Future challenges for health economics and health technology assessment of biological drugs

Abstract

In this paper, some present and future challenges of health economics and health technology assessment (HTA) of biological drugs are discussed in general with particular emphasis of their utilization in rheumatology. The development and challenges of HTA in the new European Union member states are briefly touched to evaluate how their HTA agencies are coping with the increased demand of the funders and policy makers. The most important question is whether more methodological development is needed or more local data should be required (and what kind) or more international collaboration is the solution or do we need both at the same time? Biological agents produced through genetic engineering (the so-called biologic Disease-Modifying Antirheumatic Drugs, DMARDs; i.e. tumour necrosis-alpha inhibitors, T-cell and B-cell targeted therapies, interleukin-1, -6 inhibitors) brought significant therapeutic advantages in rheumatoid arthritis (RA) treatment by improving outcomes beyond what was possible with conventional DMARDs. Therapeutic advantages are associated with increased risk of side effects as well as significant budget impact thus high attention is paid to their clinical efficacy, effectiveness and cost-effectiveness by professionals, policy makers and funders. This increasing interest can be clearly mapped in the medical literature: in PubMed, the number of publications indexed to medical subheadings ‘‘Arthritis, Rheumatoid’’ and (‘‘Cost and Cost Analysis’’ or ‘‘Economics’’ or ‘‘Cost of Illness’’) has increased from 261 to 873 since the registration of the first biological drug in RA (1998‐ 2010). However, one decade after the introduction of the first biological agent, we are facing new challenges and some ‘‘old ones’’ are still unanswered. The most important ones are as follows: