Lessons learned from a multi‐data source research collaboration: The mirabegron post‐authorization safety study program

Pharmacoepidemiology and Drug Safety, vol. 33, pp. e5799

Abstract

BACKGROUND: Many factors contribute to developing and conducting a successful multi-data source, non-interventional, post-authorization safety study (NI-PASS) for submission to multiple health authorities. Such studies are often large undertakings; evaluating and sharing lessons learned can provide useful insights to others considering similar studies. OBJECTIVES: We discuss challenges and key methodological and organizational factors that led to the delivery of a successful post-marketing requirement (PMR)/PASS program investigating the risk of cardiovascular and cancer events among users of mirabegron, an oral medication for the treatment of overactive bladder. RESULTS: We provide context and share learnings, including sections on research program collaboration, scientific transparency, organizational approach, mitigation of uncertainty around potential delays, validity of study outcomes, selection of data sources and optimizing patient numbers, choice of comparator groups and enhancing precision of estimates of associations, potential confounding and generalizability of study findings, and interpretation of results. CONCLUSIONS: This large PMR/PASS program was a long-term commitment from all parties and benefited from an effective coordinating center and extensive scientific interactions across research partners, scientific advisory board, study sponsor, and health authorities, and delivered useful learnings related to the design and organization of multi-data source NI-PASS.

Authors 15

1. Stefan de Vogel corresponding Aachen Pharmacovigilance Astellas Pharma B.V. Leiden The Netherlands Pharmacovigilance

   Astellas Pharma (Netherlands)

   Affiliation as printed

   Pharmacovigilance Astellas Pharma B.V. Leiden The Netherlands

   Pharmacovigilance, Astellas Pharma B.V., Leiden, The Netherlands

2. John D. Seeger
   Affiliation as printed

   Epidemiology Optum Boston Massachusetts USA

   Epidemiology, Optum, Boston, Massachusetts, USA

3. Alejandro Arana

   RTI Health Solutions

   Affiliation as printed

   Epidemiology RTI Health Solutions Barcelona Spain

   Epidemiology, RTI Health Solutions, Barcelona, Spain

4. Andrea V. Margulis

   RTI Health Solutions

   Affiliation as printed

   Epidemiology RTI Health Solutions Barcelona Spain

   Epidemiology, RTI Health Solutions, Barcelona, Spain

5. Lisa J. McQuay

   RTI Health Solutions

   Affiliation as printed

   Epidemiology RTI Health Solutions, Research Triangle Park Durham North Carolina USA

   Epidemiology, RTI Health Solutions, Research Triangle Park, Durham, North Carolina, USA

6. Susana Perez‐Gutthann

   RTI Health Solutions

   Affiliation as printed

   Epidemiology RTI Health Solutions Barcelona Spain

   Epidemiology, RTI Health Solutions, Barcelona, Spain

7. Jesper Hallas

   University of Southern Denmark

   Affiliation as printed

   Public Health University of Southern Denmark Odense Denmark

   Public Health, University of Southern Denmark, Odense, Denmark

8. Nina Sahlertz Kristiansen

   University of Southern Denmark

   Affiliation as printed

   Public Health University of Southern Denmark Odense Denmark

   Public Health, University of Southern Denmark, Odense, Denmark

9. Marie Linder

   Karolinska Institutet

   Affiliation as printed

   Medicine, Centre for Pharmacoepidemiology Karolinska Institutet Solna Sweden

   Medicine, Centre for Pharmacoepidemiology, Karolinska Institutet, Solna, Sweden

10. Ingvild Odsbu

    Karolinska Institutet

    Affiliation as printed

    Medicine, Centre for Pharmacoepidemiology Karolinska Institutet Solna Sweden

    Medicine, Centre for Pharmacoepidemiology, Karolinska Institutet, Solna, Sweden

11. Brandon T. Suehs

    Humana (United States)

    Affiliation as printed

    Health Economics and Outcomes Research Humana Healthcare Research Louisville Kentucky USA

    Health Economics and Outcomes Research, Humana Healthcare Research, Louisville, Kentucky, USA

12. Yihua Xu

    Humana (United States)

    Affiliation as printed

    Health Economics and Outcomes Research Humana Healthcare Research Louisville Kentucky USA

    Health Economics and Outcomes Research, Humana Healthcare Research, Louisville, Kentucky, USA

13. Claudia Uribe

    Humana (United States)

    Affiliation as printed

    Health Economics and Outcomes Research Humana Healthcare Research Louisville Kentucky USA

    Health Economics and Outcomes Research, Humana Healthcare Research, Louisville, Kentucky, USA

14. Kwame Appenteng

    Astellas Pharma (United States)

    Affiliation as printed

    Pharmacovigilance Astellas Pharma US Northbrook Illinois USA

    Pharmacovigilance, Astellas Pharma US, Northbrook, Illinois, USA

15. Noah Jamie Robinson Aachen Pharmacovigilance Astellas Pharma B.V. Leiden The Netherlands Pharmacovigilance

    Astellas Pharma (Netherlands)

    Affiliation as printed

    Pharmacovigilance Astellas Pharma B.V. Leiden The Netherlands

    Pharmacovigilance, Astellas Pharma B.V., Leiden, The Netherlands