The Alzheimer's Drug Discovery Foundation, NY, NY, USA.
J Nutr Health Aging. 2010 Oct;14(8):640-7. doi: 10.1007/s12603-010-0310-8.
The societal and individual costs of Alzheimer's disease are significant, worldwide. As the world ages, these costs are increasing rapidly, while health systems face finite budgets. As a result, many regulators and payers will require or at least consider phase III cost-effectiveness data (in addition to safety and efficacy data) for drug approval and reimbursement, increasing the risks and costs of drug development. Incorporating pharmacoeconomic studies in phase III clinical trials for Alzheimer's disease presents a number of challenges. We propose several specific suggestions to improve the design of pharmacoeconomic studies in phase III clinical trials. We propose that acute episodes of care are key outcome measures for pharmacoeconomic studies. To improve the possibility of detecting a pharmacoeconomic impact in phase III, we suggest several strategies including; study designs for enrichment of pharmacoeconomic outcomes that include co-morbidity of patients; reducing variability of care that can affect pharmacoeconomic outcomes through standardized care management; employing administrative claims data to better capture meaningful pharmacoeconomic data; and extending clinical trials in open label follow-up periods in which pharmacoeconomic data are captured electronically by administrative claims. Specific aspects of power analysis for pharmacoeconomic studies are presented. The particular pharmacoeconomic challenges caused by the use of biomarkers in clinical trials, the increasing use of multinational studies, and the pharmacoeconomic challenges presented by biologicals in development for Alzheimer's disease are discussed. In summary, since we are entering an era in which pharmacoeconomic studies will be essential in drug development for supporting regulatory approval, payor reimbursement and integration of new therapies into clinical care, we must consider the design and incorporation of pharmacoeconomic studies in phase III clinical trials more seriously and more creatively.
阿尔茨海默病在全球范围内给社会和个人带来了巨大的负担。随着世界人口老龄化，这些成本正在迅速增加，而卫生系统面临有限的预算。因此，许多监管机构和支付者将要求（或至少考虑）药物批准和报销的三期成本效益数据（除了安全性和疗效数据），这增加了药物开发的风险和成本。在三期临床试验中纳入药物经济学研究存在许多挑战。我们提出了一些具体建议，以改善阿尔茨海默病三期临床试验中的药物经济学研究设计。我们建议急性护理发作是药物经济学研究的关键结果指标。为了提高在三期临床试验中检测药物经济学影响的可能性，我们建议采用几种策略，包括：设计药物经济学结果丰富的研究，包括患者共病；通过标准化的护理管理减少护理的可变性，从而影响药物经济学结果；利用行政索赔数据更好地获取有意义的药物经济学数据；在开放标签随访期间延长临床试验，通过行政索赔电子方式捕获药物经济学数据。提出了药物经济学研究的具体方面的功效分析。讨论了临床试验中使用生物标志物引起的特定药物经济学挑战、越来越多地使用多国研究以及开发用于阿尔茨海默病的生物制剂带来的药物经济学挑战。总之，由于我们正进入一个药物开发需要药物经济学研究来支持监管批准、支付者报销和将新疗法纳入临床护理的时代，我们必须更认真、更创造性地考虑三期临床试验中的药物经济学研究设计和纳入。
