Concept

1
Concept
Mentor
Mentor
Concept
Funding
Protocol
Submission (PRMC, IRB, FDA)
Patient Accrural
Manuscript submission
2
Fellow requirements

• Guidelines roughly 1 month per 6 month period gt
  50 time devoted to a protocol
• 6 month Letter of intent
• 12 month Submission of protocol
• 24 month Protocol finalized, PRMC, IRB approval.
  Expect 1 page progress report and plans to
  publish.

3
Committee

• 4 faculty members
• Committee chair a member of advisory committee
  Ratner, Picus, Fracasso, Graudbert , Weilbaecher.

4
The Idea

• Your area of interest
• Mentors vs. your own concept

Heme
BMT
Oncology
5
Letter of Intent

• Basic study design
• Basic rationale
• Preclinical work
• Pilot studies
• STATISTICAL CONSIDERATIONS
• Provide statistical rationale for sample size and
  study design
• www.biostatist.wustl.edu

6
Funding

• No funding
• Observational/ Standard of Care
• Industry Sponsored
• Independent Funding
• Mentor with grant
• Applying for grant

7
Protocol

• Introduction
• Rationale
• Schema
• Study Design
• Inclusion/Exclusion criteria
• Endpoints

8
Intro/Rationale

• Investigators Brochure
• Drug safety
• Pharmacology
• Preclinical data
• Disease specific discussion

9
Schema/Study Design
10
Inclusion/Exclusion Criteria

• Liberal enough to allow adequate enrollment
• Restrictive enough to target certain population
  and minimize toxicities

11
Protocol Shells

• Pharmaceutical Company
• Colleagues with similar protocol
• Mentors previous protocol

12
PRMC

• Protocol Review and Monitoring Committee
• Siteman Cancer Center
• Submission required for all trials involving
  cancer patients

13
PRMC

• Premise To provide scientific peer-review of
  cancer-related studies.
• Meet monthly
• Deadline 2 weeks prior to meeting
• Reviewers
• 2 physicians (primary reviewers)
• Biostatisician
• Data manager

14
PRMC

• Submission requirements
• PRMC submission form
• Human studies committee forms
• Protocol
• Investigational drug brochure
• Data collection forms
• Consent form

15
PRMC

• Approved
• Contingent
• Respond to evaluation
• Does not go back to committee
• Deferred
• Overhaul necessary
• Full committee review necessary
• Not approved

16
IRB

• medicine.wustl.edu/hsc/
• Human studies training module
• medicine.wustl.edu/hsc/education/
• Expedited versus full committee review
• Case reports
• Expedited minimal risk
• Full committee review greater than minimal risk

17
IRB

• Reviewers
• 3 per protocol
• Various backgrounds
• Weekly meetings (deadline roughly 3 weeks prior)
• Submission requirements and responses are same as
  PRMC

18
IRB

• Responses due in 60 days
• Refer to handout
• Cost
• Free for Washington University investigators
• 2000 for pharmaceutical companies with 500
  renewal cost each year

19
IRB

• Form 1
• Additional forms based upon study

20
IND

• Necessary when
• Involve a drug, biologic, or significant risk
  device that is not approved by the FDA.
• Involve a commercially available drug to that is
  being tested to support a new indication or
  support a change in advertising or labeling of
  the product.
• Involve a commercially available drug that is
  being administered via a new route (that
  significantly increases the risks) or for use in
  a different part of the body.
• Involve a commercially available drug that is
  being given at a dosage level that might
  significantly increase the risk to the subject
  population, or that decreases the acceptability
  of the risk.
• Involve a commercially available drug that is
  going to be used in a new patient population that
  may result in a significant increase in risk(s)
  to the patient population.
• Involve a commercially available device that may
  be deemed significant risk when used for an
  unapproved purpose.

21
IND

• Does not have to be approved by FDA prior to
  PRMC/IRB submission
• Approval prior to enrollment of patients

22
GCRC

• General Clinical Research Center
• Prepare budgets
• PRMC, IRB, IND applications
• Provide support staff
• Nurse coordinators
• Phlebotomy
• Testing
• Patient rooms

23
Patient Recruitment

• Primary Sources
• Clinics and Hospitalized Patients
• Study opening announcement,
• Open clinical protocol book
• Consult service
• Referrals?
• Volunteers for Health
• Web site
• Flyers
• Health Fairs
• Advertisements

24
(No Transcript)
25
Data Collection

• Plan ahead, develop thorough CRF
• Collect all clinical data that you may need.
• Objective Biological data
• Tissue
• Imaging
• Subjective data
• Questionnaires (use validated forms if possible)

26
(No Transcript)
27
Analysis

• Plan ahead
• Data base design
• Statistical testing
• Expert support / collaboration
• pathologists, statisticians, core analysis groups
• Outsourcing
• Additional Funding

28
External Funding

• American Association for Cancer Research (AACR)-
• American Cancer Society (ACS)
• American Society of Clinical Oncology (ASCO)
• American Society of Hematology (ASH)
• BJ Hospital Foundation
• Cancer Research Institute http//www.cancerresear
  ch.org/ Postdoctoral Fellowship Program
  http//www.cancerresearch.org/postdoc.html
• The Leukemia Lymphoma Society
  http//www.leukemia-lymphoma.org/hm_lls
• National Cancer Institute (NCI)
  http//deainfo.nci.nih.gov/flash/awards.htm
• The National Institutes of Health (F30 award)
• The Ruth L. Kirschstein-NRSA individual
  fellowship application
• Prostate Cancer Foundation
• Washington University Internal Competitions
  http//internalcompetitions.wustl.edu/ocfr/grants.
  nsf/Achievement?OpenView