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To further streamline product development and facilitate post market safety surveillance, the agency has developed a new Framework for Real-World Evidence that spells out opportunities and concerns in advancing these approaches.
Patient experience with medical treatments recorded in electronic health records, insurance claims, patient registries, and newer mobile technologies can help shape the design of clinical research, expand product use, and assess the effects of new therapies in the real world, according to FDA leaders. To further streamline product development and facilitate postmarket safety surveillance, the agency has developed a new Framework for Real-World Evidence that spells out opportunities and concerns in advancing these approaches [available at https://www.fda.gov/downloads/ScienceResearch/SpecialTopics/RealWorldEvidence/UCM627769.pdf].
In unveiling the framework, FDA commissioner Scott Gottlieb emphasized the importance of leveraging information gathered from patients and providers to “inform and shape the FDA’s decisions across our drug and biologic development efforts,” [see https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627760.htm]. Expanded and appropriate use of real world data (RWD) to develop real world evidence (RWE) can support oversight of medicines throughout the product life-cycle, including clinical trial design, patient recruitment for research, expansion of product labels to include additional indications and new dosage forms, and postmarket monitoring of product effects.
FDA encourages greater use of RWD and RWE specifically to help sponsors expand product labels, such as by adding an indication, changing dose or route of administration, treating new patient populations, and providing additional safety information. The agency articulates a three-part approach to ensure that RWE is reliable, relevant and complements and supports existing information. RWD may be particularly useful in evaluating uncommon conditions or rare tumor types and, where appropriate, may replace the need for randomized controlled post-marketing studies.
In addition to encouraging use of available sources of RWE, Gottlieb looks to expand data collection through mobile technologies such as wearable devices and biosensors. Further guidance will explain more specifically how sponsors may use these methods to fill gaps in data. Additional advisories will address methods for incorporating data from routine clinical care into clinical trials and the use of observational studies to generate effectiveness data to support regulatory decisions.
The Framework fulfills requirements of the 21st Century Cures Act, which has already generated FDA projects to validate the potential of RWE to replicate the results of clinical trials. FDA has expanded its Sentinel System to make greater use of RWD and RWE generated by electronic health records, insurance claims and registries to monitor and evaluate approved drugs and biologics, and aims to further support such efforts. The agency also is working with international groups to advance efforts for making better use of information from patients and providers throughout the lifecycle of a medical product.
Despite ongoing skepticism that RWE provides comparably valid and objective data as from randomized clinical trials, the development of clear methods and standards for collecting and using this information, “may encourage others to accept high quality studies” in this area, commented Jennifer Graff, Vice President of the National Pharmaceutical Council. “There is so much quality data that is available,” she noted, “let’s not waste it.”
Jill Wechsler is Pharmaceutical Executive’s Washington Correspondent. She can be reached at email@example.com