Clinical trials are research trials conducted with healthy volunteers or patients before a therapy or drug is approved. In these trials, the efficacy and safety of drugs or other medical interventions is tested.
Legal regulations and guidelines ensure the greatest possible safety for trial participants: For instance, a clinical trial can commence only when previous laboratory and/or animal experiments have delivered sufficient safety data, and an independent ethics commission has agreed to the trial. This only occurs when the ethics commission is convinced that the trial is safe and promises to actually improve standard therapy.
The objective of clinical trials is to find answers to scientific questions and to find better methods of preventing, diagnosing and effectively treating diseases in the future. The trial results provide information on whether a new drug or therapy is effective against a certain disease and whether it is tolerated well. There is a study protocol for each trial in which its benchmarks are laid out. These include the research status on which the trial is based, its objectives and exact information on how the trial will be conducted.
In accordance with Good Clinical Practice guidelines, only clinical investigators, i.e. highly specialized and qualified physicians, may conduct a clinical trial. Meticulous controls are performed at regular intervals to ensure that adverse effects of the active substance being tested are detected immediately, so that treatment can be discontinued if necessary. In Germany, the Paul Ehrlich Institute and the German Federal Institute for Drugs and Medical Devices (BfArM) monitor clinical drug trials to make sure they comply with all the regulations.
The development of orphan drugs, i.e. drugs for the treatment of rare diseases, is very expensive and time consuming. For this reason, orphan drug development receives special support from the European Union, so that people suffering from a rare disease gain rapid access to treatment.