Source: United States House of Representatives – Representative Gus Bilirakis (FL-12)
Washington, DC-Last night, the United States House of Representatives unanimously passed the Mikaela Naylon Give Kids A Chance Act, a landmark bill to accelerate pediatric cancer treatments and expand access to life-saving therapies for children battling rare diseases. Congressman Michael McCaul (R-Texas) introduced the legislation in February alongside Representatives Gus Bilirakis (R-Fla.), Debbie Dingell (D-Mich.), Kim Schrier (D-Wash.), Diana Harshbarger (R-Tenn.), Kathy Castor (D-Fla.), Mike Kelly (R-Pa.), Doris Matsui (D-Calif.), Dan Crenshaw (R-Texas), Lori Trahan (D-Mass.) and Randy Weber (R-Texas).
This legislation combines two critical initiatives — the original Give Kids a Chance Act and the Creating Hope Reauthorization Act– to spur drug development for pediatric rare diseases, improve outcomes for patients, and close gaps in pediatric drug research.
“Today, we celebrate the House passage of this critical, bipartisan effort to give children battling cancer and rare diseases a real chance at life. By strengthening incentives for pediatric drug development, restoring essential programs such as the FDA Rare Pediatric Disease Priority Review Voucher, and ensuring that life-saving therapies reach the children who need them most, we are making meaningful progress. I am proud to have contributed to this important work and will continue fighting for children and families across our nation,” said Congressman Bilirakis.
“By passing the bipartisan Give Kids a Chance Act, the House has taken a meaningful step forward in improving health outcomes for kids fighting cancer and rare diseases,”said Rep. Castor. “This bipartisan legislation eliminates unnecessary red tape, prioritizes life-saving pediatric therapies and re-energizes pediatric drug research at no additional cost to taxpayers. As co-chair of the Childhood Cancer Caucus, I’m pleased to see the House come together to remove barriers to care. Working together is how we strengthen families, expand opportunity and build a healthier, more hopeful future for our young neighbors in Florida and across the country.”
“As a founder of the childhood cancer caucus, I’ve met with countless cancer patients and advocates who have asked me for one thing: to give kids their best chance of beating cancer. Today, the House of Representatives shined a beacon of hope for those children and their families by unanimously passing the Mikaela Naylon Give Kids A Chance Act,” said Congressman McCaul. “Mikaela represents all the children who have advocated for this bill and suffered with this heartbreaking disease. Losing some of these kids has been the hardest part of my career, but today, I’m encouraged because I know Mikaela’s legacy-and all of their legacies-will live on in this bill forever and help save countless lives.”
“Today, the House delivered long-overdue hope for the millions of Americans and their families struggling with a rare disease,” said Congresswoman Matsui. “I’m proud my RARE Act is included in this bipartisan package. It will prevent pharmaceutical companies from abusing their orphan drug status to keep other innovative drugs from coming to market. As Co-Chair of the Rare Disease Caucus, I’ve met far too many families who have been told there are no options for their child’s care. This legislation brings them real hope by ensuring investment into pediatric therapies and getting promising treatments to patients faster. Now the Senate must act quickly, because for the rare disease community, every single day matters.”
Background: Each year, nearly 16,000 children in the United States are diagnosed with cancer. In fact, children comprise as many as half of those living with rare diseases, yet treatment options for children remain extremely limited compared to those for adults. The Mikaela Naylon Give Kids a Chance Act would reauthorize the Food and Drug Administration priority review voucher (PRV) program-which allows pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases. Since the bill’s passage in 2011, 63 PRVs have been awarded for at least 39 different rare pediatric diseases.
Additionally, thousands of successful drug combination therapies are now being studied and developed for adults, but not for children. The Mikaela Naylon Give Kids a Chance Act also authorizes the FDA to direct companies to study combinations of cancer drugs and therapies in pediatric trials.