Clinical Trials Guidelines
What is a Clinical Trial – A research study designed to evaluate new cutting-edge therapies using cancer patients. Trials provide an important opportunity for patients to be part of research efforts evaluating new chemotherapy and immunotherapy drugs/agents, new radiation therapy techniques, new surgical techniques, and creative new ways to use different cancer treatments in combination.
Participation in Clinical Trials – It is most important to discuss with your oncologist the possibility of participating in a clinical trial, especially if you did not initially respond or are no longer responding to existing conventional therapies.
IMPORTANT NOTE: Before considering whether to participate in a clinical trial, learn as much as possible about the clinical trial, do as much investigation of the clinical trial as you can on your own. Then ask as many questions as you need to thoroughly understand the pros and cons of participating in the clinical trial.
Suggested Pertinent Questions to Ask Your Oncologist about a Clinical Trial:
- Are there any other existing established treatments that I should consider before participating in this or other clinical trials?
- What phase trial is this– Phase 1, Phase 2 or Phase 3?
- Do I meet the eligibility criteria for the Clinical Trial that I wish to participate in?
- What could my participation in the clinical trial mean for me in the short term / long term, based on the nature of this trial?
- Are there known side effects or risks from the treatment I will receive in this clinical trial? If so, what are they?
- Where will I receive the treatment — as a hospital inpatient or in an outpatient setting?
- Must the treatment only be administered at a participating research center requiring travel to the center?
- What is the treatment protocol — number of drugs or agents to be administered, route of administration ( oral or intravenous), length of time for the administration of the treatment, how many courses of the treatment will I receive, and what is the anticipated duration of the trial?
- Will there be patients enrolled in the trial who will not receive the drugs or agents being tested (i.e. is this a double-blind clinical trial)?
- How will I be monitored during the clinical trial — required blood tests or radiology/imaging studies and their frequency?
- Will serial biopsies be required as part of the clinical trial?
- Would I be allowed to participate in the next phase of the trial, assuming the trial progresses from the phase of the trial that I am participating in?
- Will I receive assistance/guidance on applying for participation in the trial
Patient-friendly language to help better understand the criteria / information for clinical trials:
The FDA Oncology Center of Excellence Patient-Friendly Language Project began with the need to update the definitions of common terms used in cancer clinical trials for better understanding and use by healthcare providers and patients.
This glossary addresses selected words and phrases that patients with cancer have identified as particularly difficult to understand when discussing potential participation in a clinical trial or reading an informed consent document. For terms not included in this list, please refer to the NCI Glossary.
We are continually gathering feedback on the definitions. If you have comments or suggestions, email FDAOncology@fda.hhs.gov.
UNDERSTANDING THE CLINICAL TRIAL PHASES:
- PHASE 1 – First step of the research study. Duration : 6 months – 1 year (approximate). Patient participation: 15 – 20 patients approximately (may vary somewhat). Goal: to test an experimental drug or treatment in a small group for the first time to evaluate tumor response, identify side effects, and determine the maximum tolerable dosage.
- Phase 2 – Only treatments proven to be safe in Phase 1 studies progress to the Phase 2 stage. Patient participation: 20 – 80 or 100 – 500, depending on the research facility. Duration: 6 months to 1 year or more to complete. Goal: The experimental drug or treatment is given to a larger group to evaluate its effectiveness and to further assess safety.
- Phase 3 – Progression to apply the study drug or treatment to a larger patient group. Duration: 1 – 4 years to complete depending on the type of cancer, type of study, and number of patient participants. Again, safety and side effects are well monitored and evaluated by the researchers. Goal: To compare a new drug or treatment to other standard treatments currently used to determine which may be most effective to treat cancer.
- Phase 4 – Post Marketing studies, which are conducted after a treatment is approved by the FDA, provide additional information including the treatment or drug’s risks and best use.