Pharmaceutical research and development is inherently complex and high-risk. Sponsors must ensure that investigational products demonstrate both strong therapeutic efficacy and an acceptable safety profile. At the same time, they must manage escalating costs, lengthy timelines, and increasing expectations from regulators, payers, and patients.
Despite the extraordinary levels of investment and scientific rigor applied to the drug development process, industry-wide success rates remain below 15 percent. Many promising compounds never reach the commercial stage, often after years of costly and resource-intensive effort. To change this dynamic, pharmaceutical companies are adopting new tools to improve decision-making, allocate resources more strategically, and accelerate innovation. One of the most promising of these tools is real-world data (RWD).
By integrating real-world insights throughout the R&D continuum, pharmaceutical companies are enhancing the relevance, precision, and efficiency of their efforts. Based on our collaboration with industry leaders, COTA has surfaced four key RWD strategies that are particularly useful during times of uncertainty to reduce risk and advance the development of novel therapies.
1. Prioritizing High-Potential Research Pathways
Given the low probability of success, early-stage prioritization is essential. Pharmaceutical companies are using RWD to identify areas of unmet need, better understand disease heterogeneity, and uncover novel therapeutic opportunities. RWD can also highlight real-world treatment gaps and unexpected positive outcomes in routine clinical practice. These insights may point toward new indications or line extensions for existing products.
This evidence-driven approach enables companies to focus R&D investments where they are most likely to achieve measurable clinical impact. It ensures that scientific and financial resources are directed toward programs with the highest potential to improve patient outcomes.
2. Strengthening Value Narratives for Strategic Investment
In today’s highly competitive environment, a compelling clinical data package is not always sufficient. Stakeholders, including internal portfolio leaders, payers, and providers, require a clear and credible value proposition. RWD provides essential context by showing how a therapy performs in real-world settings and how it contributes to improved patient outcomes and cost-effectiveness.
Pharmaceutical companies are using RWD to build stronger business cases for investment decisions and to support favorable market access and reimbursement. This helps ensure that the therapies with the greatest potential reach the patients who need them most.
3. Enhancing Regulatory Submissions Through External Control Arms (ECAs)
As regulatory agencies increasingly recognize the utility of real-world evidence, pharmaceutical companies are incorporating RWD into clinical trial designs through external control arms. ECAs use historical real-world patient cohorts as comparators for investigational therapies. This method allows for rigorous evaluations when randomized controls are impractical or ethically challenging, such as in rare or life-threatening conditions.
The use of ECAs can streamline development by reducing recruitment challenges, lowering patient burden, and accelerating clinical timelines. Agencies like the FDA are working closely with sponsors to define appropriate use cases for ECAs, supporting faster and more efficient pathways to approval for therapies that address urgent medical needs.
4. Proactively Monitoring Safety and Effectiveness Post-Launch
Risk management continues after a product reaches the market. Post-marketing surveillance is critical for identifying long-term or rare adverse events, understanding patterns of use in real-world settings, and ensuring continued benefit-risk balance.
Pharmaceutical companies are using comprehensive RWD sources to monitor safety and effectiveness across diverse populations. These insights allow sponsors to detect issues early, respond quickly to potential concerns, and maintain trust with regulators, providers, and patients. This type of monitoring also supports ongoing product optimization and informs future development and commercialization strategies.
The Case for Real-World Evidence
Incorporating real-world data throughout the research and development lifecycle represents a meaningful advancement for pharmaceutical innovation. By grounding decisions in real-world evidence, companies can reduce scientific and operational risk while increasing the efficiency and effectiveness of their programs. The result is a more responsive, data-informed approach that helps deliver high-value therapies to patients, supports public health goals, and ensures the continued success of the pharmaceutical industry.
