Pharmacoepidemiology Studies: what Levels of Evidence and how can They be Reached?

¹ Service de Pharmacologie médicale et clinique, Université de Toulouse, INSERM 1027, Toulouse, France
² Affaires économiques, Laboratoire Roche, Boulogne, France
³ Service hospitalo-universitaire de Pharmacologie, Université de Bordeaux, INSERM U657, Bordeaux, France
⁴ Laboratoires Boehringer Ingelheim, Reims, France
⁵ Université de Bordeaux, Bordeaux, France
⁶ Inserm Transfert, Paris, France
⁷ Laboratoires Lilly, Suresnes, France
⁸ Université Lyon 1, Hospices civils de Lyon, Lyon, France
⁹ Haute Autorité de Santé, Saint-Denis la Plaine, France
¹⁰ Laboratoires Janssen, Issy-les-Moulineaux, France
¹¹ CIC-UPCET, Hôpital la Timone, Marseille, France
¹² IMS Health, Puteaux, France
¹³ Laboratoires GlaxoSmithKline, Marly-le-Roi, France
¹⁴ Université de Rennes 1, Rennes, France
¹⁵ Laboratoires Roche, Boulogne-Billancourt, France
¹⁶ Université Paris Descartes, Assistance Publique – Hôpitaux de Paris, Paris, France
¹⁷ Laboratoires Lundbeck, Issy-les-Moulineaux, France
¹⁸ Centre Hospitalier Universitaire, Nantes, France

Correspondence and offprints: Maryse Lapeyre-Mestre, Équipe de Pharmacoépidémiologie, INSERM 1027, Université Paul Sabatier – Toulouse 3, Service de Pharmacologie clinique, Faculté de Médecine, 37 allées Jules Guesde, 31000 Toulouse, France. Email: maryse.lapeyre-mestre@univ-tlse3.fr

Received: 15 March 2013
Accepted: 7 May 2013

Abstract

In pharmacoepidemiology studies, the nature of the research question will dictate the choice of methodological approach and the conditions for optimizing the level of evidence. Thus, to document the treated population and the modes of use of a new drug in real-life prescribing conditions, a descriptive approach through cross-sectional or longitudinal studies conducted on databases, or else ad-hoc studies, will be preferred. On the other hand, evaluation of the real-life “effectiveness” of a new drug will be based on cohort, case-control or scientific modeling, depending on the drug and the disease of interest. For questions involving drug risks and safety, it is the adverse effects profile that will guide the choice of study design, both for identification of the effect (signal) and assessment of causation. In all cases, in the post-marketing authorization (MA) setting, the evidence acquired in pre-MA studies serves as the basis for generating hypotheses. Whatever the research question and the method chosen to address it, the potential biases and their impact on the results need to be identified. In certain cases, a combination of several complementary approaches may prove preferable to a single study.