Disease- vs Symptom-Modifying
From the Desk of Dr. Danielle Meadows
Vice President of Research Programs & Operations
In general, OMF’s research falls into one of three buckets: identifying underlying mechanisms, enabling accurate diagnosis, and finding effective treatments. But there are a couple ways in which a treatment could be considered “effective”, so this month, I want to talk about the difference between disease-modifying and symptom-modifying treatments. Both can be considered effective treatments, but they do different things.
Disease- vs symptom-modifying treatments
Symptom-modifying treatments reduce the burden of specific symptoms (e.g., cognitive dysfunction, pain, unrefreshing sleep). Disease-modifying treatments target the core mechanisms driving an illness, so they impact pathophysiology rather than just providing symptomatic relief.
How it applies in ME/CFS and Long COVID
As multi-system chronic complex diseases that lack a clear understanding of their underlying mechanism (or mechanisms), the majority of clinical care for ME/CFS and Long COVID focuses on symptom management. Research on these diseases often tries to identify treatable biological pathways, which may move the needle towards disease-modifying treatments. Translation of this type of research takes time, however, so disease-modifying approaches and symptom-modifying approaches—which may improve quality of life more quickly—both have value in ME/CFS and Long COVID.
OMF’s vision is a world where ME/CFS, Long COVID, and related diseases are understood, rapidly diagnosed, and effectively treated—so every patient can live fully. Finding disease-modifying treatments is a major part of that vision, but we also want to make people better faster. Therefore, through our extensive research portfolio, we are simultaneously pursuing identifying disease- and symptom-modifying treatments.
Impact on Study Design
Whether a study is exploring disease- or symptom-modifying treatments can have an impact on study design (the first stage of the research process) through the selection of outcome measures—how an effect is measured.
Symptom-modifying trials may prioritize patient-reported outcomes (e.g., surveys) to fully and accurately understand specific symptoms. Disease-modifying trials should pair clinical outcomes (e.g., function, post-exertional malaise, quality of life) with objective and mechanistic measures (e.g., autonomic testing, activity data, cardiopulmonary exercise testing) to demonstrate impact on underlying biology.
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