The digitization of healthcare has allowed previously manual processes to become both streamlined and more patient-centric. To learn about how these changes are affecting the diagnosis of rare diseases, we had a chance to sit down with Dr. Marc E. Rothenberg, MD, PhD, director at Cincinnati Children’s Hospital and principal investigator at the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR). CEGIR is an NIH-funded research network and registry that is now implementing cloud technology to improve collaboration between a nationwide network of researchers, providers, patients, and organizations interested in tackling the challenges of eosinophilic gastronintestional disorders. From improving the interactions between researchers at 9 different centers located across the country to connecting patients with clinical studies and patient advocacy groups, CEGIR is leading the charge on bringing digital health to the clinical research field.
Mike Batista, Medgadget: Let’s start with a little bit of background and how you became involved with the CEGIR registry.
Dr. Rothenberg: My background is focused on allergy, immunology and pathology of eosinophils, beginning in medical school at Harvard where I conducted studies on eosinophil hematopoiesis under Dr. Frank Austen as he developed the first culture system for human eosinophils. From there I completed my residency at Children’s Hospital in Boston and then went on to do a fellowship in immunology and hematology at Children’s Hospital and my post-doctorate training with Dr. Philip Leder at Harvard where he cloned the eotaxin chemokine. I then came to the University of Cincinnati where I serve as Professor of Pediatrics and Cincinnati Children’s Medical Center as director of the Division of Allergy and Immunology, which has since become a top program in pediatric research and out of which CEGIR was formed and is based.
Medgadget: What is the CEGIR registry and where did the idea come from?
Dr. Rothenberg: Our overall goal with CEGIR is to improve the lives of those with eosinophilic gastronintestional disorders (EGIDs) through innovative research, clinical expertise and education via collaborations between scientists, health care providers, patients and organizations. Up until now there hasn’t been a complete, NIH-backed, multidisciplinary approach leveraging the most progressive minds in gastroenterology, allergy, immunology and pathology to treat these types of rare disorders.
Through CEGIR, patients with eosinophilic gastrointestinal diseases and their family members learn about research studies that they may be able to join. These patients play a part in groundbreaking research and discover new treatment methods, as well as find resources on patient advocacy groups and related events.
This two-pronged approach – research and patient support – is crucial to our overarching mission.
Medgadget: What has changed, technology or otherwise, to make the CEGIR registry possible today?
Dr. Rothenberg: With researchers, doctors, patients and pathologists in 9 different centers spread across the U.S., we rely on technology that enables us to communicate, collaborate and ultimately reach diagnoses fast. For that we use a handful of tools, including Dropbox, and a whole image slide scanner.
EGIDs are rare diseases and involve biopsies as part of diagnosis and monitoring therapy. In CEGIR, there will be a number of studies that will be performed — both observational and interventional. These studies require that our 3 central review pathologists, located in 3 different departments across the U.S., review and score the biopsy slides for various aspects of the disease pathology.
In past years, in order to accomplish this kind of remote, collaborative research, participating institutions would need to send their glass slides to these multiple pathologists based in different locations — these are glass slides that could get broken, lost in transit, or that could pile up on desks and never be seen again. It’s costly and expensive to mail these slides the traditional way, and if problem cases arose, there would be the additional cost of the pathologists needing to travel to another site to review together at a multi-headed microscope and discuss the issue at hand.
Now, using the slide scanner to upload virtual slides to the cloud via Dropbox, our pathologists can simultaneously view and annotate slides, and share feedback in real-time, which in turn enables us to reach diagnoses and generate research data more quickly. This also allows for greater accessibility and flexibility — pathologists can log in and view the images of the slides we’re scoring from home or at work and can access the site using any device, including iPads or iPhones, which adds to the convenience. Another benefit is that participating institutions keep their patients’ glass slides onsite for easy access and archiving.
Patient security is a top concern, which is why we decided to use Dropbox to distribute the samples as the service supports HIPAA compliance. This was especially important to the NIH, who fully supports our virtual workflow system and use of Dropbox once they saw the time and cost savings it helps us achieve.
Medgadget: How does the existence of the CEGIR registry impact researchers working on clinical studies for EGIDs?
Dr. Rothenberg: CEGIR connects researchers with others conducting similar studies across the country, patients for potential participation in their own studies, and education through collaboration with scientists, health care providers, and professional organizations. We also have a core dedicated to training new investigators in clinical and translational research, enhancing the likelihood of their academic success.
At this time we have 9 participating institutions in the U.S., and one collaborating site in Switzerland. We also have a Pilot Project program, which provides grant money which CEGIR investigators can use to initiate new projects relevant to EGIDs. It is our belief that increasing the number and quality of clinician researchers studying eosinophilic diseases will improve current and future patient care.
Medgadget: What are some examples of the clinical studies for which the CEGIR registry has helped find patients?
Dr. Rothenberg: Diet manipulation is one mode of therapy for EGID, particularly eosinophilic esophagitis. Patients who eliminate offending foods from their diet often experience symptom reduction or abatement, and reduced inflammation in the esophagus. The most popular elimination diet requires elimination of 6-8 foods which is difficult to accomplish. Patients who follow the diet often report reduced quality of life, especially reduced or altered social interactions. We will test the ability of eliminating 1 food, milk, which is the food most commonly identified as an offending agent, to accomplish the same effect as more extensive food elimination. This study was requested by many patients because eliminating 1 food is simpler to follow than more extensive elimination diets and impacts others in the family less than more extensive diets.
Medgadget: How does patient advocacy play a role at CEGIR?
Dr. Rothenberg: Patient advocacy is a core aspect of our work at CEGIR. We help provide the resources and connect patients to groups specializing in eosinophilic gastrointestinal diseases that are devoted to providing support, resources and services for them and their families. Some of the organizations we partner with include the American Partnership for Eosinophillic Disorders, Campaign Urging Research for Eosinophilic Diseases (CURED), Food Allergy Research & Education, and the Eosinophilic Family Coalition.
Medgadget: Are there other examples of how you’ve seen telehealth influence the clinical research field and are there additional opportunities for telehealth to play a role in new ways?
Dr. Rothenberg: Diagnoses and drug development approval processes can often span many years. We’ve seen lots of technical advancement for researchers that can help reduce this gap, including tools like Dropbox. Clinical researchers can now collect biometric data, patient-reported outcomes and create a virtual video connection without a patient needing to leave their home. Besides increasing convenience and improving the patient experience, this is especially helpful for older or immobile patients who are unwilling or unable to do the travel required for most trials. New apps and e-clinical platforms are also transforming patient management in these trials.
Telehealth is already beginning to change the way clinical research is conducted, by breaking down barriers in collaboration that before was difficult, if not impossible, due to distance. New tools are enabling research to be done in ways that are more flexible and convenient than ever before — no longer must researchers be physically present in a lab and on a desktop in order to continue their work. They can access critical data remotely and work on tablet and mobile devices.
Secure tools that enable researchers to collaborate faster and meet patients wherever they are will mean faster diagnoses, increased retention, and improved patient engagement.
Medgadget: Is there anything else you’d like our readers to know about the CEGIR registry or CEGIR in general?
Dr. Rothenberg: We encourage patients and families interested in learning more or signing up to participate in a clinical trial to visit our website: https://www.rarediseasesnetwork.org/cms/cegir/