Pharmacotherapy in clinical trials

Generally, a patient should be prescribed a medicine that has official marketing authorisation in the patient’s age group.

According to law, the treating physician has the right to prescribe a medicine that does not have the mentioned authorisation, if no authorised medicine is available. In such a case, it is usually better for the patient that he or she is prescribed medicine that has not been investigated, rather than he or she is given no treatment at all.

The treating physician must, however, have sufficient information based on research that the administration of such medicine can be considered a better alternative than not administering it at all. A situation in which a child must be prescribed a medicine which has not been investigated in his or her age group and cannot therefore have an official marketing authorisation in the group in question, can be called experimental treatment.

The safest alternative, on the patient’s behalf, would be to administer medicine as a part of clinical trials. Clinical trials are always a safer alternative for a patient than experimental treatment.

As opposed to experimental treatment, the advantages of clinical trials for a patient are:

• in addition to the treating physician, authorities and other experts (the rest of the research group, an ethics committee, the granter of the research permission in the research unit) participate in the evaluation of risks and advantages of the medicine and the trial in question
• the efficacy and safety follow-up are systematic and well-planned
• the results of the treatment are systematically analysed
• in the trial report, the results of the treatment become more widely known and available for the benefit of other patients.