The COVID-19 pandemic is unarguably the most consequential global event of 2020. Lockdowns have crippled the global economy. The impact of pandemic will likely cascade over this decade as well. Almost all the industries are undergoing massive transformation and healthcare is no different. The COVID-19 pandemic and its enormous impact on healthcare systems has accelerated the growth in adoption of real world evidence (RWE). For the life sciences industry, this new decade will likely be known as the ‘Decade of RWE’.
Real world experience is increasingly becoming important for regulatory decision-making. A core utility for real world data (RWD) is to fill in evidence gaps remaining after randomized controlled trials (RCTs). RWE use has evolved over the years from simply post-marketing safety surveillance to regulatory decision making and to substantiate reimbursement/coverage decisions.
RWE across product lifecycle
A recent report from the Boston Consulting Group (BCG) has grouped the applications for real world experience along the product life cycle into five categories:
- Support the Understanding of Diseases and Unmet Needs,
- Reduce Clinical-Development Cycle Time and Costs
- Support Pricing and Demonstrate Value
- Improve Commercial Spending and Effectiveness
- Support Pharmacovigilance and Label Extension.
RCTs vs. RWE or RCTs and RWE?
RCTs and RWE are not mutually exclusive but complement each other. The US FDA has released the RWE framework in December 2018, part of the work of the 21st Century Cures Act from 2016. Within the context of the FDA’s “totality of evidence approach” for evaluating regulatory submissions- RWE can play a pivotal role to demonstrate safety and effectiveness. The FDA has approved both drugs and medical devices based on regulatory submissions which have included RWE. The European Medicines Agency (EMA) released the EMA regulatory science to 2025 reflection paper which included promoting the use of high-quality RWD in decision-making as one of its strategic goals.
In a review article: Randomized Controlled Trials Versus Real World Evidence: Neither Magic Nor Myth, the authors provide commentary on why the future is not about RCTs vs. RWE but RCTs and RWE- for the assessment of efficacy, safety and comparative effectiveness. To address the unique evidentiary requirements of next generation innovative products like gene & cell therapies, digital therapeutics, precision medicine etc. it will be imperative to employ evidence generation strategies utilizing best of both worlds (RCTs and RWE) to achieve regulatory approvals & reimbursement.
In an article from JAMA, researchers assessed the feasibility of replicating clinical-trial evidence using real-world data. The authors report that only 15% of the 220 US-based clinical trials published in high-impact journals in 2017 could have been feasibly replicated using currently available observational data from electronic health records (EHRs), and insurance claims. These findings suggest the potential for RWD to complement but not replace RCTs.
COVID-19 and RWE
COVID-19 presents an opportunity for real world experience to be leveraged to understand the pandemic and its long-term effect on patients, healthcare systems, and the economy. According to the US FDA, the “silver lining” of COVID-19 is that the pandemic has forced us to get comfortable with real-world data technologies like remote monitoring, telemedicine, and the ability to use RWD to fill in gaps in RCTs. In the long term, real-world data will assist in understanding the way coronavirus impairs the body, new health risks it creates and emerging long term complications. The RWE gathered from longitudinal studies of COVID-19 patients and vaccine recipients is likely to play a significant role in achieving these goals.
The way forward
Results from a recent RWE benchmarking study by Deloitte revealed that companies are expecting a shift in the application of RWE in the next two to three years with the highest impact being in R&D, including supporting regulatory filings and augmenting clinical trials. A wide range of benefits are also expected in the form of cost reduction in post-marketing commitments and execution of clinical trials. As lack of research-grade data is hindering RWE efforts in R&D, organizations are more likely to build robust centralized cloud-based platforms with comprehensive capabilities, including knowledge management and self-service analytics applications. Currently, some are entering into strategic partnerships to access new sources and to expand access to RWD globally.
The main challenges with use of RWD are quality of the data, ownership of patient data, combined with privacy requirements (HIPAA, GDPR etc), and interoperability. Some of these challenges are being tackled using integrated delivery networks. These networks are exploring use of research-centric Observational Medical Outcomes Partnership (OMOP) common data model and the Clinical Data Interchange Standards Consortium’s (CDISC) Study Data Tabulation Model (STDM) standard for regulatory submission. The Fast Healthcare Interoperability Resources (FHIR) specification is gaining traction to enable the exchange of EHRs. Linking these standards efficiently to enable better data access, improved data quality and greater insights at speed will be a game changer in the RWE world.
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