Methodological expectations for demonstration of health product effectiveness by observational studies.

The issue of assessing the effectiveness of health technologies (drugs, devices, etc.) through observational studies is becoming increasingly important as registration and market access agencies consider them in their evaluation process. In this context, observational studies must be able to provide real demonstrations of a level of reliability comparable to those produced by the conventional randomized controlled trial (RCT) approach.

Place of patient-reported outcomes & experiences measurements (PROMS/PREMS) in the assessment and pricing of Health technologies in France.

In the context of health technologies assessment, patient-reported outcome measures (PROMs) have become assessment criteria that are expected by evaluation agencies along with the other usual clinical criteria. PROMs instruments measure all aspects of patient experience in connection with their health: symptoms, activities of daily living (physical function, sleep, etc.), various aspects of health-related quality of life (QoL), compliance, global impression of change in wellbeing.

High rate of adefovir-lamivudine combination therapy in nucleoside-naïve patients with chronic hepatitis B in France: results of a national survey in 1730 patients.

Background/aims: This study describes the types of therapy used in chronic hepatitis B (CHB) in France and patient characteristics according to therapy.

Methods: This was a descriptive, multicenter, retrospective study in 1730 patients (54 centers). We collected information about demographics, epidemiology, severity of hepatitis B virus-related liver disease, antiviral therapy, response (hepatitis B viral DNA and alanine aminotransferase normalization changes), dose modification, or treatment interruption.

Clinically relevant genotype interpretation of resistance to didanosine.

We analyzed the didanosine (ddI) arm of the randomized, placebo-controlled Jaguar trial in order to define a genotypic score for ddI associated with virologic response. In this arm, 111 patients experiencing virologic failure received ddI in addition to their current combination therapy for 4 weeks. The impact of mutations in the reverse transcriptase gene on the virologic response to ddI was studied in univariate analysis.