- About Us
- Solutions
- Insights
Real-world evidence (RWE) is playing a larger role in the pharmaceutical industry. The big shift from traditional clinical trial data to integrating RWE into the drug development process creates more patient-centred and efficient approaches. This transition from traditional clinical trial settings to diverse, wider data sources has deep implications for innovation and accessibility in healthcare. From speeding up drug approvals to optimizing patient outcomes, RWE has transformed health care for all time to come.
Historically, the Randomized Controlled Trial was viewed as the "gold standard" in testing the effectiveness and safety of new medications.1 However, researchers from McKinsey & Company noted that the pharmaceuticals industry has evolved through two iterations of RWE uptake: Generation I (circa 2011) focused on safety and post-market surveillance; Generation II (2012–2015) integrated RWE into the entire product lifecycle.2 Today, in the third generation, RWE is being leveraged not only for safety and efficacy but also for regulatory decisions, economic evaluations, and personalized medicine.
A major milestone in this journey was the release of the FDA's Real-World Evidence Program framework in 2018, which defined RWE as "clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD."3 This framework has provided much-needed guidance on incorporating RWE into regulatory submissions and market access strategies.
RWD forms the backbone of RWE and encompasses data that is routinely collected from diversified sources such as:
● Electronic health records
● Claims databases
● Patient registries
● Wearable devices
Each of these sources contributes something unique to RWD.
● Clinical Data: These come from EHRs and patient registries; it is very granular, patient-level information, including lab values, diagnoses, and treatment histories. Interoperability has been increased further with the digitization of health systems.4
● Claims Data: Though originally collected for billing and reimbursement purposes, claims data contain information on diagnoses, services rendered, and payments.4
● Patient-Generated Data: Wearable devices and health apps are providing more and more patient-generated data, allowing constant monitoring of health parameters outside of clinical facilities.4
● Non-Traditional Digital Sources: Social media, health forums, and industry-sponsored platforms such as Project Data Sphere help researchers infer patient insights.4
If correctly analyzed, these datasets help bridge the gap between controlled clinical trials and the heterogeneous realities of real-world patient populations.
RWE provides actionable insights into the safety, efficacy, and cost-effectiveness of treatments in real-world settings. Here are some benefits of real-world evidence in drug development and evaluation:
One of the biggest benefits of RWE is that it helps accelerate drug approvals. RWE helps regulators take a more holistic view of how a drug performs in a diverse population of patients by augmenting RCT data.4 For instance, during the COVID-19 pandemic, RWE enabled real-time assessments of vaccine effectiveness, such as demonstrating the 91.2% effectiveness of the Pfizer-BioNTech vaccine one week after vaccination in real-world effectiveness.5 Those kinds of findings were instrumental in securing emergency use authorizations.
Drug development is a costly venture that may cost upwards of $2 billion per drug, and clinical trials account for about $1.5 billion of that.6 RWE can help reduce the skyrocketing cost of drug development. Adaptive and pragmatic trial designs in RWE could provide a way out by using existing data and focusing on real-world endpoints. This approach saves money as well as time.
RWE is revolutionizing personalized medicine because it enables patient populations to be partitioned based on genetic markers, comorbidities, or responses to treatments. This is especially needed for the area of cell and gene therapies (C>), where patient pools are small, but RWE can provide important information on treatment effects and long-term outcomes. 7 It then enables pharmaceutical companies to identify which patient subpopulation derives the greatest benefit from therapy, allowing for narrow targeting and, ultimately, improved patient outcomes by analyzing real-world data.
More and more healthcare payers and pricing managers are turning to RWE to inform pricing strategies and reimbursement decisions.8 RWE analyzes treatment performance and global pricing trends to help develop value-based pricing models.
RWE in oncology fills the gap left by traditional clinical trials in terms of representation for the elderly and those with comorbidities by showing the safety and effectiveness of treatments in these under-represented populations. For instance, the role of real-world data (RWD) in cancer clinical research is increasing. 9
Rare diseases would also substantially benefit from RWE, as traditional trials are generally not statistically powered because of the small patient populations. RWE will help to generate actionable insights using EHRs, disease registries, and patient-reported outcomes that will speed up approvals and open access to life-saving therapies. 4
Here are some trends that the healthcare industry could see for real-world evidence in drug development and evaluation in the coming years:
Digital health technologies (DHT) technologies are greatly expanding the reach and usefulness of RWE. There are huge volumes of patient-generated data flowing in from wearable devices, mobile apps, and telemedicine platforms that create new drug development opportunities, such as personalized treatment, in monitoring. According to Deloitte, integrating RWE with these technologies could bring enhanced chronic disease management, reduced healthcare costs, and tailored treatment pathways to "Precision Medicine". 10
Pharmaceutical companies now more commonly utilize RWE in efforts to pinpoint patient subpopulations likely to benefit from given treatments.4 Biomarkers and genomics now form an integral part of understanding disease stratification.10
Real-world evidence (RWE) is conducive to innovative trial designs like adaptive trials, where there are strategies that utilize interim data to modify trial characteristics as the trial progresses.10 Such trials, validated by regulators and payers, create efficiencies and lower costs.
RWE is departing from local data to global data and finding new sources of outcome data.10 Harmonization is being made possible by multinational collaborations even though differing regulatory standards and disparate data structures remain problematic.
While potentially promising, the incorporation of RWE into drug development is not without its problems. One of the biggest problems is that RWD sources are not standardized in terms of structure, completeness, and quality.4 Every database is structured differently; some provide basic information, while others offer deep insights. In the effort to harmonize records globally, many of the database-specific advantages get lost, making data analysis difficult.
