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

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 LeadsEmail

Agnes Verhoeven, Johnson & Johnson

Averho10@its.jnj.com

Alicja Budek Mark, Genmab

Amar@genmab.com
Alex Pearce, PHUSE Project Assistant

Alexandra@phuse.global

CURRENT STATUS Q4 2024

  • Discuss sub-group discussions/outcomes in all -team meeting.
  • Regrouping where needed.
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

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