Clinical research is the process for studying diseases and developing medicines to possibly prevent, treat, or help with different diseases. Before a medicine can become widely available, it must be tested in a series of clinical trials to evaluate its safety and effectiveness.
People who volunteer for clinical trials and receive an investigational medication are closely monitored. Health authorities use the information from clinical trials to decide whether or not investigational medicines are safe and effective and should be approved for routine medical use.
There are generally 3 phases of clinical trials that need to be completed before health authorities consider granting approval.
Phase 1 and 2 clinical trials mainly determine the best dose and if the investigational medication is safe when it is processed in the body.
Phase 3 clinical trials include a larger number of people, usually from all over the world, and are intended to evaluate whether the investigation medication can be used safely, and to see how well it works in a larger population of people with the condition.
For rare diseases, like acute porphyrias, there is still rigorous testing but the timelines to get the investigational medication reviewed by authorities may be shorter, and fewer participants are needed than in clinical trials for more common diseases.