Driving Value Outcomes in Orphan Drug Markets

Executive Summary

Orphan drugs, critical for treating rare diseases, often face steep challenges in pricing and reimbursement due to limited patient populations, high development costs, and stringent regulatory requirements. A global pharmaceutical company developing an innovative treatment for a rare metabolic disorder sought a comprehensive strategy to navigate these complexities and ensure a successful market entry.

Factspan designed a tailored solution using advanced AI-powered analytics, proprietary pricing models, and data from clinical trials, market research, and payer databases. This approach addressed regulatory requirements, aligned with payer expectations, and optimized pricing for sustainable revenue growth. The strategy enabled a successful product launch and established a scalable framework for future pricing in the orphan drug market.

About the Client

The client is a pharmaceutical company with a global presence specializing in treatments for rare diseases. Their recent endeavor involved the development and launch of an orphan drug targeting a rare metabolic disorder, with plans to roll it out across the United States, Europe, and Japan.

Business Challenge

The client faced significant challenges in determining an optimal pricing strategy for the orphan drug. Regulatory requirements and reimbursement frameworks varied widely across regions, making it difficult to balance affordability and profitability. Concerns over product wastage due to vial pricing complexities added another layer of complexity. Additionally, the client needed to forecast revenues accurately while addressing potential payer resistance and regulatory delays. These challenges required a robust, datadriven approach to pricing and market entry.

Our Solution

Factspan adopted a comprehensive approach to address the client’s challenges, leveraging proprietary methodologies and AI-powered tools to develop an optimized orphan drug pricing strategy. The solution was designed to align with regional regulatory frameworks and optimize revenue generation.

The core components of our solution included:

• Delivering actionable insights on reimbursement timelines and mitigating potential delays
• Conducting an in-depth market and competitor analysis to identify feasible price bands and anticipate
  payer challenges
• Developing ML-driven models such as regression analysis and time-series forecasting to predict
  revenue impacts and optimize pricing strategies for each region
• Creating tailored pricing models with managed entry agreements to facilitate market access

The solution delivered a data-driven pricing strategy, ensuring timely market entry and sustained access. Revised revenue projections stemmed from region-specific decisions, sensitivity analyses, and payer preference modeling. Insights like volume forecasts, cost impacts, and optimal pricing bands addressed regulatory challenges, while scenario-based simulations streamlined approvals. This positioned the client’s orphan drug for long-term success and set a scalable framework for future launches.