Abstract:
BACKGROUND:Orphan drug development is crucial for children, who are disproportionately affected by rare diseases. Data are lacking on the number, nature, and benefit of recently approved pediatric orphan indications. METHODS:We classified the 402 orphan indications the US Food and Drug Administration approved between 2010 and 2018 as "pediatric" if they were approved for children only or targeted pediatric diseases. We determined the number of unique diseases targeted by pediatric orphan indications and calculated the proportion that were for (1) novel drugs, (2) non-novel drugs approved to treat ≥1 common disease, and (3) non-novel drugs approved only to treat rare diseases. Among pediatric orphan indications eligible for US Food and Drug Administration breakthrough designation (granted to drugs potentially representing major therapeutic advances), we calculated the proportion receiving this designation. RESULTS:Of the 402 orphan indications, 136 (33.8%) were pediatric. These 136 indications targeted 87 unique diseases; 21 diseases were targeted by ≥1 indication. Of the 136 pediatric orphan indications, 60 (44.1%) were for novel drugs, 45 (33.1%) were for non-novel drugs approved to treat ≥1 common disease, and 31 (22.8%) were for non-novel drugs approved only to treat rare diseases. Among 97 indications eligible for breakthrough designation, 20 (20.6%) received this designation. CONCLUSIONS:Recent orphan drug development has increased the availability of treatments for pediatric rare diseases. Most pediatric orphan indications expanded use of existing drugs, and many targeted the same disease. Some indications may represent breakthroughs, but substantial unmet need for treatments remains for most pediatric rare diseases.
journal_name
Pediatricsjournal_title
Pediatricsauthors
Kimmel L,Conti RM,Volerman A,Chua KPdoi
10.1542/peds.2019-3128subject
Has Abstractpub_date
2020-04-01 00:00:00issue
4eissn
0031-4005issn
1098-4275pii
peds.2019-3128journal_volume
145pub_type
杂志文章相关文献
PEDIATRICS文献大全abstract:OBJECTIVE:We examined the association between state Medicaid and State Children's Health Insurance Program (SCHIP) income eligibility and the financial burden reported by low-income families raising children with special health care needs (CSHCN). SAMPLE AND METHODS:Data on low-income CSHCN and their families were fro...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2009-1255P
更新日期:2009-12-01 00:00:00
abstract::Standard recommendations for evaluating and treating jaundice in term babies include following all babies closely for jaundice, obtaining several laboratory tests in those with early jaundice or bilirubin levels more than 12 to 13 mg/dL (205 to 222 mumol/L), using phototherapy to try to keep bilirubin levels below 20 ...
journal_title:Pediatrics
pub_type: 杂志文章,评审
doi:
更新日期:1992-05-01 00:00:00
abstract:OBJECTIVES:To assess the relative frequency of, the clinical and pathological correlates in, and the prognosis of the subset of infants with neuroblastoma who were identified initially by prenatal ultrasonography. DESIGN:Retrospective review of all patients with neuroblastoma evaluated between 1982 and 1992. SETTING:...
journal_title:Pediatrics
pub_type: 杂志文章
doi:
更新日期:1993-09-01 00:00:00
abstract::Death from cancer is often painful. Usually, the pain can be relieved in ways that allow patients to remain awake and alert until the end. Sometimes, however, the only way to relieve pain is to sedate patients until they are unconscious. This method has been called palliative sedation therapy. Palliative sedation ther...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2017-0487
更新日期:2017-08-01 00:00:00
abstract::: media-1vid110.1542/5799875103001PEDS-VA_2017-3616Video Abstract OBJECTIVES: Noncigarette tobacco products may confer a risk of marijuana use similar to combustible cigarettes. We examined whether adolescent electronic cigarette (e-cigarette), hookah, or combustible cigarette use is associated with initiating and cur...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2017-3616
更新日期:2018-09-01 00:00:00
abstract:OBJECTIVE:The optimal treatment of neonatal hyperglycemia is unclear. The aim of this trial was to determine whether tight glycemic control with insulin improves growth in hyperglycemic preterm infants, without increasing the incidence of hypoglycemia. METHODS:Randomized, controlled, nonblinded trial of 88 infants bor...
journal_title:Pediatrics
pub_type: 杂志文章,随机对照试验
doi:10.1542/peds.2011-2470
更新日期:2012-04-01 00:00:00
abstract:CONTEXT:The growing prevalence of pediatric mental and behavioral health disorders, coupled with scarce psychiatric resources, has resulted in a substantial increase in the number of youth waiting in emergency departments (EDs) and medical units for inpatient psychiatric care. OBJECTIVE:To characterize the prevalence ...
