Clinical Trials Made Easy

 

By Scott LaFee   |   May 04, 2018

On an otherwise fine day in the winter of 2017, a young mother of two discovered a tiny lump in her left armpit. It was almost undetectable and she probably wouldn’t have even noticed it except that she had recently gone through a melanoma scare.

She saw her doctor. The lump was diagnosed as an enlarged lymph node caused by a spreading malignancy in her breast, triggered by an inherited and aggressive gene mutation.

And so, like more than 1.6 million other Americans with newly diagnosed cases of cancer each year and millions more grappling with other conditions and ailments, the young mother of two sought treatment.

But hers was a different path, in part because there was opportunity. Standard treatment called for surgery, chemotherapy using medications already on the market, and then, perhaps, radiation. But the mother of two was also eligible to participate in a clinical trial at Moores Cancer Center at UC San Diego Health, testing an experimental drug that precisely targeted her type of rare tumor.

She decided to join the trial — if not for herself, then for her young sons. It was an uncommon choice. Less than 5 percent of adult cancer patients in the United States participate in clinical trials — the final and most important step before any new drug, treatment or device is approved for general use. Moores Cancer Center conducts roughly 300 clinical trials annually. UC San Diego School of Medicine, under the auspices of the Altman Clinical and Translational Institute (ACTRI), oversees more than 1,000 trials each year investigating remedies for conditions ranging from psoriasis and diabetes to glaucoma and the mental health of caregivers of persons suffering from dementia

ATCRI

A new, easy-to-use site at UC San Diego Health Clinical Trials, co-developed by the ACTRI, lets you search through thousands of clinical trials throughout the University of California health system to find studies and opportunities relevant to you. Importantly, you can focus your search not just by disease or condition, but by closest university.

“It’s a much more streamlined and efficient approach than older options,” said Gary Firestein, MD, dean and associate vice chancellor of translational medicine at UC San Diego School of Medicine and ACTRI director.

“UC Health Clinical Trials links to nearly 4,000 trials within the UC Health system, but it also lets you zero in on relevant trials at UC San Diego. It provides not just key details about studies, but how to contact the investigators behind them. For San Diegans — and for potential patients who want to come to San Diego for treatments they cannot find anywhere else — this is a fundamentally huge step forward and allows patients to take control of their own destiny.”

Clinical Trials 101

Clinical trials look at new ways to prevent, detect or treat disease. They may test novel drugs or novel combinations of drugs, original surgical techniques or medical devices, new ways to use existing treatments or devices, new methods to change behaviors to improve health or to improve the lives of patients with chronic or acute illnesses.

Nonetheless, surveys have shown only one-third of Americans say they are willing to participate in a clinical trial and more than half of doctors do not consider recommending them until later in treatment, perhaps as a last resort.

“Clinical trials ensure that promising ideas actually deliver their desired result, and that they do so safely and effectively,” said Howard Taras, MD, director of ACTRI Community Engagement. “But there are misconceptions out there about what trials do and how they work, and the same surveys show that when patients and doctors learn more about a trial, their opinions improve significantly.”

Every approved clinical trial follows a protocol designed to balance the specific questions of researchers and their goals with the potential risks and benefits to participants. That protocol, which for most trials must be approved by an expert Institutional Review Board, describes the study’s goal, who is eligible to participate, protections against risks, details about tests, procedures and treatments, how long the trial is expected to last and what information will be gathered. By law, all clinical trial results must be reported to a public database after the trial has been completed.

Every trial requires informed consent. That is, participants must be given key information about the study before deciding whether to take part. The informed consent document includes explanatory details about the study’s purpose, length, tests and procedures, potential risks and benefits. Taking part in a clinical trial is voluntary and participants can leave a study at any time.

There are typically four phases of research, and any clinical trial will fit into one of these four phases:

The first phase tends to be small, maybe a dozen or so participants, and is designed to determine whether the experimental drug or treatment is safe, can be applied effectively and to identify side effects.

Phase two further investigates safety and efficacy in a larger group.

In phase three, the drug or treatment is tested in an even larger group, perhaps thousands of participants, to confirm its effectiveness, monitor side effects, compare results with standard or similar treatments and collect information needed to allow the drug or treatment to be used safely on a broader scale.

Phase four trials occur after a drug or treatment has been approved by the Food and Drug Administration and available to the public. These trials track safety in the general population, gather more information, address emerging questions or issues and further refine optimal use.

The gold standard for most clinical trials is the randomized, double-blind study, sometimes involving a placebo component. In other cases, the new therapy is compared to existing treatments. A placebo is an inactive product intended to resemble the tested drug or therapy, but without its treatment value. Placebos can provide a point of comparison, but they aren’t used in studies of treatments for serious illnesses or where participants might be put at risk.

In a randomized trial, participants are selected by chance to receive either the tested drug or an alternative, eliminating potential biases. Blinded or masked studies mean participants, researchers or both don’t know who is getting the tested drug and who is not, again to avoid influencing results. In single-blind studies, participants don’t know. In double-blind studies, neither participants nor researchers know.

Dial a trial

Well-designed and well-executed trials do a lot of things: They provide participants with access to new drugs or options before they are widely available — plus care and attention from a research team that includes physicians, specialists and other health professionals, often at no cost. More broadly, they advance medical progress and benefit others by contributing to knowledge about new treatments.

By their nature and design, clinical trials have traditionally represented a degree of uncertainty. If researchers knew absolutely what their results would be, there would be no need for a trial. This uncertainty has often been perceived as a significant hurdle, both in garnering sufficient participation (many studies struggle or fail because they cannot recruit enough eligible participants) and because consumers balk at not knowing whether they will receive a new treatment, an existing treatment or, sometimes, a placebo or they are skeptical about an unproven treatment. Cost is also a consideration, though trials are often free and sometimes compensate for participation.

Precision medicine has become a mantra in recent years. In cancer, it’s the idea of tailoring patient treatment based upon the genetic changes or mutations of the individual. It’s the idea that cancer is not the same in every patient, though it may have a common name.

Clinical trials are rapidly undergoing a similar metamorphosis. Historically, clinical trials have required millions, even billions of dollars, and a decade or more to deliver a new drug or treatment to market. “It’s a major reason why health care today is so expensive and often frustrating,” said Firestein.

But with rapid advances in genetic screening and new diagnostic tools, trials are becoming smaller, nimbler and more tightly focused. Rather than test thousands of participants to determine whether a drug produces a beneficial effect in a tiny sliver of recipients, new precision trials identify and recruit participants who are most deemed likely to benefit, based upon their genetics and the known mechanisms of the drug. The idea is to create specific trials for specific patients with an intended, specific result.

“The approach is sophisticated and data-driven,” said Firestein. “You have to know more about what you’re doing going in, but the results and benefits come faster and at less cost.”

Happy endings

For the young mother of two, her clinical trial went well. The process was hard, grueling, fraught with emotion and occasional moments of fear and doubt. But her experimental drug worked, eradicating her cancer faster and more fully than the standard of care. Follow-up therapies are helping to ensure it does not return.

Exhausted but defiant — “I was always going to kick cancer’s butt.” — the mother of two says she’s happy she volunteered for the trial. For herself. For future women diagnosed with breast cancer. And for her sons.

Did it save her life? She smiles. “Ask me at my boys’ weddings — in a couple of decades.”

 

THINGS TO THINK ABOUT AND ASK WHEN CONSIDERING A CLINICAL TRIAL

The study

  • What is the purpose of the study?
  • Why do researchers think the approach may be effective?
  • Who will fund the study?
  • Who has reviewed and approved the study?
  • How are study results and safety of participants being monitored?
  • How long will the study last?
  • What will my responsibilities be if I take part?
  • Who will tell me about the results of the study and how will I be informed?

Risks and possible benefits

  • What are my possible short-term benefits?
  • What are my possible long-term benefits?
  • What are my short-term risks, and side effects?
  • What are my long-term risks?
  • What other options are available?
  • How do the risks and possible benefits of this trial compare with those options?

Participation and care

  • What kinds of therapies, procedures and/or tests will I have during the trial?
  • Will they hurt, and if so, for how long?
  • How do the tests in the study compare with those I would have outside of the trial?
  • Will I be able to take my regular medications while taking part in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?

Personal issues

  • How could being in this study affect my daily life?
  • Can I talk to other people in the study?

Cost issues

  • Will I have to pay for any part of the trial such as tests or the study drug?
  • If so, what will the charges likely be?
  • What is my health insurance likely to cover?
  • Who can help answer any questions from my insurance company or health plan?
  • Will there be any travel or child care costs that I need to consider while I am in the trial?

Tips for asking your doctor about trials

  • Consider taking a family member or friend along for support and for help in asking questions or recording answers.
  • Plan what to ask — but don't hesitate to ask any new questions.
  • Write down questions in advance to remember them all.
  • Write down the answers so that they’re available when needed.
  • Ask about bringing a tape recorder to make a taped record of what's said (even if you write down answers).

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