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The Current Drug Development Paradigm: Responding to US and European Demands for Evidence of Comparative Effectiveness and Relative Effectiveness

Pharmaceutical companies face rapidly evolving regulatory and reimbursement evidentiary expectations linked to the clinical and economic realities confronting the healthcare system. Health care spending pressures, a lack of information to guide the efficient use of new technologies, and consequent political momentum are promoting the need for an enhanced generation of evidence to inform post-regulatory clinical and coverage decision-making. This white paper serves to ascertain how the current drug development paradigm at five global pharmaceutical companies is evolving in response to perceived demands for evidence of comparative effectiveness and relative effectiveness, in particular, from payers and HTA bodies, but also from clinicians and patients.

In the United States (US) and Europe, and indeed, globally, pharmaceutical companies are facing rapidly evolving regulatory and reimbursement evidentiary expectations linked in large part to the clinical and economic realities confronting their respective healthcare systems. Specifically, demands for comparative effectiveness research (CER) and relative effectiveness (RE) evidence have been driven by: (1) health care spending pressures; (2) lack of information to guide the efficient use of new technologies; and (3) consequent political momentum to promote enhanced generation of evidence to inform post-regulatory clinical and coverage decision-making. CER (the term commonly used in the US) and RE (the term commonly used in Europe) both share a focus of producing evidence about how treatments work compared with existing alternatives under circumstances of usual care.

The purpose of this work has been to ascertain how the current drug development paradigm at five global pharmaceutical companies is evolving in response to perceived demands for evidence of comparative effectiveness and relative effectiveness, in particular, from payers and HTA bodies, but also from clinicians and patients. To accomplish this goal, we first undertook a targeted literature review (primarily to provide context and help to identify some themes for the interview programme), followed by a semi-structured interview program with an international sample of 19 senior executives (12 identified as CER experts and seven as RE experts). These executives hold positions in clinical development; health economics and outcomes research (HEOR); medical affairs; and pricing, access and reimbursement (PAR) across five global pharmaceutical companies. Our goal was to develop more detailed information about how these organisations currently conceptualise CER/RE, how they have begun to modify their internal processes to respond accordingly, and what they perceive to be the enablers and barriers for continued adaptation in response to both internal and external factors confronting the pharmaceutical industry.

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