Clinical Scorecard: Building Quality into Advanced Therapies from Day One

At a Glance

Key Highlights

• Regulatory agencies are adopting risk-based approaches for rare diseases.
• GMP expectations remain high despite regulatory flexibility.
• Quality must be integrated throughout the development process.
• Data integrity is critical and requires a cultural commitment.
• Quality-by-Design should start early in research and development.

Guideline-Based Recommendations

Diagnosis

• Understand the specific needs of the target patient population.
• Utilize risk-based approaches to identify critical quality attributes.

Management

• Ensure robust communication and documentation within GMP systems.
• Collaborate transparently with CDMOs to manage quality effectively.

Monitoring & Follow-up

• Focus on data integrity and compliance with established regulations.
• Implement rigorous controls and validation for all processes.

Risks

• Underestimating the importance of foundational communication.
• Neglecting the cultural aspects of data integrity.

Patient & Prescribing Data

Individuals with rare and ultra-rare diseases requiring advanced therapies.

Therapies must be developed with a focus on safety and efficacy from the outset.

Clinical Best Practices

• Adopt a Quality-by-Design approach from the earliest stages.
• Integrate QA, manufacturing, and analytical teams for cohesive development.
• Prioritize risk-based thinking in all aspects of therapy development.

References

• The Medicine Maker