Common Data Elements (CDEs) are a set of standard questions and allowable answers that researchers use in their studies to make sure they collect the same kind of patient information in the same way every time. They ensure consistent data collection across sites, studies, clinical trials, and even diseases. During the first Myositis Common Data Elements Consensus Conference at the National Library of Medicine (NLM), researchers discussed developing CDEs for studying myositis, a group of rare autoimmune diseases involving chronic inflammation of the muscles.

An international expert panel, including myositis researchers from Canada, Germany, Italy, Sweden, the United Kingdom, and the U.S., convened at the Nov. 14 meeting.

“Developing CDEs for myositis research presents a tremendous opportunity for us to work together, so we can improve the diagnosis and treatment of myositis patients,” said conference co-host Lisa Rider, M.D., who leads the NIEHS Environmental Autoimmunity Group.

NIEHS houses the administrative arm of the International Myositis Assessment and Clinical Studies Group (IMACS), a multidisciplinary consortium that studies inflammatory muscle diseases. Advances made by the group to develop criteria for measuring how myositis is classified in patients for research studies, how to assess patients' disease activity using a core set of measures, and their responses to treatments served as the foundation for the meeting.

“National Institutes of Health (NIH) leadership views this conference as a paradigm for how CDEs could be developed across NIH Institutes and Centers, by bringing together subject matter stakeholders and data scientists, and is following our progress closely,” said Richard Scheuermann, Ph.D., scientific director at the NLM and event co-host.

Measuring disease severity and progression

The conference’s principal order of business was to discuss CDE representation of a core set of nine instruments used to measure disease activity in adult and pediatric patients with myositis. A number of the tools, as well as the core set of measures, were developed by IMACS.

All nine CDE instruments achieved greater than 80% consensus from the expert panel that will be required to forward them to the NIH CDE Governance Committee and, from there, to be deposited in the NIH CDE repository (see sidebar). Because these CDEs are based on validated clinical instruments, they meet the criteria for official endorsement by the NIH CDE Governance Committee. The nine CDEs are as follows.

• Physician Global Disease Activity Assessment
• Patient/Parent Global Disease Activity Assessment
• Manual Muscle Testing (MMT-8)
• Health Assessment Questionnaire and Childhood Health Assessment Questionnaire
• Myositis Disease Activity Assessment Tool (MDAAT)
• Serum Muscle Enzyme Measures (such as creatine kinase and aldolase)
• Childhood Myositis Assessment Scale (CMAS)
• Disease Activity Score

The NIH CDE repository groups similar CDEs into bundles of CDEs known as forms, which support the capture of data collection context and the reuse of CDEs.

After approving the first nine core set instrument CDEs, the conference participants prioritized 43 additional myositis study forms for future coding and potential inclusion in the NIH CDE repository. About one-third received high priority recommendations.

“We now have the capability to build databases around these CDEs to enhance scientific studies and clinical trials,” Scheuermann said.

Importance of patient input

Office of Autoimmune Disease Research (OADR) Director Vicki Shanmugam, M.D., said she was keen to do this work in conjunction with people affected by these diseases, their caregivers, and their advocacy groups.

Several conference attendees stressed the importance of including patient and expert input in the data collection process, using domains like mental and psychological health, social functioning, pain, fatigue, and respiratory problems. Representatives from three myositis patient advocacy groups were part of the expert panel, including from The Myositis Association, Cure JM Foundation, and Myositis Support and Understanding.

“We appreciate the need to define patient perception of disease,” said Didem Saygin, M.D., a clinical researcher and rheumatologist at Rush University.

Belinda Seto, Ph.D., deputy director of Office of Data Science Strategy (ODSS), agreed. 

“Including patient-reported outcomes and functional measures across different age groups and disease subtypes is important,” she said.

ODSS was tasked by Congress with developing CDEs in areas with limited attention, such as autoimmune diseases. It spearheads the implementation of the NIH Strategic Plan for Data Science. The work to develop CDEs for myositis and bring consensus on them has been co-focused by ODSS and OADR.

A new methodology

Organizers anticipate the workflow, methodologies of CDE creation, and bundling of data elements developed through this project can be applied to develop CDEs for other autoimmune diseases.

“Many of these core data elements can be used in a wide range of clinical study, which, if we collect in a consistent way, would support some level of interoperability across different disease areas,” Scheuermann said.

A second Myositis Common Data Elements Consensus Conference will take place in May 2025, in which the CDEs for the additional myositis study forms will be reviewed, aiming to achieve consensus in the coding from the expert myositis panel.