Creating Hope Reauthorization Act

Floor Speech

BREAK IN TRANSCRIPT

Mr. BILIRAKIS. Mr. Speaker, I rise today in support of H.R. 4439, the Creating Hope Reauthorization Act.

Tragically, 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 relating to harsh chemotherapies.

Children have significantly fewer treatment options than adults, Mr. Speaker, and oftentimes must rely on treatment regimens developed for adults because pediatric-specific treatments simply do not exist. Unfortunately, as the popular healthcare adage goes, children are not little adults.

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.

FDA's priority review voucher program has proven to be a boon to incentivizing 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, which is really great--there is still more work to be done however. Nearly 95 percent of all rare diseases do not have an FDA-approved treatment, leaving many patients without options.

As co-chair of the Rare Disease Caucus, I sincerely appreciate the good work from Ranking Member McCaul, of course, the ranking member of the Committee on Energy and Commerce, and the chairman of the Committee on Energy and Commerce for bringing this bill forward, and also Congressman Butterfield.

So, Mr. Speaker, I urge my colleagues to join us in passing the Creating Hope Reauthorization Act, which will extend this vital innovation lifeline for rare pediatric treatments and cures at FDA.

BREAK IN TRANSCRIPT

Source