Nordic Health Registers as a Source for Value-Based Evidence

Abstract

The study and approval of new drugs for use in humans has historically been based on three elements: safety, efficacy and quality. Towards the end of last century, different biomarkers and surrogate endpoints were considered appropriate for documenting new treatments. Most of our therapeutic progress during this period was developed within that viewpoint, and has been implemented in healthcare with great success. However, when it became harder and harder to demonstrate superiority of new drugs over existing therapies, combined with increasing healthcare costs, new needs came to the surface. The validity or appropriateness of surrogate endpoints was challenged, and the requirement to demonstrate value to society, in addition to safety, efficacy and quality, has become the new standard. A value proposition for new drugs will include focus on hard endpoints such as survival, but also longitudinal data on the patient’s life and the societal impact of their disease. In the Nordic countries, and also in the UK and other European countries, there is an increasing interest in using health and quality registers to establish this new type of evidence. A few examples out of the vast number of registers are presented, to illustrate their potential as a valuable source of knowledge.