1.4 Clinical Trials 101

Slide 1
Welcome to Clinical Trials 101. My name is Paul Crispin. I actively design, participate, and enroll patients in the clinical trials.

Slide 2
In this brief introduction today, I’ll be introducing you to the intent of the clinical trials that we perform, their phases, the sponsors necessary to complete these clinical trials, and how our center selects them. [slide bullet: review]

Slide 3
Clinical trials can serve multiple functions. For example, we have a scientist who discovers a new drug. This drug has the potential to cure cancer and we have Bob who, unfortunately, has been diagnosed with cancer and would like to improve his treatment options. So how do we make this new treatment available to Bob? The answer is simple: through clinical trials. Clinical trials are necessary to confirm the safety and benefit of a new treatment before making it available to all patients.

Slide 4
When we think of clinical trials, we often think of a drug or medication as given in that last example; however, we can design clinical trials to answer many important cancer care questions.
We have treatment trials that evaluate drugs, devices, and procedures;
diagnostic trials that look to improve the way that we detect cancers- for example, is a new CT scan technology better than MRI in detecting pancreatic cancers?;
we have prevention trials that look to decrease the prevalence of a cancer in a population such as looking at the routine use of multivitamin decreasing prostate cancer incidence;
there are screening trials to look to see if we can detect cancers earlier to improve outcomes- for example, looking at a chest CT being performed to decrease death rates from lung cancer;
and, finally, we have supportive care trials. Supportive care trials look to improve the quality of life of patients either before, during, or after their cancer treatment. A good example of a supportive care trial would be the evaluation of yoga and improving patient well-being following abdominal surgery.

Slide 5
A very common question we get is regarding the phase of a clinical trial. Clinical trials are phase 0-4, with each phase serving a specific purpose.
In phase 0 trials, this is the first time we’re using the drug in humans and so we’re looking to see the impact that the drug has on the body and how the body handles the medication. A small amount of drug is given to a small number of patients, generally 10 to 12. And these phase 0 trials are typically run by pharmaceutical companies.
Next, we have phase 1 trials, where the primary goal is safety: They look to see what’s the best dose of medication we give to a patient with tolerable side effects. Again, it’s typically a small number of patients, 10 to 30, and sometimes even less. And while some patients may derive benefit from this treatment, again the primary goal of phase 1 trials is safety.

Slide 6
For phase 2 trials, now that we know that the treatment is safe to give, we actually want to evaluate to see if it’s accomplishing its goal to improve the care of our treatment of our cancer patients. And these trials usually focus on a specific type of cancer and stage of cancer. This is generally the earliest you would ever see the FDA approve a new medication and when they do, it’s because it’s the only treatment available for that select group of patients.
In phase 3 trials, we compare the new treatment to the standard of care or best available care. They will enroll hundreds to thousands of patients looking to see which treatment is better and which has the fewer side effects. Patients will often be randomized in these trials so they don’t get to select which treatment they receive and they will often complete the trial without knowing which treatment they receive. And phase 3 trials are commonly required prior to FDA approval.
Finally, we have phase 4 trials. The phase 4 trials evaluate recently approved medications and will enroll thousands of patients. They provide us with a better assessment of the long-term side effects and safety of the recently approved medications. As you can imagine, these trials are extremely expensive.

Slide 7
The experimental medications and procedures will not be billed to the patient or their insurance company. Patients will often have additional visits or procedures compared to patients being treated off study. There is also the additional cost of data collection, data analysis, and close observation. For this reason, sponsors are required to help cover these costs. I have listed here the different types of sponsors available for clinical trials and, as you can see, they range from industry sponsors to private and government funding.
[slide bullets: institutional, grants, foundations, cooperative groups]

Slide 8
To help select the best trials for patients, centers typically place the greatest emphasis on the needs of the patients surrounding the center, the best science, the evaluation of institutional discoveries, and those trials that they feel are going to have the greatest impact on improving cancer care beyond their center.

Slide 9
[diagram showing a multi-step clinical trial review process, moving from disease-specific review groups to scientific review and then to Institutional Review Board (IRB) approval]
To help with the selection process, centers will often have a standardized review process in place. This review process helps to ensure that we’re not opening trials that will compete for the same patient populations and that the study is actually feasible at our site. Here’s an example of a review process which starts with the disease site groups: The disease site groups are a group of members that are experts in a specific type of cancer such as breast cancer or lung cancer. They will design, evaluate, and review multiple trials. The study would then go on to a second committee that is responsible for ensuring scientific merit, feasibility, and that the trial is meeting the patient’s needs. Once approved, it would then go on to the Institutional Review Board, or IRB, for final approval. This type of multi-step review process is required for all NCI-designated cancer centers.

Slide 10
[summary slide: why clinical trials are conducted, five phases of clinical trials, sponsors to cover costs, selection criteria, review process]
This brings us to the end of our introduction to clinical trials. I hope that it has helped you understand how and why we complete these trials. Thank you for your time and attention.