Regulatory Recommendations for Patient Experience Data in North America, Europe, and Asia Pacific
Author(s)
Poon JL1, Zschocke J2, Myrick S3, Ding D4, Hill JN5, Pérez López NG6, Alonzo C6, Wu T4, Delbecque L1
1Eli Lilly and Company, Indianapolis, IN, USA, 2Lilly Deutschland GmbH, Hesse, Germany, 3IQVIA, New York, NY, USA, 4Eli Lilly and Company, Shanghai, China, 5IQVIA, Deerfield, IL, USA, 6IQVIA, Mexico City, Mexico
Presentation Documents
OBJECTIVES:
Describe recommendations regarding Patient Experience Data (PED) in regulatory review across regulatory agencies in Australia, Canada, China, Europe, Japan, United Kingdom, and the United States (US).METHODS:
A systematic search was conducted to identify published documents that include PED-recommendations issued by regulatory agencies in scope. PED-recommendations were reviewed if they related to clinical outcomes assessment (COA) validation, analyses, interpretation, endpoint definition, or were related to other forms of PED.RESULTS:
The US FDA issued guidance documents regarding methods for capturing and measuring PED, and several disease-specific guidances addressing concepts of interest definition, and COA measures and endpoints selection. The EMA published a reflection paper and disease-specific guidelines that included COA-related recommendations and expressed plans to incorporate patient preferences data in benefit-risk assessments. The China NMPA published guidelines on the use of COA in clinical trials and draft guidelines on the design, implementation, and benefit-risk assessments in patient-centered clinical trials, which included recommendations on COA endpoints, instrument validation and interpretation, and the use of qualitative data and patient preference information to support drug registration. No official guidance on the use of PED have been issued by the Australian, Canadian, English, and Japanese regulatory agencies. However, most of these agencies are working on PED-related initiatives or have published disease-specific guidances that mentioned COA as key endpoints, without detailed COA-related recommendations.CONCLUSIONS:
The voice of the patients by means of PED plays an increasingly important role in regulatory decision-making. While FDA is leading the way in advancing PED measurement standards, Europe and China tend to be generally aligned with FDA’s approach. Other regulatory agencies demonstrate interest in PED through ongoing initiatives, but do not have published PED-related guidance. Knowing each agency’s expectations for PED is key when submitting drug applications and those expectations should be considered early in clinical trial design.Conference/Value in Health Info
2023-05, ISPOR 2023, Boston, MA, USA
Value in Health, Volume 26, Issue 6, S2 (June 2023)
Code
PCR143
Topic
Clinical Outcomes, Organizational Practices
Topic Subcategory
Clinical Outcomes Assessment
Disease
No Additional Disease & Conditions/Specialized Treatment Areas