Costs and benefits of folic acid fortification in the United States: economic analysis, regulatory action, and public health

ManuscriptThe United States Food and Drug Administration (FDA) required that as of January 1, 1998, manufacturers of enriched cereal- grain products fortify their products with folic acid to reduce the number of pregnancies affected by a neural tube defect (NTD). Prior to adoption of the regulation in 1996, three economic evaluations projected the net economic benefits or cost savings of folic acid fortification. The expected percentage decline in NTDs in these three studies was between 2.6% and 10.5%. Birth defects surveillance data indicate that since fortification there has been a 20% to 30% decline in births with either spina bifida or anencephaly. We estimate that folic acid fortification is associated with an economic benefit of $425 million per year in the United States and constitutes a major public health success that has resulted from regulatory actio

Newborn screening for congenital hypothyroidism and congenital adrenal hyperplasia : the balance of benefits and costs of a public health success

Version anglaise de : Van Vliet, G. Grosse, S. D., Dépistage néonatal de l’hypothyroïdie congénitale et de l’hyperplasie congénitale des surrénales : Bénéfices et coûts d’un programme de santé publique à succès. Médecine/Sciences n° 4, vol. 37, avril 2021. doi:10.1051/medsci/2021053.Newborn screening is an important public health program and a triumph of preventive medicine. Economic analyses show that the benefits of newborn screening clearly outweigh the costs for certain diseases but not necessarily for other ones. This is due to the great diversity of the natural history of the diseases detected, to the fact that each of these diseases considered individually is rare, and to differences in the effectiveness of interventions. In addition, the benefit cost ratio of screening for a particular disorder may differ between countries, specifically between high-income and low- and middle-income countries. The burden of a disorder may also be alleviated by increased clinical awareness and effective clinical services, even in the absence of newborn screening. In this article, the authors focus on economic analyses of newborn screening for primary congenital hypothyroidism, which has been in place in high-income countries for roughly 40 years, and for classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. Screening for the latter is not yet universal, even in high-income countries, although the lack of universal implementation may reflect factors other than economic considerations

What Contribution Did Economic Evidence Make to the Adoption of Universal Newborn Hearing Screening Policies in the United States?

Universal newborn hearing screening (UNHS), when accompanied by timely access to intervention services, can improve language outcomes for children born deaf or hard of hearing (D/HH) and result in economic benefits to society. Early Hearing Detection and Intervention (EHDI) programs promote UNHS and using information systems support access to follow-up diagnostic and early intervention services so that infants can be screened no later than 1 month of age, with those who do not pass their screen receiving diagnostic evaluation no later than 3 months of age, and those with diagnosed hearing loss receiving intervention services no later than 6 months of age. In this paper, we first document the rapid roll-out of UNHS/EHDI policies and programs at the national and state/territorial levels in the United States between 1997 and 2005. We then review cost analyses and economic arguments that were made in advancing those policies in the United States. Finally, we examine evidence on language and educational outcomes that pertain to the economic benefits of UNHS/EHDI. In conclusion, although formal cost-effectiveness analyses do not appear to have played a decisive role, informal economic assessments of costs and benefits appear to have contributed to the adoption of UNHS policies in the United States

Health policy for sickle cell disease in Africa: experience from Tanzania on interventions to reduce under-five mortality.

Tanzania has made considerable progress towards reducing childhood mortality, achieving a 57% decrease between 1980 and 2011. This epidemiological transition will cause a reduction in the contribution of infectious diseases to childhood mortality and increase in contribution from non-communicable diseases (NCDs). Haemoglobinopathies are amongst the most common childhood NCDs, with sickle cell disease (SCD) being the commonest haemoglobinopathy in Africa. In Tanzania, 10,313 children with SCD under 5 years of age (U5) are estimated to die every year, contributing an estimated 7% of overall deaths in U5 children. Key policies that governments in Africa are able to implement would reduce mortality in SCD, focusing on newborn screening and comprehensive SCD care programmes. Such programmes would ensure that interventions such as prevention of infections using penicillin plus prompt diagnosis and treatment of complications are provided to all individuals with SCD

Newborn screening for cystic fibrosis: evaluation of benefits and risks and recommendations for state newborn screening programs

In November 2003, CDC and the Cystic Fibrosis Foundation cosponsored a workshop to review the benefits and risks associated with newborn screening for cystic fibrosis (CF). This report describes new research findings and outlines the recommendations of the workshop. The peer-reviewed evidence presented at the workshop supports the clinical utility of newborn screening for CF. Demonstrated long-term benefits from early nutritional treatment as a result of newborn screening for CF include improved growth and, in one study, cognitive development. Other benefits might include reduced hospitalizations and improved survival. Mixed evidence has been reported for pulmonary outcomes. Newborn screening in the United States is associated with diagnosis of CF a median of 1 year earlier than symptomatic detection, which might reduce the expense and anxiety associated with workup for failure to thrive or other symptoms. Certain psychosocial risks for carrier children and their families (e.g., anxiety and misunderstanding) are associated with newborn screening. Exposure of young children to infectious agents through person-to-person transmission in clinical settings, although not an inherent risk of newborn screening, is a potential cause of harm from early detection. Involving specialists in CF care and infection control, genetic counseling, and communication can minimize these potential harms. Although screening decisions depend on a state\u27s individual resources and priorities, on the basis of evidence of moderate benefits and low risk of harm, CDC believes that newborn screening for CF is justified. States should consider the magnitude of benefits and costs and the need to minimize risks through careful planning and implementation, including ongoing collection and evaluation of outcome data

