WASHINGTON – Advocates for child cancer patients hope a key panel in Congress takes an important step in the decades long fight to speed up development of pediatric oncology drugs.
The Senate Health, Education, Labor & Pensions Committee was scheduled to vote on the measure Wednesday.
However, Democrats’ protest over the Republicans’ refusal to appoint a special prosecutor to investigate President Donald Trump’s campaign ties to Russia forced the postponement of the meeting. A new date has yet to be announced.
Activists said the bipartisan effort, if successful, would mark a new chapter to address the needs of children who often have not been a priority under a federally managed process geared toward treating more prevalent adult cancers.
“The vote in committee … will kick-start our national conversation around updating longstanding federal policies based on our latest scientific understanding of cancer and will clearly encourage new research and development in childhood cancer,” said Jorge Luis Lopez, a Miami attorney and member of the American Cancer Society Board of Directors.
The provisions expected to be considered are part of a broader bill known as the Research to Accelerate Cures and Equity for Children Act that advocates have been pushing since it was introduced in the last Congress. As part of a compromise, a portion of the measure is being added to a Food and Drug Administration re-authorization bill expected to win congressional approval later this year.
The children’s research act was sponsored by Republican Sens. Marco Rubio, of Florida, and Cory Gardner, of Colorado, and Democratic Sens. Michael Bennet, of Colorado, and Chris Van Hollen, of Maryland.
A similar bill has been introduced in the House by Republican Reps. Michael McCaul, of Texas, and Sean Duffy, of Wisconsin, and Democratic Reps. G.K. Butterfield, of North Carolina, and Yvette Clarke, of New York.
Congress passed the Pediatric Research Equity Act in 2003 to help address the issue. It was supposed to require the industry to conduct pediatric assessments on drugs developed for adults.
But advocates say the law has given the Food and Drug Administration too much latitude in waiving pediatric cancer studies because many of the drugs developed for the four major cancers — breast, prostate, lung and colon — are not childhood cancers.
The FDA routinely exempts drug companies from testing children, even though some experts think molecular-level studies on those adult cancers could lead to pediatric breakthroughs, according to the Alliance for Childhood Cancer, an advocacy group.
“The practical consequence of these exemptions is that no drugs have been assessed in children as a result of” the 2003 Pediatric Research Equity Act, the alliance wrote in a letter last month to the top lawmakers of key House and Senate committees. “We believe it is critical that the needs of children suffering from cancer now and in the years to come are addressed as part of this legislation.”
“Pediatric cancer impacts too many families in Florida and across the nation, including my own,” Rubio said.
The Research to Accelerate Cures and Equity for Children Act “takes a further step in extending medical advances in adult cancer treatment to children fighting this horrific disease,” he said.
The trade group for the pharmaceutical industry is not taking a formal position on the children’s research act. But a spokeswoman said the legislation passed last decade has “been successful in driving research that has led to progress against a number of pediatric diseases, including asthma, rare diseases and many forms of pediatric cancer.”
Over the last 15 years, there have been more than 680 labeling changes on medicines reflecting information on safety and effectiveness in children, said Holly Campbell, with the Pharmaceutical Research and Manufacturers of America.
“While we celebrate the continued success that America’s biopharmaceutical research companies have made in pediatric research, more work remains to be done,” she said. “We look forward to continuing to collaborate with (senators who sponsored the children’s research act) to explore ways to further enhance existing policies to support pediatric research.”
The provisions of the children’s research act that are expected to be included in the committee markup of the FDA reauthorization would:
» Allow sponsors to meet earlier with FDA on pediatric studies for drugs for serious diseases.
» Speed up agreement on pediatric studies by involving the pediatric internal review committee and requiring the FDA to respond to a proposed study in 120 days.
» Make permanent a requirement for the FDA to have neonatology expertise on the Pediatric Advisory Committee.
» Require the FDA to work with public and private stakeholders to develop guidance on how molecular targets can be used to develop pediatric cancer drugs.
Backers of the children’s research act said they still are trying to add other pieces to the FDA reauthorization bill as it makes its way through the House and the Senate.
A spokeswoman for Sen. Lamar Alexander, R-Tenn., chairman of the Senate Health, Education, Labor & Pensions Committee, said there will be opportunities beyond the committee vote to incorporate more elements of the children’s research act.
He “is committed to continuing to work with (the senators) who are leading on a critical issue — how to get more drugs approved for children with cancer,” spokeswoman Liz Wolgemuth said.
Contact Ledyard King at firstname.lastname@example.org; Twitter: @ledgeking.