discouraging example in recent history: The Caroline Pryce Walker Conquer Childhood Cancer Act, she said, authorized similar levels of pediatric cancer research funding in 2008 but the money was never appropriated.

Buenger and other critics note that the Miller bill comes on the heels of devastating cuts to the National Institutes of Health, part of last year’s federal budget cuts known collectively as sequestration.

“Sequestration was harmful to the NIH budget and it was felt disproportionately in children’s patient populations, and I doubt we will make up those cuts,” Buenger said.

Efforts to Improve Children’s Access to Cancer Medications

While the majority of pediatric cancer research funding comes from the government, “some portion comes from the money raised by childhood cancer foundations, who sponsor bake sales, fun runs, lemonade stands, and shaving events to pay for research trials,” Buenger said.

In other words, the small fundraisers — like the shaving one my fellow Babbler Meredith Carroll wrote about earlier this month — matter a lot.

On a wider scale, some children’s cancer advocates and others are urging the passage of the Patient Choice Act. The bill would allow patients faster access to experimental drugs while providing for FDA oversight. Proponents argue that the bill would also lower the cost barrier for developing new drugs, potentially allowing pharmaceutical companies to develop more drugs targeting smaller populations.

While the legislation, supporters say, would be for the benefit of all patients, it could be especially beneficial for those seeking access to the few experimental drugs that may treat pediatric cancers.

Jonathan Agin said that, had the Patient Choice Act been around years ago, it could have saved his daughter. In 2011, Alexis Agin died of an inoperable brain tumor two weeks shy of her fifth birthday after being denied access to a trial drug that doctors had recommended for her.

“Unfortunately, we will never know if this drug could have had any benefit against her cancer,” Agin told me. And, he added, there are many pediatric cancer patients like his daughter who desperately need access to experimental drugs and aren’t getting them for various reasons.

“Legislation such as the Patient Choice Act would provide access to treatments that show promise and could be targeted against specific genetic drivers,” he said. “Science is far outpacing drug development and availability for children with cancer.”

Buenger is still mulling the Patient Choice Act, but she says it’s clear that the way drug companies and the government approach drug development must be reformed, for the sake of all patients including, of course, the smallest ones.

“Childhood cancer is the number one disease killer of children in the United States,” she said. “I know we can do better.”

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Don’t miss a post! Follow Alice on Twitter and as “Mildly Inappropriate Mommy” on Facebook

More from Alice:

Mom’s Wish for Her Paralyzed Kindergartener: A Van

World-Traveling Family Rescued at Sea After Baby Becomes Ill, Face Backlash

7 Challenges Facing Working Dads

Why the Baseball Paternity Leave Uproar Is Great News

Dropping Out of the Baby Milestone Rat Race

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Photo courtesy Jonathan Agin.

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