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Oncology orphan drugs: An evidence assessment, network meta-analysis, and cost-effectiveness analysis.

机译：肿瘤学孤儿药：证据评估，网络荟萃分析和成本效益分析。

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摘要

Background;Approximately 1/3 of recently approved drugs in the United States (U.S.) were indicated for rare diseases (orphan drugs). Oncology indications comprised the largest sub-category. Overall, orphan drugs have not been well-studied and there is greater uncertainty about their value when compared to drugs for common diseases.;Objective;To critically assess the comparative effectiveness data supporting oncology orphan drugs marketed in the U.S. and to evaluate the clinical and economic value of therapies indicated for chronic lymphocytic leukemia (CLL).;Methods;I performed a systematic literature review to identify the level of evidence supporting 47 oncology orphan drugs marketed in the U.S. and applied the Grading of Recommendations Assessment, Development and Evaluation (GRADE) framework and Quality of Health Economic Studies (QHES) instrument to review the quality of evidence. I conducted a network meta-analysis using a Bayesian statistical approach to estimate the relative treatment effect of therapies for treatment-naive CLL. Lastly, I conducted a cost-effectiveness analysis of salvage therapies for double-refractory CLL. The analysis was performed from a U.S. healthcare payer perspective and used a decision analytic model to estimate life expectancy, quality-adjusted life years (QALYs), and costs over a lifetime horizon.;Results;Results from the systematic literature review and evidence assessment showed that oncology orphan drugs marketed in the U.S. have varying levels and quality of clinical evidence and a paucity of evidence demonstrating their economic value. Network meta-analysis method is useful for synthesizing available evidence and estimating relative treatment effects of competing therapies in the absence of head-to-head trials. However, because the method is based on model simulation, clinical validation of results is necessary. Based on limited clinical evidence and lack of explicit reimbursement policies for orphan drugs, it is uncertain if ofatumumab would be considered cost-effective for treatment of double-refractory CLL.;Conclusion;Current levels of evidence for oncology orphan drugs may not be sufficient to support decision-making practices consistent with evidence-based medicine. Innovative analytic and policy approaches are needed in order to implement a value assessment framework for orphan drugs that is consistent with principles of comparative effectiveness research.
机译：背景；在美国（美国），大约有1/3的近期批准的药物被指定用于罕见疾病（孤儿药）。肿瘤学适应症包括最大的子类别。总体而言，对孤儿药物的研究尚未深入，与普通疾病药物相比，其价值存在更大的不确定性。目的；严格评估支持美国市场上肿瘤孤儿药物的相对有效性数据，并评估其临床和临床应用价值。方法；方法；我进行了系统的文献综述，以鉴定支持在美国销售的47种肿瘤学孤儿药物的证据水平，并应用了推荐评估，开发和评估的等级（GRADE） ）框架和卫生经济学研究质量（QHES）工具来审查证据质量。我使用贝叶斯统计方法进行了网络荟萃分析，以评估单纯治疗CLL的相对治疗效果。最后，我对双难治性CLL的挽救疗法进行了成本效益分析。该分析是从美国医疗保健付款人的角度进行的，并使用决策分析模型来估计预期寿命，质量调整生命年（QALYs）和整个生命周期的成本。；结果；系统文献回顾和证据评估的结果表明在美国销售的肿瘤学孤儿药物的临床证据的水平和质量各不相同，并且缺乏证据表明其经济价值。网络荟萃分析方法可用于综合现有证据，并在没有进行头对头试验的情况下估算竞争疗法的相对治疗效果。但是，由于该方法基于模型仿真，因此需要对结果进行临床验证。基于有限的临床证据和缺乏明确的孤儿药补偿政策，尚不确定ofatumumab是否被认为是治疗双重难治性CLL的经济有效方法。结论：目前关于肿瘤学孤儿药的证据水平可能不足以应对支持与循证医学一致的决策实践。为了实现与比较有效性研究原则相一致的孤儿药物价值评估框架，需要创新的分析和政策方法。

著录项

  • 作者

    Cheng, Mindy M.;

  • 作者单位

    University of Washington.;

  • 授予单位 University of Washington.;
  • 学科 Health Sciences Pharmacy.;Health Sciences Oncology.
  • 学位 Ph.D.
  • 年度 2012
  • 页码 112 p.
  • 总页数 112
  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类
  • 关键词

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