Rare Disease
About Rare Disease
The Rare Disease Service examines the evolving stakeholder perspectives, financing approaches, and distribution models shaping access to rare disease and advanced cell & gene therapies. Through research with commercial payers and specialty pharmacy providers, the service provides insights into payer management strategies, emerging financing mechanisms, manufacturer engagement, and the operational considerations influencing access within this rapidly evolving market.
Latest Research Insights
For more information, or to subscribe, contact:
(408) 884-8560
Research Archive
2026 Research
Commercial Health Plans: Rare Disease & Advanced Therapy Management, Financing, and Manufacturer Engagement
September 2026. Examines commercial plans' management of high and ultra high-cost rare disease and cell & gene therapies (CGTs). Discusses financial protection solutions and manufacturer contracting, and evaluates best-in-class manufacturer support of rare disease portfolios across multiple metrics.
2025 Research
Specialty Pharmacy Providers: Rare Disease & Advanced Therapy Distribution and Manufacturer Engagement
October 2025. Reviews issues in specialty pharmacy dispensing and distribution of rare disease and gene therapy medications. Examines network considerations, specialty pharmacy capabilities, contracting approaches, and best-in-class manufacturer engagement/support.
Commercial Health Plans: Rare Disease & Advanced Therapy Management, Financing, and Manufacturer Engagement
Specialty Pharmacy Providers: Rare Disease & Advanced Therapy Distribution and Manufacturer Engagement
Go Deeper:
Rare Disease
As the pipeline of rare disease and advanced cell & gene therapies continues to expand, healthcare stakeholders are developing new approaches to evaluating, financing, distributing, and supporting these highly specialized treatments. HIRC’s Rare Disease Service helps pharmaceutical organizations better understand these developments by monitoring payer management trends, specialty pharmacy capabilities, manufacturer engagement, and emerging market dynamics. The service provides insights across 20+ disease states within the following broad therapeutic categories:
- Cell & Gene Therapy
- Rare Cardiovascular Diseases
- Rare Hematologic Disorders
- Rare Immunologic & Immune-Mediated Disorders
- Rare Neurological & Neuromuscular Disorders
- Rare Pulmonary Disorders
- Rare Oncology
- Rare Kidney Diseases
- Rare Liver Diseases
- Rare Metabolic & Lysosomal Disorders
For more information or to subscribe, contact Danielle Snook via email.
HIRC employs a triangulated research methodology that combines primary survey research, in-depth stakeholder interviews, and secondary market analysis to provide a comprehensive understanding of healthcare stakeholder perspectives, market dynamics, and emerging industry trends.
Research includes perspectives from:
- Commercial Health Plans: Management Trends, Emerging Financing & Contracting Approaches, Manufacturer Engagement
- Specialty Pharmacy Providers: Rare Disease Distribution Trends, Rare Disease Dispensing Capabilities & Services, Manufacturer Engagement, & Contracting
Subscribers can monitor emerging developments in the rare disease and gene therapy landscape, better understand payer and specialty pharmacy perspectives, identify evolving stakeholder needs, and make more informed decisions regarding market access, customer engagement, and organizational planning.