{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,22]],"date-time":"2026-06-22T13:44:48Z","timestamp":1782135888384,"version":"3.54.5"},"reference-count":26,"publisher":"American Medical Association (AMA)","issue":"1","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Netw Open"],"abstract":"<jats:sec id=\"ab-zoi231558-4\"><jats:title>Importance<\/jats:title><jats:p>The US Food and Drug Administration approved eteplirsen for Duchenne muscular dystrophy (DMD) in 2016 based on a controversial pivotal study that demonstrated a limited effect on the surrogate measure of dystrophin production. Other DMD treatments in the same class followed.<\/jats:p><\/jats:sec><jats:sec id=\"ab-zoi231558-5\"><jats:title>Objective<\/jats:title><jats:p>To assess how patients receiving novel DMD treatments in postapproval clinical settings compare with patients in the clinical trials.<\/jats:p><\/jats:sec><jats:sec id=\"ab-zoi231558-6\"><jats:title>Design, Setting, and Participants<\/jats:title><jats:p>This cross-sectional study collected data on patients who initiated 1 of 4 novel DMD treatments (eteplirsen, golodirsen, viltolarsen, and casimersen) using national claims databases of commercially insured (Merative MarketScan and Optum\u2019s Clinformatics Data Mart Database [CDM]) and Medicaid patients between September 19, 2016, and March 31, 2022. Patients were followed for 1 year after the date of first use of any novel DMD treatment. In addition, patients in pivotal DMD drug trials were identified for comparison.<\/jats:p><\/jats:sec><jats:sec id=\"ab-zoi231558-7\"><jats:title>Exposures<\/jats:title><jats:p>Age, sex, race and ethnicity, region, and DMD stage of patients receiving novel DMD treatment.<\/jats:p><\/jats:sec><jats:sec id=\"ab-zoi231558-8\"><jats:title>Main Outcome and Measures<\/jats:title><jats:p>The main outcome was health care costs and drug discontinuation as measured using descriptive statistics.<\/jats:p><\/jats:sec><jats:sec id=\"ab-zoi231558-9\"><jats:title>Results<\/jats:title><jats:p>A total of 223 routine care patients initiating novel DMD drugs (58 in MarketScan, 35 in CDM, and 130 in Medicaid) were identified. Among the 106 patients in the pivotal trials, the mean (SD) age was 8.5 (2.0) years (range, 4.0-13.0 years), which was younger than the mean age of patients in routine care (MarketScan: 13.7 [7.0] years [range, 1.8-33.3 years; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001]; CDM: 11.9 [5.7] years [range, 0.6-23.6 years; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001]; Medicaid: 13.4 [6.5] years [range, 1.8-46.1 years; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001]). The proportion of female patients identified in postapproval clinical settings was 2.9% (n\u2009=\u20091) in CDM (vs 34 male patients [97.1%]) and 1.5% (n\u2009=\u20092) in Medicaid (vs 128 male patients [98.5%]), which was not different from the pivotal trials. While nearly all patients in the pivotal trials had DMD disease stage 1 or 2 when initiating the DMD treatments (103 [97.2%]), in the postapproval clinical setting, slightly more than one-third of patients were in disease stage 3 or 4 (MarketScan, 17 [36.2%; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001]; CDM, 13 [41.9%; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001]; Medicaid, 54 [47.0%; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001]). The payer\u2019s cost for novel DMD treatments varied across the databases, with a mean (SD) of $634\u202f764 ($607\u202f101) in MarketScan, $482\u202f749 ($582\u202f350) in CDM, and $384\u202f023 ($1\u202f165\u202f730) in Medicaid. Approximately one-third of routine care patients discontinued the treatments after approximately 7 months (mean [SD], 6.1 [4.4], 6.9 [3.9], and 7.2 [4.3] months in MarketScan, CDM, and Medicaid, respectively).<\/jats:p><\/jats:sec><jats:sec id=\"ab-zoi231558-10\"><jats:title>Conclusions and Relevance<\/jats:title><jats:p>These findings raise questions about the translation of DMD drug trial findings to routine care settings, with patients in routine care discontinuing the treatment within 1 year and payers incurring substantial expenses for these medications. More data are needed on whether these high costs are accompanied by corresponding clinical benefits.<\/jats:p><\/jats:sec>","DOI":"10.1001\/jamanetworkopen.2023.53094","type":"journal-article","created":{"date-parts":[[2024,1,24]],"date-time":"2024-01-24T16:01:23Z","timestamp":1706112083000},"page":"e2353094","source":"Crossref","is-referenced-by-count":14,"title":["Characteristics of Patients Receiving Novel Muscular Dystrophy Drugs in Trials vs Routine Care"],"prefix":"10.1001","volume":"7","author":[{"given":"Dongzhe","family":"Hong","sequence":"first","affiliation":[{"name":"Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"},{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerry","family":"Avorn","sequence":"additional","affiliation":[{"name":"Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"},{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Wyss","sequence":"additional","affiliation":[{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron S.","family":"Kesselheim","sequence":"additional","affiliation":[{"name":"Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"},{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women\u2019s Hospital\/Harvard Medical School, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","published-online":{"date-parts":[[2024,1,24]]},"reference":[{"issue":"9869","key":"zoi231558r1","doi-asserted-by":"publisher","first-page":"845","DOI":"10.1016\/S0140-6736(12)61897-2","article-title":"Muscular