- Are done on MS’ers to allow the safety and effectiveness of an intervention to be assessed. The intervention can be a drug, device or procedure.
- Can only take place only after satisfactory information has been gathered on non-clinical safety (animal data), and after health authority, ethical and R&D approval is granted.
“As you can imagine this process can take years and generates an enormous amount of administrative work.”
“The red tape is unbelievable.”
The following is a brief summary of the phases of drug development:
- Pre-clinical studies – these are typically animal experiments to assess the mode of action of the drug and the effectiveness of the drug. Route of administration (oral or injection), dose effects and how the body deals with drug (metabolism) are also tested at this stage.
- Phase 0 – These are exploratory, first-in-human, microdosing studies and are designed to speed up the development of promising drugs at a very early stage. The answer whether or not the drug behaves in human subjects as was expected from preclinical studies. These studies give no data on safety or efficacy.
- Phase I – These are traditionally the first stage of testing in human subjects. Typically this phase involves healthy volunteers and are designed to assess the safety, tolerability an pharmacology of the drug in humans. A phase 1a refers to studies in normal subjects and a phase 1b study refers to studies done in subjects with the disease in question; it is sometimes necessary to test a specific attribute of a drug in the target population.
- Phase 2 – Once the initial safety of the study drug has been confirmed Phase 2 trials are performed on larger groups and are designed to assess how well the drug works, as well as to continue safety assessments in a larger group of patients. In MS trials this is typically done in two phases; phase 2A is specifically designed proof-of-principle and to assess dosing requirements (how much drug should be given). Phase 2B studies assess how well the drug works at the prescribed dose(s).
- Phase 3 – These studies are randomized controlled multicenter trials on large patient groups and are aimed at being the definitive assessment of how effective the drug is, in comparison with current ‘gold standard’ treatment. The regulatory authorities typically expected that there be at least two successful Phase 3 trials in order to obtain approval.
- Phase 4 – Phase 4 trials are also known as Post-Marketing Surveillance Trials and involve the safety surveillance and ongoing support of a drug after it receives permission to be sold.
- Phase 5 – Phase 5 is term used to refer to comparative effectiveness research and community-based research; it is used to assess how a new clinical treatment is integrated into clinical practice, i.e. the day-to-day use of the drug.