Understand The R&D Process
Clinical trials refer to the research stage in which potential therapy is tested and monitored in humans. There are 3 stages of clinical trials required before a therapy can be approved and marketed (sold) for use outside a research or clinical study. In addition to supporting clinical trial recruitment and engaging with the FDA, you or others in your patient community may be interested in becoming a member of one of the boards involved in review and monitoring of clinical trials.
- Institutional Review Boards (IRBs) and Data Safety and Monitoring Boards (DSMBs) both play important, though different, roles in reviewing and monitoring each phase of a clinical trial.
- NCATS Toolkit provides more information about IRB and DSMB in Clinical Trials and FDA Review: Serve on Boards to Review and Monitor Clinical Trials.
- Phase 1: The therapy is tested in a very small group of healthy volunteers (or sometimes patients) to confirm it is safe to use in humans, as well as to determine the therapeutic dosage. This phase usually lasts a few months.
- According to the FDA, about 70% of therapies move on to Phase 2.
- Phase 2: The therapies is tested in a small group of patients (up to several hundred) to determine whether it is effective and whether there are side effects. This phase can last anywhere from several months to 2 years.
- According to the FDA, only 33% of therapies move on to Phase 3.
- Many therapies fail in Phase 2 because they are found to be ineffective or produce unpredicted safety issues or serious side effects.
- Phase 3: The therapy is tested in a larger group of patients (from a few hundred to several thousand) to continue to gather data on the effectiveness, as well as to monitor for adverse reactions. This phase usually lasts 1 to 4 years.
- Phase 3 Trials also have a high rate of failure. According to the FDA, only 25% to 30% will receive FDA marketing approval.
- Rare diseases: When a disease is rare, certain allowances during the clinical trial process may be allowed by the FDA. For example, the number of patients required for a specific clinical trial phase will usually be much lower than required for a more common disease.
- NCATS Toolkit provides more information on clinical trials, including other key areas your group can support the process in Clinical Trials and FDA Review.