The Way We Make Progress Against Disease - Advanced (Text Version)
- Clinical Trials
The Way We Make Progress Against Disease
- What Are Clinical Trials?
- Research studies involving people
- Try to answer scientific questions and find better ways to prevent, diagnose, or treat disease
- Why Are Clinical Trials Important?
- Clinical trials translate results of
basic scientific research into better ways to prevent, diagnose, or treat disease
- The more people take part, the faster we can:
- Answer critical research questions
- Find better treatments and ways to prevent disease
- Do Many People Take Part in Clinical Trials?
- What Are the Different Types of Clinical Trials?
- Treatment
- Prevention
- Early detection/screening
- Diagnostic
- Quality of life/supportive care
- Treatment Trials
- What new treatments can help people with a particular disease?
- What is the most effective treatment for people with that disease?
- Clinical Trial Phases
Phase 1 trials
- How does the agent affect the human body?
- What dosage is safe?
- Clinical Trial Phases
Phase 2 trials
- Does the agent or intervention have an effect on the disease?
- Clinical Trial Phases
Phase 3 trials
- Is the new agent or intervention (or new use of a treatment) better than the standard?
- Participants have an equal chance to be assigned to one of two or more groups
- Randomized Trials
- Participants have an equal chance to be assigned to one of two or more groups:
One gets the most widely accepted treatment (standard treatment)
- The other gets the new treatment being tested, which researchers hope and have reason to believe will be better than the standard treatment
- Randomization
[ A graphical image (figure) is placed here. It shows a group of ten people and each person is uniquely numbered from one to ten. After a randomization process, this group divides into two subgroups made up of five people each. One is called Control Group and has the following numbered people: 5, 8, 9, 7, and 3. The other one is called Investigational Group and has the following numbered people: 4, 2, 6, 1, and 10. ]
- Why is Randomization Important?
- So all groups are as alike as possible
- Provides the best way to prove the effectiveness of a new agent or intervention
- Treatment Trials
- What new treatments can help people with a particular disease?
- What is the most of effective treatment for people with that disease?
- Treatment Trials
Placebos are almost never used:
- Placebos are used only when no standard treatment exists
- Patients are told of this possibility before deciding to take part
- Prevention Trials
- Evaluate the effectiveness of ways to reduce the risk of a particular disease
- Enroll healthy people at high risk for developing that disease
- Prevention Trials
- Action studies ("doing something")
- Agent studies ("taking something") -- also called "chemoprevention studies"
- Chemoprevention Trials
- Phase 3 chemoprevention trials compare a promising new agent with either a:
- Clinical Trial Protocol
- A recipe or blueprint
- Strict scientific guidelines:
- Purpose of study
- How many people will participate
- Who is eligible to participate
- How the study will be carried out
- What information will be gathered about participants
- Endpoints
- Benefits of Participation
Possible benefits:
- Patients will receive, at a minimum, the best standard treatment (if one exists)
- If the new treatment or intervention is proven to work, patients may be among the first to benefit
- Patients have a chance to help others and improve patient care
- Risks of Participation
Possible risks:
- New treatments or interventions under study are not always better than, or even as good as, standard care
- Even if a new treatment has benefits, it may not work for every patient
- Health insurance and managed care providers do not always cover clinical trials
- Patient Protection
- There have, unfortunately, been past abuses in patient protection
- Federal regulations ensure that people are told about the benefits, risks, and purpose of research before they agree to participate
- How Are Patients' Rights Protected?
- Informed consent
- Scientific review
- Institutional review boards (IRBs)
- Data safety and monitoring boards (DSMBs)
- How Are Patients' Rights Protected?
Informed Consent:
- Purpose
- Procedures
- Potential risks and benefits
- Individual rights
- How Are Patients' Rights Protected?
- Scientific review
- Institutional review boards (IRBs) are required by federal law for trials that are:
- Federally funded
- Subject to FDA regulation
- How Are Patients' Rights Protected?
Data and safety monitoring boards:
- Ensure that risks are minimized
- Ensure data integrity
- Stop a trial if safety concerns arise or objectives have been met
- Why Do So Few People Participate in Clinical Trials?
Sometimes patients:
- Don't know about clinical trials
- Don't have access to clinical trials
- May be afraid or suspicious of research
- Can't afford to participate
- May not want to go against health care provider's wishes
- Why Do So Few People Participate in Clinical Trials?
Health care providers might:
- Lack awareness of appropriate clinical trials
- Be unwilling to "lose control" of a person's care
- Believe that standard therapy is best
- Be concerned that clinical trials add administrative burdens
- Where to Find Clinical Trial Information
- National Institute of Dental and Craniofacial Research
National Institutes of Health
www.nidcr.nih.gov
Adapted from a slide presentation developed by the National Cancer Institute, for its Cancer Clinical Trials Education Series.