HTA agencies and EMA developed joint positions for better evidence

A key result of recent European workshops is that drug studies should be designed in such a way that they address both regulatory questions and the essential research questions of benefit assessments.

Whether a drug works better or has fewer side effects than current standard treatment is irrelevant for the approval. It is sufficient if the drug is effective and safe. An added benefit only becomes important in the second step, the benefit assessment of the drug. Then it is examined whether the new drug helps patients better than the standard treatment. For this assessment of added benefit, the drug manufacturers must submit studies in which the new drug is compared with the standard treatment.

In the German AMNOG procedure, the Institute for Quality and Efficiency in Health Care (IQWiG) examines the results of the comparative studies on behalf of the Federal Joint Committee (G-BA) and determines the added benefit of a new drug for clinical practice. On the basis of this assessment, the G-BA  determines the extent of the added benefit, which is relevant for clinical practice, and which serves as the basis for price negotiations between statutory health insurance funds and the manufacturer.