OBJECTIVES: The objectives were to a) develop a preliminary conceptual model based on qualitative research in patients with multiple sclerosis (MS) and b) identify and evaluate commonly used patient reported outcome measures (PROMs) in MS to assess the likelihood of successfully supporting future Food and Drug Administration (FDA) labeling claims.
METHODS: PubMed was searched to identify recent (since 2010) qualitative research studies in patients with MS as well as to identify relevant PROMs for further review. The development process for a selected subset of PROMs was compared against the recommendations contained in the FDA PRO guidance.
RESULTS: The PubMed search identified 3 qualitative studies focusing on key symptoms and impacts experienced by patients with MS. The most common symptoms included fatigue; pain; musculoskeletal issues such as stiffness, spasm, and difficulty walking; and balance problems. Based on these results, a preliminary conceptual model was developed displaying the relationship between the disease process, patient factors, signs/symptoms reported by patients, and the impact of MS. Nine PROMs were identified from the searches and five most relevant PROMs were selected for further evaluation (4 MS-specific, 1 generic). The measures reviewed were: MS International Quality of Life questionnaire (MusiQoL), MS Quality of Life Instrument (MSQOL-54), MS Impact Scale (MSIS-29), MS Walking Scale (MSWS-12), and the PROMIS item banks. While some of the identified measures have shown responsiveness in clinical trials, none meets all FDA requirements on PROMs.
CONCLUSIONS: Based on the preliminary conceptual model, physical functioning aspects of MS are proximal to the disease and thus a potential target for future PRO labeling claims. No existing PRO measure appears likely to support an FDA labeling claim in their current format. The next phase of the project is the conduct of clinician interviews and concept elicitation focus groups to further revise and refine the conceptual model.
