The Direct Cost of Managing a Rare Disease: Assessing Medical and Pharmacy Costs Associated with Duchenne Muscular Dystrophy in the United States

Journal of Managed Care & Specialty Pharmacy
Sarah ThayerCraig M McDonald

Abstract

A Duchenne muscular dystrophy (DMD) cohort was identified using a claims-based algorithm to estimate health care utilization and costs for commercially insured DMD patients in the United States. Previous analyses have used broad diagnosis codes that include a range of muscular dystrophy types as a proxy to estimate the burden of DMD. To estimate DMD-associated resource utilization and costs in a sample of patients identified via a claims-based algorithm using diagnosis codes, pharmacy prescriptions, and procedure codes unique to DMD management based on DMD clinical milestones. DMD patients were selected from a commercially insured claims database (2000-2009). Patients with claims suggestive of a non-DMD diagnosis or who were aged 30 years or older were excluded. Each DMD patient was matched by age, gender, and region to controls without DMD in a 1:10 ratio (DMD patients n = 75; controls n = 750). All-cause health care resource utilization, including emergency department, inpatient, outpatient, and physician office visits, and all-cause health care costs were examined over a minimum 1-year period. Costs were computed as total health-plan and patient-paid amounts of adjudicated medical claims (in annualized U.S. dollars). The ave...Continue Reading

References

Jan 1, 1991·Neuromuscular Disorders : NMD·A E Emery
May 27, 1999·Journal of Health Economics·D K BloughM C Hornbrook
Mar 7, 2002·Lancet·Alan E H Emery
Sep 13, 2005·Current Opinion in Neurology·Kate Bushby, Volker Straub
Aug 28, 2007·American Heart Journal·Denis DubocHenri-Marc Bécane
Feb 7, 2008·The Cochrane Database of Systematic Reviews·A Y ManzurA Swan
Aug 21, 2008·Journal of Neurology, Neurosurgery, and Psychiatry·M KohlerK E Bloch
Dec 1, 2009·Lancet Neurology·Katharine BushbyUNKNOWN DMD Care Considerations Working Group
Dec 1, 2009·Lancet Neurology·Katharine BushbyUNKNOWN DMD Care Considerations Working Group
Mar 28, 2012·Annals of Neurology·Jerry R MendellRobert B Weiss
May 15, 2012·Muscle & Nerve·Craig M McDonaldGregory T Carter
Jan 29, 2013·Journal of the American College of Cardiology·Gernot SchramPaul Khairy
Jul 10, 2013·Muscle & Nerve·Jane LarkindaleValerie A Cwik
Jul 22, 2014·Muscle & Nerve·Katharine BushbyUNKNOWN PTC124-GD-007-DMD STUDY GROUP
Aug 28, 2015·Neurology·Luca BelloUNKNOWN CINRG Investigators
Nov 18, 2015·Annals of Neurology·Jerry R MendellUNKNOWN Eteplirsen Study Group and Telethon Foundation DMD Italian Network

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Citations

May 19, 2018·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·Alina M AllenRozalina G McCoy

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