{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T13:17:50Z","timestamp":1785590270690,"version":"3.56.0"},"reference-count":33,"publisher":"American Medical Association (AMA)","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Pediatr"],"published-print":{"date-parts":[[2021,6,1]]},"DOI":"10.1001\/jamapediatrics.2020.7140","type":"journal-article","created":{"date-parts":[[2021,3,22]],"date-time":"2021-03-22T15:31:40Z","timestamp":1616427100000},"page":"617","source":"Crossref","is-referenced-by-count":40,"title":["A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease"],"prefix":"10.1001","volume":"175","author":[{"given":"Patrick","family":"DeMartino","sequence":"first","affiliation":[{"name":"Division of Pediatric Hematology and Oncology, Department of Pediatrics, Oregon Health & Science University, Doernbecher Children\u2019s Hospital, Portland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meredith B.","family":"Haag","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology and Oncology, Department of Pediatrics, Oregon Health & Science University, Doernbecher Children\u2019s Hospital, Portland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alyssa R.","family":"Hersh","sequence":"additional","affiliation":[{"name":"Oregon Health & Science University, Department of Obstetrics and Gynecology, Portland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron B.","family":"Caughey","sequence":"additional","affiliation":[{"name":"Oregon Health & Science University, Department of Obstetrics and Gynecology, Portland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joshua A.","family":"Roth","sequence":"additional","affiliation":[{"name":"University of Washington School of Pharmacy, Fred Hutchinson Institute for Cancer Outcomes Research, Seattle"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","reference":[{"issue":"6","key":"poi200108r1","doi-asserted-by":"publisher","first-page":"621","DOI":"10.1016\/j.jval.2019.03.014","article-title":"Estimating the clinical pipeline of cell and gene therapies and their potential economic impact on the US healthcare system.","volume":"22","author":"Quinn","year":"2019","journal-title":"Value Health"},{"issue":"19","key":"poi200108r4","doi-asserted-by":"publisher","first-page":"1977","DOI":"10.1001\/jama.2018.15782","article-title":"Alternative state-level financing for hepatitis C treatment\u2014the \u201cNetflix model\u201d.","volume":"320","author":"Trusheim","year":"2018","journal-title":"JAMA"},{"issue":"4","key":"poi200108r5","doi-asserted-by":"publisher","first-page":"952","DOI":"10.1177\/1073110518821995","article-title":"Can outcomes-based pharmaceutical contracts reduce drug prices in the US? a mixed methods assessment.","volume":"46","author":"Seeley","year":"2018","journal-title":"J Law Med Ethics"},{"issue":"2","key":"poi200108r7","doi-asserted-by":"publisher","first-page":"377","DOI":"10.1002\/pbc.24176","article-title":"Health-related quality of life in sickle cell disease: past, present, and future.","volume":"59","author":"Panepinto","year":"2012","journal-title":"Pediatr Blood Cancer"},{"key":"poi200108r8","doi-asserted-by":"publisher","first-page":"59","DOI":"10.1186\/1477-7525-4-59","article-title":"Hydroxyurea and sickle cell anemia: effect on quality of life.","volume":"4","author":"Ballas","year":"2006","journal-title":"Health Qual Life Outcomes"},{"issue":"6","key":"poi200108r9","doi-asserted-by":"publisher","first-page":"615","DOI":"10.1182\/blood-2018-10-880575","article-title":"Decreased median survival of adults with sickle cell disease after adjusting for left truncation bias: a pooled analysis.","volume":"133","author":"DeBaun","year":"2019","journal-title":"Blood"},{"issue":"3","key":"poi200108r10","doi-asserted-by":"publisher","first-page":"226","DOI":"10.1056\/NEJMoa1715971","article-title":"A phase 3 trial of l-glutamine in sickle cell disease.","volume":"379","author":"Niihara","year":"2018","journal-title":"N Engl J Med"},{"issue":"6","key":"poi200108r11","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1056\/NEJMoa1903212","article-title":"A phase 3 randomized trial of voxelotor in sickle cell disease.","volume":"381","author":"Vichinsky","year":"2019","journal-title":"N Engl J Med"},{"key":"poi200108r12","author":"Ataga","journal-title":"N Engl J Med"},{"issue":"6","key":"poi200108r13","doi-asserted-by":"publisher","first-page":"323","DOI":"10.1002\/ajh.v84:6","article-title":"The cost of health care for children and adults with sickle cell disease.","volume":"84","author":"Kauf","year":"2009","journal-title":"Am J Hematol"},{"key":"poi200108r14","author":"Kanter","year":"2019","journal-title":"Blood"},{"issue":"11","key":"poi200108r17","doi-asserted-by":"publisher","first-page":"1823","DOI":"10.3324\/haematol.2017.169581","article-title":"Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases.","volume":"102","author":"Arnold","year":"2017","journal-title":"Haematologica"},{"issue":"1","key":"poi200108r20","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1016\/j.jval.2013.08.2291","article-title":"Budget impact analysis\u2014principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force.","volume":"17","author":"Sullivan","year":"2014","journal-title":"Value Health"},{"key":"poi200108r23","author":"Broder","journal-title":"Am Health Drug Benefits"},{"issue":"12","key":"poi200108r24","doi-asserted-by":"publisher","first-page":"2147","DOI":"10.1377\/hlthaff.2015.0706","article-title":"The growing difference between public and private payment rates for inpatient hospital care.","volume":"34","author":"Selden","year":"2015","journal-title":"Health Aff (Millwood)"},{"key":"poi200108r26","doi-asserted-by":"publisher","first-page":"S336","DOI":"10.1016\/j.jval.2019.04.1644","article-title":"Use of single dose gene-replacement therapy for the treatment of spinal muscular atrophy type 1: a United States payer budget impact analysis.","volume":"22","author":"Malone","year":"2019","journal-title":"Value Health"},{"issue":"13","key":"poi200108r27","doi-asserted-by":"publisher","first-page":"1279","DOI":"10.1056\/NEJMc1506108","article-title":"Early patterns of sofosbuvir utilization by state Medicaid programs.","volume":"373","author":"Liao","year":"2015","journal-title":"N Engl J Med"},{"issue":"3","key":"poi200108r28","doi-asserted-by":"publisher","first-page":"e201737","DOI":"10.1001\/jamanetworkopen.2020.1737","article-title":"Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity.","volume":"3","author":"Farooq","year":"2020","journal-title":"JAMA Netw Open"},{"issue":"1","key":"poi200108r33","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1586\/14737167.2015.990887","article-title":"Financing cures in the United States.","volume":"15","author":"Basu","year":"2015","journal-title":"Expert Rev Pharmacoecon Outcomes Res"},{"key":"poi200108r15","author":"Walters"},{"key":"poi200108r2","unstructured":"The FoCUS Project. Survey results: Payer perspectives on financing and reimbursement of one-time high-cost durable treatments. Massachusetts Institute of Technology Center for Biomedical Innovation. Published October 11, 2019. Accessed January 20, 2020. https:\/\/newdigs.mit.edu\/sites\/default\/files\/MIT FoCUS Payer Perspectives 2019F210v044.pdf"},{"key":"poi200108r3","unstructured":"Wyden? R, Grassley? C. The price of Sovaldi and its impact on the U.S. health care system. Committee on Finance, United States Senate. Published December 2015. Accessed January 20, 2020. https:\/\/www.finance.senate.gov\/imo\/media\/doc\/1%20The%20Price%20of%20Sovaldi%20and%20Its%20Impact%20on%20the%20U.S.%20Health%20Care%20System%20(Full%20Report).pdf"},{"key":"poi200108r6","unstructured":"Centers for Disease Control and Prevention. Data & statistics on sickle cell disease. Updated October 21, 2019. Accessed April 1, 2020. https:\/\/www.cdc.gov\/ncbddd\/sicklecell\/data.html"},{"key":"poi200108r16","unstructured":"Wilson-Frederick? SH, Hulihan? M, Anderson? KK. Prevalence of sickle cell disease among Medicaid beneficiaries in 2012. Centers for Medicare & Medicaid Services Office of Minority Health. Published June 2019. Accessed November 10, 2019. https:\/\/www.cms.gov\/About-CMS\/Agency-Information\/OMH\/Downloads\/Data-Highlight-16-Sickle-Cell-Disease.pdf"},{"key":"poi200108r18","unstructured":"Beasley? DM, Mathias? T. Bluebird prices gene therapy at 1.58 million euros over 5 years. Reuters Health News. Published June 14, 2019. Accessed November 10, 2019. https:\/\/www.reuters.com\/article\/us-bluebird-bio-gene-therapy-price\/bluebird-prices-gene-therapy-at-1-575-million-euros-over-five-years-idUSKCN1TF1HP"},{"key":"poi200108r19","unstructured":"The Kaiser Family Foundation. State health facts: total Medicaid spending FY 2018. Updated August 2019. Accessed November 1, 2019. https:\/\/www.kff.org\/medicaid\/state-indicator\/total-medicaid-spending\/?currentTimeframe=0&sortModel=%7B%22colId%22:%22Location%22,%22sort%22:%22asc%22%7D"},{"key":"poi200108r21","unstructured":"Clinicaltrials.gov. A study evaluating the safety and efficacy of bb1111 in severe sickle cell disease. Published May 16, 2014. Accessed November 10, 2019. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02140554"},{"key":"poi200108r22","unstructured":"Leschly? N. In pricing our gene therapy, Bluebird weighed value, shared risk, and a lifetime cap. STAT. Published November 26, 2019. Accessed February 1, 2020. https:\/\/www.statnews.com\/2019\/11\/26\/gene-therapy-pricing-bluebird-value-shared-risk-lifetime-cap\/"},{"key":"poi200108r25","unstructured":"bluebird bio. bluebird bio Reports fourth quarter and full year 2019 financial results and highlights operational progress. Published February 18, 2020. Accessed April 1, 2020. https:\/\/investor.bluebirdbio.com\/news-releases\/news-release-details\/bluebird-bio-reports-fourth-quarter-and-full-year-2019-financial"},{"key":"poi200108r29","unstructured":"bluebird bio. bluebird bio Reports third quarter 2019 financial results and highlights operational progress. Published October 31, 2019. Accessed April 6, 2020. https:\/\/investor.bluebirdbio.com\/news-releases\/news-release-details\/bluebird-bio-reports-third-quarter-2019-financial-results-and"},{"key":"poi200108r30","unstructured":"National Institute for Health and Care Excellence. Betibeglogene autotemcel for treating transfusion-dependent beta-thalassemia [ID968]. Updated April 6, 2020. Accessed April 6, 2020. https:\/\/www.nice.org.uk\/guidance\/indevelopment\/gid-ta10334"},{"key":"poi200108r31","unstructured":"bluebird bio. bluebird bio Announces launch in Germany of Zynteglo (autologous CD34+ cells encoding \u00dfA-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent \u00df-thalassemia who do not have \u00df0\/\u00df0 genotype. Published January 13, 2020. Accessed April 6, 2020. https:\/\/investor.bluebirdbio.com\/news-releases\/news-release-details\/bluebird-bio-announces-launch-germany-zynteglotm-autologous-cd34"},{"key":"poi200108r32","unstructured":"The FoCUS Project. Precision financing solutions for durable\/potentially curative therapies. Massachusetts Institute of Technology Center for Biomedical Innovation website. Published January 24, 2019. Accessed November 10, 2020. https:\/\/newdigs.mit.edu\/sites\/default\/files\/MIT%20FoCUS%20Precision%20Financing%202019F201v023.pdf"}],"container-title":["JAMA Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jamapediatrics\/articlepdf\/2777594\/jamapediatrics_demartino_2021_oi_200108_1622130411.06068.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,6,7]],"date-time":"2021-06-07T16:18:50Z","timestamp":1623082730000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jamapediatrics\/fullarticle\/2777594"}},"subtitle":["The Medicaid Perspective"],"short-title":[],"issued":{"date-parts":[[2021,6,1]]},"references-count":33,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2021,6,1]]}},"URL":"https:\/\/doi.org\/10.1001\/jamapediatrics.2020.7140","relation":{},"ISSN":["2168-6203"],"issn-type":[{"value":"2168-6203","type":"print"}],"subject":[],"published":{"date-parts":[[2021,6,1]]}}}