Comparative and Relative Effectiveness: A Challenge for Health Systems, Regulators, or Pharmaceutical Companies?

Abstract

Looking at how well drugs work in routine clinical practice, rather than only in experimental randomised controlled clinical trials (RCTs), is increasingly seen as essential for a proper assessment of both net benefit (health gain minus harm and value (net benefit minus net cost). Establishing net benefit involves estimating effects in routine clinical practice, termed comparative effectiveness research (CER) in the US and relative effectiveness (RE) research in the European Union (EU). Yet, assessments for both market authorisation by drug regulatory authorities (DRAs) — such as the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) — and ‘at launch’ appraisals by health technology assessment (HTA) bodies acting on behalf of payers — such as France’s HAS and Germany’s IQWiG — typically use efficacy and relative efficacy data.