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Before a trial can begin enrolling patients, it requires a significant infrastructure and a skilled, dedicated team to ensure all the regulations required to assure patient safety and scientific validity are met.

Trials can differ in sources and type, and Moffitt Cancer Center investigator-initiated trials (IITs) are especially important. As a National Cancer Institute (NCI)-designated cancer center, Moffitt is charged with conducting IITs, which are designed and managed by the cancer center’s clinical faculty.

Daniel Sullivan, M.D., Associate Center Director, Clinical Science

“Investigator-initiated trials are important largely because through them we are translating laboratory findings to our patients,” said Daniel Sullivan, M.D., associate center director of Clinical Science. “For example, if we discover a new drug or a new cell therapy, that’s good. Or if we identify a molecular pathway that is important in cancer and if a pharmaceutical company has a drug that targets that pathway to interrupt the growth of cancer, that also could be important as an investigator-initiated trial.”

In addition to IITs and trials designed by pharmaceutical companies, NCI’s National Clinical Trials Network is a collection of organizations and clinicians that coordinates and supports cancer clinical trials at more than 3,000 sites, including Moffitt, across the United States and Canada.

Trials can focus on prevention, supportive care or clinical intervention. Usually referred to as clinical trials, clinical intervention trials at Moffitt are designed to test new treatments in people with cancer. These treatments investigate promising new drugs, drug combinations, new approaches to surgery or radiation therapy, and advances in new areas such as gene therapy.

"A clinical intervention trial could be investigating all kinds of things, including chemotherapy, drugs, small molecules or immunotherapies, even devices such as a prosthesis to prevent amputation in children with sarcoma," said Sullivan. The patients who participate in these clinical trials are protected by a rigorous informed consent process. They also are protected by regulations including those placed by the Institutional Review Board’s (IRB) Scientific Review Committees and the U.S. Food and Drug Administration (FDA). As an NCI-designated comprehensive cancer center, Moffitt has a Protocol Review and Monitoring System (PRMS) that oversees various committees and boards and ensures all the federal regulations for human subjects’ research are met.

Cheryl Byers, Director, Moffitt Protocol Review and Monitoring System

"All NCI-designated cancer centers have a Protocol Review and Monitoring System. It might be called something else, but the function is the same," said Cheryl Byers, who directs Moffitt’s PRMS, managing a team of people who administer the regulatory aspects of research.

"As an NCI-designated cancer center, we must have our trials reviewed by a Scientific Review Committee to evaluate the science and the statistical components," said Sullivan. This required oversight includes assurance that the research question is clear, the design is proper, the research team is qualified and the analysis is valid.

Another key safeguard, the FDA oversees clinical trials that are testing new medicines or medical devices. The federal agency reviews each trial proposal before any patient testing is performed.

"If one of our clinical faculty creates a trial involving a non- FDA-approved drug, referred to as an investigational new drug [IND], we must apply for and obtain an IND from the FDA, so that’s another regulatory step," said Sullivan. "Clearly, you need all of those regulations, and as each trial is conducted, there are annual reviews by the Protocol Monitoring Committee here at the cancer center. There is constant monitoring and auditing of the trial to be sure protocol is adhered to. There also are annual reports required by the IRB and the FDA. So there are a lot of regulatory requirements, beyond those up-front obligations, that go on throughout the life of the trial."

Another regulatory requirement is having trials pass through an IRB. These boards provide ethical and regulatory oversight of research that involves human participants. And as such, they must have high ethical and scientific standards, with patient protection at top of mind.

Moffitt uses the services of Advarra, an independent IRB, and several others. "A huge advantage of using the services of an independent IRB is the rapid turnaround time," said Byers. "Using an independent IRB improves efficiencies, which helps us activate trials quicker, so we can offer them to our patients as a treatment option."

This faster turnaround time can mean availability of a new drug through a clinical trial for patients who have relapsed and have no other treatment options. Additionally, being able to test a new drug more quickly through clinical trials helps assure quicker approval and availability of an efficient new standard of care. Advarra also has a biosafety committee, in addition to its IRB services.

Challenges of Moffitt’s regulatory compliance initiatives include keeping up with the current regulatory requirements, as well as keeping up with changes in best practices, such as newer guidelines that come from federal agencies, notes Byers.

Byers is among the first to say the benefits of this work make the effort well worth it. She happily recalls the very first trials of CAR T cell therapy and how great it was to see them approved by the FDA. "It is exciting to see that something you were reviewing and on the front end of come to fruition," said Byers.  "When we can offer new therapies that allow people to live longer and give them quality of life — that is super rewarding."