Applying real-world data from expanded-access ("compassionate use") patients to drug development

Area of Science:

• Drug Development
• Regulatory Science
• Rare Diseases

Background:

• Drug development faces challenges in meeting treatment needs for rare diseases.
• Traditional randomized clinical trials are resource-intensive and time-consuming.
• The 21st Century Cures Act encourages alternative drug approval methods.

Purpose of the Study:

• To explore the utility of real-world data and evidence in drug approval.
• To evaluate expanded access programs as a source of real-world evidence.
• To support patient-centered and personalized medicine in drug development.

Main Methods:

• Leveraging real-world evidence from expanded access ('compassionate use') programs.
• Analyzing patient outcomes data from non-approved drug treatments.
• Integrating real-world evidence with other data sources like clinical trials.

Main Results:

• Expanded access data can provide critical insights into drug safety and efficacy.
• Real-world evidence may supplement or, in some cases, form the primary basis for regulatory review.
• This approach can generate valuable data for rare disease treatments.

Conclusions:

• Expanded access programs offer a viable pathway for generating regulatory-grade real-world evidence.
• Utilizing real-world evidence can enhance drug approval processes, especially for rare conditions.
• This strategy aligns with patient-centered care and personalized medicine principles.