Real Medical Benefit Assessed by Indirect Comparison

¹ INSERM, U669, Paris, France
² Université Paris-Sud et Université Paris Descartes, UMR-S0669, Paris, France
³ AP-HP, Hôpital Paul Brousse, Département de Santé Publique, Villejuif, France
⁴ LILLY France, Suresnes, France
⁵ Faculté de Médecine Laënnec, EA 3736, Lyon, France
⁶ Haute Autorité de Santé (HAS), Saint-Denis La Plaine, France

Received: 15 October 2008
Accepted: 20 November 2008

Abstract

Frequently, in data packages submitted for Marketing Approval to the CHMP, there is a lack of relevant head-to-head comparisons of medicinal products that could enable national authorities responsible for the approval of reimbursement to assess the Added Therapeutic Value (ASMR) of new clinical entities or line extensions of existing therapies.
Indirect or mixed treatment comparisons (MTC) are methods stemming from the field of meta-analysis that have been designed to tackle this problem. Adjusted indirect comparisons, meta-regressions, mixed models, Bayesian network analyses pool results of randomised controlled trials (RCTs), enabling a quantitative synthesis.
The REAL procedure, recently developed by the HAS (French National Authority for Health), is a mixture of an MTC and effect model based on expert opinions. It is intended to translate the efficacy observed in the trials into effectiveness expected in day-to-day clinical practice in France.