E-Thesis 152 views 41 downloads
The Benefits of a United Kingdom Multiple Sclerosis Register / RODDEN MIDDLETON
Swansea University Author: RODDEN MIDDLETON
DOI (Published version): 10.23889/SUthesis.65409
Abstract
Background: In rare diseases such as multiple sclerosis (MS) the capture of data purely from clinical trials or hospital cohorts is insufficient for fully understanding the multifaceted impacts of the disease. MS is the most common non-traumatic cause of disability in young adults worldwide. The dis...
| Published: |
Swansea, Wales, UK
2023
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| Institution: | Swansea University |
| Degree level: | Doctoral |
| Degree name: | Ph.D |
| Supervisor: | Brophy, Sinead. |
| URI: | https://cronfa.swan.ac.uk/Record/cronfa65409 |
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| Abstract: |
Background: In rare diseases such as multiple sclerosis (MS) the capture of data purely from clinical trials or hospital cohorts is insufficient for fully understanding the multifaceted impacts of the disease. MS is the most common non-traumatic cause of disability in young adults worldwide. The disease is multifactorial, profoundly impacts quality of life, and life span is affected by 7 to 14 years. Diagnosis has become easier and newer treatment options have proliferated but monitoring and researching the disease's various impacts remains challenging with a largely clinical focus on ‘hard outcomes’ such as imaging, biomarkers and in-person clinically assessed scales. Disease registers are best positioned to capture data about chronic disease such as MS as they allow for longitudinal capture from a variety of sources, including clinical outcomes but also data from patients/participants. Moving disease registers away from paper-based capture has allowed for easier, more accurate and rapid capture from these sources but can also add new data sources such as novel outcome measures, data from other devices and then facilitate linkage of that data across all these domains. Aim: To communicate the learning and experiences from building a UK-wide register which captures multifaceted MS data, in order to inform the development of similar registers for other conditions. Methods: Construction of an electronic platform sufficiently flexible to capture data from people with MS in the form of patient/participant reported outcomes, from the NHS as clinical datasets, and the technology to link these datasets together in a privacy protecting way to make these datasets available to other researchers. The data capture technology must be robust enough to add additional sources or datasets as needed whilst maintaining the core elements of reproducible research. Results: A robust, flexible, privacy-protecting secure research-ready disease register was constructed containing data directly captured from more than 20,000 participants, 50 NHS sites with more than 1 million completed Patient Reported Outcomes (PRO), clinically transmitted datasets and other diverse outcomes collected. Pseudonymised elements of that data, subject to robust governance and review, are released to appropriately qualified researchers to carry out their own research on the platform. A number of important lessons were learned in the construction of this research register. The most important being that involvement of people affected by the disease in all aspects of the project is crucial for enabling key aspects of a functional register, including the collection of varied and complementary data, the levels of engagement required for longitudinal research, and assisting with the direction of research. Feedback loops in this participant-register relationship create a more holistic research instrument. Another vital aspect is the ability to carry out data linkage both within the project and to outside routinely collected datasets, expanding the scope of potential research without adding burden to participants. Flexibility of approach is particularly important for chronic disease where aetiology is uncertain and impact of lifestyle elements on the disease and the person are evolving. Maintaining these core principles of participant engagement, flexibility, and the ability to include novel datasets allows the collection of real-world data from individuals, their clinicians, and other relevant sources and stakeholders, leading to diverse and significant research into the disease. Conclusion: The UK MS Register can serve as a model for the design and construction of disease registers, capturing validated data from diverse data sources with reference to patients’ requirements and desires and satisfying researchers and clinicians needs for an unbiased, varied research ready dataset. |
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| Item Description: |
A selection of third party content is redacted/removed or is partially redacted/removed from this thesis due to copyright restrictions. |
| Keywords: |
Multiple Sclerosis, Disease Registers, Health Informatics, Epidemiology, PROS |
| College: |
Faculty of Medicine, Health and Life Sciences |
| Funders: |
Swansea University |