The Truth About Clinical Trials

How Groundbreaking Research Can Bolster Your Eczema Treatment and Improve the Lives of Tomorrow’s Patients

Erika Stalder, NEA Contributing Writer

Clinical studies. Medical research. Human Trials. For researchers in the field of emerging science, these methods of clinical inquiry can conjure thoughts of exciting breakthroughs, life-enriching cures for widespread diseases, or maybe even a discovery so significant that it gets named for the investigating doctor.

Yet, for those of us outside of the medical community, the same words can often bring to mind images of hapless guinea pigs, risky outcomes, or even failed seventh-grade science experiments. In a 2008 survey, the Center for Information & study on Clinical research Participation, a nonprofit group that educates the public about participating in clinical trials, found that only half of respondents felt that clinical research trials were “somewhat safe,” and only 17 percent felt clinical research studies and trials were “very safe” ventures.

It’s pretty unlikely that such a majority of respondents had personal or even anecdotal experience with shoddy clinical trials. After all, if that were the case, the so-called dicey clinical trial community would be making headlines left and right. so, why the shaky rep? It likely has some- thing to do with what CenterWatch, a global clinical trial research group, discovered in their own findings: 75 percent of people in the general public admit they have little to no knowledge about clinical research enterprise and the participation process—despite the fact that 94 percent agree that participating in clinical research is an important part of helping the advancement of medical science. and we often fear what we don’t know.

An Essential Part of Medical Discovery

That fear may be perfectly natural, but consider this: clinical trials are an essential part of medical discovery—one that has shaped what we know about atopic dermatitis and how it affects the skin. Ultimately, perpetuating misinformation and fear of the trial process only shrouds difficult conditions like atopic dermatitis in more uncertainty. It’s time we get real about what, exactly, clinical trials entail and how they can help both today’s and tomorrow’s suffering eczema patients overcome a burdened quality of life.

Stephen I. Katz, M.D., Ph.D., a dermatologist and director of the National Institutes of Health’s National Institute of arthritis and musculoskeletal and skin Diseases (NIAMS), points out that when it comes to atopic dermatitis, clinical trials are giving the scientific community a better under- standing of the mechanisms that cause the disease. And this means better care for patients.

clinical_trials_p5“For example, we have a better understanding of what the microbes on skin do to atopic dermatitis,” he says. “We have a better understanding of what certain types of chemicals that are produced by skin cells might be doing in atopic dermatitis. We also know that in atopic dermatitis, there are problems with the skin barrier. And these are all targets for potential intervention.”

Likewise, clinical trials have shown that a new biologic may help eczema patients and may have confirmed one of the pathways to inflammation, according to National Eczema Association Co-Founder Jon Hanifin, MD, a dermatologist at Oregon Health & Science University.

Studies funded by NIAMS continue to determine if antibodies or certain type cytokines might be used to treat adults with severe atopic dermatitis, or if certain types of emollients may change the course of disease in infants, according to Katz. (Note: The NIAMS-funded infant and emollient study was a direct result of seed funding provided by NEA to the lead investigator.)

Meanwhile, a myriad of other atopic dermatitis studies—funded by pharmaceutical companies, research centers, biotech companies, medical institutions, and patient advocacy groups like NEA—are looking to new therapies and interventions that may help prevent or reduce the day-to-day pain associated with the disease.

How Safe are they Really?

Even though research is making strides to uncover helpful treatments for atopic dermatitis, we remain largely timid and fearful about getting involved in such trials. How safe is it, really, to participate in a clinical trial? In short, quite safe. Regardless of whether they are conducted in the public or private sector, every clinical trial follows a strict protocol that ensures that the study’s central question and construct is well designed; that tests, procedures and any medications issued have minimal risks; and that participants are treated ethically. Oversight is provided by an independent Institutional Review Board, which ultimately must approve every clinical trial in the country. Oftentimes, an additional third-party committee, or Data Safety Monitoring Board, is also employed to examine a trial’s data and ensure there are no adverse side effects, along with considering other measures of patient safety and trial success.