journal_title:Pediatrics
pub_type: 杂志文章,评审
doi:10.1542/peds.2020-1174
更新日期:2020-10-01 00:00:00
abstract:BACKGROUND:Adults with childhood-onset epilepsy experience poorer adult social outcomes than their peers. The relative roles of seizures over time versus learning and psychiatric problems are unclear. METHODS:We examined independent influences of psychiatric and learning disorders and of seizure course in 241 young ad...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2015-3944
更新日期:2016-04-01 00:00:00
abstract::This statement updates the 1994 American Academy of Pediatrics policy statement on baseball and softball injuries in children. Current studies on acute, overuse, and catastrophic injuries are reviewed with emphasis on the causes and mechanisms of injury. This information serves as a basis for recommending safe trainin...
journal_title:Pediatrics
pub_type: 指南,杂志文章
doi:10.1542/peds.107.4.782
更新日期:2001-04-01 00:00:00
abstract:OBJECTIVES:To address the "millennial morbidities," pediatricians must partner with community-based organizations to develop interventions. Little is known about the capacity of the resulting programs for program evaluation or the importance of evaluation in project success and sustainability. The objective of this stu...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2006-3273
更新日期:2008-09-01 00:00:00
abstract::Urinary concentrations of beta 2-microglobulin (beta 2M) and creatinine were measured in normal term infants and in those born with meconium-stained amniotic fluid. None of the infants or their mothers had conditions known to modify beta 2M excretion. Measurements of beta 2M were made on urines collected by bagging; u...
journal_title:Pediatrics
pub_type: 杂志文章
doi:
更新日期:1985-12-01 00:00:00
abstract:OBJECTIVE:Drug trafficking among adolescents is a newly recognized high-risk behavior that seems to be involving large numbers of youths. Strategies to prevent and/or alter this behavior must be developed and evaluated. In view of the high exposure of adolescents to the mass media, interventionists seeking to reduce ad...
journal_title:Pediatrics
pub_type: 杂志文章,评审
doi:
更新日期:1994-06-01 00:00:00
abstract:OBJECTIVE:The aim of our study was to evaluate the effect of zinc supplementation on linear growth, body composition, and growth factors in premature infants. DESIGN:Thirty-six preterm infants (gestational age: 32.0 +/- 2.1 weeks, birth weight: 1704 +/- 364 g) participated in a longitudinal double-blind, randomized cl...
journal_title:Pediatrics
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.1542/peds.111.5.1002
更新日期:2003-05-01 00:00:00
abstract::Young patients and their parents often are unsure about what represents normal menstrual patterns, and clinicians also may be unsure about normal ranges for menstrual cycle length and amount and duration of flow through adolescence. It is important to be able to educate young patients and their parents regarding what ...
journal_title:Pediatrics
pub_type: 杂志文章,评审
doi:10.1542/peds.2006-2481
更新日期:2006-11-01 00:00:00
abstract:BACKGROUND:A substudy of the Management of Myelomeningocele Study evaluating urological outcomes was conducted. METHODS:Pregnant women diagnosed with fetal myelomeningocele were randomly assigned to either prenatal or standard postnatal surgical repair. The substudy included patients randomly assigned after April 18, ...
journal_title:Pediatrics
pub_type: 杂志文章,随机对照试验
doi:10.1542/peds.2015-2114
更新日期:2015-10-01 00:00:00
abstract:OBJECTIVE:To assess the physiologic response to salt depletion in subjects with cystic fibrosis (CF) and control male adolescents for sodium balance, sodium space, and stimulation of the renin-angiotensin-aldosterone axis. DESIGN:Seven subjects with CF and six controls received a salt-replete (150 or 290 mmol NaCl per...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.101.1.48
更新日期:1998-01-01 00:00:00
abstract::Two matched groups of children with a family history of atopy/allergy were observed from birth. In one group (n = 65) the mothers had a diet free from eggs, cow's milk, and fish during the first 3 months of lactation, whereas the mothers in the other group (n = 50) had a normal diet. Atopic/allergic manifestations, sk...
journal_title:Pediatrics
pub_type: 杂志文章
doi:
更新日期:1992-04-01 00:00:00
abstract:OBJECTIVE:We performed a randomized, double-masked, parallel-groups, placebo-controlled trial of recombinant granulocyte colony-stimulating factor (rG-CSF) administration to 44 preterm neonates who had blood cultures obtained and antibiotics begun because of the clinical diagnosis of early-onset sepsis. Two primary out...
