Clinical Trials

– Research studies involving patients or
populations at risk for disease
– May be directed at questions of
causation, prevention, early detection or
treatment
– Designed in a scientific manner and
conform to ethical standards

Importance of Clinical
Trials
􀂄To increase knowledgeTo knowledge
􀂄To provide ““evidenceevidence-based based” patient care or
prevention of disease
􀂄To improve the quality of careTo care
– Patients participating in a trial tend to receive
better care
– Quality of care for other patients who are not
participating in a trial can also be improved (i.e.,
supportive care)



Who Conducts Clinical
Trials?
􀂄Academic centersAcademic centers
􀂄Groups of academic centers ––cooperative groupscooperative groups
􀂄Government agencies or institutionsGovernment institutions
􀂄Corporations (pharmaceutical companies, biotechnology companies)
– Usually done via academic or private
institutions



The Conduct of Clinical
Trials
􀂄Frame a hypothesis based upon present knowledgeFrame knowledge
– Is the study question important and relevant?
􀂄Design a study to test the hypothesisDesign hypothesis
􀂄Write a detailed plan of the study (protocol)
􀂄Obtain ethical review and approvalObtain approval
􀂄Conduct study, adhering closely to the protocolConduct protocol
􀂄Collect relevant, preCollect pre-determined data elements
􀂄Ensure that the data is of the highest quality possiblepossible
􀂄Analyze the data and draw conclusionsAnalyze conclusions



The Protocol Document
􀂄Detailed plan and instructions for the conduct of the trial
􀂄Guidebook for those involved in the dayday-to to-day care of the patient enrolled
on the trial



Elements of the Protocol
Document
􀂄Title Page
􀂄Objectives (primary and secondary)
􀂄Background and rationaleBackground rationale
􀂄Eligibility Criteria (inclusion and exclusion criteria)
􀂄Evaluation parameters ––baseline and those that support and confirm eligibilitysupport eligibility
􀂄Treatment planTreatment plan
– Modifications
– Supportive care
– Concurrent therapies (including contraindications)

Treatment Plan (concon’’t)
– Surgical guidelines
– Radiation therapy guidelines
􀂄OffOff-study criteria
􀂄PostPost-study evaluation (what tests should be
done and how often)
􀂄Data collection and reporting proceduresData procedures
􀂄Statistical sectionStatistical section
– Sample size, study duration, relevant endpoints

Statistical sectionStatistical section
– How endpoints will be evaluated (e.g.,
response, adverse events)
􀂄MultiMulti-institutional guidelines (if
applicable)

Ethical ConsiderationsConsiderations
– Discussion of
rationale for risks
􀂄Are risks reasonable in relation to the anticipated benefits?
􀂄Are risks reasonable in relation to the importance of the knowledge to be gained?

􀂄Ethical Considerations (concon’’t)
– Informed consent procedures
– Conflicts of interest
􀂄Pharmaceutical SectionPharmaceutical Section
– Information about the study drug(s s)
􀂄Publication PolicyPublication Policy
– Who will be involved, registration of the trial
􀂄References



Good Clinical Practice
(GCP)
􀂄International and scientific quality standardstandardfor:for:
– Designing
– Conducting
– Recording
– Reporting
Trials that involve the participation of
human subjects



ompliance with GCP
􀂄Provides public assurance that the:
– Rights, safety and well well-being of patients
are protected and consistent with the
Declaration of Helsinki
􀂄Ensures that clinical trial dataclinical dataand and reported resultsreported resultsare:are:
– Accurate
– Credible