Real-world evidence (RWE) is defined as clinical evidence regarding a medical product that is derived from analysis of real-world data (RWD), meaning data collected in the context of the routine delivery of care as opposed to within a clinical trial. The data is usually mined from registries, electronic health records (EHRs) as well as administrative and medical claims databases.
The industry-wide shift to value-based healthcare, the growing regulatory acceptance of RWE and the numerous advances in data analytics have elevated RWE to a C-suite strategic priority, capable of unlocking broad benefits across the entire product life cycle. To that end, the pharmaceutical industry has seen an expanding number of strategic partnerships with technology firms as well as hospitals and other organisations, with the goal of ensuring access to new sources of RWD.
The present article explores how RWE can add value for pharma companies, from R&D through to commercialisation of drug products.
R&D and regulatory approvals
Deloitte’s 2020 RWE benchmarking report suggests that 94% of surveyed companies expect a shift in RWE applications in the next 2-3 years, with the highest impact being in R&D. Applications are expected to go beyond the more traditional uses in disease understanding and pharmacovigilance, with potential to cut down clinical development time and costs significantly.
Janssen’s recent collaboration with Komodo Health serves as an example of how big pharma is increasingly buying into the potential of RWE in streamlining clinical trial execution. The partnership will allow Janssen to use RWE when assessing the feasibility of new clinical trials, while also informing trial site selection and patient recruitment.
Recently, the field has also seen increasing adoption of single-arm trials with control groups synthesised entirely from RWD. Reflecting the shifting regulatory environment, some of these have already led to landmark approvals (for more on this, read a previous Sparks blog). However, a watershed event in the use of regulatory-grade RWE was the label extension of Pfizer’s Ibrance in 2019 for the treatment of male patients with breast cancer, based solely on RWE. Submitted data came from EHRs and post-marketing reports from the IQVIA Insurance, Flatiron Health Breast Cancer and Pfizer's global safety databases.
In outcomes-driven healthcare systems, RWE can provide validation of the value created for patients, potentially leading to broader coverage and more favourable formulary placement for a given product. RWE is starting to change the reimbursement model for very expensive drugs, such as Spark’s Luxturna (inherited retinal dystrophy), as well as Novartis’ Zolgensma (spinal muscular atrophy) and Kymriah (CAR-T cell therapy for haematological malignancies). All therapies have received coverage contingent on real-world benchmarks in the post-market phase. For instance, reimbursement of Zolgensma in Germany is done through a ‘pay-for-performance’ model, based on patient-relevant outcomes evaluated in the real world; if success is not achieved, the manufacturer funds up to 100% of the treatment.
Similarly, the US Prescription Drug Pricing Reduction Act of 2019 (currently stalled in Congress), includes a provision for Medicaid risk-sharing value-based agreements, with the cost of new gene therapies for rare diseases to be paid over time, according to measurements of real-world outcomes.
RWE may be crucial in the messaging and positioning strategy of drug products, commonly employed to clarify, complement, or strengthen data from randomised controlled trials (RCTs) as well as to enhance understanding of the market, inform segmentation or finetune product positioning in a competitive space.
In this context, AstraZeneca’s use of RWD in support of Forxiga is particularly interesting. Forxiga belongs to a class of drugs called SGLT2 inhibitors and has two in-class competitors in the market. Following approvals for type 2 diabetes, SGLT2 inhibitors pursued label extensions for the reduction of cardiovascular risk in type 2 diabetes. While its pivotal readout was lagging behind that of competitors, AstraZeneca used RWD to demonstrate the clinical effectiveness of all three marketed SGLT2 inhibitors in improving cardiovascular outcomes. In doing so, it attempted to strengthen perceptions of a class effect and thus mitigate potential risk of its competitors gaining ground, while Forxiga’s readout was still pending. Based on the RWE, AstraZeneca also lobbied to introduce SGLT2s earlier in the treatment algorithm of type 2 diabetes.
However, given that RCTs are generally considered superior in the evidence ladder and heavily relied upon in the context of HCP decision-making, there is a risk that singular RWE focus may be faced with HCP resistance. Therefore, RCT and RWE evidence should ideally co-exist, and be part of a balanced messaging narrative. Nevertheless, sustained improvements in the quality of data collection and analysis bodes well for the future, potentially allowing us to foresee a situation where RWE is elevated to RCT evidence grade.
RWE during the COVID-19 pandemic and beyond
The power of RWD and RWE is perhaps best illustrated in the way it enabled the pharmaceutical industry to adapt during the COVID-19 pandemic, unlocking value for pharmaceutical companies, health systems, and civilians alike. Over the last year or so, RWD have been used to study the repurposing of existing drugs to treat COVID-19, gain insights into the virus’ profile, inform decision-making and validate the effectiveness of vaccines in the real-world. In all examples, RWD has allowed the generation of valuable information at an unprecedented pace while avoiding challenges that would have hampered the execution of traditional clinical research.
Besides highlighting the value of RWE in healthcare, the pandemic has also accelerated the adoption of digital technologies worldwide. One could argue that this is the ideal time for the pharmaceutical industry to fully harness the power of RWE and…get real!
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