Coverage with Evidence Development: Application to pharmaceutical reimbursement decisions

一些新药在健康方面只有很小的改善，但成本很高。当这些药物只在少数人身上进行试验时，就会导致药物价值的不确定性，这可能会给决策者带来问题。当药物后来被发现不安全时，在存在不确定性的地方资助药物可能会造成伤害，或者当药物相对于有效性定价过高时，可能会浪费数百万美元。不资助药物可能会使药物有效的患者处于不利地位。在减少不确定性的同时使人们能够获得药品的方法将为患者、临床医生和纳税人带来重大好处。

Some new medicines produce only a small improvement in health but have considerable costs. When these medicines are trialled on only small numbers of people, uncertainty results about the value of the medicine, which can create problems for decision makers. Funding medicines where there is uncertainty may lead to harm when medicines are later found to be unsafe, or waste millions of dollars when they are overpriced relative to effectiveness. Not funding medicines may disadvantage patients in whom the medicines are effective. Methods to enable access to medicines while reducing uncertainty will offer significant benefit to patients, clinicians and taxpayers.