- An international team of researchers has published a paper focusing on ways to improve the collection, quality, and coordination of information that tracks the health experiences of people with MS in the “real world.”
- These types of efforts, also known as observational studies, can answer important questions about factors that predict disease course, long-term effectiveness of therapies, benefits of rehabilitation and many others. But because such studies are also prone to research bias – unlike controlled clinical trials – their results aren’t always accepted by regulatory agencies and healthcare providers.
- The published paper stemmed from a workshop convened in September 2018 under the auspices of the International Advisory Committee on Clinical Trials in Multiple Sclerosis, which is jointly supported by the National MS Society and the European Committee for Treatment and Research in MS. The goal was to develop recommendations to better standardize data collection and enhance the ability to answer important questions about MS and its optimal care.
- “Leveraging real-world data to investigate multiple sclerosis disease behavior, prognosis, and treatment” was published on November 28, 2019 in Multiple Sclerosis Journal. The open access paper available to anyone, without the need of a subscription.
Clinical trials in MS usually ask specific questions about the effectiveness of therapies by involving people with very specific disease activity, age range, and other characteristics over a relatively short period of time. After a therapy is approved based on clinical trials, there remain questions about who would most benefit from it, and what are its long-term safety and benefits. This is one reason why more and more efforts are underway to track health experiences of cohorts (groups) of people living with MS in the “real world” over time using patient registries and other sources of data.
There are thousands of people being followed in various types of MS cohorts around the world. These types of observational studies can answer important questions about factors that predict disease course, long-term effectiveness of therapies, benefits of rehabilitation and many others. However, the type and quality of data they collect varies significantly, as well as the way data is recorded, analyzed and used. This makes observational studies prone to research bias (unintended factors that can affect results), so their results aren’t always accepted by regulatory agencies and healthcare providers.
Workshop and Resulting Paper:
The International Conference on Data Leveraging to Answer Key Clinical Questions in Multiple Sclerosis*
was convened in September 2018 under the auspices of the International Advisory Committee on Clinical Trials in Multiple Sclerosis
, which is jointly supported by the National MS Society and the European Committee for Treatment and Research in MS.
The purpose was to evaluate how to leverage MS cohorts and the types of clinical questions that they are best at answering. The team also discussed challenges and barriers and made recommendations for improving the use of real-world data to advance the better understanding and treatment of MS. A paper was drafted by a core group based on workshop discussions.
The paper describes types of data collection that are ongoing right now, including patient registries; electronic medical records; health insurance claims; MRI and other imaging; biospecimens (such as blood); and wearable sensors. Integrating these many sources of data, and making them comparable and sharable, presents many challenges and yet would help create a holistic picture of an individual’s health experience and help guide treatment decisions, and would also enable important progress toward understanding MS.
Beyond technical challenges of data compatibility, there are overarching privacy and confidentiality issues that need to be addressed.
The paper points to some examples of MS cohorts that were established with specific research questions in mind and have very careful data collection. One is the Multiple Sclerosis Partners Advancing Technology and Health Solutions (MS-PATHS
), which captures standardized data from people during office visits and shares it on a common electronic platform, which can then be used for research studies. Another is the Pediatric MS Tool-Kit
, created to standardize the collection of data measuring environmental exposures of children with pediatric MS.
The authors offer several recommendations for improving the quality of evidence based on real-world data and its usability for informing our understanding of MS. Many involve better standardizing data to improve their compatibility. Others include:
- developing ways to incorporate additional data such as Patient Reported Outcomes, biomarkers and other information into existing cohorts;
- improving the reporting of studies such that they follow existing guidelines to reduce bias;
- making the capture of comprehensive data part of routine MS care;
- developing a searchable catalog of existing cohorts; and
- growing the workforce of data scientists, biostatisticians and epidemiologists to better enable rich data collections to be mined properly.
One effort already underway in relation to these recommendations is the creation of a “metadata catalog,” a searchable repository of cohorts and how to access them. The National MS Society is funding the development of a metadata catalog of North American cohort studies, which could be expanded to include other cohorts in the future.
The paper, “Leveraging real-world data to investigate multiple sclerosis disease behavior, prognosis, and treatment,”
was published on November 28, 2019 in Multiple Sclerosis Journal
. The open access paper is available to anyone, without the need of a subscription.
The meeting was chaired by Jeffrey A. Cohen, MD (Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic), Ruth Ann Marrie, MD, PhD (University of Manitoba Health Sciences Center), Ellen Mowry, MD, MCR (Johns Hopkins University), Maria Trojano, MD (University of Bari), and Bernard Uitdehaag, MD, PhD (VU University Medical Center).