Real World Evidence (RWE) is defined by the FDA as ‘clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD)’.1RWD is the data collected from sources other than traditional clinical trials. These data are being obtained from electronic tracking systems used to capture patient experiences during care (Patient Stories).
RWE is increasingly crucial for life science companies to ensure patient access and commercial success. Generating and leveraging RWE requires technical expertise along with strategic insights for informed healthcare decisions and optimal outcomes. However, as the sources of RWD are disparate and the quality can vary greatly, accessing, analyzing and drawing conclusions from the data is more challenging than ever. Going forward, Patient Stories will become increasingly more patient centric, as highlighted through the growing emphasis by payers and regulators for advances in this area.
RWE in clinical development and post approval
RWE has a role in both pre- and post-approval. In the clinical development stage RWE can enhance the effectiveness of randomized clinical trials, for example in the initial phases (Phase I and Phase II) it can aid in the identification of patient sub-populations and in understanding patient pathway, disease progression, treatment preferences and patient related outcomes (PRO’s). RWE facilitates operational aspects of drug development by optimizing patient recruitment and patient access.
Post-approval RWE can assist in generating further insights to improve safety and effectiveness of products, prescribing patterns and adherence, along with real world health outcomes.
A value-based, Patient Stories approach is driving the role of RWE in formulating a product’s evidence requirements across the development continuum. RWE may potentially explain facets like head-to-head comparative effectiveness, differentiation in sub-population and effects of switching. For example, data from an observational study among insulin-naïve patients with uncontrolled Type 2 diabetes was used to assess health outcomes of Toujeo (insulin glargine) compared to basal insulins.2RWD pooled from US insurance claims databases was used to demonstrate effectiveness of Eliquis (apixaban) among non-valvular atrial fibrillation (NVAF) patients
RWE can play a positive role in improving patient access by providing payers with information on the effectiveness in the real world and the cost-effectiveness in comparison to other drugs on the market. RWD can improve health outcomes for patients and help pharmaceutical companies to develop a more efficient and streamlined commercialization process.
The scope of RWE in market access includes achieving optimal price and full reimbursement, favorable recommendation from health technology assessment (HTA) authority and inclusion in various formularies at hospital, regional and national levels. As an example, RWE from US insurance claims helped Zytiga (albiraterone acetate) receive recommendation by National Institute for Health and Care Excellence (NICE) for prostate cancer patients.
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Going forward, drug pricing will be based on health outcomes rather than payment per pill. The current constraints of using and leveraging RWE will be mitigated by the use of technologies like big data, machine learning and blockchain technology. The evolution of the Internet of Things (IoT) means that the future sources of RWE are likely to be much more diverse. Furthermore, blockchain technology will enable more secure storage of patient health data and the direct transmission of anonymized data to pharma companies, helping to incentivize the right health outcomes in the system.
