Client
- Top 20 pharma company, Global, Canada and UK cross-functional teams
Challenge
- Our client was facing potential reimbursement challenges for a novel therapy targeting two rare diseases with profound patient and family impacts
- Paediatric indications were expected to lead to challenges around endpoint selection and measurement, and the company wanted to explore how to supplement their randomised clinical trial programme
Approach
- redthread supported a joint early dialogue process with NICE and CADTH, one of the first real-world evidence focused advice projects commissioned by CADTH under their new drive to incorporate this evidence into their decision making
- redthread facilitated developing and refining the questions and company positions based on our extensive in-market HTA experience, and developed a succinct briefing book across the two indications well within the submission timelines
- redthread pushed to achieve consensus across cross-functional stakeholders, from programme leads and commercial, through to R&D and in-country access leads
Result
- Our client benefitted from a smooth engagement and briefing book development process, and received actionable feedback on their evidence generation plan
- All internal stakeholders involved heard powerful first-hand testimony of the real-life patient and family impact of these debilitating rare diseases
