Deparment of Pediatrics and Medicine, Stony Brook University Hospital, Stony Brook, New York.
Division of Pediatric Pulmonology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland.
JAMA Netw Open. 2020 Mar 2;3(3):e201737. doi: 10.1001/jamanetworkopen.2020.1737.
Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal recessive disorders associated with intermittent disease exacerbations that require hospitalizations, progressive chronic organ injury, and substantial premature mortality. Research funding is a limited resource and may contribute to health care disparities, especially for rare diseases that disproportionally affect economically disadvantaged groups.
To compare disease-specific funding between SCD and CF and the association between funding and research productivity.
DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study examined federal and foundation funding, publications indexed in PubMed, clinical trials registered in ClinicalTrials.gov, and new drug approvals from January 1, 2008, to December 31, 2018, in an estimated US population of approximately 90 000 individuals with SCD and approximately 30 000 individuals with CF.
Federal and foundation funding, publications indexed in PubMed, clinical trial registrations, and new drug approvals.
From 2008 through 2018, federal funding was greater per person with CF compared with SCD (mean [SD], $2807 [$175] vs $812 [$147]; P < .001). Foundation expenditures were greater for CF than for SCD (mean [SD], $7690 [$3974] vs $102 [$13.7]; P < .001). Significantly more research articles (mean [SD], 1594 [225] vs 926 [157]; P < .001) and US Food and Drug Administration drug approvals (4 vs 1) were found for CF compared with SCD, but the total number of clinical trials was similar (mean [SD], 27.3 [6.9] vs 23.8 [6.3]; P = .22).
The findings show that disparities in funding between SCD and CF may be associated with decreased research productivity and novel drug development for SCD. Increased federal and foundation funding is needed for SCD and other diseases that disproportionately affect economically disadvantaged groups to address health care disparities.
镰状细胞病（SCD）和囊性纤维化（CF）是严重的常染色体隐性疾病，与间歇性疾病恶化有关，需要住院治疗、进行慢性器官渐进性损伤以及过早死亡。研究资金是一种有限的资源，可能导致医疗保健方面的差距，尤其是对于那些不成比例地影响经济弱势群体的罕见疾病。
比较 SCD 和 CF 之间的疾病特异性资金以及资金与研究生产力之间的关系。
设计、地点和参与者：这项横断面研究调查了 2008 年 1 月 1 日至 2018 年 12 月 31 日期间联邦和基金会资金、在 PubMed 中索引的出版物、在 ClinicalTrials.gov 中注册的临床试验以及新批准的药物，研究对象为美国约 90000 名 SCD 患者和约 30000 名 CF 患者。
联邦和基金会资金、在 PubMed 中索引的出版物、临床试验注册和新批准的药物。
从 2008 年到 2018 年，每例 CF 患者的联邦资金高于 SCD（平均[标准差]，$2807 [$175]比$812 [$147]；P < .001）。基金会支出 CF 比 SCD 多（平均[标准差]，$7690 [$3974]比$102 [$13.7]；P < .001）。与 SCD 相比，CF 有更多的研究文章（平均[标准差]，1594 [225]比 926 [157]；P < .001）和美国食品和药物管理局的药物批准（4 比 1），但临床试验总数相似（平均[标准差]，27.3 [6.9]比 23.8 [6.3]；P = .22）。
研究结果表明，SCD 和 CF 之间的资金差距可能与 SCD 研究生产力下降和新药开发减少有关。需要增加 SCD 和其他不成比例地影响经济弱势群体的疾病的联邦和基金会资金，以解决医疗保健差距。
