Remote Monitoring, Evaluation, Recruitment, and Outcomes Measures: Creating a New Clinical Trial Model for Myositis and Other Rare Autoimmune Diseases

The structure of clinical trials in rare autoimmune diseases, and for that matter, rare diseases in general, has led to suboptimal enrollment and durable participation, which in turn has limited knowledge, and slowed the pace of therapeutic progress. For patients with idiopathic inflammatory myopathies (IIMs), but more prominently those living outside major urban centers and proximity to academic medical centers, participation in research and clinical trials has been historically underrepresented, leading to the exclusion of most trial-eligible individuals, particularly those with progressive or mobility-limiting disease. In this sense, and in a lot of ways across medical fields and specialties, rheumatology or otherwise, geography can, unfortunately, be destiny.

To address these limitations, the UPMC Division of Rheumatology and Clinical Immunology is pursuing research with the goal of building the evidence base to support developing a new framework for conducting clinical trials in IIM and related conditions. Led by Rohit Aggarwal, MD, MS, professor of medicine and codirector of the UPMC Myositis Center, and colleagues from the Division, this effort is working to integrate remote patient evaluation tools, create decentralized trial enrollment protocols, validate virtual outcome measures, and find ways to leverage and incorporate the rapidly growing field of artificial intelligence (AI) tools for enhanced monitoring and trial design.

“We have to change the way we design clinical trials if we want to make them accessible to the full spectrum of rare disease patients,” says Dr. Aggarwal. “This effort goes way beyond the realm of convenience. And beyond our world in rheumatology. It cuts across the ideas of equity, generalizability, and the quality and translatability of scientific knowledge into meaningful improvements in patient care and quality of life. And that’s just the beginning of the list.”

Remote Feasibility and Recruitment in Clinical Myositis Trials

To support the development of a more inclusive clinical trial framework, Dr. Aggarwal and colleagues have conducted research, including several observational studies over the past several years that explore the feasibility, validity, and operational performance of decentralized trial elements in patients with IIM. One study, titled “Internet-based Enrollment of a Myositis Patient Cohort — A National Experience,” was published in Clinical Rheumatology in 2024. The study showed that patients could be successfully screened, consented, and longitudinally followed using fully remote infrastructure and telemedicine platforms.

A prospective observational study published by the Division in Rheumatology in 2025, titled “Disease Characteristics and Medications Use in Idiopathic Inflammatory Myopathy: A Multi-Centre Prospective Observational Study of Decentralized Remote Vs. Traditional Clinic Enrolment,” compared the clinical and demographic characteristics of patients enrolled through remote mechanisms with those enrolled at academic sites. The two populations were found to be clinically comparable, reinforcing the potential for decentralized trial models to reach a broader and more representative patient population.

“We were able to show that patients enrolling remotely look no different clinically than those coming to the site,” Dr. Aggarwal says. “That gave us the confidence to build out the model further.”

Another study published in Rheumatology in 2025, titled “Recruitment Rate Comparison Between a Virtual Tele-Research Cohort and a Traditional Centre-Based Cohort in Idiopathic Inflammatory Myopathy,” evaluated recruitment patterns and found that monthly enrollment rates were significantly higher in the virtual cohort. The analysis also showed that the geographic spread of participants extended far beyond the traditional 200-mile catchment zone typical of single-site or multicenter trials.

“Recruitment is faster, more efficient, and more inclusive when you remove the geographic barrier,” Dr. Aggarwal says. “That’s not a theory anymore. We have data that support this idea and will be working to collect further measures along similar lines as we progress with these areas of investigation for improving how clinical trials are conducted.”

Developing Tools for Remote Outcome Assessment

Along with their studies on feasibility and recruitment, Dr. Aggarwal and colleagues have also focused on developing and validating outcome measures that can support remote assessment of patients in decentralized clinical trials. One study published by the Division in Rheumatology in 2025, “Short Form-36 Psychometric Properties in Idiopathic Inflammatory Myopathies: Reliability, Validity and Responsiveness,” evaluated the reliability and responsiveness of the Short Form(SF)-36 in patients with idiopathic inflammatory myopathies. The SF-36 is a widely used health-related quality-of-life measure that captures data, including physical functioning, pain, vitality, general health perceptions, and emotional well-being. The analysis performed by Dr. Aggarwal and colleagues confirmed that the instrument performs well in the myositis patient population and has the potential to be used to track outcomes in different types of encounters: in-person visits and virtual or remote settings.

In earlier work published in Clinical Rheumatology in 2022, Dr. Aggarwal and the Division collaborated with international partners to validate two simplified patient-reported outcome measures for use in remote monitoring. That study on the “Validation of Two Simple Patient-centered Outcome Measures for Virtual Monitoring of Patients With Idiopathic Inflammatory Myositis,” was designed to assess fatigue and physical function and could be deployed asynchronously through digital platforms, reducing the reliance on in-clinic assessments.

