FDA, Gottlieb Get Real About Real-world evidence

Area of Science:

• Regulatory Science
• Health Informatics
• Pharmaceutical Policy

Background:

• The integration of real-world data (RWD) into regulatory decision-making is a developing area.
• The pharmaceutical industry and regulatory bodies are exploring the potential of RWD.
• Current adoption of RWE by regulatory agencies is limited.

Purpose of the Study:

• To assess the anticipated acceptance of real-world evidence (RWE) and real-world data (RWD) by the FDA.
• To identify barriers to the integration of RWD into regulatory processes.
• To examine the necessary changes in FDA regulations and culture for RWE adoption.

Main Methods:

• Qualitative assessment of the current regulatory landscape in early 2019.
• Analysis of industry trends and statements regarding RWD.
• Review of existing FDA guidelines and cultural practices.

Main Results:

• The embrace of RWE and RWD by the FDA and pharmaceutical industry in early 2019 is not yet definitive.
• Existing regulatory frameworks and internal culture may impede the full utilization of RWD.
• Substantive changes in FDA rules and culture are required for meaningful RWE integration.

Conclusions:

• The future role of RWE in pharmaceutical regulation is contingent on significant FDA adaptation.
• Overcoming cultural and regulatory inertia is crucial for leveraging RWD effectively.
• A proactive approach from the FDA is needed to foster the reliable use of RWE.