The need and challenges of complementing observed efficacy from RCTs with effectiveness from RWD: The case example of bone therapies in Post-menopausal osteoporosis population

Over the past 15 years, a number of bone remodelling agents have become available, offering osteoporosis patients and their treating physicians more options. It is also now well recognized that, following regulatory approval, data regarding the effectiveness of these agents in actually preventing fractures, in routine clinical practices, is necessary. The case for these evidences of effectiveness using RWD is well articulated by different reimbursement and HTAs bodies. Sponsors, at time, can also see these effectiveness studies and their comparison, as additional differentiation opportunity. However, there are numerous practical and methodological challenges in obtaining such data, among which that of confounding. After a brief review and discussion of some of the above-mentioned challenges, and a brief review of the limitation of some of the classical statistical tools in addressing some of these challenges, this talk exemplifies the use of patients as their own control design to effectively assess the effectiveness of bone remodeling agents usin RWD and thus facilitate an indirect comparative effectiveness instead.

About the speaker
Maurille Feudjo Tepie

Dr. Maurille Feudjo Tepie is an Observational Research Director at Amgen and leads the Amgen European Team of the Centre for Observational Research. Prior to joining Amgen in 2009, he spent six years in the Worldwide Epidemiology Department at GSK. Maurille obtained his PhD in Medical Statistics from the London School of Hygiene and Tropical Medicine and then completed a one year post-doctoral fellowship working on a collaborative methodological project between GSK and the London School of Hygiene and Tropical Medicine, under the supervision of Profs Stuart Pocock and Steven Evans. He has considerable experience designing and executing both retrospective and prospective observational studies to inform drug development and drug commercialisation and is a keen contributor to scientific discussions pertaining to the challenges and/or opportunities offered by such studies. He has been active in European-wide initiatives aimed at improving biomedical research (IMI PROTECT and EMIF) and is also the chair of the PSI Epidemiology Special Interest Group.

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