Real-World Evidence Generation and Evaluation of Therapeutics Proceedings of a Workshop (2017) / Chapter Skim
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4 Generating and Incorporating Real-World Evidence into Medical Product Development and Evaluation: Building from Successful Case Studies
4 Generating and Incorporating Real-World Evidence into Medical Product Development and Evaluation: Building from Successful Case Studies
Pages 27-38
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From page 27... ...
• Preapproval pragmatic trials have been carried out in the United Kingdom, where the National Health System infra structure enables the real-time monitoring of safety outcomes. Clear guidance from FDA on whether such studies would be acceptable in the United States would be helpful.
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From page 28... ...
The question drove the approach: Because the comparator arm was continuing treatment with usual care, which would have involved multiple inhalers, it would not have been possible to conduct this evaluation through a traditional RCT in a controlled setting, observed Roddam. The investigators adopted broad inclusion criteria and, importantly, no other aspects of the care being rendered were changed, so the study provided a truly representative assessment of real-world effectiveness in the clinical setting in which the medication would be used.
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From page 29... ...
. Thus, through this real-world evidence approach, the first Salford Lung Study provided information that could be used to have meaningful conversations with patients about treatment options for COPD.
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From page 30... ...
In its national coverage determination, CMS approved the TAVR treatment with coverage under evidence development, whereby CMS requires that services or items be provided in the context of clinical study participation or that additional clinical data be collected to support further evidence development (CMS, 2014)
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From page 31... ...
• Research -- registry data are being incorporated into several differ ent research studies, including an evaluation of volume-to-outcome relationships that may inform the optimal number of TAVR sites, and a comparative effectiveness study comparing outcomes for surgical and non-surgical interventions. Workshop participants discussed opportunities and challenges to scaling the TVT Registry model as a mechanism for real-world evidence generation.
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From page 32... ...
Additionally, she emphasized that the full potential of registries in a learning health system depends on their ability to provide actionable information to providers in real-time, for example, by connecting to decision support tools. In the context of bringing use cases to scale, Mack showed a model proposed by John Laschinger, medical officer, FDA, representing a vision for a tiered national device registry infrastructure (see Figure 4-1)
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From page 33... ...
As examples of the kinds of studies that can be conducted using these large linked datasets to generate real-world evidence, Lesley Curtis, professor of medicine and director for Pragmatic Health Services Research, Duke Clinical Research Institute, shared her experiences with the FDA Sentinel Initiative and Shah discussed the Observational Health Data Sciences and Informatics (OHDSI) program.
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From page 34... ...
Additionally, distributed data networks for safety surveillance across Europe are being considered under the auspices of the Innovative Medicines Initiative, a public–private partnership involving the European Medicines Agency and the European pharmaceutical industry. Observational Health Data Sciences and Informatics Initiative The OHDSI community is a multistakeholder, interdisciplinary group of investigators working collaboratively to bring out the value of observational research and generate evidence that will improve health decision making through building open-access tools, best practice methods, and a large data network.
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From page 35... ...
3. Reusable Tools -- Recognizing that the kinds of queries the Mini-Sentinel operation center would need to run to extract data from health plans for safety surveillance would take a fairly standard form, efficiencies were created by generating a library of reusable tools as part of the Sentinel infrastructure.
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From page 36... ...
. Figure 4-2 depicts a model that FIGURE 4-2 Schema for leveraging OHDSI resources to inform patient care decisions and clinical studies.
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From page 37... ...
However, for that same patient, there may not be an obvious second-line treatment, and evidence generation becomes necessary, as depicted by central rings. Thus, when no clinical guideline based on high-quality RCT data is available, health care providers could use a decision support tool, represented by the flow chart shown in Figure 4-2, that leverages OHDSI's existing data networks to aggregate data sources -- such as EHRs, claims data, and even social media data -- and conduct analyses of treatment results for large cohorts of similar patients (when available in OHDSI databases)
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