Small Molecule Orphan Drugs: Balancing Financial Incentives And Complex Challenges

By Kevin Kane, Global SME, Thermo Fisher Scientific

Executive Summary

The orphan drug industry focuses on developing life-changing and potentially life-saving treatments for patients living with rare diseases. Previously, the needs of these patients often went unmet because of the prohibitively high costs of developing drugs for small populations. The Orphan Drug Act of 1983 ushered in an era of incentives, government support, and regulatory agency assistance, making the development of drugs for rare diseases financially feasible and changing the pharmaceutical landscape significantly. Nearly half of all drugs approved by the FDA in 2019 were orphan drugs, and the market value of this industry segment is anticipated to be $262 billion by 2024¹.

Achieving clinical and commercial success with orphan drugs requires overcoming numerous development and manufacturing challenges related to fast-tracked timelines, active pharmaceutical ingredient (API) supply lines, formulation scale-up, clinical trial establishment, and regional variation in legislation and regulatory policies. Meeting these challenges requires specialized skills, resources, and infrastructure, as well as agile supply chain management with the flexibility to accommodate commercial needs.

This white paper offers a roadmap for navigating the complexities of orphan drug development based on the following key features.

• close alignment of clinical and CMC teams
• a continuous regulatory feedback loop
• predictive API modeling
• a robust formulation development program
• an integrated, single-vendor strategy

Changing the lives of patients living with rare diseases requires collaboration, communication, and careful planning that incorporates regulatory, clinical, scientific, and commercial strategy. It also requires a commitment to advancing science to speed solutions.