Real-World Assessment Process to Identify Fit-for-Purpose Data Sources to Meet Milestones for Post-Marketing Requirements (PMR), Post Authorization Safety Studies (PASS), and Health Technology Assessment (HTA) Submissions Utilizing Source Exploration...
Author(s)
Summer Drummond, DNP MSN1, Marta Viana, PhD2, Anna Castelo-Branco, PhD3, Sydney Willis, PhD1, Eileen Thorley, MPH1, Dony Patel, PhD4, Pierre Engel, PharmD PhD5, Tahereh Kamalati, PhD4, Ashley Lau, MPH6, Mariana Amaro, PhD2, Intissar Bourahla, MSc7, Anna Glans Lundin, PhD8.
1IQVIA, Durham, NC, USA, 2IQVIA, Oeiras, Portugal, 3IQVIA, Stockholm, Sweden, 4IQVIA, London, United Kingdom, 5IQVIA, Madrid, Spain, 6IQVIA, Ontario, ON, Canada, 7IQVIA, Paris, France, 8IQVIA, Solna, Sweden.
1IQVIA, Durham, NC, USA, 2IQVIA, Oeiras, Portugal, 3IQVIA, Stockholm, Sweden, 4IQVIA, London, United Kingdom, 5IQVIA, Madrid, Spain, 6IQVIA, Ontario, ON, Canada, 7IQVIA, Paris, France, 8IQVIA, Solna, Sweden.
OBJECTIVES: Noncompliance due to missed PMR/PASS/HTA milestones can occur due to inadequate feasibility assessments or inability to evaluate data prior to protocol submission. Due to these common issues, we aimed to develop an expeditious and effective process for systematic data source identification, feasibility assessment, and dissemination of results, to meet regulatory/HTA deadlines, without relying on data holders to provide information.
METHODS: We mapped data access requirements/timelines (CDA, contracting, scientific committee, EC/IRB, and data extraction/transfer/report provision) against 108 databases commonly considered for PASS across North America, South America, Asia Pacific, and Europe to justify the need for an alternative Feasibility process. Next, we developed desktop research focused data collection/evaluation methods and a regulator/HTA-facing result reporting structure.
RESULTS: Mapping determined that data provision from databases commonly occurs within 1-4 months for 18.5%, within 5-8 months for 23.1%, and in 9 months or greater for 58.3%. The mean time for data access was 8.8 months (SD=5.1), with values ranging from 1 to 19 months, confirming data access may not be possible during a feasibility assessment. The RAPID feasibility process allowed researchers to effectively assess relevance (population coverage and representativeness, adequate sample size, variable availability, and timeliness) and reliability (data provenance and quality) across data sources to meet milestones imposed. RAPID Feasibility was successfully piloted for a complex PASS submitted to FDA, EMA, MHRA, and HTA bodies in which data sources were unable to provide information prior to milestones. Provision of RAPID feasibility results during protocol submission mitigated concerns related to the synopsis, and the PRAC assessor stated data source selection was “informed by a comprehensive feasibility assessment.”
CONCLUSIONS: Researchers should anticipate data access timelines and feasibility questions from regulators/HTA bodies. However, if time is compressed, the RAPID Feasibility process may be deployed for selection of fit-for-purpose data sources that meet regulatory/HTA requirements/timelines.
METHODS: We mapped data access requirements/timelines (CDA, contracting, scientific committee, EC/IRB, and data extraction/transfer/report provision) against 108 databases commonly considered for PASS across North America, South America, Asia Pacific, and Europe to justify the need for an alternative Feasibility process. Next, we developed desktop research focused data collection/evaluation methods and a regulator/HTA-facing result reporting structure.
RESULTS: Mapping determined that data provision from databases commonly occurs within 1-4 months for 18.5%, within 5-8 months for 23.1%, and in 9 months or greater for 58.3%. The mean time for data access was 8.8 months (SD=5.1), with values ranging from 1 to 19 months, confirming data access may not be possible during a feasibility assessment. The RAPID feasibility process allowed researchers to effectively assess relevance (population coverage and representativeness, adequate sample size, variable availability, and timeliness) and reliability (data provenance and quality) across data sources to meet milestones imposed. RAPID Feasibility was successfully piloted for a complex PASS submitted to FDA, EMA, MHRA, and HTA bodies in which data sources were unable to provide information prior to milestones. Provision of RAPID feasibility results during protocol submission mitigated concerns related to the synopsis, and the PRAC assessor stated data source selection was “informed by a comprehensive feasibility assessment.”
CONCLUSIONS: Researchers should anticipate data access timelines and feasibility questions from regulators/HTA bodies. However, if time is compressed, the RAPID Feasibility process may be deployed for selection of fit-for-purpose data sources that meet regulatory/HTA requirements/timelines.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD147
Topic
Epidemiology & Public Health, Real World Data & Information Systems, Study Approaches
Topic Subcategory
Data Protection, Integrity, & Quality Assurance
Disease
No Additional Disease & Conditions/Specialized Treatment Areas