Examining the Impact of Real-World Evidence on Medical Product Development Proceedings of a Workshop Series (2019) / Chapter Skim
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2 Perspectives on Real-World Evidence
2 Perspectives on Real-World Evidence
Pages 13-24
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From page 13... ...
• Delivery systems value medical practices that are supported by quality, relevant evidence that demonstrates value for patients; typical evidence generation practices for such evidence include traditional clinical trials, delivery system data collection, and reviews of current practices and literature. (Ford, Horberg)
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From page 14... ...
Andrew Bindman, professor of medicine, epidemiology, and biostatistics at the University of California, San Francisco, said there are many ways to arrive at scientifically valid evidence, so the means by which evidence is generated should be adaptable to the needs of the end user. Robert Califf, vice chancellor of health data science at Duke University and scientific advisor at Verily Life Sciences, agreed, and said that different end users not only have different evidence needs, but they also need different degrees of certainty in the data.
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From page 15... ...
For some rare conditions, there will never be the possibility of a highquality, randomized clinical trial of sufficient size; for other conditions, the time that it would take to generate proper data is significant, and during that time, patients are suffering. This is an ethical "tightrope" that patients, providers, regulators, and payers all must walk, said Sherman.
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From page 16... ...
. In particular, he agreed that a "clinical trial is often an idealized version of what we would all hope the care would be." In the real world, there are issues with patient adherence and care delivery that can impact the effects of a medical intervention.
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From page 17... ...
In addition, despite the emphasis on evidence-based medicine, individual care decisions are often based on discussions and experiences of providers and patients. Overall, KP aims for "collaboration" within the organization, with the goal of "getting what's best for the patient," concluded Horberg.
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From page 18... ...
In the system's three community hospitals, there are about 450 patients in a clinical trial at any one time, with 23 research coordinators supporting them. This project marks a transition to collecting evidence outside of the traditional academic health center, said Ford.
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From page 19... ...
than we do when we sit here primarily as professionals." Terry briefly told the story of how she transitioned from a mother of two with a background in religious studies to a researcher who is involved in clinical trials with four different therapies. Terry's children were born with a rare genetic condition called pseudoxanthoma elasticum (PXE)
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From page 20... ...
However, they all share the same underlying data structures and are therefore interoperable across diseases. There are currently 45 communities using PEER to build the database they need to get industry attention or to start clinical trials themselves, said Terry.
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From page 21... ...
Stakeholders could build on the successes of these communities, and help to facilitate the rigorous collection and analysis of patient-generated data. DISCUSSION Joanne Waldstreicher, chief medical officer at Johnson & Johnson, and Eleanor Perfetto offered some reflections on the presentations by Sherman, Horberg, Ford, and Terry, as well as their own perspectives on the issue of RWE generation.
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From page 22... ...
In a prospective study, researchers obtain active consent from patients, whereas in studies involving RWD, patient consent and privacy are much less central, he said. Roundtable contributors also said that EHRs and claims are "not authentic sources of real patient data" because these sources do not include information about patient preferences or experiences.
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From page 23... ...
When observational study designs are used, the bar for rigor and quality should be as high as it is for clinical trials, said Waldstreicher: Clinical trials have a number of requirements that grant collective confidence in the data, and observational trials could have similar requirements. Observational trials should be reported with transparency about the sponsor of the trial, the study design, the methodology, the protocol, and the analysis plan, and observational trials should be registered just like clinical trials, said Waldstreicher.
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From page 24... ...
Horberg concurred that payers have a unique role to play; he said that KP feels "a strong sense of obligation to contribute to medical knowledge." For Horberg, changing the system comes down to improving research practices. Research must be based on a sound scientific question that is the "right" question for the community and the patients, he said.
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