Case Study

Opportunity Assessment and Patient
Mapping for an Asset in Rare Diseases

Background and Objectives

A global pharmaceutical company, with a marketed product for a rare disease, wanted to assess the opportunity of the asset in two other rare disease indications. SmartAnalyst supported the client by conducting a comprehensive commercial assessment to quantify the size of the opportunity in these two new indications for the US, EU5, and 5 other countries of interest.

Approach and Recommendations (Methodology)

SmartAnalyst utilized a rigourous methodology to develop the commercial assessment and conducted primary and secondary market research to support the analysis.

Phase I: Secondary and Qualitative Primary Research

  • Conducted secondary research to understand the current and future disease landscape in the selected countries. This included a thorough analysis of the disease etiology, epidemiology (patient flow between different treatment lines), diagnosis (key stakeholders involved, key assays, and the limitations/advantages of each test), treatment (key stakeholders involved in management, current treatment modalities, and associated benefits and challenges), and competitive landscape (emerging treatment options) for the two indications.
  • Qualitative in-depth interviews with specialists in target geographies were conducted to validate unmet needs, treatment paradigms and patient journey mapping, as well as to test the Target Product Profile (TPP) for each of the two indications

Phase II: Semi-Quantitative Primary Research and Revenue Forecast

  • Carried out semi-quantitative primary research with 65 KOLs in target geographies to update the forecast model and estimate the revenue potential based on the expected utilization (share, uptake) of the client’s asset in each of the two new indications.

Time Frame

21 Weeks


Based on high unmet need, positive reaction to the TPP, and expected level of utilization, a recommendation was made to proceed with both new indications. Based on the assessment, these two new indications could significantly enhance asset value in most countries of interest. Potential risks to product adoption in specific countries were identified (reimbursement and dosing regimen) that would need to be addressed.

About SmartAnalyst: Driving pipeline and portfolio value for global biopharma and life science companies

SmartAnalyst helps biopharma companies make key decisions at the Disease, Asset, and Portfolio levels, utilizing proprietary strategic frameworks. We provide comprehensive, integrated solutions based on our deep understanding of the science, the market, and the key business decisions that our clients face. We deploy agile, cross-functional project teams that are responsive to client needs.