Another challenge is data privacy and accessibility. Regulations such as the Health Insurance Portability and Accountability Act (HIPAA) impose stringent requirements on the use of patient data.11 Tokenization and anonymization techniques are emerging as solutions to ensure compliance while enabling robust data analysis.
The fragmented nature of the regulatory guidelines is another challenge for real-world evidence in drug development and evaluation. Companies need to navigate differences across various jurisdictions, such as FDA regimes in the U.S. and EMA's published guidance on registry-based studies in Europe. 12 13
Real-world evidence in drug development and evaluation has had its fair share of advancements over the years:
RWE derives its legitimacy as a decision-making tool by endorsements and support from global regulatory agencies. For example:
● The U.S. FDA's 21st Century Cures Act (2016) underlined the role of RWE in drug approvals.14
● In 2020, the European Medicines Agency initiated programs to incorporate real-world evidence in marketing authorizations.15
These frameworks not only highlight the increasing importance of real-world evidence (RWE) but also provide a systematic way to utilize it.
Technological advancements are a significant factor in the adoption of RWE. Here are some examples:
● Digital health platforms such as wearables/telehealth applications produce large amounts of patient-level data. 4
● Project Data Sphere is a platform where data from cancer trials is shared so that it can be used for other analyses, and researchers can conduct more types of research studies with that data. 10
● Tokenization protects patients' privacy by allowing tracked data to circulate through healthcare systems. 4
RWE's evolution has led to its application across the drug lifecycle—from very early R&D to post-market surveillance. It enhances efficiency and accuracy by bridging fragmented datasets into holistic insights at companies.16
Real-world evidence is no longer a complementary tool but rather a mainstay of drug development. RWE brings in diverse sources of data, offering a holistic view of treatment efficacy and safety and narrowing the gap between clinical trials and real-world practice. With continuing improvement in data analytics, digital health technologies, and regulatory frameworks, RWE will surely carve out much more space for itself in the future development of drugs and give better results to patients while easing the burden on healthcare systems.
References
1. Zabor EC, Kaizer AM, Hobbs BP. Randomized controlled trials. CHEST Journal. 2020;158(1):S79-S87. doi:10.1016/j.chest.2020.03.013
2. Cavlan O, Chilukuri S, Evers M, Westra A. Real-world evidence: From activity to impact in healthcare decision making. McKinsey & Company. Published May 7, 2018. https://www.mckinsey.com/industries/life-sciences/our-insights/real-world-evidence-from-activity-to-impact-in-healthcare-decision-making
3. Purpura CA, Garry EM, Honig N, Case A, Rassen JA. The role of Real‐World evidence in FDA‐Approved new drug and biologics license applications. Clinical Pharmacology & Therapeutics. 2021;111(1):135-144. doi:10.1002/cpt.2474
4. Dang A. Real-World evidence: A primer. Pharmaceutical Medicine. 2023;37(1):25-36. doi:10.1007/s40290-022-00456-6
5. Zheng C, Shao W, Chen X, Zhang B, Wang G, Zhang W. Real-world effectiveness of COVID-19 vaccines: a literature review and meta-analysis. International Journal of Infectious Diseases. 2021;114:252-260. doi:10.1016/j.ijid.2021.11.009
6. Examination of clinical trial costs and barriers for drug development. ASPE. Published July 24, 2014. https://aspe.hhs.gov/reports/examination-clinical-trial-costs-barriers-drug-development-0
7. Honig N, Arena P, Lederer NM, Jaksa A. RWD84 The Role of Real-World Evidence in Cell and Gene Therapy Regulatory and Health Technology Assessment Decisions. Value in Health. 2022;25(12):S465. doi:10.1016/j.jval.2022.09.2309
8. Khosla S, White R, Medina J, et al. Real world evidence (RWE) – a disruptive innovation or the quiet evolution of medical evidence generation? F1000Research. 2018;7:111. doi:10.12688/f1000research.13585.2
9. Saesen R, Van Hemelrijck M, Bogaerts J, et al. Defining the role of real-world data in cancer clinical research: The position of the European Organisation for Research and Treatment of Cancer. European Journal of Cancer. 2023;186:52-61. doi:10.1016/j.ejca.2023.03.013
10. Nightingale F. Real World Evidence Enabling the life sciences industry to transform patient care.; 2018. https://www2.deloitte.com/content/dam/Deloitte/uk/Documents/life-sciences-health-care/deloitte-uk-rwe-enabling-the-life-sciences-industry-to-transform-patient-care.pdf
11. Edemekong PF, Annamaraju P, Afzal M, Haydel MJ. Health Insurance Portability and Accountability Act (HIPAA) compliance. StatPearls - NCBI Bookshelf. Published November 24, 2024. https://www.ncbi.nlm.nih.gov/books/NBK500019/
12. Office of the Commissioner. Real-World Evidence. U.S. Food And Drug Administration. Published September 19, 2024. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
13. European Medicines Agency. Guideline on registry-based studies.; 2021. https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-registry-based-studies_en.pdf
14. Goble JA. The potential effect of the 21st century cures act on drug development. Journal of Managed Care & Specialty Pharmacy. 2018;24(7):677-681. doi:10.18553/jmcp.2018.24.7.677
15. Flynn R, Plueschke K, Quinten C, et al. Marketing Authorization Applications Made to the European Medicines Agency in 2018–2019: What was the Contribution of Real‐World Evidence? Clinical Pharmacology & Therapeutics. 2021;111(1):90-97. doi:10.1002/cpt.2461
16. Khosla S, White R, Medina J, et al. Real world evidence (RWE) – a disruptive innovation or the quiet evolution of medical evidence generation? F1000Research. 2018;7:111. doi:10.12688/f1000research.13585.2