Insight Gathering for Clinically Meaningful Change
Introduction
Our pharmaceutical client has completed a Phase 2 trial for a rare genetic disorder. Based on this study, the FDA recommended that our client update their primary endpoint (the Clinical Global Impression-Improvement subscale) for their planned Phase III clinical study.
The Challenge
To this end, our client sought our support in modifying the Clinical Global Impression – Severity (CGI-S) and Clinical Global Impression – Improvement (CGI-I) subscales to include condition-specific anchors to provide clinicians with a scale for assessing symptom severity at baseline as well as clinically meaningful improvement of symptoms for caregivers and health care providers.
The recommendations for a revised clinical assessment scale was issued as a report, informed regulatory interactions, as well as the design of the Phase III study.
Our Solution
- Literature Review: First we performed a comprehensive literature review to understand the extent CGI-I has been used to record clinically meaningful change in other neurological and neurodevelopmental diseases, including the relationship between CGI-I score and clinically meaningful thresholds.
- Next we engaged healthcare providers in qualitative one-on-one interviews as well as facilitated a live working group session. Findings from the literature review were presented to help inform discussion.
- The objectives of these engagements were to:
- Define minimally clinically meaningful change and clinically meaningful change for healthcare providers and caregivers in the context of the disorder.
- Gather input on the CGI scale developed specifically for rare genetic disorder to ensure it is appropriate to measure treatment effect.
- Insights from the one-on-one interviews and working group session were used to modify the CGI scale.
- In collaboration with our client, we developed the interview guide, as well as designed, facilitated, and reported the session. Our team managed all logistics and event management throughout the project.
- The insights gathered from the one-on-one interviews and working group session, were essential to develop a custom slide deck which was submitted to the FDA for consideration.
Value Delivered
- Provided strategic, adaptable and collaborative partnership that was attuned to the needs of our client.
- We excel at asking the right questions, probing assumptions, offering creative solutions and facilitating ‘big picture’ discussions.
- Delivered a thoughtful, strategic, and customized summary of the healthcare provider engagement which supported key regulatory interactions with the FDA and informed clinical study design.
- Fostered strong relationships by ensuring our client and their stakeholders received comprehensive support through seamless event management, while meeting all regulatory and compliance needs.
