Download The Clinical Trials Process - International Myeloma Foundation

Cancer Clinical Trials:
In-Depth Information
The Drug Development and
Approval Process
1. Early research and preclinical testing
2. IND application filed with FDA
3. Clinical trials (phases 1, 2, and 3)
4. NDA filed with FDA
5. FDA validates claim and approves drug
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Phases of Clinical Trials
• Phase 1: 15-30 people
– What dosage is safe?
– How should treatment be given?
– How does treatment affect the body?
• Phase 2: Less than 100 people
– Does treatment do what it is supposed to?
– How does treatment affect the body?
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Phases of Clinical Trials
• Phase 3: From 100 to thousands of people
– Compare new treatment with current standard
• Phase 4: From hundreds to thousands of people
– Usually takes place after drug is approved
– Used to further evaluate long-term safety and
effectiveness of new treatment
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Types of Clinical Trials
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Treatment
Prevention
Screening and early detection
Diagnostic
Genetics
Quality-of-life / supportive care
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Treatment Trials
• Test safety and effectiveness
of new agents or interventions
in people with cancer
• Possible benefit:
– Early access to new treatments
• Possible risk:
– Occurrence of unknown side
effects
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Prevention Trials
• For people at risk of developing
cancer
• Action studies vs. agent studies
• Possible benefit:
– Early access to new
interventions
• Possible risk:
– Unknown side effects and
effectiveness
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Screening and Early-Detection Trials
• Assess new means of detecting cancer earlier
in healthy people
• Possible benefit:
– Detecting disease at an earlier stage, resulting in
improved outcomes
• Possible risks:
– Discomfort and inconvenience
– If imaging technique is studied, exposure to xrays or radioactive substances
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Diagnostic Trials
• Develop better tools for classifying types and
phases of cancer and managing patient care
• Possible benefits:
– New technology may be better and less invasive
– Earlier detection of recurrences
• Possible risk:
– May require people to take multiple tests
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Genetics Trials
• These trials seek to:
– Determine how one’s genetic makeup can
influence detection, diagnosis, prognosis, and
treatment
– Broaden understanding of causes of cancer
– Develop targeted treatments based on the genetics
of a tumor
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Quality-of-Life / Supportive Care
Trials
• Aim to improve quality of life for patients and
their families
• Possible benefit:
– Early access to new treatment
• Possible risk:
– May not benefit from participation
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Clinical Trial Protocol
A written, detailed action plan that:
• Provides background about the trial
• Specifies trial objectives
• Describes trial’s design and organization
• Ensures that trial procedures are consistently
carried out
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Investigational Drug Use
Outside of a Clinical Trial
• Group C drugs
• Treatment Investigational New Drug
application
• Compassionate use program
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Clinical Trial Design
• Eligibility criteria: Can range from general
(age, sex, type of cancer) to specific (prior
treatment, tumor characteristics, blood cell
counts, organ function); eligibility criteria also
vary with trial phase
– Varies with protocol and phases
• Endpoint: Measurable outcome that indicates
an intervention’s effectiveness
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Clinical Trial Design
• Randomization: A method used to prevent
bias in research; a computer or a table of
random numbers generates treatment
assignments, and participants have an equal
chance to be assigned to one of two or more
groups (e.g., the control group or the
investigational group)
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Randomization
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Clinical Trial Design
• Stratification: Categorizing subjects into
subgroups by specific characteristics
– Enables researchers to look into separate
subgroups to see whether differences exist
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Stratification
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The Final Step: FDA Approval
• Review of New Drug Application (NDA) or
Biologics License Application (BLA)
• Labeling
• Continued monitoring
• Feedback
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Releasing the Results
of Clinical Trials
• Peer-reviewed journals
• Public announcements
– Results not made public until end of trial
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Improving Cancer Prevention,
Detection, and Treatment
Once proven safe and effective in a clinical trial,
an intervention may become the new standard of
care
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Advancing Cancer Care
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Gleevec for chronic myelogenous leukemia
Cervical cancer: improved survival rates
Breast cancer: less extensive surgery
Intron-A for melanoma
Biological therapy
Monoclonal antibodies
Cancer vaccines
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Evolution of Participant Protection
• Nuremberg
• Tuskegee Syphilis Study
• National Commission for the Protection of
Human Subjects of Biomedical and Behavioral
Research
• Belmont Report
• National Research Act
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Government Oversight of
Safeguards for Participants
• Office for Human Research Protections
(OHRP)
– The Common Rule
• FDA Regulations
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Protecting Participants
Before a Trial
• Scientific review by sponsoring organization
• Institutional review board approval
• Informed consent
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Protecting Participants
During a Clinical Trial
• Institutional review boards (IRBs)
• Data and safety monitoring boards (DSMBs)
– Minimize risks
– Ensure integrity of data
– Can stop study if necessary
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Barriers to, Benefits of, and
Risks of Participation in Clinical
Trials
Barriers to Adult Participation in
Clinical Trials
Physicians and other health professionals may:
• Be unaware of appropriate trials
• Be unwilling to lose control of patient’s care
• Believe that standard therapy is best
• Believe that clinical trials are more work
• Harbor concerns about the patient’s care or
how the person will react to suggestion of
clinical trial participation
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Barriers to Adult Participation in
Clinical Trials
Patients may:
• Be unaware of clinical trials
• Lack access to trials
• Fear, distrust, or be suspicious of research
• Have practical or personal obstacles
• Face insurance or cost problems
• Be unwilling to go against their physicians’
wishes
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Benefits of Participating in
Clinical Trials
• Early access to new treatments
• Active role in own health care
• Participation in advancing medical knowledge
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Risks of Participating
in Clinical Trials
• New treatments are not always better than
standard care
• Unexpected side effects
• May not work for everyone
• Additional cost
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NCI-Sponsored
Clinical Trial Programs
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Clinical Trials Cooperative Group Program
Clinical Trials Support Unit (CTSU)
Community Clinical Oncology Program (CCOP)
Minority-Based Community Clinical Oncology
Program
• Cancer Centers Program
• Clinical Grants Program
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Referring Patients to Clinical
Trials
Health care professionals should know:
• Trial objectives
• Eligibility criteria
• Treatment and prevention options
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Locating NCI Clinical Trials
in the Community
NCI clinical trial registry:
• Nation’s most comprehensive resource on
cancer clinical trials
• www.cancer.gov
• 1-800-4-CANCER (1-800-422-6237)
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