Patient Experiences with Relapsing-Remitting and Primary Progressive Multiple Sclerosis: Development of a Conceptual Model to Inform Clinical Outcome Assessment Evaluation.

introductionMultiple sclerosis (MS) is an autoimmune-mediated neurodegenerative disorder. The United States Food and Drug Administration recommends using clinical outcome assessments (COAs) to measure concepts that matter to patients in clinical trials and to document the content validity of the COAs using conceptual models of patient experience. This study aimed to explore and document the patient experience of signs, symptoms, and health-related quality of life impacts of relapsing-remitting MS (RRMS) and primary progressive MS (PPMS) to inform future COA validation research.

methodsWe conducted a targeted literature review (TLR) and qualitative semi-structured interviews of patients with RRMS and PPMS in the United States of America (USA). Publications and interview transcripts were analyzed, synthesized, and inductively categorized to develop a conceptual model of patient experiences of RRMS and PPMS.

resultsTen publications were reviewed as part of the TLR, and 16 participants (RRMS, n = 7; PPMS, n = 9) were interviewed. Most participants reported fatigue, muscle weakness, difficulty walking, sleep disturbance, and bladder issues. Many participants experienced negative emotional, physical, and social impacts due to the disease. Most participants also described needing to rest and plan/avoid activities, as well as requiring support from their family to manage their symptoms.

conclusionThis study highlights the multidimensional burden of living with RRMS and PPMS. A provisional conceptual model was developed, harmonizing the TLR and interview findings. This conceptual model may be used to promote a better understanding of the patient experience of RRMS and PPMS and to evaluate the content validity of COAs during the development of new drug treatments.