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Challenges_ISR_IDSSFINAL
This document summarizes a presentation about challenges and experiences with multinational investigator-sponsored research (ISR) trials. It discusses the roles of investigators and sponsors in ISRs. It outlines key elements of a successful multinational ISR, including study design, infrastructure, site selection, and data analysis. Potential challenges include feasibility of treatment across countries, sample size, regulatory compliance, contractual issues, and limited resources. However, multinational ISRs also provide opportunities to expand medical knowledge through independent research and promote further studies.
Challenges_ISR_IDSSFINAL
- 1. Challenges and Experiencein the Execution of Multinational ISRs Valentina Kutyifa MD, PhD, FESC, FHRS, FACC University of Rochester Medical Center, Rochester, NY Semmelweis University, Budapest, Hungary International Dead Sea Symposium Tel-Aviv, March 8th 2016
- 2. Disclosures Valentina Kutyifa MD,PhD, FESC, FHRS, FACC Company Relationship Boston Scientific Research Grant Zoll Research Grant Hold no stock or stock options in any device company and not a member of any corporate advisory group or speakers’ bureau. This presentation represents my personal perspective of ISRs.
- 4. Introduction In opportunities, there arechallenges… In challenges, there are opportunities…
- 5. Multinational ISRs -Outline 1 Multinational ISRs.Who are involved and what are their roles? II How should a multinational ISR be executed with success? ISR Success Plan. Key elements and potential challenges III Multinational ISRs. Risks and Rewards.
- 6. Definition Investigator Sponsored Research Researchindependently proposed and conducted by institutions, physicians, and researchers who seek to advance medical and scientific knowledge. Goals: Expand Knowledge Base on Current Use/Indication„ Evaluate Different Treatment Strategies„ Assess Potential New Use/Indication „ IIT made easy,Yung Jue Bhan et al.
- 7. Definition Sponsor and Investigator Sponsor: a person who takes responsibility for and initiates a clinical investigation. It may be an individual or company, government agency, academic institution, private organization, or other organization. Investigator: An individual who actually conducts the clinical investigation (i.e., under whose immediate direction the drug is administered or dispensed to a subject). OR the device under investigation is implanted). US FDA 21 CFR 312.3
- 8. Individual whoboth initiates & conducts a clinical investigation, and under whose immediate direction the investigational drug is administered or dispensed (or medical devices are implanted). The term does not include any person other than an individual. The requirements applicable to a sponsor investigator under this part include both those applicable to an investigator and a sponsor. Definition Investigator Sponsor US FDA 21 CFR 312.3
- 9. Responsibilities Investigator Sponsor Preparing abstract(s),manuscript(s), presentations at scientific meetings Communication of the study results, safety reporting Analysis/interpretation of the data Status updates, timely communication with sponsor Site selection/initiation, study conduct Appropriate regulatory and ethics approval Study design, protocol Adapted from Lori T. Gilmartin
- 10. Responsibilities “Sponsor” Preparing abstract(s), manuscript(s),presentations at scientific meetings Communication of the study results, safety reporting Analysis/interpretation of the data Status updates, timely communication with sponsor Site selection/initiation, study conduct Appropriate regulatory and ethics approval Study design, protocol
- 11. Responsibilities “Sponsor” Keeps contactwith the investigator sponsor to faciliate the conduct of independent research Provides sufficient financial support for the study Works closely with investigator sponsor to identify research scope
- 12. Multinational ISRs -Outline 1 Multinational ISRs.Who are involved and what are their roles? II How should a multinational ISR be executed with success? ISR Success Plan. Key elements and potential challenges III Multinational ISRs. Risks and Rewards.
- 13. ISR Success Plan Planto Succeed Step 1. Make a Plan Adapted from Lori T. Gilmartin Make a Plan
- 14. ISR Success Plan Planto Succeed Step 2, 3, 4.Work the Plan Follow the Plan Record the Conduct of the Plan Report on the Plan Adapted from Lori T. Gilmartin Make a Plan
- 15. Key Elements ofMultinational ISRs Preparing abstract(s), manuscript(s), presentations at scientific meetings Communication of the study results, safety reporting Analysis/interpretation of the data Status updates, timely communication with sponsor Site selection/initiation, study conduct Appropriate regulatory and ethics approval Study design, protocol Adapted from Lori T. Gilmartin
- 16. Key Elements ofMultinational ISRs Study Design/Protocol Relevant study question Appropriate sample size (statistician) Appropriate end points Detailed plan for study conduct, countries/sites in the study Needs Feasibility/acceptance of treatment across countries Lack of statistical consultants Sample size indirectly influences the number of countries/sites When to go to another country?Which country? How many countries? Challenges Study design, protocol
- 17. Key Elements ofMultinational ISRs Infrastructure and Know-How Appropriate regulatory and ethics approval Infrastructure and resources Know-How Personnel Resources Facility (space, services and equipment) Financial Resources (given) Functional Infrastructure Needs Not always established Just for one study? Lack of know how, consider CRO Cost considerations Financial Resources – given? New teams Challenges
- 18. Key Elements ofMultinational ISRs Site selection/initiation, study conduct Selected countries Selected sites Contracts Motivation Data quality Needs Feasible for the study Contracts often need input from legal Country-specific Governing laws Indemnification Motivating sites might vary by country and culture Cultural/ language barriers Monitoring is costly Challenges Site selection/initiation, study conduct
- 19. Key Elements ofMultinational ISRs Database/ Analytical Resources Resources Database programmer Data analyst PhD biostatistician Involvement of study site investigators Needs Significant costs and only short-term Sub-study activity might be challenged by very limited resources provided after study completion Academic competition Challenges Analysis/interpretation of the data Preparing abstract(s), manuscript(s), presentations at scientific meetings
- 20. Multinational ISRs -Outline 1 Multinational ISRs.Who are involved and what are their roles? II How should a multinational ISR be executed with success? ISR Success Plan. Key elements and potential challenges III Multinational ISRs. Risks and Rewards.
- 21. Multinational ISRs. Risksand Rewards. RISKS REWARDS Inadequate infrastructure Lack of know-how Lack of indemnification Risk of regulatory non- compliance Non-validated data (false claims) Expansion of medical knowledge Independent research Promotes further research
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- 23. Synthesis In the ISRopportunities, there are ISR challenges… But in those ISR challenges, there are NEW opportunities… THANK YOU