Pharmacoepidemiology studies: what levels of evidence and how can they be reached?
Maryse Lapeyre-Mestre
(1, 2)
,
Claudine Sapède
(3)
,
Nicholas Moore
(4, 5)
,
Pascal Bilbault
(6)
,
Patrick Blin
(7)
,
Damien Chopy
(8)
,
David Evans
(9)
,
François Gueyffier
(10)
,
Laure Lacoin
(11)
,
Muriel Malbezin
(12)
,
Joëlle Micallef
(13)
,
Dana Morlet-Vigier
(14)
,
Sophie Muller
(15)
,
Emmanuel Oger
(16, 17)
,
Yannick Plétan
(3)
,
Gérard Pons
(18)
,
Patrice Verpillat
(19)
,
Caroline Vigneau
(20)
1
LEASP -
Epidémiologie et analyses en santé publique : risques, maladies chroniques et handicaps
2 Service Pharmacologie Clinique [CHU Toulouse]
3 Laboratoire Roche [Boulogne-Billancourt]
4 Department of Pharmacology
5 Laboratoire de Pharmacologie Expérimentale
6 Hôpital de Strasbourg
7 Département Hospitalo-Universitaire de Pharmacologie de Bordeaux
8 Neuro-Immunologie Virale
9 Laboratoire pharmaceutique Lilly France [Neuilly-sur-Seine]
10 Evaluation et modélisation des effets thérapeutiques
11 HAS - Haute Autorité de Santé [Saint-Denis La Plaine]
12 Janssen-Cilag [Issy-les-Moulineaux]
13 EuroMov - Euromov
14 IMS Health [Puteaux]
15 Laboratoire GlaxoSmithKline [ Marly-le-Roi]
16 CIC-IT Rennes
17 Service de Pharmacologie [Rennes]
18 U1129 - Epilepsies de l'Enfant et Plasticité Cérébrale
19 Neurologie et thérapeutique expérimentale
20 IGDR - Institut de Génétique et Développement de Rennes
2 Service Pharmacologie Clinique [CHU Toulouse]
3 Laboratoire Roche [Boulogne-Billancourt]
4 Department of Pharmacology
5 Laboratoire de Pharmacologie Expérimentale
6 Hôpital de Strasbourg
7 Département Hospitalo-Universitaire de Pharmacologie de Bordeaux
8 Neuro-Immunologie Virale
9 Laboratoire pharmaceutique Lilly France [Neuilly-sur-Seine]
10 Evaluation et modélisation des effets thérapeutiques
11 HAS - Haute Autorité de Santé [Saint-Denis La Plaine]
12 Janssen-Cilag [Issy-les-Moulineaux]
13 EuroMov - Euromov
14 IMS Health [Puteaux]
15 Laboratoire GlaxoSmithKline [ Marly-le-Roi]
16 CIC-IT Rennes
17 Service de Pharmacologie [Rennes]
18 U1129 - Epilepsies de l'Enfant et Plasticité Cérébrale
19 Neurologie et thérapeutique expérimentale
20 IGDR - Institut de Génétique et Développement de Rennes
Maryse Lapeyre-Mestre
- Function : Author
- PersonId : 11291
- IdHAL : maryse-lapeyremestre
- ORCID : 0000-0002-5494-5873
- IdRef : 057940118
Pascal Bilbault
- Function : Author
- PersonId : 773279
- ORCID : 0000-0003-0904-4600
François Gueyffier
- Function : Author
- PersonId : 178661
- IdHAL : francois-gueyffier
- ORCID : 0000-0002-9921-0977
- IdRef : 091433061
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.