In addition to the checks and balances that the review and monitoring boards provide, the FDA plays an additional role in participants’ protection. Its regulations are designed to foster good clinical practice and ensure the safety of human subjects and of products used in all clinical trials. Further, its Bioresearch Monitoring (BIMO) program protects the safety and rights of participants within FDA-regulated clinical trials.

“Through the BIMO program, the FDA conducts on-site inspections of entities involved in clinical trials (clinical investigators, sponsors, contract research organizations, monitors and Institutional review boards) to determine if the studies were conducted in compliance with applicable good clinical practice and human subject protection regulations,” notes FDA spokesperson Tara Goodin.

AD Trial Investigators 
are Often Leaders in their Fields

Since the patients in a clinical trial don’t see the review and ethics boards working for their rights firsthand, they often look to the lead researcher, or principal investigator, as the central figurehead, representing how the trial
is run, both practically and ethically. Perhaps because clinical trials aim to try new treatments or find answers
to unsolved health questions, prospective participants sometimes presume that a trial’s doctor may also be new or in training. Quite the opposite is often true. Generally, for atopic dermatitis studies, the principal investigators are physicians who have trained both as dermatologists or allergists and as clinical investigators, according to Katz (as they must understand the nature of the disease, along with, say, regulatory issues in regard to investigational new drugs and new drug application).

In addition to being well qualified as physicians and executors of a clinical trial, these doctors are often the leading professionals in their fields. They have not only designed a prospective trial, but have had it scrutinized by peers and Institutional review boards and approved for funding. They are often the individuals who are asking the most compelling medical questions—and working to seek answers.

What this Means for the AD Patient

In addition to helping the medical community and the community of patients with atopic dermatitis by contributing to medical research, eczema patients who qualify for and enroll in clinical trials can also reap the benefit of receiving individual care from a leading doctor in the field. and while some clinical trials can be more profit-focused than patient-focused, doctors who run clinical trials at many research institutions dutifully extend care to clinical trial participants outside of what a study might demand.

Hanifin is one such doctor. as an internationally recognized atopic dermatitis researcher who has led dozens
of clinical trials, Hanifin views his study participants as patients, not simply subjects, and treats them accordingly.

“Most of the time, people are coming to see us [in a study] because what they’re doing [to treat their atopic dermatitis] isn’t working. So I try to always make myself available for help. Our goal is to get the patient better,” Hanifin says. “I don’t stop being a doctor because they come in for a study. I want to make sure they get better—even if it’s with other health problems (and a lot of people don’t qualify because they have other problems). I want to help them with their skin problems, but if they need a referral or special test for other problems, then I try to help with that. Doctors have to be doctors. ”

In addition to getting supplemental health care from a leading doctor in the field and helping to further medical research, clinical trial participants may also get the benefit of being treated with a promising new medicine first. While this can be a particularly attractive perk for those who have long suffered with itchy and painful skin, it’s important to be thoughtful when entering a clinical study, since by the very nature of the word “trial,” outcomes aren’t guaranteed.

Questions to Ponder when Considering Participation in a Clinical Trial
 Though

Though there are designated boards and specially trained staff to protect the safety of patients in clinical trials, it’s important that participants understand the scope of the study (whether it is designed to learn more about the disease itself or to test a specific treatment or intervention), how it’s set up (whether a study is randomized, double blind, or uses a placebo) and whether it carries any potential risks (such as going off of a current medication to begin a new one).

