Real-World Evidence (RWE) refers to clinical evidence obtained from real-world data (RWD) on the use and potential benefits or risks of a medical product. This information is derived from sources like electronic health records (EHRs), claims and billing activities, product and disease registries, claims databases, patient registries, and data from wearable devices.
In the pharmaceutical industry, the growing importance of RWE stems from its ability to complement clinical trials by offering insights into how drugs perform outside the controlled settings of randomized clinical trials (RCTs). RWE also helps pharmaceutical companies advance new treatments, optimize drug development, and enhance patient outcomes, playing an increasingly vital role in driving innovations.
Reasons for the Growing Importance of Real World Evidence (RWE) in Pharma Innovations
Regulatory Decision-Making: Regulatory agencies, such as the FDA and EMA, are increasingly using RWE to support regulatory approvals, label expansions, and post-marketing safety monitoring. The 21st Century Cures Act in the U.S. encourages the use of RWE for approving new indications of existing drugs.
[The 21st Century Cures Act of 2016 is a U.S. law designed to accelerate medical innovation, streamline drug and medical device approvals, and enhance patient access to new therapies. The Act encourages the FDA to integrate RWE into regulatory decision-making, allowing real-world data to support approvals for new indications and post-market monitoring of drugs and devices.]
Support for Market Access: Payers and health technology assessment (HTA) bodies are relying on RWE to evaluate the value and cost-effectiveness of therapies.
Post-Market Surveillance and Pharmacovigilance: RWE helps monitor its safety and effectiveness of the drug in a broader patient population, identifying adverse events or long-term outcomes that may not have been observed during clinical trials.
Clinical Trial Optimization: RWE can help design more efficient clinical trials by identifying patient populations, informing endpoint selection, and creating historical controls.
Real-World Impact of Novel Therapies: With the rise of personalized medicine and innovative therapies like gene and cell therapies, RWE is critical in understanding their performance outside the structured environment of trials. And RWE also helps identify genetic markers, patient subgroups, and treatment responses, allowing for better personalization of therapies.
Accelerating Drug Development: Pharmaceutical companies use RWE to streamline drug development, improve protocol design, and enhance recruitment for clinical trials. RWE enables pharma companies to explore new uses for approved drugs beyond their initial indications by analyzing real-world treatment outcomes.
Companies Relying on RWE for Pharma Innovations
Several pharmaceutical companies are leveraging Real-World Evidence (RWE) to drive innovations. Here are key players utilizing RWE for pharmaceutical innovations:
Pfizer: The company utilizes RWE for optimizing clinical trial designs and expanding indications for existing therapies. Example: Pfizer collaborated with healthcare providers to gather RWE for its COVID-19 vaccine to monitor its safety and efficacy across diverse populations post-approval.
Roche: Roche integrates real-world data from cancer patient registries, electronic health records (EHRs), and other data sources to support drug development and regulatory filings. Example: Roche used RWE to gain FDA approval for Tecentriq (atezolizumab) for the treatment of advanced cancer.
Novartis: The company incorporates RWE in its development and commercial strategies for innovative treatments. Example: Novartis used RWE to support regulatory submissions and market access for Kymriah, its CAR-T cell therapy for treating certain cancers. RWE provided evidence of safety and efficacy beyond clinical trials.
Johnson & Johnson (Janssen): Janssen incorporates RWE into regulatory filings and post-market safety monitoring. Example: Janssen used RWE from EHRs and claims data to support the development of Imbruvica (ibrutinib), a drug used to treat various types of blood cancer. Janssen has integrated RWE into its Data Science Council to leverage data analytics, including AI, in advancing drug discovery and improving patient outcomes.
Sanofi: Sanofi has adopted real-world data to complement clinical trials and demonstrate the real-world effectiveness of its therapies. Example: Sanofi utilized RWE to study patient adherence and the real-world impact of Dupixent (dupilumab) in asthma and atopic dermatitis, providing valuable data for pricing and reimbursement discussions.
AstraZeneca: AstraZeneca uses RWE to inform regulatory submissions and demonstrate real-world effectiveness. Example: AstraZeneca used RWE to gain FDA approval for Tagrisso (osimertinib), a lung cancer drug, by presenting data on real-world patient outcomes.
GSK (GlaxoSmithKline): GSK uses real-world data to monitor vaccine safety, improve drug adherence, and support regulatory and reimbursement decisions. Example: GSK applied RWE in the development of Shingrix, a vaccine for shingles, which demonstrated real-world effectiveness and helped secure approval in broader patient populations.
Eli Lilly: Eli Lilly integrates RWE into its R&D processes to optimize trial design and to gather post-market evidence. Example: Lilly used RWE to demonstrate the real-world benefits of Trulicity (dulaglutide), a diabetes treatment, and supported its approval for reducing cardiovascular risks.
Amgen: Amgen is leveraging RWE to complement clinical trials and support regulatory and payer decisions. Example: Amgen used RWE to support the approval of Repatha (evolocumab), a cholesterol-lowering drug, by showing its real-world effectiveness in reducing cardiovascular events.
Takeda: Takeda is using RWE to drive innovation in rare diseases, immunology, and oncology. The company integrates real-world data into its R&D and market access strategies to improve patient outcomes. Example: Takeda applied RWE in its development of treatments for rare genetic disorders, such as Ninlaro (ixazomib).
Real-World Evidence (RWE) is transforming pharmaceutical innovation by bridging the gap between clinical trials and real-world patient outcomes. Ultimately, RWE is not only reshaping how pharmaceutical companies develop and market drugs but also improving patient outcomes and healthcare efficiency on a global scale. In conclusion, RWE is enhancing every stage of the drug lifecycle—from early development and regulatory approvals to post-market surveillance and value-based healthcare models.
Stay ahead of the curve with Artixio’s expert regulatory insights. Subscribe now to receive the latest updates on evolving compliance standards, guidelines, and industry best practices, tailored to your business needs. Don’t miss out—stay informed and compliant with Artixio!
References:
Comments