{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,6]],"date-time":"2025-10-06T18:16:19Z","timestamp":1759774579923,"version":"3.41.2"},"reference-count":26,"publisher":"American Academy of Pediatrics (AAP)","issue":"2","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2019,2,1]]},"abstract":"<jats:p>In 2016, the EveryLife Foundation for Rare Diseases, in partnership with Dr Pan, who is a pediatrician and state senator in California, launched legislation to advance and expand newborn screening. Researchers have shown that newborn screening can be cost-effective and can greatly improve health outcomes for patients with rare diseases. However, adding additional diseases in newborn screening is a long process, requiring legislative approval in addition to new state funding. Such process delays can lead to protracted diagnostic odysseys for patients, especially those with rare diseases. These delays can result in irreversible morbidity and, in some cases, early mortality for patients. To improve this process, legislation known as Senate Bill 1095 was introduced to require California to adhere to the latest federal recommendations for newborn screening within 2 years. We provide insight and describe the process of advancing state legislation, coalition building, and managing opposition. Senate Bill 1095 would become law in 2016, requiring California to screen for 2 new rare diseases by August 2018: mucopolysaccharidosis type I and Pompe disease. This case study can serve as a model for advocates looking to expand state newborn-screening programs.<\/jats:p>","DOI":"10.1542\/peds.2018-1886","type":"journal-article","created":{"date-parts":[[2019,1,3]],"date-time":"2019-01-03T13:25:22Z","timestamp":1546521922000},"source":"Crossref","is-referenced-by-count":7,"title":["Leveraging Evidence-Based Public Policy and Advocacy to Advance Newborn Screening in California"],"prefix":"10.1542","volume":"143","author":[{"given":"Max G.","family":"Bronstein","sequence":"first","affiliation":[{"name":"Audentes Therapeutics, San Francisco, California;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard J.","family":"Pan","sequence":"additional","affiliation":[{"name":"California State Senate, Sacramento, California;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark","family":"Dant","sequence":"additional","affiliation":[{"name":"The Ryan Foundation and EveryLife Foundation for Rare Diseases, Washington, District of Columbia; and"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bertrand","family":"Lubin","sequence":"additional","affiliation":[{"name":"University of California, San Francisco Benioff Children\u2019s Hospital, San Francisco, California"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"417","published-online":{"date-parts":[[2019,2,1]]},"reference":[{"issue":"19","key":"2021121021320727500_B1","first-page":"619","article-title":"Ten great public health achievements","volume":"60","author":"Centers for Disease Control and Prevention","year":"2011","journal-title":"MMWR"},{"key":"2021121021320727500_B2","unstructured":"National Institutes of Health Eunice Kennedy Shriver National Institute of Child Health and Human Development . Brief history of newborn screening website. 2017. Available at: https:\/\/www.nichd.nih.gov\/health\/topics\/newborn\/conditioninfo\/history. Accessed April 13, 2018"},{"issue":"4","key":"2021121021320727500_B3","doi-asserted-by":"crossref","first-page":"486","DOI":"10.1002\/pbc.21883","article-title":"Newborn screening for hemoglobinopathies in California.","volume":"52","author":"Michlitsch","year":"2009","journal-title":"Pediatr Blood Cancer"},{"issue":"6","key":"2021121021320727500_B4","doi-asserted-by":"crossref","first-page":"749","DOI":"10.1542\/peds.81.6.749","article-title":"Newborn screening for sickle cell disease: effect on mortality.","volume":"81","author":"Vichinsky","year":"1988","journal-title":"Pediatrics"},{"key":"2021121021320727500_B5","unstructured":"National Institutes of Health Eunice Kennedy Shriver National Institute of Child Health and Human Development . How is newborn screening done? 2017. 