Clinical and economic challenges facing pharmacogenomics
Clinical and economic challenges facing pharmacogenomics
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DOI：
10.1038/tpj.2011.63
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发表时间：
2013-08-01
影响因子：
2.8
通讯作者：
Manzolillo, K.
中科院分区：
文献类型：
--
作者：
Cohen, J.;Wilson, A.;Manzolillo, K.
在本文中，我们研究了个性化药物和伴随诊断的开发者和支付者所面临的临床和经济挑战。我们回顾并总结了八种备受瞩目的个性化药物及其伴随诊断的临床、监管和报销问题。随后，我们确定医疗保险部分B和D报销的八种药物从公开的数据库。最后，我们利用调查-每个定制的三个关键利益相关者;付款人，药物和诊断开发人员，和药物基因组学专家分析师-评估报销的诊断，分析的作用，不同种类的证据在通知处方和报销决策，以及具体的临床，监管和经济的挑战，面临药物基因组学，因为它向前发展。我们发现，医疗保险受益人获得医生管理（医疗保险B部分）的药物是相对不受约束的，与固定的病人共同保险的比例为20%。对自我管理的药物（医疗保险D部分）实行更多的报销限制，这意味着更高和更可变的费用分摊，更多地使用事先授权和数量限制。对伴随诊断缺乏全面的报销，即使是在标签上有诊断并由食品和药物管理局推荐或要求的情况下。由于缺乏将诊断测试与健康结果联系起来的证据，付款人对测试的临床实用性持怀疑态度。专家分析师预测，伴随诊断的事后开发将适度增长，以个性化已经批准的药物，而伴随诊断和个性化药物的同时共同开发将有限增长。缺乏临床上有用的诊断以及在药物和诊断临床有效性知识方面的证据缺口似乎阻碍了个性化医疗的发展。增加比较有效性研究可能有助于缩小证据差距。
In this paper, we examine the clinical and economic challenges that face developers of and payers for personalized drugs and companion diagnostics. We review and summarize clinical, regulatory and reimbursement issues with respect to eight, high profile personalized medicines and their companion diagnostics. Subsequently, we determine Medicare parts B and D reimbursement of the eight drugs from publicly available databases. Finally, we utilize surveys-each tailored to three key stakeholders; payers, drug and diagnostic developers, and pharmacogenomic expert analysts-to assess reimbursement of diagnostics, analyze the role that different kinds of evidence have in informing prescribing and reimbursement decisions, as well as the specific clinical, regulatory and economic challenges that confront pharmacogenomics as it moves forward. We found that Medicare beneficiary access to physician-administered (Medicare part B) drugs is relatively unfettered, with a fixed patient co-insurance percentage of 20%. More reimbursement restrictions are placed on self-administered (Medicare part D) drugs, which translates into higher and more variable cost sharing, more use of prior authorization and quantity limits. There is a lack of comprehensive reimbursement of companion diagnostics, even in cases in which the diagnostic is on the label and recommended or required by the Food and Drug Administration. Lack of evidence linking diagnostic tests to health outcomes has caused payers to be skeptical about the clinical usefulness of tests. Expert analysts foresee moderate growth in post-hoc development of companion diagnostics to personalize already approved drugs, and limited growth in the concurrent co-development of companion diagnostics and personalized medicines. Lack of clinically useful diagnostics as well as an evidence gap in terms of knowledge of drug and diagnostic clinical effectiveness appear to be hindering growth in personalized medicine. An increase in comparative effectiveness research may help to close the evidence gap.