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By: Donna R. Rivera, PharmD., M.S., Associate Director for Pharmacoepidemiology, Oncology Center of Excellence (OCE) and Paul G. Kluetz, M.D., Deputy Director, OCE 

Expanding ways to generate evidence can lead to an array of benefits for medical product development, including more diverse patient representation and the potential to expedite the availability of safe and effective therapies. As data sources outside of traditional clinical trials become more accessible and methods for analyzing such sources become more robust, there are further opportunities to use real-world data (RWD) to evaluate safety and support evidence of effectiveness. 

Submissions to the U.S. Food and Drug Administration that incorporate the use of RWD are rapidly increasing, particularly in the field of oncology. When selecting a data source, RWD should be fit-for-purpose for the prespecified scientific study objective. To effectively review a study proposal, there should be sufficient information to allow the FDA to provide useful feedback to sponsors. Sponsors should consult relevant FDA guidance documents for specific recommendations regarding studies using RWD.

Our initial experience with oncology RWD submissions suggests that early proposals for studies incorporating RWD often lack basic elements of the study design and characterization of the data source to facilitate an appropriate evaluation from our review teams. Moreover, there is a wide heterogeneity in the types and formats of information received. This process often creates requests for additional information that leads to inefficiencies and unnecessary delays. 

The Oncology Quality, Characterization, and Assessment of Real-world Data (QCARD) Initiative

The cancer drug development community could benefit from an enhanced understanding of information needs that can facilitate the evaluation of initial RWD study proposals. To address this issue, the FDA Oncology Center of Excellence (OCE) established a collaboration with the Reagan-Udall Foundation to evaluate essential data elements that could provide a starting point for characterizing the data source(s) and study design of an early RWD study proposal. Importantly, the Oncology Quality, Characterization, and Assessment of Real-world Data (QCARD) Initiative effort is intended to improve the quality of initial study proposals and is not an evaluation of the quality of real-world data submitted within a regulatory review. 

The Oncology QCARD Initiative was designed to facilitate effective communication between study sponsors and protocol reviewers by evaluating the potential minimum elements for sponsors to consider when assembling submissions for review of a proposed study and RWD source(s). The Oncology QCARD Initiative was informed by input from an external group of epidemiology, biostatistics, clinical, and health services research experts. 

The objectives of the Oncology QCARD Initiative included:

• To review the published literature, guidelines, and guidances on RWD and dimensions of quality measurement.
• To engage stakeholders and obtain feedback on essential RWD study design elements and data characteristics. 
• To generate key study and data elements (Oncology QCARD) to facilitate initial review of studies incorporating RWD.

Insights from the Oncology QCARD Initiative have informed information requests sent by oncology review teams when they have received RWD study proposals that lack sufficient information. The FDA OCE is providing example data elements contained within these information requests to share these lessons learned. Details about the Oncology QCARD Initiative are available on the OCE Real World Evidence (RWE) website. This effort illustrates the commitment of the OCE Oncology RWE Program to collaborate with the community to provide practical ways to advance the appropriate use of RWD in oncology product development. A publication describing the Oncology QCARD Initiative collaboration and methodological approach is in development. 

The OCE believes defining relevant data elements to facilitate review of initial RWD study proposals can introduce review efficiencies given the increasing volume of oncology RWD submissions and may be able to inform broader efforts across therapeutic areas. Imperative to successful multidisciplinary drug development, we advise sponsors to engage early with the relevant oncology review division to obtain feedback on their individual development program. 

Questions? Please email OCERWE@fda.hhs.gov