One of the traditionally popular sessions of the Interchange is the FDA Panel, which took place on Thursday, 4 November, in Baltimore. In the prior session that afternoon, chaired by CDISC Technical Architect, Dave Iberson-Hurst, presentations were made by Dr. Vicki Seyfert-Margolis, Senior Advisor of Science and Technology, Office of the Commissioner; Amy Malla (CBER); Dr. Chuck Cooper (CDER/Computational Science Center) and Dr. Steve Wilson (Biometrics, CDER).
Dr. Seyfert-Margolis spoke of the Vision:
- FDA has been working towards an electronic approach to acquire, receive, and analyze study data
- Electronic capture of study data is vital to integrate premarketing study data and post-marketing safety data to improve public health and patient safety
- FDA is working towards development of a scientific computing environment to support research and a development environment for our data and to combine other disparate data sets - science enclave
The Science Enclave and Data Repository (formerly Janus) are central to the Vision. She spoke about this being a hub for integrating data across the Agency to support the Science Enclave and regulatory decisions.
As a starting point for the Science Enclave, ARRA funding will be used to support legacy data conversion to populate this database which will be used to support Comparative Effectiveness Research (CER), which is NOT (she stated): solely about effectiveness; cost-effectiveness; intended as regulatory or directive; restricted to randomized controlled trials; exclusionary of clinical judgment or the circumstances of the individual patient; aimed at limiting or restricting health services. Rather, CER IS: focused on real-world circumstances and decisions; intended to help make decisions more consistent,
transparent and rational; useful in identifying gaps and uncertainties; fruitful ground for statistical involvement, including meta-analyses, analyses of retrospective or observational data, design/analysis of pragmatic, adaptive trials and consideration of heterogeneity in treatment effects. PACES is a Partnership in Applied Comparative Effectiveness Science for Medical Products (PACES).
[NOTE: Although not stated in this presentation, the Legacy Data Conversion contract has been awarded by FDA to ScenPro (management) partnering with Octagon (for the conversion) and CDISC (for development of any new standards needed) and the PACES work will be done with Johns Hopkins.]
The Long-term Benefits of this work to enable FDA Computing will take place over many years and are anticipated to be the following:
To the Agency…
- Credibility… improved scientific support for regulatory decisions by ensuring access to all available, relevant data (e.g., all clinical studies with Vioxx) and enhancing analytical, visualization, and other computational tools and techniques
- Efficiency… reuse data and tools
To the Public…
- Improve product safety
- Proactive surveillance where data standards move toward interoperability with wide-variety of internal (e.g., marketing application data, adverse event reports) and external (e.g., electronic health record) data sources
- Correlate animal toxicity studies with the human experience
- Faster, better informed regulatory decisions
Amy Malla spoke of implementation experiences at CBER with eSubmissions using CDISC standards. She specified the lessons learned and the ‘dos and don’ts’ for such submissions. Chuck Cooper spoke of the Computational Science Center Activities, particularly with respect to the Data Standards Needs and a checklist that FDA is going through with CDISC representatives. He explained how they are evaluating submissions with respect to their use of the CDISC standards and the common errors they are seeing. Steve Wilson spoke of the history of the use of standards within CDER and the progress made over the past decade. He gave evidence that we are getting to ‘the hard parts’ and that CDISC is focusing on addressing ‘the hard parts’. He ended his presentation with is signature surfing slide, this time featuring yours truly.
The panel was comprised of these four speakers along with Dr. Sean Kassim (DSI), Dr. Jay Levine (OC), and Ranjit Thomas (CDER). It was introduced by Mr. Frank Newby and chaired by Dr. Edward Helton, past-chair of the CDISC Board of Directors.
The audience asked questions of the panel, either by going to the microphones or by passing along paper versions to a ‘runner’ so that they could ask their questions anonymously. The panelists responded to these questions and encouraged the use of CDISC standards for eSubmissions and to keep open communications with FDA representatives during the process.
Dr. Rebecca Kush
CDISC CEO & President
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