NOKC, Norway

Efficacy refers to the trial setting, and thus any HTA need to consider whether the results obtained in clinical trials can be generalized outside to clinical practice. In some instances “real world” studies are conducted to evaluate real world effectiveness, such studies are however often registry based and thus not of a design comparable to study designs most often included to analyse efficacy.

We and probably other HTA-agencies are often faced with the question – what is sufficient documentation of effectiveness? This question would be important to discuss within EUnetHTA, and one might consider some approaches that include consideration of the amount and quality of clinical trials, use of surrogate measures, time for follow up etc.

Another potential problem arises when efficacy is derived from confidential information. How do we handle the fact that agencies within Europe have different approaches to the use of confidential information and how would this influence the sharing and using of reports from other agencies?