Rare Disease Opportunity Assessment

Evaluate a new therapeutic for a rare debilitating neuromuscular disorder.

Challenge

Opportunity assessment in a rare disease presents challenges, especially where a novel therapeutic (new MOA) would be the first product to treat a long unaddressed disease. From unsubstantiated epidemiology, to a lengthy and highly variable diagnostic and treatment experience–much is uncertain and needs to be explored and calibrated.

Our client, a development stage biotech start-up, sought to explore the landscape for their novel disease-modifying therapeutic–all to shape their near-term strategy as well as to quantify the opportunity for their lead asset.

The client team had a strong sense of the clinical development challenges and an emerging plan, but they were in need of objective feedback on their target product profile, as well as insight into variables that would likely drive utilization. Additionally, to inform a comprehensive global strategy and financial model, they needed input from the payer community to inform assumptions and clinical program priorities around pricing and access. Developing a clear and well-supported narrative supporting the value of the asset will be critical as they continue to communicate with current and prospective investors and partners.

Solution

Clarion took a comprehensive, multi-pronged research approach to support the opportunity assessment, combining an extensive literature review, robust analog prioritization and characterization, and in-depth primary research interviews with KOLs and payer experts. Collaborative engagement with our client in the development of the primary research plan ensured critical nuance

around the product attributes and clinical development plan were appropriately conveyed and explored. Findings from across the primary, secondary, and analog research were synthesized to substantiate key assumptions in the final opportunity forecast and define likely drivers of or barriers to uptake.

Global opportunity assessment

  • Summary presentation materials for internal and external uses
  • Comprehensive analysis of epidemiology sources across key geographies
  • Summary KOL/physician findings
  • Target product profile and proposed clinical endpoints
  • Competitive pipeline analysis
  • Key drivers of utilization, pricing, and access
  • Detailed appendix including all the secondary research and analog analysis supporting forecast assumptions

Global forecast model

  • Excel-based forecast model with detailed assumptions and scenarios organized by geography
  • Sensitivity analysis
  • Scenario modeling

Impact

The assessment enabled our client to tell a compelling story to investors around the commercial opportunity for their lead asset, backed up by rigorous research and analysis. Insights from primary research with physicians and payers will also help guide the design of the clinical development program, and support

our client in future strategic decision-making around the asset. The final presentation was made to the executive team, who were impressed with the intellectual rigor and thoroughness of the assessment.

The Clarion Difference

  • Cross-disciplinary expertise: Clarion’s broad expertise across scientific, clinical, and market access domains enabled a rigorous assessment of the opportunity to support the final forecast.
  • Strategic insights: Clarion provides not only financial projections of the commercial opportunity, but additionally leverages market research to generate strategic insights into likely drivers of success and actionable recommendations.
  • Collaborative approach: Clarion consistently works to engage key stakeholders, drawing on diverse viewpoints across the client team in crafting the final assessment and ensuring the resulting model will be widely endorsed throughout the organization.

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