Enhanced Risk-based Quality Management (RBQM) Approaches for First-in-Human and/or Small Clinical Trials

Problem Statement

Early development (ED), first-in-human or small clinical trials (e.g. rare diseases) often have a complex and dynamic study design and are fast-paced. It can be challenging to find the appropriate balance in risk-based approaches and timing of implementation to assure value-added oversight.

Participant safety in first-in-human clinical trials is of utmost importance, which makes implementation of risk-based approaches challenging for the study teams and, therefore, often results in a conservative approach where everything is considered critical.

While trials that focus on rare diseases may not be fast-paced, they are challenging due to the limited data available to provide insights into emerging risks.

Project Scope

To discuss and focus on a robust strategy/process and best practices for:

Identifying critical items
Risk Based data review approaches
Risk Based Monitoring approach strategy (tSDV/tSDR)
Usage of analytical tools and methods for data review & central monitoring, including limitations on (statistical) methodologies
Identification of thresholds or equivalent on limited patient data
Targeted medical review
Strategy to adequately anticipate on highly dynamic data
Establish links with the other work streams on FIH/small studies focus

Project Leads	Email
Agnes Verhoeven, Johnson & Johnson	Averho10@its.jnj.com
Alicja Budek Mark, Genmab	Amar@genmab.com
Alex Pearce, PHUSE Project Assistant	Alexandra@phuse.global

Click above for the latest project status.

Objectives	Timelines
Establish membership Host Kick off meeting	Q2 2024
Regular meetings to assess RQBM in small trials Determine appropriate processes Establish links to other work-streams	Q3 2024
Outline guidance for RBQM in small trials	Q4 2024 – Q1 2025
Present at PHUSE connect session White paper or guidance document with a mutual agreed process for RBQM on FIH/Small studies	2025