Wearable technologies have also been explored as part of the efforts by Dr. Aggarwal and colleagues to find ways in which clinical trial participation for myositis patients can be improved, and by extension trials for other conditions. In a separate 2022 study published in Rheumatology, the team examined the utility and efficacy of consumer-grade step counters for capturing physical activity data in patients with myositis. The findings from this research project showed a correlation between step count and clinician-assessed and patient-reported function. That finding suggests that these types of readily available devices and technologies may serve as viable alternatives to more traditional outcome measures in decentralized trials conducted with patients far from the enrolling center’s physical location.

“We now have a set of tools that are patient-reported, wearable, and validated that can support real measurement in a remote context,” Dr. Aggarwal says. “And we’ve tested them specifically in the myositis patient population.”

Transitioning to Interventional Trials

In 2024, Dr. Aggarwal and colleagues, in a multicenter collaborative effort, launched a prospective randomized interventional clinical trial using a hybrid design that incorporated remote infrastructure. The study, conducted under the Myositis Interstitial Lung Disease Nintedanib Trial (MINT) network, is currently evaluating the efficacy and safety of nintedanib in patients with progressive myositis-associated interstitial lung disease. The trial design combines remote enrollment and digital follow-up tools without requiring in-person assessments for most patients. The study’s design and further details were published in an open access article in BMC Pulmonary Medicine.

The study is serving as a proof of concept that decentralized and hybrid clinical trial designs can be conducted in complex autoimmune populations.

While initial efforts focused on idiopathic inflammatory myopathies, the model and approaches that Dr. Aggarwal and colleagues are working to design, test, and validate have the dual goals of translatability to other domains in and out of rheumatology, as well as scalability. The long-term goal is building a cross-disease platform for rare disease trial access and participation.

“The barriers we see to more effective clinical trials with broader participation are not necessarily disease specific. Many times, they are entrenched structural constructs,” Dr. Aggarwal says. “If we solve them in myositis clinical trials, we can use that platform to open up research access and insights in other rare diseases, too.”

References and Further Reading

• Silva RL, Keret S, Chandra T, Sharma A, Pongtarakulpanit N, Moghadam-Kia S, Oddis CV, Aggarwal R. Internet-based enrollment of a myositis patient cohort—a national experience. Clin Rheumatol. 2024 Oct; 43(10): 3157–3166.
• Keret S, Silva RL, Chandra T, Gkiaouraki E, Pongtarakulpanit N, Sriram S, Moghadam-Kia S, Oddis CV, Aggarwal R. Disease characteristics and medications use in idiopathic inflammatory myopathy: a multi-centre prospective observational study of decentralized remote vs. traditional clinic enrolment. Rheumatology (Oxford). 2025 Apr 1; 64(4): 2099–2105.
• Moghadam-Kia S, Aggarwal A, Venuturupalli S, Keret S, Pongtarakulpanit N, Riley M, Mahajan S, Scaramangas-Plumley D, Patel A, Kelley J, Tiniakou E, Oddis CV, Aggarwal R; MyPacer Study Group. Recruitment rate comparison between a virtual tele-research cohort and a traditional centre-based cohort in idiopathic inflammatory myopathy. Rheumatology (Oxford). 2025 Aug 1; 64(8): 4687–4694.
• Keret S, Aggarwal A, Almackenzie M, Bijoy George T, Sriram S, Chandra T, Lomanto Silva R, Gkiaouraki E, Pongtarakulpanit N, Moghadam-Kia S, Oddis CV, Aggarwal R. Short Form-36 psychometric properties in idiopathic inflammatory myopathies: reliability, validity and responsiveness. Rheumatology (Oxford). 2025 Aug 1; 64(8): 4695–4702.
• Naveen R, Thakare DR, Agarwal V, Aggarwal R, Gupta L. Validation of two simple patient-centered outcome measures for virtual monitoring of patients with idiopathic inflammatory myositis. Clin Rheumatol. 2022 Mar; 41(3): 765–772.
• Saygin D, Rockette-Wagner B, Oddis C, Neiman N, Koontz D, Moghadam-Kia S, Aggarwal R. Consumer-based activity trackers in evaluation of physical activity in myositis patients. Rheumatology (Oxford). 2022 Jul 6; 61(7): 2951–2958.
• Aggarwal R, Oddis CV, Sullivan DI, Moghadam-Kia S, Saygin D, Kass DJ, Koontz DC, Li P, Conoscenti CS, Olson AL; MINT investigators. Design of a randomized controlled hybrid trial of nintedanib in patients with progressive myositis-associated interstitial lung disease. BMC Pulm Med. 2024 Oct 30; 24(1): 544.