CDISC Standards Key to Meeting FDA Regulations, Research Goals

Despite considerable potential benefits from using common standards for collecting and reporting information from clinical trials, research sponsors and investigators have been slow to move in this direction. That appears to be changing as the December 2016 deadline looms for meeting the FDA requirement that all applications for new drugs and biologics compile and submit clinical trial data electronically and according to standardized formats. At the same time, electronic health records utilizing common nomenclature, content and structure provide a ready source of patient information with potential to facilitate the discovery of new therapies.   

Jill Wechsler

These developments are focusing more attention on efforts by the Clinical Data Interchange Standards Consortium (CDISC) to develop consensus-based standards for collecting data on certain critical diseases that facilitates regulatory submissions and reporting adverse events. The expectation is that such efforts will reduce the time and resources required to conduct clinical studies and thus accelerate biomedical R&D. And these developments stand to support calls for greater transparency in biomedical clinical research and in reporting study results.    These initiatives fit efforts by FDA commissioner Robert Califf to modernize the clinical research system. Califf has long championed strategies to replace the traditional “top-down” hub-and-spoke study model with a “neural network” system that shares data among multiple sites, including healthcare systems holding records data on thousands of individuals. Califf commented at the CDISC International Interchange in September in Bethesda, Md. that the current system for conducting clinical research is slow, unreliable, and labor intensive. Instead, he urged adopting approaches that can extrapolate data from a small number of study subjects to answer multiple research questions. Diverse data systems at different health operations need to bridge to a national network, Califf advised, adding that these methods should be tested to ensure that analysts draw sound conclusions from the data.    Such approaches appear to hold great promise for developing targeted cancer therapies, particularly personalized treatments that may be tested on small patient cohorts in streamlined clinical trials. These kind of studies can be informed by data from millions of cancer patients through the National Institutes of Health (NIH) Data Commons. But this strategy, explained Warren Kibbe, deputy director of the National Cancer Institute, at the CDISC conference, requires standards for collecting information from clinical trials, imaging studies, proteomics and other procedures.    Data standards are “at the heart of linking different data streams” to assess safety issues from thousands of safety reports and to “connect the dots” from pre-clinical and clinical studies to real world health issues, added Sean Khozin, senior medical officer in the Office of Hematology & Oncology Products (OHOP) in the Center for Drug Evaluation and Research (CDER). Thus, randomized clinical trials, he noted, may not be needed to answer every health care issue.    Many sponsors already are submitting applications to FDA that conform to the electronic Common Technical Document (eCTD) format, and in December that will be required for all submissions for new and generic drugs and for biologics, including amendments, supplements, reports and drug master files. In May 2018, the electronic submission requirement will extend to all commercial INDs. The hope is, as Califf noted at the CDISC conference, that e-submission of data to FDA will make it easier for agency reviewers to examine disparate information on different drug classes, and to monitor safety issues related to therapies new to the market.     A 2015 survey of biopharma company executives and researchers in academia and industry indicates that data standards adoption has been slow, but is increasing due to the need to ensure compliance with FDA’s e-filing policy, as well as similar requirements under consideration in Europe and Japan. The study by Accenture found that more firms have moved to adopt standards in the last five years, but many find the process challenging due to diversity in data repositories and management systems. While regulatory compliance is the key force driving adoption of CDISC standards, industry leaders also recognize that such efforts will enhance research and business operations.