In 2014, Congress passed The Gabriella Miller Kids First Pediatric Research Act. The act was intended to be a 10-year program. With $88 million of non-taxpayer funding provided by the act over the past seven years, the National Institutes of Health (NIH) Common Fund established the very successful Gabriella Miller Kids First Pediatric Research Program (Kids First). Another $38 million is expected to be appropriated over the remaining four years of the Act.
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, more than two-dozen 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. This First Act is just the beginning, we must continue and enhance 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 6 drugs have been developed exclusiverly to treat childhood cancer.
On January 28, 2021, Representatives Jennifer Wexton (D-VA10) and Tom Cole (R-OK4) introduced the bipartisan Gabriella Miller Kids First Research Act 2.0 (HR 623) and in April, Senators Tim Kaine (D-VA) and Jerry Moran (R-KS) introduced (S 1521), a bipartisan Senate companion bill. Both bills take the penalties collected by the U.S. Securities and Exchange Commission (SEC) from pharmaceutical, cosmetic, food supplement, and medical device companies that violate the existing Foreign Corrupt Practices Act (FCPA) and redirects them to critical rare pediatric disease research.
The Gabriella Miller Kids First Research Act 2.0 would redirect funds from the penalties 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 since 2013 – hundreds of millions of dollars from six violations against pharmaceutical companies alone.
With the new 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.
With this legislation, penalties from companies that break the law would be channeled directly to critical medical research.
Cosponsors include more than 80 Representatives and 7 Senators . As of this writing, there are over 300 foundations and organizations including the Alliance for Childhood Cancer and more than 1,600 advocates covering all 50 states, who have signed a letter of support to the House and Senate for this new bill.
Your support is essential now to build upon the successful work that has already been done and to expand and enhance our capabilities in the future. Please consider cosponsoring H.R. 623/ S. 1523 – Gabriella Miller Kids First Research Act 2.0.
Ellyn Miller, always Gabriella’s mom