Since there are so many different types of research institutions that conduct clinical trials, Hanifin suggests that prospective participants learn about the style of the principal investigator before signing a consent form to participate. Does the investigator refer to you as a patient or a subject? Is he or she willing to provide attention and care outside of what the study mandates? Does he or she consult with you when explaining the study or rely on printed materials to convey the study’s goals, parameters, and details? Does the principal investigator seem person- ally invested and connected to the work? Whether you feel comfortable with the doctor’s style will likely help you decide whether the study may be a good fit for you.

clinical_trials_p6For his part, Hanifin strives to work directly with participants, when possible, to ensure their inclusion and safety. “If I see a patient in the study and their atopic dermatitis is flaring out of control and they have to be off medication for a week, then I might delay the start of the study for that patient, in order to get them cleared up and to where they can sleep comfortably at night,” he says.

By law, participants are able to withdraw from a clinical trial at any time. However, Katz, Hanifin, and Goodin strongly suggest that potential participants take the time to understand a study’s purpose and scope and
ask additional questions of the clinical staff or principal investigator before signing on. Though each prospective participant must be given basic facts about how a trial works before they agree to participate (also called giving informed consent), asking additional questions (like whether there’s any already available information as to whether the drug works, or whether the drug may have known side effects) can help you make the right decision for you.

A New Perspective: Adjusting Personal Expectations and Recognizing Hidden Benefits

Finally, after being accepted into a clinical trial and choosing to participate in it, it’s important to keep some perspective about what may or may not be achieved—and to adjust expectations accordingly. Many people feel that unless a new cure-all is found, a participant’s time in a clinical trial is wasted or without merit. But those who participate in clinical trials that address questions of basic biology (such as why a child may have atopic dermatitis) might
be of assistance in findings that offer a greater knowledge base—something that will serve the wider population of sufferers for generations, instead of a possible treatment or targeted solution that might help just a small segment of people now. Additionally, even the most promising of therapies don’t always reveal themselves in an initial study.

As Hanifin points out, “The main new drugs [for atopic dermatitis] that have come out in the last 20 years are the topical calcineurin inhibitors. I don’t think there was much promise there when they started. But we quickly found that the drugs were helpful.”

“But much more frequently, we’ve struck out, and things haven’t been useful,” he continues. “Sometimes, we find what seems like a really exciting pathway to inflammation and you hope the drug being tested will block that. Then in the trial, it doesn’t work. So you kind of move on and start looking at other pathways. And that helps us. Sometimes, when a drug fails, it’s an indication that we don’t know enough and we have to think about other ways the disease happens.”

Likewise, while many of the successful clinical trial discoveries for atopic dermatitis can help both current and future patients, there is just as much to be learned from what may not work as from what may.

“There’s benefit of participating retrospectively,” Katz reminds us. “Even if a trial doesn’t work, well, that’s a benefit— so people aren’t treated with something that doesn’t work.”

No matter what the result of a given atopic dermatitis trial, both patients and researchers alike benefit from some pretty great odds: the buildable and newfound knowledge that’s gained is always a win.

The National Eczema Association extends a special thank you to Dr. Stephen Katz. Dr. Katz is a tireless advocate for skin disease research including eczema and we are grateful for his dedication. A special thank you, as always, to Dr. Jon Hanifin for his eternal and unsurpassed dedication to the eczema patient.

 

Questions to Ask

Before Participating in a Clinical Trial

  • What are the available treatments for my disease or condition?
  • How well have the available treatments been studied? How beneficial are they? What are their risks?
  • Are there any alternatives to therapies available on the market or through clinical trial?
  • What is the goal of the research being conducted in this clinical trial?
  • What are the stated benefits of the product being investigated?
  • What are the stated risks associated with the product being investigated?
  • How serious are these risks and how likely are they to occur?
  • What’s being done by clinical trial administrators to reduce risks as much as possible?
  • Will all participants receive the study treatment or the standard of care? Will a placebo (an inactive substance that looks like the drug being tested) also be administered?
  • Do I stand to personally benefit from the outcomes of the trial, or will my participation benefit others, or eczema research in general?

Additionally, the National Institutes of Health (NIH) offers a range of questions to ask when considering clinical trial participation. Go to the NIH website and search “Clinical Research Trials and You” or visit: http://www.nih.gov/health/clinicaltrials/basics.htm