shutterstock_208628524Ishita Srivastava and Divya Babbar co-authored this blog post with Qin Ye.

The FDA considers the use of real-world data and real-world evidence a top strategic priority, and on Dec. 6, it unveiled its much-anticipated RWE framework to support the development of drugs and biologics. The framework is aimed at evaluating the potential use of RWE for assessing a product’s effectiveness and focuses on increased stakeholder engagement to effectively leverage RWD and RWE for public health purposes.

The framework seems to be a continuation of various efforts that the agency is undertaking to drive the uptake of evidence-based decision-making: initiating a series of public workshops on RWE in early 2016, releasing guidance on the use of RWE for medical devices, and launching pilot projects on RWE methodology in 2017. The FDA believes that the RWE framework will serve as a road map for increased incorporation of RWD and RWE into the regulatory paradigm. 

Here are four key themes from the framework:

1. The efficacy of clinical trials: The FDA has experience using RWE to monitor and evaluate the post-marketing safety of drug products. However, there have been only limited instances where the FDA has accepted RWE to support drug product approvals, primarily in the setting of oncology and rare diseases. The framework shows that the FDA’s efforts are shifting to explore various study designs for efficacy assessments, including pragmatic clinical trials, observational studies and non-randomized, single-arm trials with external RWD control. 

To ensure the maximum uptake of these studies, the FDA is formulating guidance around various considerations for designing clinical trials that include pragmatic design elements, studies utilizing an external RWD control arm, and observational study designs using RWD.

2. Multiple dimensions of data (reliability, relevance and standardization): When it comes to RWD, the FDA is focused not only on the evaluation of the methodologies used to analyze the data but also on the reliability and relevance of the underlying data sources. While a reliability assessment includes checking the data for completeness, consistency and trends over time, a relevance assessment considers whether the data is fit for purpose. The FDA also stresses the data standards to be used for the efficient processing of RWD, focusing on how data should be structured, defined, formatted or exchanged between computer systems. The FDA will soon release formal guidance to address these critical parameters.

3. Novel data sources: The FDA shows immense interest in exploring strategies for addressing gaps in its commonly used data sources—EMR, claims data and registry data—and is set to expand its radar to include the use of mobile technologies, electronic patient-reported-outcome tools, wearables and biosensors. It’s also working toward publishing guidance on potential gaps in various RWD sources and identifying strategies to address them.

4. Stakeholder engagement: The framework acts as a foundation for both internal and external groups to collaborate on developing strategies to efficiently use RWD in drug development. The FDA is working on multiple programs to promote shared learning and collaboration among FDA staff and external stakeholders, including representatives from the industry, academia and patient advocacy groups.

The FDA’s framework presents many opportunities for pharma companies to harness RWD and RWE to the fullest. Here’s what pharma companies can do to get started:

  • Insist on collaboration. The FDA has been quite vocal about the collaborative approach that it’s aiming to take and calls out for collaboration among pharma, the FDA and industry experts to co-develop strategies for RWD- and RWE-driven solutions for the betterment of patients. 
  • Leverage RWD and RWE to improve clinical development strategy. The framework has signaled the industry to start including RWD and RWE as a part of pharma’s clinical development strategy. Organizations should proactively leverage this framework to establish a road map to build data partnerships and expand the clinical development team’s capability to better leverage RWD sources and data science. 
  • Focus on data expertise, standardization and federation. Creating an in-depth understanding of the data, widespread use of common data standards, and best practices for collecting, sharing and using RWD throughout the organization are the prerequisites in this strategic move toward RWE. The FDA is already working on identifying standards and methodologies for the collection and analysis of RWD, which clearly signals pharma companies to include standardization and a federated RWD model in their data strategy. 
  • Understand the infrastructure requirements. With the directive to continue incorporating RWE in future study designs, we envision a considerable reduction in cost and time requirements for conducting a clinical trial. This would require organizations to redirect their investments toward maintaining a well-established infrastructure to capture, integrate and analyze the plethora of RWD.
  • Move toward patient centricity. The framework’s focus on exploring patient-reported outcomes is a signal to the industry to look at patient-centric study designs and bring the patient’s voice into trials. Organizations could look at ramping up their data collection and analytical capabilities to handle novel data sources like patient-reported outcomes and wearables. Better capabilities for capturing data from these sources could not only help companies make drug development processes more efficient but also can help patients be better informed about the use of new treatments, and the benefits and limitations of the products for addressing specific needs.

The FDA’s RWE framework shows the organization’s gradual progress on the use of RWD and RWE for regulatory decision-making and gives direction to pharma companies to start incorporating RWD in their end-to-end processes, ranging from infrastructure to people development to devising the right data strategy. While the FDA continues the development of its formal RWE guidance, now is the time for the pharma industry to proactively partner with the FDA and others to prepare for the new era of drug development and help create better treatment options for patients.


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Topics: RWE, real-world evidence, RWD, real-world data, FDA, Pharma, pharma companies, patient centricity, pharma decision-making, data driven decisions