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Scientific and Regulatory Portfolio Prioritization in Rare Disease

About Client

The client is a biopharmaceutical company with a rare disease pipeline spanning multiple development stages and constrained development resources

Study: A cross-functional portfolio prioritization effort evaluating scientific, regulatory, and commercial risk across a rare disease (e.g., rare metabolic diseases, kidney, liver) pipeline

Challenge

Leadership needed an objective framework to prioritize assets under uncertainty

  • Limited internal alignment on which assets merited continued investment

  • Need to assess development readiness, regulatory feasibility, and competitive positioning

  • Requirement for an objective, risk-adjusted prioritization framework

Solution

Led a cross-functional diligence and prioritization effort, including:

  • Built a comprehensive asset and clinical trial database using Citeline TrialTrove and PharmaTrove

  • Conducted key opinion leader interviews to validate scientific rationale and unmet need

  • Applied risk-adjusted valuation using clinical analogs and analyst reports

Results

The work enabled clearer, more defensible portfolio decisions agreed upon by the company’s board

  • Enabled data-driven portfolio prioritization decisions

  • Clarified key de-risking milestones for lower-confidence programs

  • Improved leadership alignment around resource allocation and development sequencing