journal_title:Pediatrics
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1542/peds.107.1.30
更新日期:2001-01-01 00:00:00
abstract:OBJECTIVE:Increased physical activity and menstrual irregularity have been associated with increased risk for stress fracture among adult women active in athletics. The purposes of this study were to determine whether menstrual irregularity is also a risk factor for stress fracture in active female adolescents and to e...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2006-2145
更新日期:2007-08-01 00:00:00
abstract:BACKGROUND:Some parents are requesting aluminum testing in their children with developmental issues. Although aluminum can be measured in plasma, serum, or urine, there is scant scientific information about normal ranges. We sought to determine the basis for laboratory reference ranges and whether these ranges are appl...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2010-3481
更新日期:2012-01-01 00:00:00
abstract::A workgroup meeting on the use of family history information in pediatric primary care and public health sponsored by the National Center on Birth Defects and Developmental Disabilities at the Centers for Disease Control and Prevention was held February 24 to 25, 2006. The workgroup participants met to discuss how to ...
journal_title:Pediatrics
pub_type:
doi:10.1542/peds.2007-1010H
更新日期:2007-09-01 00:00:00
abstract:BACKGROUND AND OBJECTIVES:Children with special health care needs (CSHCN) face unique challenges in accessing affordable health care. Massachusetts implemented major health reform in 2006; little is known about the impact of this state's health reform on uninsurance, access to care, and financial protection for private...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2013-3884
更新日期:2014-08-01 00:00:00
abstract:OBJECTIVES:The reported increasing prevalence of autism spectrum disorder (ASD) and attendant health and family impact make monitoring of ASD prevalence a public health priority. METHODS:The prevalence of parent-reported diagnosis of ASD among US children aged 3 to 17 years was estimated from the 2007 National Survey ...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2009-1522
更新日期:2009-11-01 00:00:00
abstract::Social networking web sites are popular among adolescents and may represent a new venue for conducting adolescent health research. Conducting research by using social networking web sites raises several concerns, including the social value of this research, fair subject selection, confidentiality, privacy, and informe...
journal_title:Pediatrics
pub_type: 杂志文章,评审
doi:10.1542/peds.2007-3015
更新日期:2008-01-01 00:00:00
abstract:OBJECTIVE:There is no safe level of exposure to tobacco smoke. Thirdhand smoke is residual tobacco smoke contamination that remains after the cigarette is extinguished. Children are uniquely susceptible to thirdhand smoke exposure. The objective of this study was to assess health beliefs of adults regarding thirdhand s...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2008-2184
更新日期:2009-01-01 00:00:00
abstract:OBJECTIVE:To evaluate a simple, relatively inexpensive method using the fluid bed to provide high-intensity double-surface (HIDS) phototherapy and to confirm that the method described is more effective than conventional phototherapy (CPT) in the treatment of neonatal hyperbilirubinemia. DESIGN:Prospective controlled s...
journal_title:Pediatrics
pub_type: 临床试验,杂志文章
doi:
更新日期:1995-06-01 00:00:00
abstract:OBJECTIVE:Lifelong functional, adaptive, and economic outcomes of moderate to severe infantile malnutrition are not well known. We assessed social status and income at midlife in a cohort of Barbadian adults, hospitalized for protein-energy malnutrition (PEM) during the first year of life, with good nutrition and healt...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2012-0073
更新日期:2012-07-01 00:00:00
abstract::The Vaccine Safety Datalink (VSD) project is a collaborative project between the Centers for Disease Control and Prevention and 8 managed care organizations (MCOs) in the United States. Established in 1990 to conduct postmarketing evaluations of vaccine safety, the project has created an infrastructure that allows for...
journal_title:Pediatrics
pub_type: 杂志文章,评审
doi:10.1542/peds.2010-1722H
更新日期:2011-05-01 00:00:00
abstract:OBJECTIVE:We followed a cohort (N = 306) of infants at well-baby visits in two suburban pediatric practices to assess the relation of exclusive breastfeeding, and other environmental exposures, to episodes of acute otitis media (AOM) and otitis media with effusion (OME). METHODS:Detailed prospective information about ...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.100.4.e7
更新日期:1997-10-01 00:00:00
abstract:OBJECTIVE:Preterm birth is associated with respiratory morbidity later in life, including asthma. Previous studies have mainly focused on asthma in early childhood in children born extremely preterm. In this study, we examined the risk of asthma in a national cohort of schoolchildren grouped according to degree of imma...
journal_title:Pediatrics
pub_type: 杂志文章
doi:10.1542/peds.2010-3083
更新日期:2011-06-01 00:00:00