Clinical trials

General information:
Before a new drug or medicine becomes available through hospital doctors, family doctors or pharmacies, it has had to undergo a long process of research and testing anything up to 10-15 years. New therapeutic agents, new combinations of drugs or new uses of known drugs, should first be tested in a laboratory setting and in animal models (preclinical phase), before proceeding to test on humans in clinical trials is allowed. In clinical trials, the goal is to test the efficacy and safety of a new drug in human participants.

Clinical studies have three phases:

  • In Phase I, the new drug or treatment is tested for its safety in humans and the maximum dose will be determined. In general, patients for whom a standard treatment no longer exists are asked to participate in phase 1 trials.
  • In a phase II study, the effectiveness and the optimal dose of the new drug are studied. In a phase III study, the effectiveness of the new drug will be compared to the currently defined standard or best available therapy.
  • In Phase lll trials larger patient groups will be used to examine whether the new drug is more effective than the current best treatment. After successful phase III trials, drugs can then be submitted for approval by medical authorities such as the European Medicines Agency (EMA), or the Food and Drug Administration (the FDA) in America.

After a drug is launched, the long-term effects may still be monitored, sometimes referred to as postmarketing studies or phase IV studies.

The decision to participate in a clinical trial is one you should carefully consider. It is important to discuss clinical trial participation with your family and your physician. If you are considering participating in a clinical trial click here to read a clinical trial FAQ page. You can find a data base of clinical trials on the MPN Research Foundation website here, or if you look for a special country at