Assignment No 1
CRP102SA 2/10/2013
G Vellasamy
Clinical Study Design – Critical Issues:
A clinical study is a planned experiment for the purpose of evaluating one or more beneficial therapies or treatments. Study designs are the critical elements of a clinical study and they are important in enhancing the reliability and validity of experiments. Some important elements are listed below:
Phases
Objective of the clinical study
Clinical study elements and description o For scientific evidence and knowledge o Prove the concept o Evaluation of product features and capabilities o Obtain initial safety data o Substantiate claim and indication for use o Establish degree of efficacy or effectiveness o Compare with competitor product o Marketing evaluation o Finance and insurance
Types of the clinical study o o o o o o o o o o o
Prospective or review
Blinded/masked or open label
Randomized
Non-Randomized
Single-Center
Multi-Center
Phase I, II, III Trials
Active control or placebo
Normal subjects or patients
Actual or surrogate clinical endpoints
Statistically significant or un reliable
Ethical considerations to consider in the study o o o o o o o o IRB requirements (approvals)
Declaration of Helsinki
Informed consent
Compliance
Use of placebos and positive control
Use of normal subjects
Use of investigational material and standard human care
Vulnerable populations eg. Children
Regulatory requirement in the study o o Overall investigation Plan
Phase 1 - Normal subjects: usually < 30 subjects, at one facility, safety parameters Phase 2 - Patients: about 50 - 100 subjects; safety and initial efficacy; blinding, randomization, controls used
Phase 3 - Patients: 100->1000; multiple sites; main study; blinding, randomization, controls used
Phase 4 - Patients (post-marketing): Surveillance or study; labeling issues
o o o
Elements to consider in devising the study
o
Monitoring visit/ reports
o o o o o o o o o o o o o o o o o o o
Pilot study
Main study
Study population
ICH - GCP requirement
Investigator’s Brochure
Study Protocol
Finance and Insurance
Subject compensation
Investigator qualifications/ certifications certifications) Sponsor
FDA approvals
Subject screening and enrolment procedures
Investigational products accountability
Study safety
Investigators
Study site coordinators
Research nurses
Pharmacist and dispensing staff
Laboratory staff
(eg
CV,
Monitoring elements o o o o o o Pre-study visit; on-site; visits; telephone; e-mail; fax
Inclusion/exclusion criteria
Adverse effects informed
Informed consent
Signed, dated and completed Case Report Forms
Audit certificates
