[USA Today] By
WASHINGTON – Advocates for child cancer patients hope a key congressional panel takes an important step in the decades-long fight to speed up development of pediatric oncology drugs.
The Senate Health Education, Labor & Pensions Committee is scheduled to vote Wednesday on several provisions aimed at shortening Food and Drug Administration review periods for pediatric treatments and delivering drugs to market faster.
Activists say the bipartisan effort, if successful, marks a new chapter to address the needs of children who have often not been a priority under a federally managed process geared toward treating adults where cancer rates are much more prevalent.
“The vote in committee tomorrow 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 (RACE) 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 reauthorization bill expected to win congressional approval later this year.
The RACE Act was sponsored by Democratic Sens. Michael Bennet of Colorado and Chris Van Hollen of Maryland, and Republican Sens. Cory Gardner of Colorado and Marco Rubio of Florida.
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.
Child cancer advocates have been frustrated at what they see as the slow pace of drug development by the pharmaceutical industry.
Congress passed the Pediatric Research Equity Act (PREA) 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 practical consequence of these exemptions is that no drugs have been assessed in children as a result of PREA,” 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.”
The RACE Act would update PREA by increasing the number of pediatric studies developed from the molecular work on adult cancer drugs, Bennet said.
“This legislation is a necessary update to our laws and has the potential to save thousands of children’s lives,” he said when the bill was introduced in February.
The trade group for the pharmaceutical industry is not taking a formal position on the RACE 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, known as PhRMA.
“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 Race Act) to explore ways to further enhance existing policies to support pediatric research.”
The provisions of RACE that are expected to be included in Wednesday’s committee markup of the FDA reauthorization would:
— Allow sponsors to meet earlier with FDA on pediatric studies for drugs for serious or life-threatening diseases;
— Speed up agreement on pediatric studies by involving the pediatric internal review committee and requiring FDA to respond to a proposed study in 120 days.
— Make permanent a requirement for 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 RACE Act say they are still trying to add other pieces to the FDA reauthorization bill as it makes its way through the House and the Senate.
A spokeswoman for Tennessee Republican Sen. Lamar Alexander, who chairs the Senate HELP Committee, said there will be opportunities beyond this week’s committee vote to incorporate more elements of the RACE 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,” said Liz Wolgemuth. “He looks forward to discussing the issue further at Wednesday’s markup.”