Landscape assessment to identify existing clinical outcome measures and regulatory and payer perceptions

Insight and direction for strategic outcome assessment to optimise the clinical development program of an early phase asset through synthesising previous payer and regulatory insights.

The challenge

Our client had an early phase asset for use in cystic fibrosis and bronchiectasis. They sought our help in identifying:
> The management of the respiratory conditions of interest with a focus upon clinical outcome assessments (COAs) used to assess symptoms, impact function and general health-related quality of life.
> Appraisal of different COAs from stakeholders at both regulatory and Health Technology Assessment (HTA) levels.

The solution

We conducted a thorough literature and landscape review to understand and explore existing outcome measures available for these respiratory conditions. Alongside this, we critically reviewed HTA appraisals and regulatory documents for products in the relevant indications to understand the potential weaknesses of competitor outcome assessments and inform a robust outcome assessment strategy.

Key results

Our research provided evidence for the COAs used across the indications and highlighted where previous products have been criticised on the appropriateness of their outcome assessments during regulatory and HTA appraisals. Findings were synthesised into a detailed technical report, a digestible summary slide deck and a userfriendly database that could be easily updated as the landscape emerged. Our research highlighted the most commonly used outcome measures across the indications of interest and provided recommendations for our client for the outcome assessment strategy to best support future market access.

Value to the client

Our evidence-based approach, combined with our knowledge of the regulatory and payer landscape, enabled our client to make informed decisions on the choice of outcome measures for their clinical development program for an early-stage asset, helping prepare them for future market access.