The mucopolysaccharidoses (MPS) are a group of rare genetic disorders that cause chronic, progressive cellular damage, which can affect multiple organ systems and lead to reduced life expectancy. Diagnosis and treatment of MPS are often delayed, during which irreversible organ damage can occur. Treatments are not currently available for all MPS subtypes, and those that are available are associated with significant limitations. The therapeutic pipeline for MPS is active; however, bringing new treatments for rare conditions to market is difficult. To discuss the role of managed care in improving patient care and outcomes in MPS, AMCP Market Insights virtually convened an expert panel of managed care stakeholders in September 2025. Key insights from the discussion were that the earliest possible intervention is crucial to improving outcomes in MPS; expertise is limited because of the rarity of these disorders; patients and caregivers face significant challenges accessing treatments and other interventions for MPS; and the therapeutic pipeline for MPS holds promise for addressing some critical treatment gaps but leaves others unmet. Suggested payer practices for MPS also emerged from the discussion.
Flavin et al. (Sun,) studied this question.