Over the past couple of years, the U.S. Food and Drug Administration (FDA) has taken a hard-line stance against drug products that were approved under the accelerated approval pathway, but have not met their post-marketing or confirmatory trial commitments. Drug developers have often balked at the cost and difficulty of conducting confirmatory trials once a product is already widely available. Many at the FDA and at other regulatory agencies such as the European Medicines Agency (EMA) are considering the use of real-world data (RWD) as an alternative to traditional confirmatory studies. These agencies are already using real-world evidence (RWE) to make faster decisions for cancer drugs by leveraging data from various sources outside of traditional clinical trials. RWE in this case refers to a broad definition of data collected from sources such as electronic health records (EHRs), claims data, patient-generated data, and social media.
One example of the use of RWE in cancer drug approvals is the recent approval of pembrolizumab for the treatment of solid tumors with high microsatellite instability or mismatch repair deficiency. The FDA based its decision on data from a basket trial, which used a novel trial design that allowed for the inclusion of patients with various cancer types and was based on RWE from a cancer genomic database.
For cancer drugs, the FDA is also interested in using RWE to support the approval of new indications for existing drugs, to evaluate the safety and effectiveness of drugs in real-world populations, and to inform regulatory decision-making in situations where traditional clinical trials are not feasible or ethical. With the ongoing review by Health Canada (HC) of the “Terms and Conditions” regulations for products approved under the Notice of Compliance with conditions (NOC/c) pathway, there is an opportunity for Canada to align its policies with other major regulatory agencies. More importantly, an ongoing pathway for continuous evidence generation may also help Canada accelerate our drug approval process for drugs that can deliver meaningful improvements to the quality of life and life expectancy of patients with cancer.
While RWE can accelerate the development and approval of cancer drugs by providing high-quality evidence throughout a drug’s life cycle, it also requires the appropriate infrastructure and governance to ensure the right data is available when needed. This is the role that Exactis has chosen to play in the Canadian cancer- treatment ecosystem. Since 2015, we have quietly built a network of clinical partners and patients whose data will facilitate access to the next generation of cancer therapies. It’s time for RWE to take centre stage not only for reimbursement decisions but also for regulatory decisions.
Exactis is ready to do its part. If you’re ready, let’s talk.
Kostas Trakas, PhD
CEO, Exactis Innovation