dystrophies.","volume":"381","author":"Mercuri","year":"2013","journal-title":"Lancet"},{"issue":"2","key":"zoi231558r2","doi-asserted-by":"publisher","first-page":"177","DOI":"10.1016\/S1474-4422(09)70272-8","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care.","volume":"9","author":"Bushby","year":"2010","journal-title":"Lancet Neurol"},{"issue":"1","key":"zoi231558r3","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1016\/S1474-4422(09)70271-6","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.","volume":"9","author":"Bushby","year":"2010","journal-title":"Lancet Neurol"},{"issue":"2","key":"zoi231558r4","doi-asserted-by":"publisher","first-page":"257","DOI":"10.1002\/ana.v79.2","article-title":"Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.","volume":"79","author":"Mendell","year":"2016","journal-title":"Ann Neurol"},{"issue":"1","key":"zoi231558r6","doi-asserted-by":"publisher","first-page":"162","DOI":"10.1002\/ana.v81.1","article-title":"Regarding \u201ceteplirsen for the treatment of Duchenne muscular dystrophy.\u201d","volume":"81","author":"Unger","year":"2017","journal-title":"Ann Neurol"},{"issue":"22","key":"zoi231558r7","doi-asserted-by":"publisher","first-page":"2357","DOI":"10.1001\/jama.2016.16437","article-title":"Approving a problematic muscular dystrophy drug: implications for FDA policy.","volume":"316","author":"Kesselheim","year":"2016","journal-title":"JAMA"},{"issue":"9","key":"zoi231558r9","doi-asserted-by":"publisher","first-page":"1251","DOI":"10.7326\/M23-1073","article-title":"The regulatory repercussions of approving muscular dystrophy medications on the basis of limited evidence.","volume":"176","author":"Bendicksen","year":"2023","journal-title":"Ann Intern Med"},{"issue":"10","key":"zoi231558r13","doi-asserted-by":"publisher","first-page":"1426","DOI":"10.18553\/jmcp.2021.27.10.1426","article-title":"Characterizing demographics, comorbidities, and costs of care among populations with Duchenne muscular dystrophy with Medicaid and commercial coverage.","volume":"27","author":"Klimchak","year":"2021","journal-title":"J Manag Care Spec Pharm"},{"issue":"6","key":"zoi231558r14","doi-asserted-by":"publisher","first-page":"2906","DOI":"10.1007\/s12325-022-02117-1","article-title":"Disease progression stages and burden in patients with Duchenne muscular dystrophy using administrative claims supplemented by electronic medical records.","volume":"39","author":"Iff","year":"2022","journal-title":"Adv Ther"},{"key":"zoi231558r20","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.jocn.2017.10.082","article-title":"Eteplirsen for paediatric patients with Duchenne muscular dystrophy: a pooled-analysis.","volume":"49","author":"Randeree","year":"2018","journal-title":"J Clin Neurosci"},{"issue":"6","key":"zoi231558r21","doi-asserted-by":"publisher","first-page":"989","DOI":"10.3233\/JND-210643","article-title":"Open-label evaluation of eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: PROMOVI trial.","volume":"8","author":"McDonald","year":"2021","journal-title":"J Neuromuscul Dis"},{"issue":"2","key":"zoi231558r22","doi-asserted-by":"publisher","first-page":"167","DOI":"10.1080\/13543784.2021.1868434","article-title":"Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies.","volume":"30","author":"Sheikh","year":"2021","journal-title":"Expert Opin Investig Drugs"},{"issue":"4","key":"zoi231558r23","doi-asserted-by":"publisher","first-page":"462","DOI":"10.1002\/mus.v66.4","article-title":"Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: a systematic review and meta-analysis.","volume":"66","author":"Weber","year":"2022","journal-title":"Muscle Nerve"},{"issue":"12","key":"zoi231558r24","doi-asserted-by":"publisher","first-page":"1229","DOI":"10.2165\/00019053-200523120-00007","article-title":"Estimating mean total costs in the presence of censoring: a comparative assessment of methods.","volume":"23","author":"Young","year":"2005","journal-title":"Pharmacoeconomics"},{"issue":"7","key":"zoi231558r26","doi-asserted-by":"publisher","DOI":"10.1001\/jamahealthforum.2022.1812","article-title":"Cautions when using race and ethnicity in administrative claims data sets.","volume":"3","author":"Nead","year":"2022","journal-title":"JAMA Health Forum"},{"key":"zoi231558r8","first-page":"115","volume-title":"Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value-Evidence Report","author":"Lin","year":"2019"},{"key":"zoi231558r5","unstructured":"Exondys 51 (eteplirsen) approval letter. US Food and Drug Administration. Accessed December 19, 2023. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/appletter\/2016\/206488Orig1s000ltr.pdf"},{"key":"zoi231558r10","unstructured":"Vyondys 53 (golodirsen) approval letter. US Food and Drug Administration. Accessed December 19, 2023. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/appletter\/2019\/211970Orig1s000ltr.pdf"},{"key":"zoi231558r11","unstructured":"Viltepso (viltolarsen) approval letter. US Food and Drug Administration. Accessed December 19, 2023. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/appletter\/2020\/212154Orig1s000ltr.pdf"},{"key":"zoi231558r12","unstructured":"Amondys 45 (casimersen) approval letter. US Food and Drug Administration. Accessed December 19, 2023. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/appletter\/2021\/213026Orig1s000ltr.pdf"},{"key":"zoi231558r15","unstructured":"Exondys 51 (eteplirsen) medical review. US Food and Drug Administration. 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