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Available at: https:\/\/www.hrsa.gov\/advisory-committees\/heritable-disorders\/recommendations-reports\/index.html. Accessed April 13, 2018"},{"issue":"5, pt 2","key":"2021121021320727500_B9","first-page":"S296","article-title":"Newborn screening: toward a uniform screening panel and system\u2013executive summary.","volume":"117","author":"American College of Medical Genetics Newborn Screening Expert Group","year":"2006","journal-title":"Pediatrics"},{"key":"2021121021320727500_B10","unstructured":"Advisory Committee on Heritable Disorders in Newborns and Children . ACHDNC Form for Nomination of a Condition for Inclusion in the Uniform Screening Panel. Available at: https:\/\/www.hrsa.gov\/sites\/default\/files\/hrsa\/advisory-committees\/heritable-disorders\/rusp\/Nominate-condition\/nomination-form.pdf. Accessed December 12, 2018"},{"key":"2021121021320727500_B11","unstructured":"Eunice Kennedy Shriver National Institute of Child Health and Human Development . How many newborns are screened in the United States? Available at: https:\/\/www.nichd.nih.gov\/health\/topics\/newborn\/conditioninfo\/infants-screened. Accessed June 8, 2018"},{"issue":"1","key":"2021121021320727500_B12","doi-asserted-by":"crossref","first-page":"e20170300","DOI":"10.1542\/peds.2017-0300","article-title":"Delay in state adoption of newborn screening tests [published correction appears in Pediatrics. 2018;141(4):e20174305].","volume":"141","author":"Xu","year":"2018","journal-title":"Pediatrics"},{"issue":"1","key":"2021121021320727500_B13","doi-asserted-by":"crossref","first-page":"54","DOI":"10.1002\/ajmg.a.36197","article-title":"A large-scale nationwide newborn screening program for Pompe disease in Taiwan: towards effective diagnosis and treatment.","volume":"164A","author":"Yang","year":"2014","journal-title":"Am J Med Genet A"},{"issue":"5, pt 2","key":"2021121021320727500_B14","doi-asserted-by":"crossref","first-page":"S287","DOI":"10.1542\/peds.2005-2633H","article-title":"Comprehensive cost-utility analysis of newborn screening strategies.","volume":"117","author":"Carroll","year":"2006","journal-title":"Pediatrics"},{"issue":"5","key":"2021121021320727500_B15","doi-asserted-by":"crossref","first-page":"613","DOI":"10.1016\/j.jval.2012.02.007","article-title":"Cost-effectiveness of expanded newborn screening in Texas.","volume":"15","author":"Tiwana","year":"2012","journal-title":"Value Health"},{"key":"2021121021320727500_B16","unstructured":"California State Legislature . SB-1095 newborn screening program. 2016. Available at: https:\/\/leginfo.legislature.ca.gov\/faces\/billNavClient.xhtml?bill_id=201520160SB1095. Accessed April 13, 2018"},{"key":"2021121021320727500_B17","unstructured":"March of Dimes . Newborn screening funding. 2018. Available at: www.marchofdimes.org\/advocacy\/newborn-screening-funding.aspx. Accessed April 13, 2018"},{"issue":"5, pt 2","key":"2021121021320727500_B18","doi-asserted-by":"crossref","first-page":"S280","DOI":"10.1542\/peds.2005-2633G","article-title":"Economic evaluation of tandem mass spectrometry screening in California.","volume":"117","author":"Feuchtbaum","year":"2006","journal-title":"Pediatrics"},{"key":"2021121021320727500_B19","unstructured":"Association of Maternal and Child Health Programs . Newborn screening contingency plan (CONPLAN). 2010. Available at: www.amchp.org\/programsandtopics\/CHILD-HEALTH\/projects\/newborn-screening\/Documents\/NBS-CONPLAN_2010.pdf. Accessed April 13, 2018"},{"key":"2021121021320727500_B20","unstructured":"Health Resources and Services Administration Advisory Committee on Heritable Disorders in Newborns and Children . Summary of nominated conditions to the Recommended Uniform Screening Panel (RUSP). 2016. Available at: https:\/\/www.hrsa.gov\/sites\/default\/files\/hrsa\/advisory-committees\/heritable-disorders\/rusp\/previous-nominations\/summary-previously-nominated-conditions.pdf. 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