Real-World Evidence Generation and Evaluation of Therapeutics Proceedings of a Workshop (2017) / Chapter Skim
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5 Potential Strategies for a Way Forward
5 Potential Strategies for a Way Forward
Pages 39-50
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From page 39... ...
With proper controls, calibration, and demonstrated repeatability using independent datasets, observational studies can yield robust evidence that can be used for causal inference in specific circumstances. (Berger, Califf, McClellan)
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From page 40... ...
• Some individual workshop participants suggested straightfor ward next steps: connecting existing initiatives and databases; developing and iterating an idealized test case for using real world evidence throughout product development; and stan dardizing inputs for stakeholder engagement. During the closing session of the workshop, individual panelists reflected on the day's presentations and discussions, and identified practical strategies to generate momentum toward the evidence generation paradigm of the future, in which real-world evidence is systematically incorporated into processes for the development and evaluation of medical products.
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From page 41... ...
The ability to share data seamlessly across systems involves broad stakeholder engagement and a commitment to transparency. It was emphasized by several individual workshop speakers, however, that robust engagement of stakeholders in a learning health system will be bolstered if data partners, whether they are patients, con umers, s providers, health plans, or health systems, trust that their data will be protected and that their confidentiality and privacy needs will be responsibly addressed.
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From page 42... ...
. Several individual workshop participants noted that bringing to scale the kinds of successful initiatives highlighted at the workshop will be bolstered by a significant investment in infrastructure.
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From page 43... ...
In the context of these discussions, a number of individual participants emphasized that evidence should be fit-for-purpose, and the optimal methodology will depend on the research question. Study Design Discussing the relative value of different study designs, Califf emphasized that although RCTs are not the answer to every question, randomization has a critical role to play in ensuring that clinical trials provide definitive results.
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From page 44... ...
McClellan added that, for validation purposes, results from observational studies can be compared to RCT results if available, as was done during the early years of FDA's Sentinel Initiative. Recognizing that the strength of evidence from observational studies falls short of that from well-controlled clinical trials, Berger and McClellan emphasized that with proper controls, calibration, and demonstrated repeatability using independent datasets, secondary data can nevertheless be used for causal inference.
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From page 45... ...
Noting that common data models have been validated by application to a wide range of datasets used to conduct realworld studies, Shah suggested that a shared standard dataset -- on the scale of millions of patients -- could be used for benchmarking to facilitate more rapid progress in methods development in the real-world evidence realm. McClellan added that with more validated methods, there may be less need to share or aggregate sensitive raw data and more evidence could be generated using "cloud-based" approaches as described for Sentinel and OHDSI, where patient-level data are retained behind the firewalls of data partners.
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From page 46... ...
Tunis underscored the need to understand what motivates clinicians to engage. The following possible incentives for health care providers were suggested by individual workshop participants: • Communicate the importance of provider participation -- If health care providers are asked to take on additional responsibilities beyond their clinical burden to assist with patient recruitment and data collection for research purposes, it will be important for them to understand why their participation is critical and how the research could benefit their patients.
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From page 47... ...
to advance the use of real-world evidence for the evaluation of medical devices, McClellan cited the NEST initiative. The Role of Payers Aligning payments for drugs and devices with payment reforms that are taking place in the health care industry would put drug and device manufacturers "on the hook" along with providers for demonstrating better patient population outcomes and lower total cost of care, said McClellan.
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From page 48... ...
SOME PRACTICAL NEXT STEPS McClellan brought the workshop to a close by asking panelists to identify practical next steps that can be initiated now that would significantly advance the generation and application of real-world evidence and our understanding of how medical products -- those on the market today and those in the pipeline -- perform in the real world. Connecting Existing Systems and Initiatives At the federal level, different agencies are spending significant energy and resources creating separate systems and initiatives to address similar and connected goals (see Box 5-1)
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From page 49... ...
Creating a Standard Process for Stakeholder Engagement Several workshop participants expressed frustration with the current sequential process for evidence generation, which is inefficient and does not meet the needs of all stakeholders, often necessitating post-hoc efforts to address gaps. The evidence generated to support regulatory approval, for example, may not meet payer requirements for coverage determinations, resulting in a need for additional studies.
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