Clinical-Regulatory Path: Finding Solutions
Clementia works with urgency and compassion to advance palovarotene, an investigational therapy for people with fibrodysplasia ossificans progressiva (FOP), Multiple Osteochondroma (MO) and other diseases.
For a drug to obtain approval for use in humans, it must first make its way through laboratory testing (also known as preclinical or nonclinical studies) and through rigorous clinical testing phases. Pre-clinical trials involve testing in animal models to determine safety and potential effectiveness, after which the medication may advance to testing in humans, typically consisting of three phases.
The Path to Approval
All drugs must pass through a rigorous development process and regulatory review before being approved for use in humans. The typical path to approval is outlined below.
6 to 18 months
- Evaluates safety in humans by administering medication to healthy volunteers
- Collects information about potential side effects
18 to 36 months
- Evaluates safety and efficacy in people with targeted disease
18 to 36 months
- Evaluates safety and confirms efficacy in people with targeted disease
- Involves a larger number of affected individuals than a Phase 2 trial
- Data package can be submitted to regulatory agencies for review and potential approval
- Drug approved to market to affected individuals
- Country-by-country procedures to ensure commercialization and patient access to treatment
For more information about the clinical trial process, visit the United States Food and Drug Administration website: http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm394839.htm