gms | German Medical Science

There is no difference in methodology between the benefit assessment of older (i.e. established) and newer and thus less established drugs. The key elements of a benefit assessment following evidence-based medicine (EBM) are a precisely worded medical question and a definition of benefit and harm according to, for example, patient-relevant outcomes such as life expectancy, symptoms, quality of life, and adverse effects. Furthermore, a systematic search is needed to retrieve all relevant studies.

An important question in a benefit assessment concerns the added benefit of a drug versus a treatment alternative. Not only the added benefit of a new drug needs to be proven; the same applies to the established drug (also versus other established drugs). This means that new research questions could arise for established drugs, for example, through current medical developments.

The benefit of an intervention has to be assessed according to patients’ needs and the current status of scientific knowledge (e.g. on EBM, statistical methods, and drugs). There is thus basically no difference in the benefit assessment of new versus established drugs. The only potential differences could be a better evidence base for new drugs regarding, for example, quality of life or the use of different statistical methods.

In the benefit assessment it is insufficient to rely on data collected since market entry (e.g. Periodic Safety Update Reports). These data provide information on whether a drug is safe enough to remain on the market. They do not provide sufficient information on the (added) benefit of a drug versus other interventions, no matter whether the drug is established or not.