Christiana Care Health System, Wilmington, DE 19718, USA.
Value Health. 2013 Jul-Aug;16(5):901-6. doi: 10.1016/j.jval.2013.03.1623. Epub 2013 May 15.
Hospitals, physicians, payers, and patients face economic and ethical decisions about the use of biotechnology drugs, commonly called specialty medications. These often target a small population, have data based on smaller clinical trials, are expensive, and may have questionable advantage. This is a result of how the Food and Drug Administration (FDA) approves medications, which is based only on safety and efficacy. Cancer drugs, once approved by the FDA, regardless of cost or value must be covered by Medicare. Some states have laws requiring additional coverage as well. All of this has created an unintended consequence: It has driven up costs with questionable evidence to support the medication's value, placing patients, payers, and providers in an ethical conflict. In this new era of health care transformation, health care leaders must focus on creating value to support a sustainable health system. Christiana Care Health System's Value Institute has designed a new model to evaluate specialty medications, using value as its main criterion.
This article describes the process and outcomes using a new value model for evaluating specialty medications for a hospital formulary. It also introduces a new criterion of evaluation entitled "Societal Benefit" that provides a rating on quality- of-life issues. With measurable factors of efficacy, risk, cost, and quality-of-life concerns, our methodology provides a more balanced approach in the evaluation of specialty medications.
Specialty medications are the fastest growing segment of drug expense, and it is hard to understand how these medications will be sustainable under health care reforms. Unlike other countries, the United States has no national agency providing cost-effectiveness review; review occurs, if at all, at a local level. Laws governing Medicare and most private insurers' coverage of FDA-approved medication and some clinical quality standards conflict with cost-effectiveness, making this type of review difficult. Finally, because these medications affect the health system as a whole, it is a great example to begin to support health care reform.
Hospitals need to challenge the value of specialty medication. Although our model will continue to evolve, value is now our central consideration when selecting specialty medications to be added to the formulary. We share this experience to encourage other hospitals to design their own approach to this vital issue.
医院、医生、支付方和患者在生物技术药物（通常称为专科药物）的使用方面面临着经济和伦理决策。这些药物通常针对一小部分人群，基于较小的临床试验数据，价格昂贵，并且可能具有可疑的优势。这是食品和药物管理局 (FDA) 批准药物的方式的结果，该方式仅基于安全性和疗效。一旦 FDA 批准了癌症药物，无论成本或价值如何，都必须由医疗保险覆盖。一些州也有法律要求额外覆盖。所有这些都造成了一个意想不到的后果：它推高了成本，而对药物价值的支持证据却值得怀疑，使患者、支付方和提供者陷入了伦理冲突之中。在医疗保健转型的新时代，医疗保健领导者必须专注于创造价值，以支持可持续的医疗体系。Christiana Care Health System 的价值研究所设计了一种新的模型来评估专科药物，以价值作为主要标准。
本文描述了使用评估医院处方药物的新价值模型的过程和结果。它还引入了一个新的评估标准，称为“社会效益”，它提供了生活质量问题的评级。通过可衡量的疗效、风险、成本和生活质量因素，我们的方法为专科药物的评估提供了更平衡的方法。
专科药物是药物支出增长最快的部分，很难理解在医疗改革下这些药物将如何持续。与其他国家不同，美国没有提供成本效益评估的国家机构；如果有的话，评估也是在地方一级进行的。管理医疗保险和大多数私人保险公司对 FDA 批准药物的覆盖范围以及一些临床质量标准的法律与成本效益相冲突，使得此类审查变得困难。最后，由于这些药物影响整个医疗体系，因此这是一个很好的例子，可以开始支持医疗改革。
医院需要对专科药物的价值提出质疑。尽管我们的模型将继续发展，但在选择要添加到处方中的专科药物时，价值现在是我们的主要考虑因素。我们分享这一经验，以鼓励其他医院针对这一重要问题设计自己的方法。
