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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: 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 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 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