SITUATION
A clinical-stage biotech company was advancing a novel biologic targeting an under-researched, immune-driven rare disease with both dermatological and systemic manifestations. The therapy had demonstrated favorable safety and biomarker response in Phase I, and the team was preparing to move quickly into a multi-country Phase II study.
Given the lack of historical benchmarks, limited prevalence data, and the absence of well-defined clinical endpoints in this disease area, the company needed external support to confidently plan their Phase II trial — from site selection to protocol design.
CHALLENGE
Planning clinical trials in rare diseases is often constrained by low patient numbers, inconsistent diagnostic practices, and unclear regulatory expectations.
The client faced several specific challenges:
- No established clinical trial precedent for the indication, making endpoint selection difficult
- Limited clarity on patient availability and diagnostic infrastructure across target regions
- Uncertainty about what data would satisfy regulatory agencies and support future access discussions
- Internal pressure to move quickly while still building confidence with investors and scientific partners
The team needed a data-backed strategy to design an efficient, defensible, and scalable Phase II program.
APPROACH
ConsaInsights partnered with the cross-functional development and clinical operations teams to build a clear Phase II trial strategy using deep competitive intelligence, KOL insights, and operational feasibility analysis.
Competitive and Historical Trial Benchmarking
We identified and analyzed 12 trials across adjacent immunological and rare dermatological conditions. This included dose-finding strategies, patient stratification methods, endpoint selection, and geographic recruitment performance.
Site and Feasibility Mapping
Using a global investigator database and published trial performance data, we assessed the experience, recruitment capacity, and diagnostic familiarity of sites across North America, Europe, and Asia-Pacific. This helped the client narrow their initial list to high-probability locations.
KOL and Advisory Alignment
We coordinated structured interviews and advisory sessions with disease-area experts to refine the proposed inclusion/exclusion criteria and define clinically meaningful endpoints. Their input was integrated directly into protocol drafts and supported later regulatory dialogues.