Navigating the Complexities of Adding Rare Disease Products to Your Company’s Portfolio

1. Accelerated Time to Commercialization: Rare disease products typically have shorter or accelerated clinical development timelines, lower development and commercialization costs, and fewer life-cycle challenges compared to other specialty products.
2. Patient Focus: Rare disease companies often provide holistic services to meet the needs of patients and their families, which requires significant planning and organizational implications.
3. Challenging Market Access: Gaining early and broad access for high-cost treatments with limited clinical data sets and health economics data can be difficult for rare disease products.
4. Payer Contract Strategy: Manufacturers are employing value-based or outcomes-based contracting to demonstrate the value of their treatments and share risk with payers, who are increasingly concerned about the high costs of rare disease therapies.
5. Regulatory Incentives: The Orphan Drug Tax Credit and R&D tax credit, as well as FDA designations like priority review vouchers and fast track, encourage larger organizations to invest in rare disease research and development.
6. Emerging Technology: AI, genetic sequencing, and biomarkers are being leveraged to accelerate rare disease research, improve diagnosis, and enhance patient outcomes.
7. Market Growth: The rare disease market is expected to reach $262 billion by 2024, with a CAGR of 11.3% from 2018 to 2024, driven by increased investment and innovation in the space.