Global Burden of Sickle Cell Anaemia in Children under Five, 2010-2050: Modelling Based on Demographics, Excess Mortality, and Interventions

The global burden of sickle cell anaemia (SCA) is set to rise as a consequence of improved survival in high-prevalence low- and middle-income countries and population migration to higher-income countries. The host of quantitative evidence documenting these changes has not been assembled at the global level. The purpose of this study is to estimate trends in the future number of newborns with SCA and the number of lives that could be saved in under-five children with SCA by the implementation of different levels of health interventions.First, we calculated projected numbers of newborns with SCA for each 5-y interval between 2010 and 2050 by combining estimates of national SCA frequencies with projected demographic data. We then accounted for under-five mortality (U5m) projections and tested different levels of excess mortality for children with SCA, reflecting the benefits of implementing specific health interventions for under-five patients in 2015, to assess the number of lives that could be saved with appropriate health care services. The estimated number of newborns with SCA globally will increase from 305,800 (confidence interval [CI]: 238,400-398,800) in 2010 to 404,200 (CI: 242,500-657,600) in 2050. It is likely that Nigeria (2010: 91,000 newborns with SCA [CI: 77,900-106,100]; 2050: 140,800 [CI: 95,500-200,600]) and the Democratic Republic of the Congo (2010: 39,700 [CI: 32,600-48,800]; 2050: 44,700 [CI: 27,100-70,500]) will remain the countries most in need of policies for the prevention and management of SCA. We predict a decrease in the annual number of newborns with SCA in India (2010: 44,400 [CI: 33,700-59,100]; 2050: 33,900 [CI: 15,900-64,700]). The implementation of basic health interventions (e.g., prenatal diagnosis, penicillin prophylaxis, and vaccination) for SCA in 2015, leading to significant reductions in excess mortality among under-five children with SCA, could, by 2050, prolong the lives of 5,302,900 [CI: 3,174,800-6,699,100] newborns with SCA. Similarly, large-scale universal screening could save the lives of up to 9,806,000 (CI: 6,745,800-14,232,700) newborns with SCA globally, 85% (CI: 81%-88%) of whom will be born in sub-Saharan Africa. The study findings are limited by the uncertainty in the estimates and the assumptions around mortality reductions associated with interventions.Our quantitative approach confirms that the global burden of SCA is increasing, and highlights the need to develop specific national policies for appropriate public health planning, particularly in low- and middle-income countries. Further empirical collaborative epidemiological studies are vital to assess current and future health care needs, especially in Nigeria, the Democratic Republic of the Congo, and India

Depressive and anxiety disorders and antidepressant prescriptions among insured children and young adults with congenital adrenal hyperplasia in the United States

BackgroundDysfunction in the hypothalamic-pituitary-adrenal axis has been associated with depressive and anxiety disorders. Little is known about the risk for these disorders among individuals with congenital adrenal hyperplasia (CAH), a form of primary adrenal insufficiency.ObjectiveWe investigated the prevalence of depressive and anxiety disorders and antidepressant prescriptions in two large healthcare databases of insured children, adolescents, and young adults with CAH in the United States.MethodsWe conducted a retrospective cohort study using administrative data from October 2015 through December 2019 for individuals aged 4–25 years enrolled in employer-sponsored or Medicaid health plans.ResultsAdjusting for age, the prevalence of depressive disorders [adjusted prevalence ratio (aPR) = 1.7, 95% confidence interval (CI): 1.4-2.0, p<0.001], anxiety disorders [aPR = 1.7, 95% CI: 1.4-1.9, p<0.001], and filled antidepressant prescriptions [aPR = 1.7, 95% CI: 1.4-2.0, p<0.001] was higher among privately insured youth with CAH as compared to their non-CAH peers. Prevalence estimates were also higher among publicly insured youth with CAH for depressive disorders [aPR = 2.3, 95% CI: 1.9-2.9, p<0.001], anxiety disorders [aPR = 2.0, 95% CI: 1.6-2.5, p<0.001], and filled antidepressant prescriptions [aPR = 2.5, 95% CI: 1.9-3.1, p<0.001] as compared to their non-CAH peers.ConclusionsThe elevated prevalence of depressive and anxiety disorders and antidepressant prescriptions among youth with CAH suggests that screening for symptoms of depression and anxiety among this population might be warranted