2. Introduction
► Clinical research is the scientific investigation of new medical treatments and
therapies to enhance patient care.
► It involves systematic investigation to understand diseases, treatments, and their
impact on patients.
► Clinical research is vital for several reasons:
► Advancing Medical Knowledge: It expands our understanding of diseases, therapies,
and medical technologies.
► Improving Patient Care: Findings from clinical research lead to better treatments and
care protocols.
► Key Objectives:
► To identify new treatments and therapies.
► To enhance existing medical practices.
► To ensure patient safety and well-being.
3. Introduction
► Patient centric approach :
► Puts patients at the heart of healthcare progress.
► Patient participation shapes future treatments.
► Improves health outcomes through research.
► Collaboration and Innovation:
► Collaborative efforts among healthcare experts.
► Catalyst for medical innovation.
► Ethical Foundations:
► Ethical guidelines protect participant rights.
► Ensure the well-being of those involved.
► Ensures ethical integrity and transparency.
4. Clinical Trials & their phases
► Clinical trials are scientific studies of the safety and efficacy of a new medical drug or
other treatment, conducted on human volunteers.
► Clinical trials are typically divided into four phases, each serving a specific purpose in
the development process.
► Clinical trials consist of four main phases :
► Phase 1
► Phase 2
► Phase 3
► Phase 4
► Clinical trials progress through four phases, from initial safety assessment in Phase 1
to large-scale testing in Phase 3, and finally, ongoing monitoring in Phase 4.
5. Phase 1: Early Safety Evaluation
► Objectives:
Assess safety, dosage and side effects.
► Small Group:
Involves a small number of healthy volunteers or patients (typically 20-80).
For cytotoxic candidates(eg-anti cancer drugs),the phase 1 trials are conducted on
patients)
► Duration:
Several months.
► Outcomes:
Identifies the maximum tolerated dose and initial safety profile.
6. Phase 2: Efficacy and Side Effects
► Objectives:
Evaluate effectiveness and further assess safety.
► Larger Group:
Enrolls a larger group of patients (around 100-300).
► Duration:
Several months to two years.
► Outcomes:
Determines the drug's effectiveness, dosage and side effects.
7. Phase 3: Large-Scale Testing
► Objectives:
Confirm effectiveness, monitor side effects and compare to existing treatments.
► Large-Scale:
Involves a much larger patient population (often thousands).
► Duration:
Several years.
► Outcomes:
Provides robust data on safety and efficacy for regulatory approval.
8. Phase 4: Post-Market Surveillance
► Objectives:
Monitor long-term safety, efficacy, and less common side effects.
► After Approval:
Conducted after regulatory approval and market release.
► Duration:
Ongoing for as long as the drug or treatment is in use.
► Outcomes:
Continuously assesses the treatment's real-world performance.
9.
10. Key Players
► Role: Lead the research study.
► Responsibilities:
► Designing the study protocol.
► Ensuring patient safety and care.
► Collecting and analyzing data.
► Interpreting results.
► Key Figure: Often a medical doctor or
principal investigator.
► Role: Ensure regulatory compliance.
► Responsibilities:
► Conduct site visits for quality
assurance.
► Verify data accuracy and
participant safety.
► Ensure adherence to study
protocols.
► Collaborate with investigators
and coordinators.
► Key Figure: Represents the sponsor or
regulatory agency.
Clinical investigators Clinical Research Associates
11. Key Players
► Role: Organize and manage study
activities.
► Responsibilities:
► Recruitment of participants.
► Scheduling visits and procedures.
► Maintaining study
documentation.
► Liaising with regulatory
authorities.
► Key Figure: Ensures smooth day-to-
day study operations.
► Role: Ensure ethical and regulatory
compliance.
► Responsibilities:
► Review and approve study
protocols.
► Protect participants' rights
and safety.
► Monitor ongoing studies for
compliance.
► Key Figure: Independent ethics
committees.
Clinical Research Coordinators Institutional Review Boards
12. Different stakeholders
in clinical research
► Sponsor
► Contract Research
Organization
► Investigators
► Ethics Committee
► Regulatory Agencies
► Clinical Research Coordinators
► Clinical Research Associates
► Data Manager
► Data Analyst
► Data Entry Operator
► Quality Assurance & Quality
Control Personnel
► Auditor
13. Documents Needed
► Purpose: To ensure participants
understand the study and
voluntarily agree.
