Framework for FDA’s Real-World Evidence Program

Blog Posts - 29 January 2019


In December of 2016, the 21st Century Cures Act was signed into law and included regulations that hoped to make the production of medical technology and drug development more efficient, as a way to bring deliverable results to patients more quickly. Under the jurisdiction of the Cures Act, Section 505f was added to the Federal Food, Drug, and Cosmetic Act (FD&C Act) which required the Food and Drug Administration (FDA) to develop a framework for the Real World Evidence Program, a framework that would be used to analyze the viability of using real world evidence to identify additional indications for drugs already on the market.

On December 7, 2018, the FDA released the Framework for FDA’s Real World Evidence program, and the regulatory team at Veradigm has provided a brief overview below, including information from the report related to definitions, usability, and stakeholder involvement.

What is real-world data?

According to the FDA, real world data is routinely collected from a variety of sources, related to a patient’s health status and/ or delivery of health care. Real world evidence is clinical evidence of product usage, benefits, and/or risks, derived from analysis of real world data. Currently, real world data is defined to include patient generated data, medical claims and billing data, data generated from electronic health records (EHR), and product and disease registry data.

How is this data currently used?

The FDA has a long history of utilizing real world evidence to monitor and evaluate the safety of marketed drugs. The FDA Sentinel System, developed in response to a mandate in the 2007 FDA Amendments Act (FDAAA), has established a network of more than 18 research partners, including healthcare payers and academic institutions, with access to administrative claims data for over 100 million patients. It is analyzed both through rapid, distributed queries as well as by protocol-driven custom studies. The Sentinel System contains electronic health data from multiple queries and is used for the evaluation of drug product safety post-approval. Depending on the severity of the safety concerns of a drug, the FDA may develop a study utilizing the Sentinel system’s data prior to the approval of the drug.

How will the Framework for Evaluating Real World Data and Real World Evidence be Used in Regulatory Decisions?

With the growing availability of real world data and steeply increasing costs for bringing a drug to the market, currently greater than one billion dollars, the FDA is actively assessing the use of real world evidence for regulatory decision making. The initial focus of the real world evidence program will be for approval of new indications of drugs that are already on the market. To uphold the integrity of the FDA’s study program and consequential results, they have stated the following details will be considered under their new framework: the reliability and validity of the real world data being examined, whether enough real world evidence is generated from the study or trial to establish scientific evidence, and whether the regulatory requirements of the FDA are being met by the study.

What are potential gaps in gaps in RWD Sources?

While real world evidence documents diagnosis and treatment in “the real world”, the limitations on the use of the data for clinical evidence are widely known and well documented. Patient changes, such as an increase in joint pain, a worsening skin condition, or the continued decline into depression are not always recorded, and if recorded tend to be in differing methods by doctors as is required by their EHRs. Differences in medical coding vocabularies and free form text in physician notes make analyses and interpretation of evidence difficult. In administrative claims, the diagnoses are coded for the purposes of charge capture, not necessarily for accurately identifying patient status. To address the issue of the absence of standardization, the FDA will seek out new methods for reliably filling data gaps.

How will real world data be used in regulatory considerations for study designs?

The FDA is evaluating whether clinical study efficiency may possibly be improved under the deployment of the FDA’s new study and trial design. This efficiency may be enhanced through the inclusion of EHRs, mobile health technology, and other electronic data capture technology. The FDA has stressed that when utilizing these new technologies, it will be critically important to pay attention to an electronic records’ reliability, quality, and authenticity. To guarantee reliability of data, the FDA will utilize audit trails as well as record archival.

As it is established, the framework will work to advance the continued progression of medical technology, allowing companies like Veradigm to advance and continue transforming health, insightfully. Just recently Veradigm, in concert with Microsoft, published a whitepaper titled “Integrated Clinical Research”, which will serve as an operating model to enable research as a natural output of clinical practice. In the aggregation of information, the additional inclusion of various stakeholders will provide more data and will help to alleviate any bias potential. Detailed and defined, the FDA framework offers real promise to improving the future capabilities of medicine and the capabilities of associated partners, such as Veradigm.

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