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

Published on Apr 1, 2014
· DOI :10.2139/SSRN.2637916
Jorge Mestre-Ferrandiz1
Estimated H-index: 1
,
Patricia Deverka1
Estimated H-index: 1
+ 1 AuthorsEmily Rosenberg1
Estimated H-index: 1
Abstract
The project reported in this Occasional Paper was intended to determine how changing demands for evidence are affecting drug development in five global pharmaceutical companies - Amgen, Eli Lilly, GSK, Novartis and Sanofi-Aventis. A literature review helped elucidate concepts and define focus. The authors then conducted semi-structured interviews with an international sample of 19 senior pharmaceutical executives in various positions in the five companies - R&D, outcomes research, medical affairs, and pricing and reimbursement. The intent was to capture information about the effect of CER/RE requirements on the drug development process now and in the future.
  • References (29)
  • Citations (4)
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References29
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#1Ranell MylesH-Index: 1
Qualitative data is extremely varied in nature. It includes virtually any information that can be captured that is not numerical in nature. Here are some of the major categories or types: In-Depth Interviews In-Depth Interviews include both individual interviews (e.g., one-on-one) as well as "group" interviews (including focus groups). The data can be recorded in a wide variety of ways including stenography, audio recording, video recording or written notes. In depth interviews differ from direc...
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Aim: Explore key factors influencing future expectations for the production of evidence from comparative effectiveness research for drugs in the USA in 2020 and construct three plausible future scenarios. Materials & methods: Semistructured key informant interviews and three rounds of modified Delphi with systematic scenario-building methods. Results & conclusion: Most influential key factors were: health delivery system integration; electronic health record development; exploitation of very lar...
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#1Adrian TowseH-Index: 24
#2Bengt Jönsson (HHS: Stockholm School of Economics)H-Index: 71
Last. Nancy DevlinH-Index: 42
view all 8 authors...
Background: Relative effectiveness has become a key concern of health policy. In Europe, this is because of the need for early information to guide reimbursement and funding decisions about new medical technologies. However, ways that effectiveness (does it work?) and efficacy (can it work?) might differ across health systems are poorly understood. Methods: This study proposes an analytical framework, drawing on production function theory, to systematically identify and quantify the determinants...
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#1Koonal ShahH-Index: 16
Last. Emily Nash Smyth (Eli Lilly and Company)H-Index: 8
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#1Sarah KleijnenH-Index: 5
#2Elisabeth George (National Institute for Health and Care Excellence)H-Index: 7
Last. Wim GoettschH-Index: 27
view all 13 authors...
Objective: Assessment of the effectiveness compared with alternative treatment(s)playsanimportantroleinmanyjurisdictionsindetermining the reimbursement status of pharmaceuticals. This type of assessment is often referred to as a relative effectiveness assessment (REA) and is carried out by many jurisdictions. Increased sharing of information across jurisdictions may save costs and reduce duplication. The objective of this study was to explore the main similarities and differences in the major me...
34 CitationsSource
#2S. Holmstrom (CGD: Center for Global Development)H-Index: 5
Last. Teresa K. WilcoxH-Index: 8
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Healthcare decision makers who determine funding for new medical technologies depend on manufacturers to provide evidence of the technology's efficacy, safety and cost-effectiveness. Constrained budgets and increasing reliance on formal health technology assessment (HTA) have created an abundance of external hurdles that manufacturers must navigate to ensure successful product commercialization. These demands have pushed pharmaceutical companies to adjust their internal structures to coordinate ...
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#1Robert Temple (CDER: Center for Drug Evaluation and Research)H-Index: 41
Background ‘Comparative effectiveness’ is the current enthusiasm, and for good reason. After knowing a treatment works, the most critical question is how it compares with alternatives. Comparative studies are not commonly conducted by drug companies and they represent a significant methodological challenge. Comparative data could include evidence of overall superiority to an alternative or advantages in identifiable subsets, for example, people who do not respond to or tolerate alternatives, or ...
21 CitationsSource
Last. Adrian TowseH-Index: 1
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The cost of R&D for a successful new medicine has been an important policy issue at least since the 1960s. Cost estimates matter not just because of intellectual curiosity or for industry understanding of its performance, but because they are a key aspect of the international debate about the reasonableness of pharmaceutical prices and the magnitude of the long-term investments involved. This publication reviews research published over the last three decades, which shows an increase in costs fro...
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In the current healthcare environment, bringing a new drug to market infers that it has not only received regulatory approval, but also a favorable decision from payers and health technology assessors. As healthcare costs across the globe escalate and the demand for better control of spending and balanced budgets increase, it has become a necessity that the therapeutic value of an innovative technology be demonstrated. Payers play an increasingly important role in the decision-making process of ...
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#1Amr MakadyH-Index: 6
Last. Wim GoettschH-Index: 27
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Abstract Background Randomized controlled trials provide robust data on the efficacy of interventions rather than on effectiveness. Health technology assessment (HTA) agencies worldwide are thus exploring whether real-world data (RWD) may provide alternative sources of data on effectiveness of interventions. Presently, an overview of HTA agencies' policies for RWD use in relative effectiveness assessments (REA) is lacking. Objectives To review policies of six European HTA agencies on RWD use in ...
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#1Derek J. Ward (University of Birmingham)H-Index: 8
#2Edward Hammond (University of Birmingham)H-Index: 1
Last. Andrew Stevens (University of Birmingham)H-Index: 30
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Objectives Recent decades have witnessed the development of highly innovative new antiviral drug therapies. However, there are concerns that rising costs and lengthening development times could have implications for future patient access to innovative new drugs. We sought to establish whether the time taken for the clinical development of new antiviral drugs launched in the UK had increased since the 1980s. Design and setting Retrospective observational study of all new antiviral drugs licensed ...
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#1Christopher-Paul Milne (Tufts Center for the Study of Drug Development)H-Index: 11
#2Joshua Cohen (Tufts Center for the Study of Drug Development)H-Index: 16
Last. Ranjana Chakravarthy (Tufts Center for the Study of Drug Development)H-Index: 4
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Abstract Purpose Meeting marketplace demands for proving the value of new products requires more data than the industry has routinely produced. These data include evidence from comparative effectiveness research (CER), including randomized, controlled trials; pragmatic clinical trials; observational studies; and meta-analyses. Methods We designed and conducted a survey to examine the industry’s perceptions on new data requirements regarding CER evidence, the acceptability of postapproval study t...
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Is there a broad, all-encompassing structure that meets the regulatory needs of medical innovations? This chapter will try to make the case that the answer is indisputably no, and provide some groundwork for improving and integrating regulation of drugs, devices, diagnostics and surgical innovations.
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