► Content: Study objectives,
procedures, risks, benefits,
participant rights.
► Importance: Protects participant
rights and ensures ethical research.
► Purpose: Provides a structured
plan for the study.
► Content: Study objectives, design,
criteria, endpoints.
► Importance: Ensures consistent
study execution and clear
guidelines.
Informed Consent Forms (ICF) Protocol Documents
14. Documents Needed
► Purpose: Collects and records
patient data during the study.
► Content: Data fields for study-
specific information.
► Importance: Crucial for analyzing
study outcomes accurately.
► Purpose: Contains essential info
about investigational product.
► Content: Pharmacology, safety,
prior research findings.
► Importance: Guides investigators
for safe and effective study
conduct.
Case Report Forms (CRFs) Investigator's Brochure (IB)
15. Informed consent form
► Informed Consent Form: A document that explains a study and gets your
permission before you decide to participate.
► It includes key details of a research study such as its purpose, procedures,
potential risks, and benefits.
► Participants sign the form to indicate their understanding and voluntary
agreement to participate.
► Components of Informed Consent :
► Study Purpose: Clearly state the purpose of the research.
► Procedures: Describe the research procedures participants will undergo.
► Risks and Benefits: Outline potential risks and benefits.
► Voluntary Participation: Emphasize that participation is voluntary.
► Confidentiality: Explain how participant data will be handled.
► Contact Information: Provide researcher contact details for questions or
concerns.
16. Informed consent form
Informed Consent Process
► Discussion: Researchers must
engage in a meaningful dialogue
with participants.
► Comprehension: Ensure
participants understand the
information provided.
► Voluntary Agreement:
Participants should voluntarily
agree to participate without
coercion.
Ongoing Informed Consent
► Continuous Process: Consent is
not a one-time event; participants
should be informed of any
changes.
► Withdrawal: Participants have the
right to withdraw consent at any
time without consequences.
17. Data Collection
Methods for Data Collection
► Patient Interviews: Gathering information
directly from participants through structured
interviews.
► Medical Records Review: Extracting data
from patients' medical histories.
► Observational Studies: Observing
participants in real-life settings.
► Laboratory Tests: Collecting biological
samples for analysis.
► Surveys and Questionnaires: Administering
standardized forms to gather specific data.
► Electronic Health Records (EHRs): Utilizing
digital patient records for data extraction.
Tools for Data Collection
► Electronic Data Capture (EDC) Systems:
Streamlining data entry and storage.
► Mobile Apps: Enabling real-time data
input in remote locations.
► Wearable Devices: Monitoring patient
metrics continuously.
► Online Surveys: Efficiently collecting
responses from a geographically dispersed
group.
► Paper-Based Forms: Traditional method
when digital options aren't feasible.
18. Importance of Data Integrity
► Accurate Results: Ensures that research findings are reliable
and credible.
► Patient Safety: Inaccurate data can lead to incorrect medical
decisions.
► Regulatory Compliance: Maintaining data integrity is
essential for meeting regulatory requirements.
► Scientific Validity: Valid conclusions and contributions to
medical knowledge depend on data integrity.
19. Regulatory Compliance
Adherence to Guidelines
► Strict adherence to FDA (or
applicable regulatory authority)
guidelines is paramount.
► These guidelines ensure the
safety and ethical conduct of
clinical research.
Ethical Review Boards (ERBs)
► ERBs play a vital role in
participant safety.
► They review and approve
research protocols, ensuring
ethical standards are met.
► ERBs safeguard the rights and
well-being of study participants.
20. Challenges during research
► Patient Recruitment:
Challenge: Finding suitable participants can be difficult.
Importance: Adequate representation is crucial for study validity.
► Data Quality:
Challenge: Ensuring data accuracy and completeness is a constant effort.
Importance: Reliable data is essential for drawing valid conclusions.
► Regulatory Hurdles:
Challenge: Navigating complex regulations can be time-consuming.
Importance: Compliance is vital to ensure ethical and legal standards.
21. Benefits
Medical Advancements
► Research drives innovation in
medicine.
► New treatments and therapies
emerge.
► Life-saving drugs and
breakthroughs happen.
Improved Patient Care
► Research enhances the quality of
healthcare.
► Better understanding of diseases.
► Tailored treatments for
individual patients.
