1. Background and general principles of the regulatory approval of medicines

Basic philosophy behind the approval of medicines

For a new medicine to be approved it is necessary to submit sufficient documentation to establish that the following criteria have been met:

In addition, the applicant must demonstrate a positive benefit-risk ratio for the new medicine.

1.1. Quality

The quality of the active substance in the medicine must be high. The term ‘quality’ in this context should not be confused with the effect of the medicine. The company should describe how they produce the substance. It is important that they also document the nature and amount of any impurity in the substance – and how such amounts are controlled. New impurities could be potentially dangerous and toxic.

A description of the manufacturing of the medicinal product (also called: finished product,

 Pharmaceutical product, Drug product) must also be present, as well as test procedures to ensure good quality.

Quality requirements for the medicine can ensure that the marketed product is identical to the product that was tested during all the clinical trials.

1.2. Safety

During development of the medicine, the safety of the active substance and the finished product needs to be demonstrated. The company should test the medicine first in animals, and later in humans. During such testing, the company should investigate, to which extent the medicine could cause adverse reactions (side effects), which would be harmful to patients. In addition, the company needs to collect and report all information on adverse reactions observed in any clinical trial.

1.3. Efficacy

The company needs to perform a series of clinical trials on many patients. During the analysis of the trials, the desired effect should be clearly demonstrated.