Examining the Impact of Real-World Evidence on Medical Product Development Proceedings of a Workshop Series (2019) / Chapter Skim
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4 Barriers and Disincentives to the Use of Real-World Evidence and Real-World Data
4 Barriers and Disincentives to the Use of Real-World Evidence and Real-World Data
Pages 39-50
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From page 39... ...
• Randomized controlled trials (RCTs) are the traditional design for evidence-generating clinical research, while observational database studies are seen as riskier due in part to less precise data; hybrid approaches such as pragmatic trials and cluster randomized designs require some real-world considerations, but combine some advantages of both RCTs and RWE studies.
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From page 40... ...
Presenters included researchers, product developers, and data aggregator and nalytics a companies. THE USE OF REAL-WORLD EVIDENCE AND REAL-WORLD DATA IN PRODUCT DEVELOPMENT Elliott Levy, senior vice president of global development at Amgen Inc., began by challenging the notion that there are perverse incentives that encourage companies to develop drugs in ways that are unnecessarily time consuming and costly.
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From page 41... ...
Lack of Knowledge and Awareness Levy noted that many of the people working in product development are those "who are steeped in the randomized clinical trial model, who have benefited from the prestige and scientific cache of the randomized clinical trial, and who have deep faith in it and, to some degree, distrust for noninterventional methods." The physician scientists who lead product teams are accustomed to and comfortable with working with traditional forms of evidence generation, and simply are not aware of the potential uses and benefits of RWE. Talents and Capabilities Few people working in product development have experience conducting observational or non-interventional research, said Levy.
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From page 42... ...
In particular, he said, the lack of a clear regulatory pathway means there is less willingness to mobilize resources and develop capabilities for an approach that may not be acceptable to regulators. MAKING CHOICES ABOUT RESEARCH DESIGN Clinical researchers have choices about how to design their research, said Daniel Ford.
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From page 43... ...
Clinical researchers need support, training, and tools to choose appropriate methodologies and to feel comfortable working with RWE and hybrid methods, concluded Ford. ACCELERATING EVIDENCE GENERATION THROUGH DEFRAGMENTATION One of the biggest barriers to the adoption of RWE and RWD, said Marcus Wilson, president of HealthCore, Inc., is the fact that there is a "natural gravitational pull" back to the old, traditional ways of doing things -- in this case, RCTs.
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From page 44... ...
Data sharing among stakeholders -- even if it is beneficial 1 Cal INDEX eventually merged with another health information exchange organization and the project is now known as Manifest MedEx.
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From page 45... ...
EVIDENCE HIERARCHIES A major barrier to the adoption of RWE, said Hui Cao, executive director, Center of Excellence for RWE, Global Medical Affairs, Novartis, is a "fixed mindset" introduced in medical school that evidence from RCTs is the best possible evidence. There are "evidence hierarchies," said Cao, that place RCTs at the top, and put RWE at level 2 or even lower.
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From page 46... ...
For example, patients and payers are demanding proof of value of new interventions compared with standard of care, and the move to precision medicine requires generating evidence for smaller and more diverse subgroups. The traditional approach to health research, said Doyle, involved a systematic, methodical process of solving a problem for a single, isolated stakeholder.
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From page 47... ...
Researchers have already realized some of the advantages of the use of RWD -- anecdotally, Doyle reported that recruitment times are being reduced and recruitment rates are increasing, start-up time lines are compressed, and the cost of evidence generation has been reduced. Using RWD and RWE in clinical research, said Doyle, addresses one simple but often overlooked fact: "Real-world patients are fundamentally different than clinical trial patients." No matter how well a clinical trial is conducted, questions remain about how the findings will extend to more diverse patient populations, how the lack of perfect adherence will affect outcomes, or what the longer term outcomes will be.
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From page 48... ...
is more efficient Agent + comp. FIGURE 4-3 Mosaic methodologies to blend randomized controlled trial (RCT)
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From page 49... ...
QuintilesIMS was building an evidence platform for lung cancer, and had designed it with input from multiple experts, including oncologists, epidemiologists, and patient advocates. After developing a dozen protocols, the designers decided to "hit the pause button" and test the design with patients and physicians in the real world.
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From page 50... ...
A primary goal for the research community, said John Graham, is to understand and accept that there is a role for RCTs, prospective observational studies, retrospective observational studies, and many other methodologies that span the spectrum. Together, the wealth of different data sources and methodologies can come together to give the full answer to the research question.
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