Meers S, Selleslag D, Potier H, Glasmacher A, Mineur P, Voelter V
AZ Klina , Brasschaat , Belgium.
Curr Med Res Opin. 2015 Jan;31(1):35-42. doi: 10.1185/03007995.2014.972499. Epub 2014 Oct 17.
Azacitidine (Vidaza *) is approved in Europe for treatment of myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) with 20-30% bone marrow (BM) blasts, and chronic myelomonocytic leukemia (CMML) with 10-29% BM blasts and no myeloproliferative syndrome (i.e. <13.000/μL white blood cells). In Belgium, the azacitidine reimbursement process can take several months, and is often delayed at submission for medical assessment by the Belgian National Institute for Health and Disability Insurance of incomplete patient dossiers, due to disease complexity and classification, and administrative burden. We describe the Vidaza Access Program and its application to an initial 175 patients. Individual medical dossiers were reviewed for completeness to facilitate patient access to treatment in Belgium.
A standardized anonymized patient information form is completed by the physician and sent for review to the Belgian Celgene Medical Department. The form is reviewed within three working days and, for complete dossiers, Celgene grants a financial guarantee for treatment with azacitidine. The patient can then be treated without the hospital being subjected to financial risk.
Between January 2013 and June 2014, 63 physicians (53 Belgian hospitals) recruited 175 patients. In total, 163 patient dossiers were approved by Celgene (120 MDS, 36 AML, and 7 CMML), of which 104 dossiers were also approved by the review committee and 49 have been waiting for a final decision for a median of 6 months; no information is currently available for the remaining 10. No dossiers approved by Celgene have been rejected by the review committee.
The Celgene Vidaza Access Program offers support to healthcare professionals in the appropriate use of azacitidine. By facilitating the assessment of patient dossiers and providing a financial guarantee for prescribers and hospitals, treatment can be initiated more rapidly and patients may better benefit from azacitidine treatment.
阿扎胞苷（维达莎*）在欧洲被批准用于治疗骨髓增生异常综合征（MDS）、骨髓原始细胞占20%-30%的急性髓系白血病（AML）以及骨髓原始细胞占10%-29%且无骨髓增殖综合征（即白细胞<13,000/μL）的慢性粒-单核细胞白血病（CMML）。在比利时，阿扎胞苷的报销流程可能需要数月时间，且由于疾病的复杂性、分类以及行政负担，患者档案不完整时，向比利时国家健康与残疾保险研究所提交进行医学评估时常常会延迟。我们描述了维达莎准入计划及其在最初175例患者中的应用情况。对个体医学档案进行完整性审查，以促进比利时患者获得治疗。
医生填写标准化匿名患者信息表，并将其送交比利时新基医药部门进行审查。该表格在三个工作日内进行审查，对于完整的档案，新基为阿扎胞苷治疗提供财务担保。然后患者可以接受治疗，而医院无需承担财务风险。
2013年1月至2014年6月期间，63名医生（来自53家比利时医院）招募了175例患者。新基总共批准了163份患者档案（120例MDS、36例AML和7例CMML），其中104份档案也得到了审查委员会的批准，49份档案正在等待最终决定，中位等待时间为6个月；其余10份档案目前暂无信息。新基批准的档案没有被审查委员会拒绝。
新基维达莎准入计划为医疗保健专业人员合理使用阿扎胞苷提供支持。通过促进患者档案评估并为开处方者和医院提供财务担保，可以更快地开始治疗，患者可能会更好地从阿扎胞苷治疗中获益。
