For review and approval of new drug products, substantial evidence regarding safety and effectiveness of the drug products under investigation are necessarily provided. A traditional approach is to test a null hypothesis of ineffectiveness against an alternative hypothesis of effectiveness at the 5% level of significance. The rejection of the null hypothesis of ineffectiveness is in favor of the alternative hypothesis of effectiveness. This approach, however, is somewhat misleading because the rejection of the null hypothesis of ineffectiveness leads to the conclusion of not ineffectiveness, which consists of the proportion of inconclusiveness and the proportion of effectiveness. In this article, we explore the potential use of the concept of demonstrating not ineffectiveness and then effectiveness for regulatory approval of new drug products, especially for rare disease drug products. For rare disease drug product development, one of the major obstacles and challenges is how to use small patient population available for achieving the same standards for regulatory approval. To address this problem, a two-stage approach by first demonstrating not ineffectiveness and then ruling out (or controlling) the probability of inconclusiveness for demonstrating effectiveness is proposed. The proposed two-stage approach is useful with small patient population available for achieving the same standards for regulatory approval of rare disease drug products.

为了审查和批准新药品，必须提供有关所调查药品的安全性和有效性的大量证据。传统方法是在5％的显着性水平上，对无效的无效假设与有效性的另一假设进行检验。对无效无效假设的否定是对有效性的另一种假设的支持。但是，这种方法在某种程度上具有误导性，因为对无效无效假设的拒绝会导致得出非无效结论，该结论包括不确定性部分和有效性部分。在本文中，我们探讨了演示概念的潜在用途不是无效的，然后是新药品的监管批准的有效性，特别是对于罕见病药品。对于稀有疾病药物产品的开发，主要的障碍和挑战之一是如何利用小的患者群体来实现相同的标准以进行监管审批。为了解决这个问题，提出了一种分为两个阶段的方法，首先证明不是无效的，然后排除（或控制）不确定性的概率来证明有效性。提议的两阶段方法对于可用于实现罕见病药物产品监管批准的相同标准的小患者群体很有用。