Global Go-to-Market Strategy for an Innovative Rare Disease Therapy

A clinical-stage biopharma company was developing two therapies (phase 1 and 2) for a rare disease with a high unmet need. The company needed to define the market opportunity and assess the market structure across a range of regions globally. It also needed to develop a go-to-market strategy (direct, partner, etc.) for these assets by market region.

Our team began by conducting a qualitative and quantitative evaluation (epidemiology, treatment rates, patient journey, treatment flow, access environment, etc.) of the priority market regions. We determined the overall opportunity size by market region. In addition, we categorized / archetyped the regions. For each, we assessed the individual market structure including treatment centers, referral patterns, patient journey, patient population sizing, stakeholder mapping, and the competitive environment. This helped to determine the optimal market footprint (sales/medical/access) for launch and commercial success. We then identified and analyzed various market entry options. This included financial evaluations to assess the full cost of market entry and a review of P&L scenarios for both direct (self) and partner (distributors / out-licensing) commercialization across markets. Finally, we reviewed the financial scenarios by market and entry method with company leaders via a series of workshops to help guide strategic decision-making.

Our work illuminated the optimal development and go-to-market pathways for the company’s leaders. They incorporated our recommendations into their strategic decision-making and continued to rely on Blue Matter for guidance and support as these assets progressed through the development process.