By Amy Ratner, Beyond Celiac Medical and Science News Analyst
Celiac disease is entering a new phase that recognizes it as a serious, burdensome condition with a high unmet need for treatment options beyond the gluten-free diet, says an article published in the journal, Gastroenterology.
Additionally, scientists, government regulators, pharmaceutical companies, patient advocates and patients agree that all need to collaborate to improve the lives of those living with celiac disease. Beyond Celiac is developing technology to bring these stakeholders together to create solutions.
The journal article, published in the September issue, is a summary of findings of the 2015 GREAT3 workshop sponsored by the Food and Drug Administration (FDA) and several gastroenterological associations. Beyond Celiac also provided financial support to the workshop.
GREAT3 stands for Gastroenterology Regulatory Endpoints and Advancement of Therapeutics. Celiac disease took center stage on the second day of the two-day meeting (the first focused on inflammatory bowel disease).
The workshop focused on:
- Identifying patients with celiac disease who would be candidates for drug treatments
- Defining what patients, gastroenterologists and the FDA perceive as the benefit of these treatments
- Defining how the clinical benefits could be measured in drug trials in ways that would lead to drug approval
Alice Bast, president and chief executive officer of Beyond Celiac and an FDA Patient Representative for the field of celiac disease, represented patients, speaking about their experiences living with celiac disease and their expectations of celiac disease treatment.
"GREAT3 was an important and essential step that will support both current and future scientific studies and clinical trials to advance celiac disease research and provide celiac disease patients with alternative therapeutic solutions," Bast says.
At the time of the workshop, fewer than a dozen randomized controlled therapeutic trials had been published, the summary article says. “Major historical obstacles to drug development include misperceptions that celiac disease is rare and mild and that the gluten-free diet is a near-optimal therapy,” the summary notes.
Despite these misperceptions, those living with celiac disease face the reality that the gluten-free diet is expensive, socially limiting and fraught with uncertainties caused by the potential for cross-contact when dining out.
“The constant vigilance required by this diet can be a source of great anxiety and have substantial impact on adherence,” the article notes. Additionally, patients report persistent symptoms despite trying to follow the diet, it says.
Consequently, surveys have shown that patients want non-dietary treatments. One study found they are more interested in those that protect against cross-contact with gluten than those that would eliminate the need for the gluten-free diet.
Path to drug approval
Patients and clinicians at the workshop agreed that typical gastrointestinal symptoms should be the focus of initial studies looking for clinical benefits. These symptoms are common and can be measured in a reasonable time frame, the article says. Meanwhile, “the FDA emphasized that a clear definition of clinical benefit and a reliable method of measuring it are key” to designing drug trials that could be successful in getting market approval.
The role of health-related quality of life, called HRQOL and defined as the effect of illness and its treatment on physical, psychological and social aspects of life, was also addressed. Celiac disease is unique in that both symptoms and the gluten-free diet have a negative impact on quality of life, the article says. The FDA acknowledged that quality of life is important, but said that it is a broad concept that includes impacts of a disease that might not be able to be modified. The FDA noted for a drug to be approved it would be key to show that it allows gluten to be consumed without disease exacerbation.
The role of the biopsy to measure intestinal damage and blood tests to measure gluten antibodies, called serology, were also discussed in the context of measuring clinical benefits. “Currently, the only FDA-cleared use for celiac serologies is as an aid for diagnosis of patients with suspected celiac disease,” the article says. “This limits how serologies can be used in regulatory trials, although they are routinely used for monitoring in clinical practice.”
George Duke, MD, a representative of the Pharmaceutical Research and Manufacturers of America, described some obstacles to celiac disease drug approval, including the lack of precedent for a product to treat celiac disease, according to the article. And he recommended support for development of a patient registry as “a mechanism to better understand and support therapeutic trials.”
Beyond Celiac is addressing this need and in early 2017 will launch an innovative and unique technology to collect valuable patient-reported data and advance research.
“We intend to provide a pathway and proactive strategy to educate patients and empower them to become engaged in their healthcare and actively participate in research,” says Bast. “The technology will be a dynamic learning system where, together, patients and researchers will work towards solutions for everyday challenges caused by living with celiac disease.”
Just a few weeks after GREAT3, Beyond Celiac launched its innovative Research Summit, a workshop months in the making, which brought together patients, caregivers, clinicians, scientists, the diagnostics and biopharmaceutical industries and a federal research agency. Participants identified that existing research tools and instruments do not accurately capture the patient’s experience or satisfy the needs and interests of these key stakeholder groups.
Together, attendees helped to develop innovative questions and concepts that will produce new, meaningful information that researchers can use to solve the field’s most pressing challenges. The Summit sparked ideas for a new research technology and helped form the design of the organization’s long-term, patient-centered agenda.
The questions and concepts developed at the Summit are forming a new Beyond Celiac web-based technology that will capture the experiences of the community, through qualitative and quantitative data. This data will be used to build a knowledge base about celiac disease. The knowledge base will lead patients to relevant tools and resources based on their experiences and concerns and incorporate health behavior change theories to ensure success.
The technology will allow patients to enroll in a registry where they can provide data that will help researchers develop new treatment options. The registry will also link patients to clinical trials and other research studies.
The first step will be a beta, or small, limited, version of the technology, and Beyond Celiac will be recruiting those who have been diagnosed with celiac disease by a physician. Sign up for the Research Opt-In to stay informed and learn how you can get involved in 2017.
And you can read more about the GREAT3 meeting here.