New opportunities of real-world data from clinical routine settings in life-cycle management of drugs: example of an integrative approach in multiple sclerosis

Abstract

The assessment and demonstration of a positive benefit–risk balance of a drug is a life-long process and includes specific data from preclinical, clinical development and post-launch experience. However, new integrative approaches are needed to enrich evidence from clinical trials and sponsor-initiated observational studies with information from multiple additional sources, including registry information and other existing observational data and, more recently, health-related administrative claims and medical records databases. To illustrate the value of this approach, this paper exemplifies such a cross-package approach to the area of multiple sclerosis, exploring also possible analytic strategies when using these multiple sources of information.

Keywords: :

• Benefit
• risk assessment
• Comparative effectiveness
• life-cycle management
• Multiple sclerosis
• Observational data
• Real-world evidence

Transparency

Declaration of funding

The study was funded by an unrestricted grant from Novartis Pharma AG, Basel, Switzerland. The funders (except the coauthors) had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

Declaration of financial relationships

G.C. and Y.G. have disclosed that they are paid employees and own shares in Novartis Pharma AG, Basel, Switzerland. D.R. has disclosed that he is a consultant for Novartis Pharma, Roche and MEDA Pharma. H.U. has disclosed that she has no significant relationships with or financial interests in any commercial companies related to this study or article. H.T. (H. Tumani) has disclosed that he is a consultant for Biogen Idec, Novartis Pharma, Merck Serono, Siemens, Teva, Genzyme. H.T. (H. Tilson) has disclosed that he is a member of the Novartis-sponsored Gilenya multiple sclerosis pregnancy advisory board.

CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgments

The authors take full responsibility for the content of the paper.