临床试验於研究设计和计画撰写方面之法规科学考量.ppt

Active Control 要選擇 current standard therapy。 Trial 可採 superiority, non-inferiority 或 equivalence design。 亦可以由 non-inferiority 成功後，再測試 superiority。 有時 active control 僅作為 assay sensitivity 之用 (主要療效假說仍是 test product 優於 placebo，例如 3-arm design)。 參考資料: www.ema.europa.eu/pdfs/human/ewp/215899en.pdf www.ema.europa.eu/pdfs/human/ewp/048299en.pdf /downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM202140.pdf * When to Break The Code? Randomization procedures must be followed to ensure that the treatment code is only able to be broken in the event of any of the following occurrences (unless otherwise specified): Adverse event or medical emergency where the treatment of the patient is dependent on knowledge of the study treatment code Accidental administration of the investigational product to a patient not participating in the research Any other instance as specified in the study protocol * 5. Selection and Withdrawal of Subjects Subject inclusion criteria Subject exclusion criteria Subject withdrawal criteria (i.e. terminating investigational product treatment/trial treatment) and procedures specifying: (a) When and how to withdraw subjects from the trial/ investigational product treatment (b) The type and timing of the data to be collected for  withdrawn subjects (c) Whether and how subjects are to be replaced. (d) The follow-up for subjects withdrawn from investigational product treatment/trial treatment * Patient Selection Inclusion/Exclusion (Eligibility) Criteria: Clear and unambiguous statement of subject selection criteria Include subjects depending on their risk of event of interest likely responsiveness to trial intervention Exclude subjects who may be placed at some increased risk already manifest outcome * Eligibility Requirements 診斷要明確，診斷方式要為目前之 gold standard。 疾病嚴重程度與目前有無現行標準治療，皆會影響試驗設計與療效分析。 收納族群太窄，代表性太差，很容易日後再重複做試驗。 收納族群太寬，容易因為納入不適合之次族群而被法規單位要求停止試驗。 排除條件依照藥品特性機轉列出可能之禁忌症清單。 可能產生藥物交互作用之清單(考慮禁止或有適當規範)。 考量上述限制後，要是否可能收納足夠