Global Regulatory Milestones in Real World Data and Evidence
Real-world evidence (RWE) is clinical evidence about the use and potential benefits or risks of a medical product, derived from real-world data (RWD). In 2018, the FDA established a framework for evaluating RWE to support new drug indications or post-approval study requirements. The FDA has a history of using RWD and RWE for postmarket safety monitoring and, though less frequently, for supporting effectiveness. Recent advances in RWD availability and analysis have enhanced the generation of robust RWE for FDA regulatory decisions.
RWD and analytics can speed up drug development and offer insights beyond what clinical trials can provide. It refers to healthcare information collected outside of clinical research environments. This includes:
Electronic health records (HER)
EHRs are electronic systems that store health-related data collected during medical care in practices, clinics, and other settings from various sources, creating a network of patient clinical information. They can also include software for streamlined physician ordering (CPOEs) with safety features, as well as tools that assist physicians in clinical decision-making using the latest guidelines (CDSS). Examples- lab results, treatment plans, and radiology images.
Disease and product registries
Disease and product registries that systematically gather data on all eligible patients are valuable resources for capturing crucial safety information. These registries often enroll patients who are not only different from those in clinical trials but also more complex, considering factors like the severity of their underlying conditions, comorbidities, and concurrent medications. Examples- the Alzheimer’s Prevention Registry, GenomeConnect, and Rare Disease Registry Program
Claims and billing data
These are data generated when healthcare providers submit payment requests to health plans, offering insights into the interactions among patients, providers, and insurers. Example- which encompasses information from health insurance companies about procedures and demographics
Data from wearables and health monitoring devices
Wearable device data plays a crucial role in drug development and regulatory approvals by delivering continuous, real-time information on patient responses to medications, tracking side effects, and assessing medication adherence. This data enhances traditional trial methods and provides valuable insights during clinical trials. For example, heart rate tracking from devices like an Apple Watch or Fitbit.
In 2014, Japan’s PMDA issued its first guidelines on using real-world data (RWD) for pharmacoepidemiological studies in drug safety assessment. Global regulators like the US FDA, Health Canada, EMA, and China’s NMPA are leading efforts to incorporate RWD into regulatory decision-making. The US FDA and Health Canada now accept observational data for efficacy assessments, while the EMA is exploring registry data for rare diseases, emphasizing the need for standardized approaches. The 21st Century Cures Act of 2016 aimed to speed up medical product development, prompting the US FDA to create a framework in 2018 for utilizing real-world evidence (RWE) to support new drug indications and meet post-approval study requirements. This framework also applies to biological products under the Public Health Service Act.