On Saturday, Dec. 21, Congress passed a new spending package, narrowly averting a government shutdown.
Approval of the package, however, came at potentially great cost to the pediatric cancer community.
Republicans removed pediatric cancer bills that would have reauthorized the priority review voucher, authorized FDA to direct companies to undertake pediatric cancer novel clinical trials of approved drugs, and ensured that kids on Medicaid could access treatment across state lines.
“I’m just so disappointed that our bills couldn’t stay in the [continuing resolution], because the pediatric cancer community is not a resource-rich community,” Nancy Goodman, founder and executive director of Kids v. Cancer, said to The Cancer Letter.
It’s a community of many bereaved parents, trying to affect change with minimal financial resources, she said.
“We don’t hire professional lobbyists. It’s really a lot of work for us to put these ideas forward,” Goodman said. “I’m just incredibly disappointed that that wasn’t taken into consideration. It’s just an incredible burden for this community to do everything we’re doing in terms of taking care of our kids or missing our kids, and then to try to work with Congress.”
The bills were jettisoned following Elon Musk’s demands to cut frivolous expenditure for the sake of “government efficiency.” The pediatric cancer provisions were among the many health programs cut when the spending bill was reduced from 1,500 pages to just 116, which Musk later gloated about on his platform, X.
The spending package originally included the following provisions:
- The Gabriella Miller Kids First Research Act 2.0., which would have extended the National Institutes of Health’s Gabriella Miller Kids First Research Program.
- The Accelerating Kids to Research Act, which would have made it easier for children from low-income families on Medicaid to receive specialized cancer treatment across state lines.
- The Give Kids a Chance Act, which would have:
- Authorized FDA to direct companies with newly approved cancer drugs to undertake studies of combinations of those drugs with other drugs that the same company owns,
- Reauthorized the pediatric priority review voucher program and,
- Extended the penalties imposed on companies that fail to complete required pediatric studies to equal the penalties imposed on companies that fail to complete required adult studies.
The Gabriella Miller Kids First Research Program, named after a 10-year-old girl who died from an inoperable tumor in 2013, passed during the Obama administration in 2014. The $126 million bill was in response to $1.55 billion being cut from the NIH budget.
Although the Gabriella Miller Kids First Research Act 2.0 was ultimately passed as a standalone bill to provide for $63 million in the funding for pediatric research over five years, the other provisions remain in limbo.
Goodman spent four years working on the Give Kids a Chance Act, which was also cut. The bill would have allowed FDA to direct companies to undertake combination cancer treatments for kids.
The science shows that a combination of cancer therapies are necessary to finding a cure, Goodman said. It’s unlikely that a single therapy will be curative.
Pursuant to the Give Kids a Chance Act, required pediatric studies under the Race for Children Act, which are single-drug studies at this time, might instead be studies of combinations of two or more drugs owned by the same company.
This is critical because there are very few opportunities for clinicians to do studies of two or more precision drugs for pediatric cancer. The new precision drugs are too expensive for academics to purchase at this time, and there’s no incentive for companies to cooperate, Goodman said.
In a statement, Goodman said the past three weeks have been a rollercoaster for her and the legislation she has fought for for many years:
We were elated on Dec. 17, when Congress included our pediatric cancer drug development bills, the reauthorization of the pediatric priority review voucher program and the Give Kids a Chance Act, in the federal end-of-year funding bill. We were so close to victory.
But three hours before a scheduled vote, to the surprise of the country, the funding bill fell apart. What Congress ultimately passed into law was a stripped down bill from which the reauthorization of the pediatric priority review voucher program and the Give Kids a Chance Act had been dropped.
Other critical pediatric cancer bills had been stripped as well including Accelerating Kids Access to Drugs Act, a bill to ensure kids with cancer on Medicaid can cross state lines for treatment and Innovation in Pediatric Drugs Act, a bill to impose on companies that fail to complete required pediatric studies the same penalties as companies that fail to complete required adult studies.
We went into action. Social media stories blew up the internet. We were covered by CNN, ABC, MSNBC, STATNews, Newsweek, Rolling Stones, The Bulwark.
We had one more chance. At 11:50 p.m. on Dec. 20, the last hour of the last day of this Congress, the Senate took up our bills. Senator Rand Paul objected. The vote failed.
We have gone backwards.
It doesn’t cost taxpayers a penny. None of this stuff does. It’s bipartisan. We just really hope that Republicans and Democrats can get together and do the right thing to help seriously ill kids.
Nancy Goodman
Today, the pediatric priority review voucher program has expired for the first time in 12 years. Now, after yielding 65 new drugs for kids, there is no incentive for companies to develop drugs expressly for kids with cancer and other life threatening illnesses.
The Give Kids a Chance Act, which had 235 House cosponsors, had gone through two House hearings, a markup and a House floor vote, is no more.
Now, there’s no opportunity for companies to do studies of cocktails of new cancer drugs for kids.
The current continuing resolution to fund the government extends until March 14. Congress is going to have to pass another funding bill on or before that date, and the pediatric cancer community aims to be included in that, said Goodman, using the exact same language of Give Kids a Chance Act, which includes the priority review voucher reauthorization and the penalties for noncompletion of pediatric studies.
“It doesn’t cost taxpayers a penny. None of this stuff does. It’s bipartisan. We just really hope that Republicans and Democrats can get together and do the right thing to help seriously ill kids,” said Goodman. “You know, it’s not enough for Congress to say they care about seriously ill kids. They’ve got to pass bills to protect children who are seriously ill.”
Leading up to the funding bill, the Give Kids a Chance Act had passed in the House unanimously. It’s not controversial—everyone supports it, Goodman said.
Without these bills there is no opportunity for new drugs to be developed because there are no paths forward for companies to develop drugs for kids with cancer, Goodman said.
“We got bumped out of the CR because of political winds that had nothing to do with us,” said Goodman. “But Congress has just got to sit down and do the right thing by kids with cancer.”
