U.S. Rep. Gus Bilirakis, R-Fla., continues to champion health proposals on Capitol Hill, including one to reauthorize the cell transplantation program named after a longtime Florida congressman.
Back in October, Bilirakis announced his support for U.S. Rep. Doris Matsui’s, D-Calif., “Timely Re-Authorization of Necessary Stem-cell Programs Lends Access to Needed Therapies (TRANSPLANT) Act.” U.S. Rep. Chellie Pingree, D-Maine, is also cosponsoring the bill which would reauthorize the C.W. Bill Young Cell Transplantation Program and the National Cord Blood Inventory. Longtime U.S. Rep. Bill Young, R-Fla., served in Congress for more than four decades until his death in 2013 and led the U.S. House Appropriations Committee.
“Through renewed federal funding for the vital national bone marrow and cord blood registry and coordinating centers, which facilitate bone marrow and cord blood donations, the TRANSPLANT Act ensures the continued provision of lifesaving transplants to treat diseases like blood cancer, sickle cell anemia, and inherited metabolic or immune system disorders,” Bilirakis’ office noted.
“For the 1.3 million Americans fighting painful and potentially life-threatening blood cancer or bone marrow disorders, a successful transplant brings hope and a second chance at life,” said Matsui in October. “Reauthorizing the C.W. Bill Young Cell Transplantation Program, the ‘Be a Match’ National Registry, and the National Cord Blood Inventory are critical in connecting patients with donors and funding life-saving research to eradicate these devastating diseases. I am proud to introduce the TRANSPLANT Act so we can continue to support patients in need and those willing to give the gift of life.”
The bill was sent to the U.S. House Energy and Commerce Committee on which Bilirakis sits back in October. This week, the committee’s Health Subcommittee examined the bill.
“This bill is about providing hope to those who are struggling with life-threatening illnesses. This federal program provides critical support in the advancement of research for better treatments and the infrastructure necessary to organize registries which help ensure transplant patients have access to life saving procedures. Its reauthorization is vital, and I hope our colleagues will join us in expediting passage of this important legislation,” said Bilirakis on Wednesday.
Matsui, Bilirakis and Pingree have been able to reel in more than 40 cosponsors including U.S. Reps. Charlie Crist, D-Fla., Alcee Hastings, D-Fla.. and Michael Waltz, R-Fla. So far, there is no counterpart over in the U.S. Senate.
The Health Subcommittee also reviewed another bill backed by Bilirakis, the “Creating Hope Reauthorization Act” from U.S. Rep. G.K. Butterfield, D-NC. The bill would make permanent the Priority Review Voucher (PRV) program for pediatric diseases.
“The PRV was first created in 2012, with the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) to incentivize the development of therapies to treat rare pediatric diseases. While progress has been made in the development of pediatric therapies – in fact, 22 therapies have been approved for the treatment of 18 rare pediatric diseases since 2012 – there is still more work to be done. Nearly 95-percent of all rare diseases do not have an FDA-approved treatment, leaving many patients without options,” the Florida Republican’s office noted.
“Pediatric cancer remains the number one disease that leads to the death of American children. While survival rates have improved for some types of pediatric cancers, thousands of children are lost to cancer each year and many more encounter life threatening complications related to harsh chemotherapies. Children have significantly fewer treatment options than adults and oftentimes must rely on treatment regimens developed for adults because pediatric-specific treatments simply do not exist. Children are not little adults, and despite their significant need, pharmaceutical companies have had trouble developing treatments for pediatric cancer and rare diseases because of the small population and high cost of bringing these specific treatments to market,” said Bilirakis.
“The PRV program has been an effective tool for incentivizing the development of better treatments for children with cancer. Our legislation makes sure this successful program continues,” he added.
Butterfield’s bill has more than 20 cosponsors including U.S. Reps. John Rutherford, R-Fla., and Darren Soto, D-Fla. U.S. Sen. Robert Casey, D-Penn., and U.S. Sen. Susan Collins, R-Maine, are championing the proposal in the U.S. Senate.
Reach Kevin Derby at kevin.derby@floridadaily.com.
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