Brosgart C L, Mitchell T, Charlebois E, Coleman R, Mehalko S, Young J, Abrams D I
East Bay AIDS Center, Alta Bates Medical Center, Berkeley, CA, USA.
J Acquir Immune Defic Syndr Hum Retrovirol. 1996 May 1;12(1):56-62. doi: 10.1097/00042560-199605010-00008.
We wished to determine the extent to which drugs used to treat HIV disease and its clinical manifestations are prescribed for conditions other than those listed on the U.S. Food and Drug Administration's approved drug label, how such "off-label" use varies by patient characteristics and type of HIV-related medical condition, and the extent to which physicians alter the way they treat HIV-related conditions because of reimbursement problems associated with off-label drug use. We surveyed 1,530 primary care providers for people with HIV disease between February and May 1993. A three-part survey instrument was used to obtain data on the drugs prescribed for the last three patients with HIV disease treated by the provider, the preferred choice of therapy for 32 specific HIV-related conditions, and the extent to which providers faced reimbursement problems regarding the use of drugs for off-label indications. Three drug compendia were used as cited sources of off-label drug uses. In all, 387 (32%) evaluable surveys were returned, yielding data on 1,148 patients. The majority (81%) of patients received at least one drug off-label, and almost half (40%) of all reported drug therapy was off-label. Most off-label drug use was for treatment and prevention of HIV-related opportunistic infections, which frequently represented the community standard of practice (e.g., trimethoprim/sulfamethoxazole for prevention of Pneumocystis carinii pneumonia), or the de facto standard of practice when no licensed therapies were available (e.g., drugs for treatment of Mycobacterium avium complex, MAC). More than 75% of off-label usage was cited in at least one of the three authoritative medical compendia. The use of drugs for off-label indications in HIV care is common and frequently represents community standards of care. Reliance on drug compendia for support of off-label drug use accounts for the majority of such uses, although many legitimate off-label uses may not be included because of compendia publication lag. The prevalence of off-label drug use in routine clinical practice and the development of newer and more costly drugs for treatment of HIV and its medical complications argues for the articulation of an explicit national reimbursement policy for off-label uses of prescription drugs so that medically appropriate therapies will be available to those with insurance in a rational, consistent way.
我们希望确定用于治疗HIV疾病及其临床表现的药物，在用于美国食品药品监督管理局批准的药品标签所列病症以外的其他病症时的使用程度，这种“标签外”使用因患者特征和HIV相关医疗状况类型的不同而有何差异，以及医生因标签外用药相关的报销问题而改变其治疗HIV相关病症方式的程度。我们在1993年2月至5月期间对1530名HIV疾病患者的初级保健提供者进行了调查。使用一份由三部分组成的调查问卷来获取关于提供者治疗的最后三名HIV疾病患者所开药物的数据、针对32种特定HIV相关病症的首选治疗方案，以及提供者在使用标签外适应症药物方面面临报销问题的程度。三份药物手册被用作标签外用药的引用来源。总共回收了387份（32%）可评估的调查问卷，得到了1148名患者的数据。大多数（81%）患者至少接受了一种标签外用药，所有报告的药物治疗中近一半（40%）为标签外用药。大多数标签外用药用于治疗和预防HIV相关的机会性感染，这常常代表了社区的治疗标准（例如，甲氧苄啶/磺胺甲恶唑用于预防卡氏肺孢子虫肺炎），或者在没有许可疗法可用时的实际治疗标准（例如，用于治疗鸟分枝杆菌复合体感染的药物）。超过75%的标签外用药在至少一份权威医学手册中被引用。在HIV治疗中使用标签外适应症药物很常见，且常常代表了社区护理标准。尽管由于手册出版滞后，许多合理的标签外用药可能未被收录，但依靠药物手册支持标签外用药占了此类用药的大部分。在常规临床实践中标签外用药的普遍情况以及用于治疗HIV及其并发症的更新、更昂贵药物的研发，都表明需要明确制定一项关于处方药标签外使用的国家报销政策，以便使有保险的人能够以合理、一致的方式获得医学上合适的治疗。
