Clinical Trial FAQs
At the Center for Cancer Care at Griffin Hospital, we are committed to providing access to a full range of cancer services, and this includes access to clinical trials. Our multidisciplinary tumor boards are an opportunity for your physician to discuss the details of your results with a team of colleagues and develop a recommendation for treatment, including clinical trials. Your physician will discuss with you whether there are open clinical trials that may be right for you. Please read the information below to learn more about clinical trials.
Clinical trials for cancer are research studies that involve people who either have or are at risk of
getting cancer. They are designed to evaluate promising new treatments. Clinical trials are the most
important tool that doctors and researchers have in their effort to find new and more effective ways to
treat and possibly cure cancer. Many of today’s standard treatments for cancer began in clinical
trials, and many people treated for cancer are now survivors thanks to the knowledge gained through
those clinical trials.
According to the American Society of Clinical Oncology nine out of ten women with breast
cancer are alive 5 years after their diagnosis and breast cancer mortality has fallen by more
than a third since its peak in the 1980’s. Listed below are just a few of the many treatments
that have prolonged lives, boosted cure rates and improved quality of life for women
diagnosed with breast cancer:
- 1977 – Breakthrough drug, Tamoxifen, approved for advanced breast cancer
- 1986 – Tamoxifen is approved as standard therapy for post menopausal women following breast cancer surgery as studies showed that use of the drug for 5 years after surgery significantly reduced the risk of recurrence
- 1998 – FDA approves groundbreaking drug, trastuzumab (Herceptin), first targeted anti‐cancer drug, and dramatically improves survival for women with advanced cancer that over produce a protein called HER2.
- 1998 - Tamoxifen approved to reduce the risk of developing breast cancer in women identified as high risk for the disease.
- 2006 – Herceptin is approved as part of therapy after surgery for all women with Her2‐positive breast cancer reducing risk of recurrence by 50%
- 2011 – A large phase III trial shows Aromasin greatly reduces the risk of developing invasive breast cancer in post-menopausal women at very risk for breast cancer. This includes women with BRCA gene mutations and carries milder
side effects than
- 2013 – FDA approves T‐DM1, a next‐generation targeted drug called Trastuzumab emtanine which extends survival in women with HER2 positive cancer that has progressed despite other standard treatments.
Clinical trials are conducted in distinct phases as described below. In order for a treatment to become standard, it must first go through 3 or 4 phases. Each phase has a distinct purpose. The early phases make sure the treatment is safe. The later phases are evaluating if new treatment works better than the standard treatment. On average it takes at least 6 years of research before a cancer drug makes it to a clinical trial and on average another 8 years from the time a cancer drug enters a clinical trial until it is approved by the Food and Drug Administration (FDA) as the new standard treatment.
This is the first step in testing a new treatment in humans. The main objective is to determine the
highest dose of a new treatment that can be given safely without serious side effects. Phase 1
clinical trials usually involve a small number or volunteers (15- 30) and take place in large teaching
hospitals or cancer centers, where volunteers can be watched closely.
Phase I Clinical Trials:
- What route is the safest to administer this new drug (by mouth, injected into the blood or in
injected into a muscle)
- How often should the drug be given
- What dosage is safe
- What are the side effects on the human body
Once a new treatment has been found to be reasonably safe in a phase I trial it will move into a
phase II trial. Here researchers learn more about its effectiveness and safety. They are looking for
evidence that the treatment is working on the cancer (tumor shrinks, disappears or there’s a longer
period of time before the cancer gets larger or comes back). In phase II trails the new treatment is
given to larger numbers of volunteers (25-100) and can take place in local community hospitals or
Phase II Clinical Trials:
- Designed to assess if the treatment works on a specific type of cancer
- All volunteers get the same dose by the same method found to be the safest and most effective in
the phase I trial
- No placebo is used (placebos are generally not used in cancer clinical trials)
In this phase the safety and effectiveness of the new treatment is now compared against the current
standard treatment. Most phase III trials involve a large number of volunteers (hundreds to
thousands) in order to determine the true effectiveness of the treatment. Phase III clinical trials are
needed before the FDA will approve a new drug for use by the general public.
Phase III clinical Trials:
- Larger number of trails available to volunteers geographically
- Trials can be found in local oncology offices, clinics and cancer centers across the country
- Phase III trials are randomized in which one treatment group receives the new treatment being
studied and the other group receives the
standard treatment currently in use.
- Participants continue to be monitored very closely
Phase IV trials take place after a new treatment has been approved by the FDA and made available to the public. The purpose of these trails are to see how safe and useful the treatment will be over the long term, how well the drug works in combination with other treatments and its effectiveness on other types of cancer.
Usually insurance will cover all standard of care treatments such as exams, laboratory work, X-ray, etc. (standard of care is the routine care provided for a disease). All additional clinical trials related therapies or laboratory studies may be paid for by the sponsor of the clinical trial. Patients should ask their doctor what is covered by their insurance to ensure that their out-of-pocket costs have been explained to fully.
Myths and Truths About Clinical Trials
People who take part in clinical trials stand to benefit from promising new treatments that are not
available to everyone. Participants also have the opportunity to contribute valuable information to
research that might help other cancer patients in the future. Some common myths and facts about
clinical trials provided by the National Comprehensive Cancer Network include:
Myth In a clinical trial, you are little more than a human guinea pig in a lab experiment.
Truth In designing clinical trials, physicians have two concerns: that all patients who participate
receive the highest quality medical care, and that rigorous scientific principles are followed to allow
meaningful conclusions to be drawn. Compelling evidence must exist that suggests that the
investigational drug or other therapy is at least as good as the standard. In every clinical trial, patient
welfare and safety are the major considerations.
Myth Once you are enrolled in a clinical trial, you must continue, even if you change your mind.
Truth Clinical trial participants always have the right to withdraw—at any time, for any reason.
Myth If you take part in a clinical trial, you might receive a placebo (a pill, liquid or powder that
contains no medicine; sometimes called a “sugar pill”) instead of real medicine.
Truth Few cancer clinical trials use placebos, and they are never given to cancer patients in place of
treatment. Cancer patients who take part in clinical trials must receive at least the best standard
treatment available. In many cases, they will receive a new and better therapy being evaluated.
Myth If you are thinking of enrolling on a clinical trial, you won’t be told in advance about everything
that’s going to happen.
Truth The rights and interests of people who enroll in clinical trials are carefully protected by an
Internal Review Board (IRB) and the Food & Drug Administration (FDA). In a process called informed
consent, anyone who is thinking about enrolling in a clinical trial must receive detailed information
about the study so they can make an informed decision about participating.
Myth You should enroll in a clinical trial only if there are no other treatment options left.
Truth Most cancer patients should consider participating in a clinical trial as one of their treatment
options. There are trials for all different types and stages of cancer. Participants are monitored
carefully throughout the trial, so they receive excellent care and can be the first to benefit from new
treatments before they are available to everyone.
Your Rights and Protection
There are multiple safeguards in place to protect the welfare of the people who take part in clinical
trials. Government agencies such as The Office Human Research Protections, Food and Drug
Administration (FDA) and The National Cancer Institute who play a role in ensuring that all research
is conducted with patient safety in mind. In addition, each institution, cancer center or hospital that
conducts clinical trials has a committee called an Institutional Review Board (IRB) that reviews each
trial to make sure it is safe and ethical.
Another, and probably the most important, protection for participants, is the process of informed
consent. Informed consent is a process by which the people (doctors, researchers, nurses) running
the study are required to thoroughly explain all the details about the study including the purpose of
the study, tests and procedures used and the possible risks and benefits of the new treatment. Once
you feel fully informed you will be asked to sign a written consent form so you can take part in the
Learn More About Clinical Trials
Coalition of Cancer Cooperative Groups
The National Cancer Institute
National Cancer Institute’s Center for Cancer Research
National Institutes of Health
American Cancer Society
Patient Information from the American Society of Clinical Oncology