We’re pleased to share that Donald White (Head of Translational Science) and Mark Mayhew (Head of Portfolio Strategy) of Treehill Partners contributed their expertise to FirstWord’s September 2025 ExpertViews Report: “Biomarkers and Diagnostics: Foundations for Rare Disease Therapies.”
About the Report
The comprehensive report examines how biomarkers and diagnostics serve as the foundation for developing therapies for rare diseases—a market valued at $224 billion in 2024 and projected to reach $527 billion by 2034.
Key Areas of Treehill Expertise
Our experts provided insights across the drug development lifecycle, addressing:
Strategic Development Considerations:
- The critical role of biomarkers from early drug discovery through regulatory approval and market access
- How biomarkers can accelerate clinical trial processes by approximately 50%, reducing timelines from five years to 2.5 years
- The importance of integrating biomarker strategy early in development programs
Validation and Implementation Challenges:
- Technical and strategic considerations for biomarker qualification in small, heterogeneous rare disease populations
- The evolving regulatory landscape, noting the FDA’s more advanced guidance compared to EMA requirements
- Real-world applications in conditions like ALS, where biomarker validation supports faster patient recruitment and earlier treatment access
Future Opportunities:
- The transformative potential of AI/ML and multiomics approaches in biomarker discovery
- How disease-agnostic biomarkers can address the needs of over 500 million people globally affected by rare diseases
- The critical importance of industry investment and public-private consortia to advance the field
Market Context
The report highlights that 52% of all drugs approved in 2024 targeted rare diseases, with 35% of the biological pipeline consisting of rare disease projects—underscoring significant industry momentum and the growing importance of sophisticated biomarker strategies.
Industry Leadership
Don and Mark’s contributions reflect Treehill Partners’ unique approach: integrating drug development expertise with corporate development strategy to help companies optimize the commercial relevance, transactability, and fundability of their rare disease programs.
Full Report Details:
FirstWord ExpertViews Report: “Biomarkers and Diagnostics: Foundations for Rare Disease Therapies”
Published: September 2025
Learn more about & obtain the report here