Unformatted text preview: Human Clinical Studies
S. Laguette ME 128 Fall 2007 November 19, 2007
11/19/07 ME 128 1 Human Clinical Studies
Clinical Trials Types of Clinical Trials Patient Protection Phases 11/19/07 ME 128 2 Clinical Trial
A Clinical Trial (also Clinical Research) is a research study in human volunteers to address the safety and effectiveness of a device or system. Regulatory requirements will help define the scope and magnitude of the trial. Studies are conducted under Investigational Device Exemption (IDE)
11/19/07 ME 128 3 What Are Clinical Trials?
Research studies involving people Try to answer scientific questions and find better ways to prevent, diagnose, or treat disease
11/19/07 ME 128 4 Why Are Clinical Trials Important?
Clinical trials translate results of basic scientific research into better ways to prevent, diagnose, or treat disease 11/19/07 ME 128 5 What Are the Different Types of Clinical Trials?
Treatment Prevention Early detection/screening Diagnostic Quality of life/supportive care
11/19/07 ME 128 6 Treatment Trials
What new treatments can help people with a particular disease? What is the most effective treatment for people with that disease?
11/19/07 ME 128 7 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
11/19/07 ME 128 8 Randomization 11/19/07 ME 128 9 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
11/19/07 ME 128 10 Treatment Trials
What new treatments can help people with a particular disease? What is the most of effective treatment for people with that disease? 11/19/07 ME 128 11 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 11/19/07 ME 128 12 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
11/19/07 ME 128 13 Prevention Trials
Action studies ("doing something") Agent studies ("taking something")--also called "chemoprevention studies" 11/19/07 ME 128 14 Chemoprevention Trials
Phase 3 chemoprevention trials compare a promising new agent with either a: --Standard agent --Placebo
11/19/07 ME 128 15 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
11/19/07 ME 128 16 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
11/19/07 ME 128 17 How Are Patients' Rights Protected?
Informed consent Scientific review Institutional review boards (IRBs) Data safety and monitoring boards (DSMBs)
11/19/07 ME 128 18 How Are Patients' Rights Protected?
Informed Consent: Purpose Procedures Potential risks and benefits Individual rights
11/19/07 ME 128 19 How Are Patients' Rights Protected?
Scientific review Institutional review boards (IRBs) are required by federal law for trials that are: --Subject to FDA regulation
11/19/07 ME 128 20 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
11/19/07 ME 128 21 Clinical Trial
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. 11/19/07 ME 128 22 Clinical Trial
All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called "inclusion criteria" Those that disallow someone from participating are called "exclusion criteria". These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.
11/19/07 ME 128 23 Benefits
Clinical trials that are well-designed and wellexecuted are the best approach for eligible participants to: Play an active role in their own health care. Gain access to new research treatments before they are widely available. Obtain expert medical care at leading health care facilities during the trial. Help others by contributing to medical research.
11/19/07 ME 128 24 Risks
There are risks to clinical trials. There may be unpleasant, serious or even lifethreatening side effects to experimental treatment. The experimental treatment may not be effective for the participant. The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays
11/19/07 ME 128 25 Phases
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions: Phase I trials, researchers test a in a small group of people (10-80) for the first time to evaluate its safety. Phase II trials, the study is expanded to a larger group of people (30-200) to see if it is effective and to further evaluate its safety.
11/19/07 ME 128 26 Phases
Phase III trials, the study is expanded to large groups of people (100 to 300) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow review and approval. Phase IV trials, post marketing studies provide additional information including the risks, benefits, and optimal use. 11/19/07 ME 128 27 Phase I
Prior to Phase I significant efforts have previously been conducted:
Bench studies Computer models and studies Animal studies Research studies However, design changes are anticipated during this phase. Assessment of safety is critical during this phase Typically 3 sites with about 3 subjects per site.
11/19/07 ME 128 28 Phase II
Successful completion of Phase I results in design improvements and revisions if needed Regulatory review typically is needed to enter Phase II to expand the study with more sites and subjects Effectiveness is assessed during this phase. Typically 3 or more sites with about 10 subjects per site
11/19/07 ME 128 29 Phase III
Successful completion of Phase II results in the finalization of the design Regulatory review is needed to enter Phase III to expand the study with more sites and subjects Effectiveness is statistically assessed during this phase. Typically 3 or more sites with about 30 subjects per site
11/19/07 ME 128 30 Phase IV
Post marketing studies are conducted to better understand customer needs These are not intended to address safety of effectiveness May be used to help support Marketing and Sales programs These studies are not conducted under IDE.
11/19/07 ME 128 31 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.
11/19/07 ME 128 32 ...
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