CRED Understanding Clinical Development 2024

14/10/2024

Study Design: Controlled Studies

Randomised controlled studies (RCTs) = gold standard for Phase 3

Controlled Studies

● Concurrently controlled – Control and test group chosen from same population and treated concurrently – With standard of care (SOC)/placebo ● External (historical) control – Compares subjects receiving test treatment with a group of subjects external to the study (e.g. earlier time point (historical) or other setting) – Rare diseases, oncology, chronic conditions

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Study Design: Uncontrolled Studies

More common in earlier phase studies of new and untried therapies where standard relatively ineffective May be appropriate when: ● No longer genuine uncertainty over whether a treatment will be beneficial ● Truly dramatic response Most uncontrolled studies performed in Oncology/ Hematology 1 e.g.

● acute myeloid leukemia (AML): Uncontrolled studies = 66% ● chronic heart failure (CHF): Uncontrolled studies = 13%

The Organisation for Professionals in Regulatory Affairs

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