Rob Freistat, President of Uncommon Cures, discusses how clinical trials are transforming to accelerate drug development and ensure patients receive treatments faster, particularly in the rare disease space.
- Current market uncertainty and investment challenges in biotech, especially for rare disease research
- Approximately two-thirds of clinical trial processes add no value and aren’t required by regulations
- Many clinical trial failures occur not because drugs don’t work but because trials can’t recruit enough patients
- Companies often treat development phases as “islands” rather than planning for commercial success from the beginning
- Rare disease economics require greater efficiency since development costs must be recovered from much smaller patient populations
- Reviving shelved drug candidates represents a potentially valuable opportunity
- Patients should have greater input in trial design and outcome measures that matter to them
- Better communication between sponsors and regulators could significantly improve drug development
