Incorporating the patient voice in clinical research: Validation of COAs for a rare disease NDA

Clinical Outcome Assessments (COAs) are vital in evaluating the effectiveness of new drug products by directly measuring patient experiences and outcomes, ensuring that treatments address meaningful aspects of patients' lives. The FDA's Patient-Focused Drug Development (PFDD)¹ guidance underscores the importance of COAs in capturing patients' perspectives and experiences throughout the drug development lifecycle. 

Proper validation of COAs is crucial to ensure their reliability, relevance, and ability to detect meaningful changes, thereby strengthening regulatory submissions and increasing the likelihood of product approval. 

Case study: Validation of COAs for a rare neurodegenerative condition 

To illustrate the practical application and benefits of implementing robustly validated COAs in rare disease clinical research, we share a case study where Parexel supported the validation of key COAs in a pivotal study. This work supported our biotech customer’s New Drug Application (NDA) submission, which the FDA accepted and assigned priority review. 

Challenge: 

A biotech company faced a regulatory obligation to validate two COAs for the assessment of a rare, hereditary, neurodegenerative condition, in accordance with PFDD guidance. This validation was crucial to support the acceptability of Phase III study findings and needed to be included in the NDA submission. Given tight filing timelines, the company also needed to efficiently leverage existing data. 

Solution: 

We recommended a multi-faceted approach, combining comprehensive research methodologies, expert collaborations, innovative techniques, and rigorous analysis to ensure a thorough and patient-centric validation process for the COA endpoints. This included – 

Comprehensive research approach: Conducted patient (n=7) and HCP (n=8) interviews, analyzed existing survey (n=145) and focus group (n=8) data, performed meaningful change interviews (n=24) 
Expert collaboration: Engaged SMEs with rare disease experience, partnered with Parexel Patient Insights team 
Innovative techniques: Developed interactive visual aids, designed patient-centric interview methods 
Rigorous analysis: Applied qualitative analysis and reporting standards 

Results: 

We successfully delivered regulatory-grade evidence for COA validity and patient-centric meaningful change thresholds. This supported a successful NDA filing with robust data on patient-centered measurement of disease progression and treatment benefits. 

We exceeded planned FDA submission timelines, delivering study reports within three months. Additionally, we disseminated findings through peer-reviewed publications and conference presentations, reaching key audiences including KOLs, FDA, and patient advocacy groups. The study results were also cited by FDA in a review of another rare disease product approval. 

Advancing patient-focused research – the impact and value of COAs 

By leveraging expertise in rare diseases and innovative patient engagement approaches, sponsors can conduct rigorous qualitative research with patients and healthcare professionals. When COAs are properly validated, this approach can meet regulatory requirements for incorporating the patient voice – as well as contribute to the broader understanding of how to assess treatment benefits for patients in specific disease areas. 

COAs allow sponsors to measure patient experiences and perceptions that have not been measured by traditional clinical research endpoints. Nothing could be more patient-centric than that. 

Ready to discuss the next steps to implement COAs in your drug development program? Please get in touch, we’re always available for a conversation. 

References

1. FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making 

Disclaimer: Parexel provides the information contained in this document for educational purposes only. The information does not constitute legal or regulatory advice. Readers should not act upon this information without seeking advice from professional advisers. 

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