The increased Regulatory consideration of RWE in NDA/BLA submissions represents a fundamental shift toward a more patient-centric, evidence-based approach to drug evaluation. This approach has the potential to expedite access to life-saving therapies, particularly for those facing rare diseases and unmet medical needs. Gaining momentum in Real-World Evidence (RWE) in NDA (New Drug Applications)/ Biologics License Application (BLA) submissions represents a transformative approach to evaluating the safety and efficacy of new therapies and has the potential to revolutionize drug development and Regulatory decision-making.

The FDA's Shifting Perspective

There are several reasons why the FDA is increasing its consideration of RWE.

  1. RWE can provide data on the safety and effectiveness of drugs and biologics in real-world settings, where patients are taking the drugs for their intended use. This data can complement the data collected from clinical trials, which are often conducted in controlled settings with a limited number of patients.
  2. RWE can be used to generate data on the long-term safety and effectiveness of drugs and biologics. Clinical trials are often of relatively short duration, so they may not be able to detect all of the potential long-term side effects of a drug. RWE can provide data on the safety and effectiveness of drugs and biologics over longer periods.
  3. RWE can be used to study the safety and effectiveness of drugs and biologics in specific populations, such as children, pregnant women, and the elderly. These populations are often excluded from clinical trials, so RWE can provide valuable data on how drugs and biologics work in these populations.

How can RWE be used to support NDA/BLA submissions?

RWE can be used to support NDA/BLA submissions in several ways. For example, RWE can be used to:

  • Provide evidence of the safety and effectiveness of a drug or biologic in a real-world setting.
  • Identify new populations of patients who could benefit from a drug or biologic.
  • Generate data on the long-term safety and effectiveness of a drug or biologic.
  • Compare the safety and effectiveness of two or more drugs or biologics.

Key Advantages of RWE in NDA/BLA Submissions

  • Broader Patient Representation: RCTs often have strict eligibility criteria, limiting the diversity of patients included. RWE includes data from a more diverse patient population, providing a broader perspective on a drug's performance.
  • Long-Term Data: RWE offers insights into a drug's performance over extended periods, allowing for the assessment of long-term safety and effectiveness—an aspect often missed in traditional clinical trials.
  • Cost-Effectiveness: Leveraging RWE can be more cost-effective than conducting additional clinical trials, reducing the financial burden on drug developers and potentially lowering drug prices.
  • Rare Diseases and Special Populations: RWE is particularly valuable in evaluating drugs for rare diseases or assessing the performance of therapies in specific patient groups where conducting large-scale RCTs may be impractical.

Challenges and Considerations

While the integration of RWE into NDA/BLA submissions is a positive development, it comes with challenges. Ensuring the quality and reliability of RWE, maintaining patient privacy, and establishing rigorous methodologies for data analysis are essential considerations.

Additionally, there's a need for continued collaboration between the FDA, drug developers, and healthcare stakeholders to establish clear guidelines and standards for the use of RWE in Regulatory decision-making.


The integration of RWE into Regulatory decision-making promises to enhance the evaluation of drug safety and effectiveness, speed up the drug development process, and ensure that patients receive the most appropriate treatments. While challenges remain, the benefits of this approach are undeniable, and the pharmaceutical industry is on the cusp of a new era in drug development—one that is more patient-focused, data-driven, and efficient.

Collaborating with a Regulatory expert like Freyr, pharmaceutical and biologics companies can confidently embrace the era of RWE and leverage this valuable resource to enhance their drug development efforts.


Sonal Gadekar


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