Tubestop - Efficacy

Description

Efficacy is defined as the ability of a new treatment to produce a desired effect in a clinical trial. It is measured by comparing the results of the new treatment to a control group, which is typically given a standard treatment or a placebo.
When planning a clinical trial protocol, it is important to carefully consider how efficacy will be measured. The following factors should be taken into account:

The primary outcome: The primary outcome is the main measure of efficacy that will be used to evaluate the new treatment. I
The secondary outcomes: Secondary outcomes are additional measures of efficacy that may be used to evaluate the new treatment.
The control group: The control group is the group of patients who are not given the new treatment; they may receive standard of care treatment or placebo.
The statistical analysis: The statistical analysis will be used to determine whether the new treatment is more effective than the control group.

DCT-specific Considerations/Inputs

The patient population: The patient population for a decentralized clinical trial is likely to be more diverse than the patient population for a traditional clinical trial. This is because decentralized clinical trials can be conducted remotely, which makes it easier to recruit underrepresented patients. Regulatory authorities are interested in increasing diverse trial participation to help ensure the trial populations better align to the populations impacted by disease.
The study design: Decentralized clinical trials often use a hybrid design, which combines elements of both traditional and remote clinical trials. Consider which efficacy assessments can be conducted remotely, any training and oversight requirements to ensure they are conducted appropriately.
The data collection methods: Decentralized clinical trials often use technology tools and systems to collect data. This can make it easier to collect data and to monitor patient adherence to the study protocol. Consider which efficacy measures might be collected directly from patients using appropriate measurement instruments (e.g surveys and diaries) or digital tools (e.g. wearables)
The statistical analysis: Decentralized clinical trials often require more complex statistical analyses than traditional clinical trials. The number of data sources or data collection methods used may increase, and may need to be considered in the analysis plan.

U.S. FDA Final Guidance Document: Conducting Clinical Trials With Decentralized Elements Guidance for Industry, Investigators, and Other Interested Parties - September 2024

Efficacy

Description

DCT-specific Considerations/Inputs

Tools

Regulatory Documents

FDA/U.S.

Europe/EMA

Reference Documents