Clinical trials are essential to cancer research as the critical link for developing scientific discoveries into new treatments for patients.

Yale Cancer Center’s clinical trial office is led by two people with five decades of experience between them in this specialized realm of cancer treatment. They recently had a conversation with Yale Cancer Center Director Eric P. Winer, MD, host of the weekly Connecticut Public Radio show Yale Cancer Answers.

Director of the Clinical Trials Office (CTO) Ian Krop, MD, PhD, began as a cancer researcher in the lab and transitioned to clinical trials about 25 years ago because he wanted to more directly impact patients.

Alyssa Gateman, MPH, executive director of the CTO, has worked with cancer researchers and clinical trial specialists, as well as data and compliance to ensure patient protection over the last 25 years.

Following is a synopsis of the Yale Cancer Answers conversation between Drs. Krop and Winer and Ms. Gateman on Connecticut Public Radio. The questions are from Dr. Winer.

What is involved when a patient joins a clinical trial?

Patients first discuss treatment options, which could include clinical trials, with their healthcare providers. If a trial is recommended, the patient receives detailed information about the trial through an informed consent document. If interested, they sign the consent and then there are checks to ensure their eligibility before starting the trial.

How are patients protected from feeling pressured into clinical trials?

There is a lot of information coming at patients. Patients have the right to ask questions and take their time to consider participation and to discuss it with friends and family. Each trial’s informed consent is reviewed by an Institutional Review Board (IRB) to ensure ethical standards, and that accurate information is provided to patients. This includes that patients can withdraw from trials at any time without affecting future care.

I often tell patients that the clinical trials are not something that some doctor, or other researcher, dreamt up and then offered to patients the next day. These have been developed over years and repeatedly reviewed at each step.

That is a very good point. It is a very thorough process. There are multiple committees of cancer experts, experts in logistics and in ethics on the IRB. The IRB includes researchers, physicians, and members of the community. It is all for the safety of the patients and to make sure we’re actually answering the question for which the trial was designed.

What are the phases of clinical trials?

Phase 1 trials test a drug's safety and dosage with a few dozen patients. If that leads to Phase 2 trials, those have slightly larger numbers of patients and are focused more on determining a drug's effectiveness. Phase 3 trials, often involve hundreds or thousands of patients and compare the new drug to the current standard treatment to determine which is more effective.

Why is it important to have people of different backgrounds in clinical trials?

We know that drugs affect different people in different ways. If everyone in a trial is similar, then the results could be read as specific to that group. But if the trial group includes patients of different genders, ethnicities, ages, races and backgrounds it yields information on the possible drug side effects in different populations or it shows that it is broadly applicable to all.

Can you give us some examples of the breakthroughs that we’ve seen in the last five to 10 years and how clinical trials were involved?

All of the breakthroughs in the last five to 10 years are a result of clinical trials. About 20-25 years ago, in almost all cancers, the treatments were nearly all various kinds of chemotherapy. Chemotherapy can be effective, but it has a substantial risk of side effects because it doesn’t distinguish well between cancer cells and normal cells. Now, we’ve changed our treatment to drugs that target something specific in the cancer cells and leave the other cells alone so there are fewer side effects. That’s all come about from scientific research and then clinical trials.

For patients on clinical trials, is there more work such as additional costs or visits?

In some trials there are additional visits required, but it is uncommon for trials to have significantly added costs for the patient. Participating in a trial also has benefits beyond potentially receiving a more effective treatment. Patients also have a bigger care team to support them while they are on the study. If there are any additional expenses, they can often be offset by stipends or reimbursements.

What happens if a trial shows one treatment is clearly better than another when it’s a comparison trial?

A data safety monitoring committee oversees trials. If a clear winner emerges, they can stop the trial and often allow patients on the less effective treatment to switch to the better one, ensuring access to the superior therapy.