Real-world evidence: Driving a new drug development paradigm in oncology

Real-world evidence: Driving a new drug development paradigm in oncology

Real-world evidence: Driving a new drug development paradigm in oncology

The potential for real-world evidence is expanding, particularly in oncology drug development. To compete, some companies are investing in robust real-world data and analytic capabilities.

Evidence generation in oncology is at a key inflection point. Given the rapid pace of scientific innovation, historical approaches to drug development are increasingly burdensome, with randomized controlled trials remaining incredibly costly and time intensive to conduct, and having a low certainty of success. Biopharmaceutical companies will collectively invest $50 billion to support oncology research and development in 2018, with a ~3 percent probability of success for any individual product from preclinical through registration phases.1 Despite this investment, many completed trials fail to answer critical questions for payors and healthcare providers, exacerbating the wide evidence gap that already exists between clinical research and practice.

Patients, providers, and payors lack answers to fundamental questions—“What treatment is best for me?”; “How do patients treated in the ‘real world’ perform on this therapy?”; “What is the value of this therapy relative to other treatment options?” The evidence gap persists despite a richness of available data, novel analytic methods, and inexpensive computing and genomic sequencing power. Real-world evidence (RWE)—insights generated from data collected during routine clinical practice—provides a platform with which to close the evidence gap between clinical research and practice.

The role of RWE in drug development is expanding, driven in part by biotechnology and pharmaceutical manufacturers’ embrace of digital solutions to realize gains in speed and efficiency from innovation. RWE teams have taken root across the pharmaceutical industry with industry-wide investments in talent and technical infrastructure. Successful biopharmaceutical companies have coupled investment with the belief that RWE is a critical component of development and life cycle management, and committed to this by building RWE generation capabilities on a large scale. Providers have similarly embraced RWE to inform clinical practice, and clinical guidelines (eg, National Comprehensive Cancer Network) increasingly incorporate RWE-generated insights. Recognizing the need for a more flexible framework for therapy evaluation, regulators are developing approaches to incorporate RWE in their decision making, as outlined in frameworks such as the 21st Century Cures Act. These changes are accompanied by the emergence of high-quality data providers, including those with distinct data sources and analytic approaches.

The potential for RWE is perhaps greatest in oncology. Cancer will soon overtake cardiovascular disease as the leading cause of death in the US,2 with a similarly increasing disease burden in other geographies. Governmental support for addressing the burden of cancer has increased, and includes record levels of federal funding (the National Cancer Institute is the most heavily funded of the 27 National Institutes of Health) and regulatory support through the creation of the Oncology Center of Excellence within the Food and Drug Administration (FDA).3 Clinical breakthroughs have led to the development of novel modalities of therapy (ie, chimeric antigen receptor T (CAR-T)/cell therapy) that offer the potential for cure-like responses, but at financial costs that raise questions regarding the role and, ultimately, the value of such therapy. RWE is well positioned to help address these questions in a manner aligned with the interests of all stakeholders. Effectively deployed, RWE can accelerate the pace of discovery—and patient impact—of new oncology therapies.

Download Real-world evidence: Driving a new drug development paradigm in oncology, the full report on which this article is based (PDF–1.1MB).

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