White Paper:

Addressing the Special Commercial Challenges in Rare Diseases

Rare disease therapies face different market realities than other specialty products.  Patients are rare and hard to find, physicians and disease experts are often few and far between, robust data are typically limited.  These realities present unique challenges for commercial decision makers.  To succeed in the difficult rare disease marketplace, biopharmaceutical companies must develop a tailored set of tools and approaches.

This paper outlines the special commercial challenges facing rare disease therapies. It also offers some approaches for overcoming them. The challenges highlighted here are those that were listed time and again in conversations that the Blue Matter team has conducted with decision makers in rare disease companies.

Key Contents:

  1. Introduction to Rare Diseases
  2. Why Rare Diseases are Different
  3. Challenge 1: Finding Patients
  4. Challenge 2: Leveraging Centers of Excellence and Referral Networks
  5. Challenge 3: Demonstrating Product Value
  6. Challenge 4: Organizing to Support the “High-Touch” Rare Disease Business Model
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