WEST PALM BEACH, Fla. — Patient-reported outcomes (PROs) define the issues that matter to the patient, but their potential in multiple sclerosis (MS) is likely to remain unfulfilled until barriers, including a lack of incentives, are addressed systematically, according to experts participating in a symposium at the 2024 Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum.
These barriers include a lack of consensus on how and which PROs to collect, lack of a systematic method of interpreting the meaning of PRO changes, and lack of reimbursement for the time to collect PRO data and enter it into the medical record, said Robert McBurney, PhD, president of the nonprofit Accelerated Cure Project for Multiple Sclerosis, Washington, DC.
Useful Clinical Information
PROs can identify hidden symptoms of MS as well as provide information on the relative importance of the standard measures of disease progression, such as disability, but at the current time “these are rarely captured or used in shared decision-making to guide treatment,” McBurney said.
A reasonable analogy can be made between MS and musculoskeletal diseases, such as arthritis, according to McBurney. In both, not all patients experience the burden of disease in the same way, whether measured with traditional laboratory or imaging evidence of disease activity or by PROs that capture anxiety, depression, and specific impairments affecting activities of daily living.
Yet, the Centers for Medicare and Medicaid Services (CMS) is now mandating the entry of PRO data for the reimbursement of some forms of orthopedic surgery, while MS is lagging behind, according to McBurney.
The difference between orthopedics and MS is evidence submitted to CMS showing that improvement in PROs matter for patient outcome and well-being. McBurney argued that the same type of data is lacking for MS.
More well-designed clinical trials are needed to confirm that beneficial effects on PROs can improve patient outcomes, but McBurney suggested that PRO data from the many MS patient registries might be an easier first step. He reported that 24 of 43 MS registries around the globe are now capturing PRO data.
Unfortunately, the AXON registry, which is managed by the American Academy of Neurology, is not one of them, McBurney said. This is not an oversight. McBurney explained that the first effort to add PROs to data collected by AXON was initiated more than 5 years ago, but several complications thwarted the process. A new effort has been recently scheduled.
By developing data showing that PROs matter, AAN “might lead the charge” for establishing the collection of PRO data as a standard of care and eliciting reimbursement from third-party payers for doing so, McBurney said.
Nevertheless, he cautioned that validated methodology for collecting PRO data and identifying clinically meaningful changes in scores will be fundamental to PRO utility.
A Path Forward
In the best circumstance, PRO data captured at a patient visit would be analogous to a lab test. Just as blood tests generate data in the context of normative ranges for a dozen or more parameters, the PRO data could be displayed with the same type of context, allowing physicians and patients to see a specific PRO measure displayed against a normative range so results are easily interpreted, said McBurney.
But, again, there are barriers. Numerous validated sets of PROs are available with no consensus on which might serve as a standard. While Dr McBurney singled out the SymptoMScreen tool as one that is already recommended by the MS Data Alliance, a nonprofit organization supported by the European Charcot Foundation to transform real-world MS data into evidence suitable for MS care, he acknowledged it is just one of many options.
“The SymptoMScreen has been used in several clinical studies and it is relatively simple to use,” McBurney said. Even if there is no single “best” instrument for measuring PROs, a standard might move the process forward.
The president of the European Charcot Foundation, Giancarlo Comi, MD, agreed that PROs are almost certainly coming to the routine management of MS as each of the current barriers described by McBurney are addressed.
Comi said PROs are particularly important in managing progressive MS, for which he thinks that traditional biomarkers, such as brain images, are particularly poor at capturing the burden of disease.
“The EMA [European Medicines Agency] and the FDA [Food and Drug Administration] are both very interested in using PROs to evaluate treatments in MS,” he said.
PROs might be incorporated into routine care by clinicians convinced that they help in guiding treatment choices, but McBurney and Comi agree that some approach, including financial incentives, to encourage clinicians to capture and record PROs is probably needed before they are used routinely.
Dr McBurney reports no relevant financial disclosures. Dr Comi reports financial relationships with Almirall, Celgene, Genzyme, Hoffman-LaRoche, Janssen, Merck, Novartis, and Sanofi.