Transcript Research Pharmacy

Yesterday, today, and
tomorrow
Goals of this presentation :
A look at clinical research from a
pharmacy point of view.
 What will NOT be a focus of this presentation:
 Social and ethical questions and considerations
 Legal and regulatory issues
 Protocol development and IND applications
 Statistical considerations in clinical trials
Why do we do research?
Improve patient health through
improved patient care
 Types of studies:
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Observational: e.g. epidemiologic, cohort, case
control, longitudinal, etc
Interventional
Interventional clinical trials
 Clinical trials allow us to evaluate the
efficacy and safety of new medications or
agents
 Usually in comparison to an established
standard of care
 Randomized, double-blind studies (the
“Gold Standard”)
Why do we do interventional
clinical trials?
 To obtain data to present to the FDA !
Drug companies are trading drug (and
medical care) for data on patient outcomes
Stages in drug development
 Drug discovery / development
 Pre-clinical testing
 Investigational New Drug application (IND)
 Clinical testing
 Phase I
 Phase II
 Phase III
 Phase IV
Phase I trials
 First use of a new drug in humans
 Small numbers of patients, usually healthy volunteers
 Looking primarily at safety and dose determination
 Safety – dose limiting toxicity (DLT)
 Dose determination – increase dose to toxicity
 Pharmacokinetic and pharmacodynamic studies
Phase II trials
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Determine short-term risks and safety
Looking at effectiveness and best tolerated dose level
Dose(s) based on information from Phase I studies
Also looking at side effects
Study subjects are the types of patients the agent is
intended to treat
Phase III
 Large scale trials (e.g. 300 – 1000+ subjects) looking at
safety and efficacy usually in a randomized, controlled
fashion often involving multiple centers
 Seek to assess risk/benefit ratio and gather date for
FDA approval of the agent / labeling
 If data gathered from Phase I, Phase II, and Phase III
trials demonstrate safety and efficacy => New Drug
Application (NDA) may be filed with the FDA
Phase IV trials
 Post approval studies
 May be required as a condition of approval
 Long term safety studies
 Comparison studies
Important elements in clinical trials
 The protocol
 The informed consent
 Regulatory bodies
 Blinding
 Randomization
 Finance
The Study Protocol
 Common elements:
 Title
 Protocol summary or synopsis
 Background and rationale
 Study design / investigational plan
 Randomization and blinding
 The investigational product
 Inclusion and exclusion criteria
 Adverse events
 Data analysis
Informed Consent Form (the ICF)
 Background
 Elements
 Description of trial
 Voluntariness / withdrawal of consent / alternatives
 Risk / benefit ratio
 Questions
 Contacts
 Signatures
Regulatory bodies
 Institutional review board (IRB) – patient protection
 Institutional bio-safety committee (IBC) and the
Recombinant DNA Advisory Committee (RAC)
 Food and drug administration (FDA)
 Compliance office / office of research integrity (ORI)
Blinding
 Single vs double blind
 Physical blinding: Size, shape, taste, appearance,
smell, etc.
 Time considerations
 Double dummy designs
Randomization
 History
 IVR and IWR systems
 Stratification
 Blocks
Finance
 Contracts and grants (OCGM)
 Cost of doing a study
 Conflict of interest concerns
The “Players”
 Patients
 The PI (and sub I’s), the 1572 form (Statement of
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Investigator)
Monitors
Research Coordinators
Regulatory entities (IRB, IBC, FDA, ORI / compliance,
etc)
CRO’s
The “Players” (continued)
 Support services
 Laboratory
 Radiology
 Pharmacy
 Statistics
 Finance
In the beginning…
 “What’s this and where did it come from?” (x2)
 “…and you’re also in charge of investigational drugs.”
 “I think it’s in that cabinet over there next to the sink”
 “We are missing a week’s worth of entries on the temp
record.”
 “Is this the next envelope?”
 “I’m going to San Diego for a meeting!”
 “Budgets???”
The present
 Personnel
 Types of studies
 Training
 Records and documentation
 Monitoring
 Disposal of study materials
Elements of a Research Pharmacy
 Personnel
 Storage space / equipment
 Temperature monitoring
 Documentation / record retention
 The “information sheet” or “summary sheet”
 Study budget
Pharmacy study budgets
 Common Elements
 Start-up
 Maintenance
 Randomization
 Study drug preparation and dispensing
 Close-out fee
 On – call fees
What’s ahead?
 Why utilize pharmacy services?
 Exotic agents
 Electronic data capture and records
 Impact of healthcare systems
 Community based research