Investigator-Initiated Clinical Research

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Investigator-InitiatedClinical Research

• Planning, Developing, Conducting, Managing, and
  Succeeding
• Wm. Hirschhorn, M.S.
• Director, OCT RQI and
• Adj. Prof of Regulatory Affairs
• Office of Clinical Trials Research
• Quality Improvement
• 2-5245
• 2-3106

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Objectives

• To begin to be able to
• Describe the differences between
  investigator-initiated clinical research and
  sponsor-initiated clinical research.
• Successfully plan your own clinical research
  project and assume ownership for your work and
  achievement.
• Thoroughly develop your research proposal.

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Objectives

• Successfully initiate and conduct your clinical
  research program.
• Comply with institutional, federal, and other
  policies, guidances, and regulations in managing
  your clinical research program.
• Count on the Offices of Clinical Trials and
  BioStatistics to assist you.

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Isnt Clinical Research All Alike?

• Basic science NIH, bench-top and other. Grant
  from department or government.
• Industry (sponsored) - initiated clinical
  research. FDA regulated.
• Usually leads to marketing registration
• Investigator-initiated
• research
• Its your project - ownership!
• Various funding?
• Multiple responsibilities!!

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(1) Examples of Investigator-Initiated Research

• Sponsor-Investigator or Investigator-IND
• Investigator submits a Letter of Intent (LOI) to
  industrial sponsor and OCT/BioStatistics
• Your interest!!
• Project proposal Youre the expert
• Technical and logistical support Kinetics,
  clinical/therapeutic section, investigational
  drug, Drug Master File (Inv.-IND), GCRC, etc.
• Address Federal regulatory requirements!!
• OCT will help you
• Human research participant population??
• Possible funding??
• Data ownership??

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(2) Investigator-Initiated Research Funded

• Proposal (LOI)
• Resources
• Application process
• NIH funded
• Refer to Dr. Steven Housers presentation titled
  Planning a Successful NIH Research Application
  (12/11/03)
• Other funding (for-profit, private sources)
• Publications??

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(3) Institutional Supported Research

• Department funded possibly no outside funding
• LOI
• Resources
• Assistance from BioStatistics
• Assistance from OCT and GCRC

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Remember!!

• Wherever you go, whatever you do - your research
  needs should satisfy
• Your unswerving attention and complete ownership
• Department chairs
• knowledge and approval
• Interaction with Sponsored
• Projects
• Assistance from OCT
• IRB review and approval

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Who is an Investigator? (Sponsor-Investigator)

• An individual (not a corporation) who both
  initiates (plans and designs) and conducts an
  investigation, and under whose immediate
  direction the study drug is administered or
  dispensed. Investigator IND.
• 21 CFR 312.3 and ICH E-6 (GCPs) 1.54
• Plans, designs, conducts, monitors, manages the
  data, prepares reports, and oversees all
  regulatory and ethical matters
• 21 CFR 312 Subpart D
• Absolute responsibility and
• accountability required!!

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So, how do you go about doing this?

• Take ownership
• Devote sufficient time and initiative
• Dont lose focus of your objective
• Ask yourself Do I really want to do this?
• Work with OCT BioStatistics
• Work with Sponsored Projects and the IRB
• Plan for the long-haul

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Steps to Success

• Development Conduct Management
• Chronological steps
• Project Development
• Letter of Intent
• Proposal
• Timeline
• Protocol development and data collection forms
• Budget development
• Funding
• Contract
• IRB review and approval
• Study conduct and monitoring
• Data Review and Analysis

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Steps to Success

• What is the Letter of Intent (LOI)?
• Statement of your research interest in a
  particular drug or device
• Your interest in conducting a clinical trial with
  the drug or device
• Type of human research participant population
• Anticipated time to conduct the trial
• Funding source, if known
• Who will supply the drug or device?

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Steps to Success

• What should be contained in the LOI?
• Rationale for the proposed trial
• Design for the trial
• Dose, blinding, schedule, and comparison groups
• Characteristics of the population
• Patient enrollment feasibility
• Any unique features of the proposed trial
• Who would benefit from the new information?
• Patients
• Industry
• Temple

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Steps to Success

• The LOI Points to Consider
• Be clear, brief and to the point
• Avoid overuse of jargon
• Be unique
• Here creativity is appropriate
• Appeal to your audience(s)
• Follow all guidelines
• Show that you own the proposal

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Steps to Success

• Developing the Protocol
• Format from company or OCT
• Build in flexibility
• Revisions will absolutely occur
• Make it informative
• Utilize the appendix section
• Supply important supplemental reading
• See BioStatistics for valuable assistance
• Consult with experts beside yourself
• Carefully edit the final edition
• Consult with OCT for template and IRB submission
  assistance
• What do I want to do with the data?
• Publication?
• Where? When? Requirements?

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Steps to Success

• The Budget
• Think about the following things
• Department interest
• Industry interest
• OCT can assist in identifying outside sources
• Staff resources
• Co-investigator, CRC
• Consultants and resources (i.e., laboratory,
  special services, data management, monitoring,
  technical)
• OCT benchmarks budgets successfully
• Complete the SPAF at the appropriate time

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Steps to Success

• Some resources I need to identify?
• Clinical Laboratory and special lab support
• Therapeutic specialties (i.e., cardiology, D.I.,
  G.I., etc
• Regulatory guidance
• Project management (i.e., QA, timeline adherence)
• Patient recruitment and advertising
• Data management
• Pharmacy
• Facility charges
• Computer support

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Steps to Success

• Project timeline
• Development phase Plan your work and work your
  plan!
• Regulatory (IRB, FDA, NIH) and legal (contract)
• Project conduct phase
• Recruitment
• Trial conduct
• Data collection
• Data Quality Assurance
• Data review and analysis
• Publication

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Steps to Ensure Success

• Study conduct and Monitoring
• KNOW YOUR OBLIGATIONS and RESPONSIBILITIES
• Administrative
• Federal
• Temple, State and local
• Take charge of your project
• Champion your expertise
• Mentor
• See it through to completion
• Understand the requirement for monitoring and
  Quality Assurance - OCT

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Steps to Ensure Success

• Tips to ensure you have fun and succeed
• Frequently review your project for schedule
  adherence
• On-track in what areas?
• Off-track in what areas?
• Finances
• Logistics
• Prepare for the worst case scenario
• Get use to paperwork its a fact of life
• Unexpected delays or issues
• Count on this happening
• Identify the source
• Contingency planning work with OCT/OCR

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Whats next?

• In March 2004 Investigator initiated clinical
  research
• Data management and analysis
• Monitoring
• Quality Assurance
• Study outcomes
• Publications
• Other really exciting and useful topics