NIH and National Foundation Expenditures For Sickle Cell Disease and Cystic Fibrosis Are Associated With Pubmed Publications and FDA Approvals

摘要: Sickle cell disease (SCD) and cystic fibrosis are both rare inherited diseases most often diagnosed in the United States by newborn screening. Early diagnosis treatment have led to substantial improvements survival, but adult mortality remains high with median survival of 40 45 years for sickle anemia 35 fibrosis. Disparities funding between these two similar severity been long recognized, relationship research productivity has not rigorously evaluated. We hypothesized that expenditures NIH national foundations would be associated number publications indexed PubMed, active clinical trials, FDA approvals. estimated disease-specific career development awards using RePORT (http://www.report.nih.gov/categorical_spending.aspx) Form 990 financial reports year Cell Disease Association America Cystic Fibrosis Foundation Therapeutics, Inc. calculated per individual published estimates people SCD (89,079) (30,000) States. developed a comprehensive search strategy medical informationist identify relevant PubMed new postings trials (clinicaltrials.gov) disease. reviewed approvals from 2009 July 2013 indications or Funding affected was 7.6 (2010) 11.4 fold (2011) greater than included 3.5-fold higher 370 440 foundation funding. were despite nearly 3-fold more individuals SCD. There twice as many slightly listings No drugs approved 2010 compared 5 confirmed widely disparate Research measured articles drug substantially fibrosis, fewer This likely reflects increased resources available quality improvement efforts. Disclosures: Strouse:NIH: Funding; Doris Duke Charitable Foundation: Masimo Corporation: Membership on an entity’s Board Directors advisory committees, Funding. Lanzkron:NIH: Haywood:NIH: