دورية أكاديمية
أعرض في EDS

Can you hear us now? The impact of health-care utilization by rare disease patients in the United States.

التفاصيل البيبلوغرافية
العنوان: Can you hear us now? The impact of health-care utilization by rare disease patients in the United States.
المؤلفون: Navarrete-Opazo AA; Advocate Aurora Health, Advocate Aurora Research Institute, Milwaukee, WI, USA., Singh M; Advocate Aurora Health, Advocate Aurora Research Institute, Milwaukee, WI, USA.; School of Dentistry, Marquette University, Milwaukee, WI, USA., Tisdale A; National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA., Cutillo CM; National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA., Garrison SR; Advocate Aurora Health, Advocate Aurora Research Institute, Milwaukee, WI, USA. sheldon.garrison@aah.org.
المصدر: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2021 Nov; Vol. 23 (11), pp. 2194-2201. Date of Electronic Publication: 2021 Jun 28.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Elsevier Country of Publication: United States NLM ID: 9815831 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1530-0366 (Electronic) Linking ISSN: 10983600 NLM ISO Abbreviation: Genet Med Subsets: MEDLINE
أسماء مطبوعة: Publication: 2022- : [New York] : Elsevier
Original Publication: Baltimore, MD : Lippincott, Williams & Wilkins, c1998-
مواضيع طبية MeSH: Hospitalization* , Rare Diseases*/diagnosis , Rare Diseases*/epidemiology , Rare Diseases*/genetics, Adult ; Child ; Health Care Costs ; Humans ; Length of Stay ; Patient Acceptance of Health Care ; United States
مستخلص: Purpose: The vast majority of rare diseases (RDs) are complex, disabling, and life-threatening conditions with a genetic origin. RD patients face significant health challenges and limited treatments, yet the extent of their impact within health care is not well known. One direct method to gauge the disease burden of RDs is their overall cost and utilization within health-care systems.
Methods: The 2016 Healthcare Cost and Utilization Project (HCUP) databases were used to extract health-care utilization data using International Classification of Diseases, Tenth Revision (ICD-10) codes.
Results: Of 35.6 million national hospital weighted discharges in the HCUP Nationwide Inpatient Sample, 32% corresponded to RD-associated ICD-10 codes. Total charges were nearly equal between RDs ($768 billion) compared to common conditions (CCs) ($880 billion) (p < 0.0001). These charges were a result of higher charges per discharge and longer length of stay (LOS) for RD patients compared to those with CCs (p < 0.0001). Health-care cost and utilization was similarly higher for RDs with pediatric inpatient stays, readmissions, and emergency visits.
Conclusion: Pediatric and adult discharges with RDs show substantially higher health-care utilization compared to discharges with CCs diagnoses, accounting for nearly half of the US national bill.
(© 2021. The Author(s).)
References: Stoller JK. The challenge of rare diseases. Chest. 2018;153:1309–14. (PMID: 10.1016/j.chest.2017.12.018)
Rodwell C, Ayme S. Rare disease policies to improve care for patients in Europe. Biochim Biophys Acta. 2015;1852:2329–35. (PMID: 10.1016/j.bbadis.2015.02.008)
Shah RR. Regulatory framework for the treatment of orphan diseases. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford. 2006.
Haendel M, Vasilevsky N, Unni D, Bologa C, Harris N, Rehm H, et al. How many rare diseases are there? Nat Rev Drug Discov. 2020;19:77–8. (PMID: 10.1038/d41573-019-00180-y)
Nguengang Wakap S, Lambert DM, Olry A, Rodwell C, Gueydan C, Lanneau V, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2020;28:165–73. (PMID: 10.1038/s41431-019-0508-0)
EURODIS. Rare Diseases: understanding this Public Health Priority. 2005;1-27.
Herder M. What is the purpose of the orphan drug act? PLoS Med. 2017;14:e1002191. (PMID: 10.1371/journal.pmed.1002191)
Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, et al. Rare disease terminology and definitions—a systematic global review: report of the ISPOR rare disease special interest group. Value Health. 2015;18:906–14. (PMID: 10.1016/j.jval.2015.05.008)
King S, Lehrman SN, Lehrman S. Newborn screening and early intervention for the treatment of rare diseases a Win-Win for children with rare diseases and North Carolina. 2018.
