Real-world evidence (RWE) is increasingly valued by pharmaceutical companies for its ability to complement and enhance the evidence generated from traditional clinical trials. RWE provides insights into how drugs and treatments perform in routine clinical practice, considering diverse patient populations, healthcare settings, and treatment patterns. However, persistent challenges have, and continue to impact implementation, especially around data.
RWE can support regulatory decision-making processes, such as drug approvals, label extensions, and post-approval commitments. By demonstrating the real-world effectiveness and safety of a drug, pharmaceutical companies can improve the efficacy of their submissions to regulatory agencies. RWE allows for the comparison of different treatment options and interventions in real-world populations. Monitoring and analysing adverse events and other safety outcomes in real-world settings, pharmaceutical companies can identify potential risks, implement risk management strategies, and ensure patient safety.
A recent McKinsey report estimated that, over the next three to five years, an average top-20 pharmaceutical company could unlock more than US$300 million a year by adopting RWE across its value chain.
RWE plays a critical role in unlocking the real-world benefits and value of drugs to payers and reimbursement authorities. By leveraging RWE, pharmaceutical companies can strengthen their case for market access, negotiate favourable pricing and reimbursement agreements, and overcome barriers to formulary inclusion. It also helps pharmaceutical companies conduct post-marketing studies to further evaluate drug safety, efficacy, and effectiveness in real-world populations. These studies can provide additional data to support label expansions, identify new patient populations, optimise dosing regimens, and inform clinical practice guidelines.
In real-world settings, RWE allows pharmaceutical companies to understand patient needs, preferences, and treatment outcomes. This patient-centric approach enables the development of more patient-centered therapies, improved treatment guidelines, and better-informed decision-making throughout the drug development and commercialisation process.
RWE is essential in conducting Health technology assessment (HTA), which evaluate the clinical and economic value of healthcare technologies, treatments, and interventions. By analysing real-world data on outcomes, costs, and patient experiences, policymakers can make evidence-based decisions on the adoption, reimbursement, and utilisation of healthcare technologies.
Nevertheless, implementation challenges abound. Realworld data is subject to various sources of bias and confounding factors. These include, selection bias, treatment allocation bias, and confounding variables that can affect the accuracy and reliability of RWE studies. Integrating data from various sources and systems with different formats and structures is also a significant challenge. Demonstrating the reliability, validity, and generalisability of RWE studies to regulatory and reimbursement authorities is crucial to support decision-making and gain acceptance. Respecting patient privacy, ensuring informed consent, and maintaining data confidentiality are critical ethical considerations when working with real-world data. Collaboration among stakeholders, including pharmaceutical companies, healthcare providers, regulatory bodies, and patient advocacy groups can address these challenges and unleash the full potential of RWE.
The cover story “Real-world Evidence - How patientled insights are transforming the way we develop health policy”, Julie Cini, CEO, Advocacy Beyond Borders and Ruth Kuguru, Executive Director, Communications & Patient Engagement, Novartis Asia Pacific, Middle East, and Africa, highlights why the Healthcare leaders in the Asia-Pacific region are keen to address the perception gap that exists around RWE. To bridge the current gap, the article offers two perspectives on the relevance of RWE to patient communities and strategies to foster greater multidisciplinary participation in RWE collection. How patient organisations use RWE to gain disease awareness traction and shape health policy and the importance of collaboration among multiple stakeholders.
Prasanthi Sadhu, Editor, Pharma Focus Asia
