On April 17, 2020, Congresswoman Jennifer Wexton (D-VA-10) introduced the bipartisan Gabriella Miller Kids First Research Act 2.0 (HR 6556) which would redirect penalties collected from pharmaceutical companies that break the law to critical rare pediatric disease research. Representatives Tom Cole (R-OK-04), Peter Welch (D-VT-At Large) and Gus Bilirakis (R-FL-12) joined in introducing the legislation. In addition, Representatives Bill Flores TX-17, Sheila Jackson Lee TX-18,and Pete Olson TX-22 have recently joined 18 others who have cosponsored this bill. As of this writing, we already have over 200 foundations and organizations and more than 1,000 advocates covering all 50 states, who have signed a letter of support for this new bill.
Your support is needed to continue to build upon established, proven methods of research, data collection and data sharing that will lead to more refined diagnostic capabilities and ultimately more targeted therapies. The original Gabriella Miller Kids First Pediatric Research Act was passed by Congress in 2014 and President Obama signed it into law the very same year. The act was intended to be a 10-year program. With $75 million of non-taxpayer funding provided by the act over the past six years, and another $50 million expected over the remaining four years, the National Institutes of Health (NIH) Common Fund established the very successful Gabriella Miller Kids First Pediatric Research Program (Kids First).
The Kids First Program fosters collaborative research to uncover the genetic etiology of childhood cancer and structural birth defects. In pursuit of this goal, NIH developed the Gabriella Miller Kids First Pediatric Data Resource Center (Kids First Data Resource Center), and is in the process of expanding a public-facing, web-based portal that allows researchers to search, access, aggregate, analyze, and share annotated genomic sequence, variant, and phenotypic datasets. This resource is of high value for the pediatric research community and will facilitate analyses across diverse conditions to uncover shared developmental pathways. Under this program, The University of Texas – Southwestern Medical Center, Baylor, and two-dozen other pediatric research focused laboratories have generated vital genomic data that will be shared with the entire research community. The overall goal is to help researchers understand the underlying mechanisms of these conditions leading to more refined diagnostic capabilities and ultimately more targeted therapies or interventions. We must continue this vital work, because while cancer is the single leading cause of death by disease among American children, currently only 4% of the National Cancer Institute’s $4.9 billion budget goes towards development of cures and treatments for childhood cancer. Because of limited research in the past 30 years, only 4 drugs have been developed exclusiverly to treat childhood cancer.
With H.R. 6556 – The Gabriella Kids First Research Act 2.0, again with non-taxpayer funding, we will be able to expand this program to develop a truly comprehensive shared-data resource for scientists researching the majority of pediatric cancers and structural birth defects, and support development of computational tools to analyze these very large, complex genomic, and clinical data sets.
Simple as 1,2, 3, More funding for Pediatric Cancer and Rare Disease Research
Here’s how the Gabriella Miller Kids First Act 2.0 will create more research for childhood cancer and rare pediatric diseases: Under the Food, Drug, and Cosmetic Act, related companies under this act that violate its laws must pay fines. Under the new Kids First 2.0 Act, the fines are collected and transferred to the National Institutes of Health (NIH), which funds the GMKF Pediatric Research Initiative Fund. The new HR 6556 Gabriella Miller Kids First Act 2.0 is expected to generate much more funding. The Gabriella Miller Kids First Research Act 2.0 (H.R. 6556) would redirect penalties levied against the pharmaceutical compnies by the U.S. Securities and Exchange Commission (SEC) for violating the Foreign Corrupt Practice Act (FCPA). The funds from the penalties would be transferred to the Kids First Pediatric Research Program (Kids First) at the National Institutes of Health (NIH). There have been consistent and large civil sanctions leveraged against pharmaceutical companies since 2013 – hundreds of millions of dollars from six violations alone. With this legislation, penalties from pharmaceutical companies that break the law would be channeled directly to critical medical research.
Lend Your Support Now!
Your support is essential now to build upon the successful work that has already been done and to expand and enhance our research and data collection capabilities in the future. Please cosponsor H.R. 6556 – Kids First Research Act 2.0 and help kids fight cancer and rare diseases.
Ellyn Miller, always Gabriella’s Mom