Quantification et valorisation du progrès thérapeutique médicamenteux par la Haute Autorité de santé

Abstract

The French National Health Authority has delegated to the Transparency Commission (TC) responsibility for defining and quantifying therapeutic progress and for certifying the therapeutic added value of new drugs. It is essential to distinguish between pharmacologic innovation and therapeutic progress. The TC considers that a new product offers therapeutic progress if it: 1) improves patient management, 2) represents a significant clinical breakthrough, and/or 3) meets a previously uncovered need. Therapeutic progress must be supported by quantitative or qualitative clinical evidence of improved therapeutic tolerance, compliance or maintenance. The TC measures progress in terms of the enhancement of therapeutic value (ETV) relative to existing products. Ideally, ETV should be evaluated in head-to-head comparisons, but companies often prefer placebo-controlled studies, meaning that indirect comparisons are unavoidable. ETV is graded in five levels, ranging from I (major progress) to V (no progress), and is attributed for a specific target population. The ETV only measures expected therapeutic progress, to be confirmed in post-registration studies. TC decisions are taken into account by the commission that determines drug prices. One pending economic issue involves "me-too" products. Indeed, scientific committees cannot be expected to regulate market competition--for example, to decide when to inform decision-makers that the number of statins or betablockers on the market is sufficient to cover requirements. Another delicate problem concerns efforts by brand-name drug companies to circumvent generic drugs, notably by introducing fixed-dose combinations. Although the assessment of therapeutic progress is based on objective, verifiable and reproducible criteria, it can only be carried out fairly within a collaborative framework independent of pharmaceutical companies, healthcare insurers and consumer associations.