Clinical Trials 101: Everything You Wanted to Know

Dr. Shannon Westin–Associate Professor in the Department of Gynecologic Oncology and Reproductive Medicine at UT MD Anderson Cancer Center, and Director of Early Drug Development/Phase I Trials–has a motto: Research is what cures cancer.

Dr. Westin spoke at our 2020 Uniting for Hope Ovarian Cancer National Conference, sharing everything from what exactly a clinical trial is and how it helps propel science forward, to how to find one and what questions to ask your doctor about them.

Below are some highlights from her presentation, which you can watch in our National Conference video collection >>

What is a clinical trial?

Sir Bradford Hill, considered to be the father of clinical trials, defined it in 1962 as “a carefully and ethically designed experiment in humans with the aim of answering some precisely framed question.” Broken down, a clinical trial:

Is a research study that involves patients
Focuses on an improvement in healthcare and/or outcomes
Is actually the tail end of a long process (could be decades!) of trying to get a drug to market
Has objectives, endpoints and a rationale
Has eligibility criteria and strict safety considerations
Has a study design with specific details as to how many people are involved and for how long, and how they will be treated
Has toxicity modifications (what doctors should do if they see certain side effects)
Defines ahead of time a set of measurements

Sponsors of a clinical trial could include the National Cancer Institute, pharmaceutical companies, non-profit organizations (like OCRA) or local institutions/medical centers.

What is the pathway to FDA approval for clinical trials?

It all starts with an idea or hypothesis. Preclinical studies and research take place in test tubes (cell lines) and animal models. From there, theories that show merit move onto human studies. These are as follows:

Phase I are small studies that determine the best dose while avoiding side effects and whether it is working. There are many Phase I studies, and only a select few move onto Phase II.
Phase II studies are a bit larger and determine whether a particular drug or therapy works for a particular type of cancer (or other illness). If the answer is yes, it will move onto Phase III.
Phase III studies are very large, randomized trials that compare a new drug to standard of care treatment

These studies, or clinical trials, look for measurable data involving survival rates, symptom control, drug-to-drug interactions, response, toxicity or side effects, and metabolism. At the time of this presentation, there were 500 ovarian cancer Phase I, II and III trials taking place.

Why would someone want to participate in a clinical trial?

Clinical trials are a great way to access cutting edge therapy, as they may allow patients to get access to new interventions sooner than the rest of the population, and can have a positive effect on one’s outcome. Those involved with clinical trials have more doctors’ visits and are closely monitored. Furthermore, the therapy or drug being tested may be better than the current standard of care regimen.

Of course, more doctors’ visits can bring about its own burdens, and being involved in a clinical trial may lead to unknown side effects. And there’s a chance that the therapy won’t work.

How are patients in a clinical trial protected?

While there is an element of unknown involved in participating in a clinical trial, there are certain protections that are standard:

The study is always voluntary, and a patient can withdraw at any time
The trial is monitored by the sponsor
Costs to patient are explained ahead of time. Trials don’t compensate patients for participating, but travel costs may be covered.
Patients will be made aware if anyone in the trial has had a bad side effect
Patients are closely monitored by their healthcare professionals

How to find clinical trials?

Patients who are interested in participating in a clinical trial are encouraged to speak to their doctor or visit OCRA’s Clinical Trial Finder.

You can play Dr. Westin’s presentation, as well as a presentation about the future of ovarian cancer clinical trials, in our 2020 National Conference videos.