Grosse SD, Thompson JD, Ding Y, Glass M. The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience. The Milbank Q. 2016;94:366–91. (PMID: 10.1111/1468-0009.12196)
Murdock S, Lee JY, Guckes A, Wright JT. A costs analysis of dental treatment for ectodermal dysplasia. J Am Dent Assoc. 2005;136:1273–6. (PMID: 10.14219/jada.archive.2005.0343)
Friedlander L, Berdal A, Boizeau P, Licht BA, Manière MC, Picard A, et al. Oral health related quality of life of children and adolescents affected by rare orofacial diseases: a questionnaire-based cohort study. Orphanet J Rare Dis. 2019;14:124. (PMID: 10.1186/s13023-019-1109-2)
López-Bastida J, Oliva-Moreno J. Cost of illness and economic evaluation in rare diseases. Adv Exp Med Biol. 2010;686:273–82. (PMID: 10.1007/978-90-481-9485-8_16)
Chiu ATG, Chung CCY, Wong WHS, Lee SL, Chung BHY. Healthcare burden of rare diseases in Hong Kong - adopting ORPHAcodes in ICD-10 based healthcare administrative datasets. Orphanet J Rare Dis. 2018;13:147. (PMID: 10.1186/s13023-018-0892-5)
Angelis A, Tordrup D, Kanavos P. Socio-economic burden of rare diseases: A systematic review of cost of illness evidence. Health Policy. 2015;119:964–79. (PMID: 10.1016/j.healthpol.2014.12.016)
Michaels-Igbokwe C, McInnes B, MacDonald KV, Currie GR, Omar F, Shewchuk B, et al. (Un)standardized testing: the diagnostic odyssey of children with rare genetic disorders in Alberta, Canada. Genet Med. 2021;23:272–9. (PMID: 10.1038/s41436-020-00975-0)
Zurynski Y, Deverell M, Dalkeith T, Johnson S, Christodoulou J, Leonard H, et al. Australian children living with rare diseases: experiences of diagnosis and perceived consequences of diagnostic delays. Orphanet J Rare Dis. 2017;12:68. (PMID: 10.1186/s13023-017-0622-4)
Gonzaludo N, Belmont JW, Gainullin VG, Taft RJ. Estimating the burden and economic impact of pediatric genetic disease. Genet Med. 2019;21:1781–9. (PMID: 10.1038/s41436-018-0398-5)
http://www.orphadata.org/cgi-bin/index.php (2018). Accessed September 15, 2018.
Orphanet. Free datasets powered by Orphanet. In:2019.
https://omim.org (2021). Accessed March 26, 2021.
HCUP National Inpatient Sample (NIS). In: Rockville, MD, editor. Quality AfHRa. Agency for Healthcare Research and Quality. 2016.
HCUP Kids’ Inpatient Database (KID). In: Rockville, MD, editor. Quality AfHRa. Agency for Healthcare Research and Quality. 2016.
Barrett ML, Bailey MB. https://www.hcup-us.ahrq.gov/db/nation/nis/NISChangesforDataYear2016.pdf (2016). Accessed September 18, 2020.
Yoon F, Sheng M, Jiang HJ, Steiner CA, Barrett ML Calculating nationwide readmissions database (nrd) variances. US Agency for Healthcare Research and Quality. 2017.
HCUP Nationwide Emergency Department Sample (NEDS). In: Rockville, MD, editor. Quality AfHRa. Agency for Healthcare Research and Quality. 2016.
Robinson SW, Brantley K, Liow C, Teagarden JR. An early examination of access to select orphan drugs treating rare diseases in health insurance exchange plans. J Manag Care Spec Pharm. 2014;20:997–1004. (PMID: 25278322)
Handfield R, Feldstein J. Insurance companies’ perspectives on the orphan drug pipeline. Am Health Drug Benefits. 2013;6:589–98. (PMID: 249913854046481)
Divino V, DeKoven M, Kleinrock M, Wade RL, Kaura S. Orphan drug expenditures in the United States: a historical and prospective analysis, 2007-18. Health Aff (Millwood). 2016;35:1588–94. (PMID: 10.1377/hlthaff.2016.0030)
Molster C, Urwin D, Di Pietro L, Fookes M, Petrie D, van der Laan S, et al. Survey of healthcare experiences of Australian adults living with rare diseases. Orphanet J Rare Dis. 2016;11:30. (PMID: 10.1186/s13023-016-0409-z)
Baynam G, Pachter N, McKenzie F, Townshend S, Slee J, Kiraly-Borri C, et al. The rare and undiagnosed diseases diagnostic service - application of massively parallel sequencing in a state-wide clinical service. Orphanet J Rare Dis. 2016;11:77. (PMID: 10.1186/s13023-016-0462-7)
Gahl WA, Mulvihill JJ, Toro C, Markello TC, Wise AL, Ramoni RB, et al. The NIH Undiagnosed Diseases Program and Network: Applications to modern medicine. Mol Genet Metab. 2016;117:393–400. (PMID: 10.1016/j.ymgme.2016.01.007)
Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe’s disease.New Engl J Med. 2005;352:2069–81. (PMID: 10.1056/NEJMoa042604)
Allewelt H, Taskindoust M, Troy J, Page K, Wood S, Parikh S, et al. Long-term functional outcomes after hematopoietic stem cell transplant for early infantile Krabbe disease. Biol Blood Marrow Transplant. 2018;24:2233–8. (PMID: 10.1016/j.bbmt.2018.06.020)
تواريخ الأحداث: Date Created: 20210629 Date Completed: 20211111 Latest Revision: 20220210
رمز التحديث: 20221213
مُعرف محوري في PubMed: PMC8553605
DOI: 10.1038/s41436-021-01241-7
PMID: 34183788
قاعدة البيانات: MEDLINE
الوصف
تدمد:1530-0366
DOI:10.1038/s41436-021-01241-7