OBJECTIVES: Medications that treat the motor symptoms of Parkinson’s disease (PD) do not necessarily yield short-term, clinically significant direct medical cost offsets. However, these medications may minimize other negative effects of motor dysfunction on the daily lives of patients living with PD and of their caregivers. Many of these effects are historically not well-captured in economic models of PD pharmacotherapy, although they are consistent with “Augmented CEA” and with the newest constructs included in ICER’s value assessment framework (“Other Benefits or Disadvantages” and “Contextual Considerations”). Due to the breadth of motor symptoms’ direct impact on activities of daily living, the early age of onset of PD (relative to other neurodegenerative diseases), the unrelenting progressive nature of PD, the length of time patients live with PD, and the severity of late-stage PD, we believe the capture of these effects is critical.

METHODS: We conducted a targeted literature review, exploring eight often-overlooked elements that should be consistently included in PD models of pharmacotherapy cost-utility: Patients’ early retirement; absenteeism and presenteeism among patients remaining in the labor force; caregivers’ unplanned retirement; absenteeism and presenteeism among caregivers remaining in the labor force; paid nonmedical care and home modification expenditures; proportional QALY shortfalls; and the value of hope (in treatment effectiveness), noting that the value of hope is especially relevant in a disease where high heterogeneity of treatment effects is positively correlated with disease progression.

RESULTS: A typology matching motor symptom effects with the design requirements of PD clinical trials, open-label trial extensions, and prospective observational studies followed from the literature review.

CONCLUSIONS: In this presentation, we describe the typology, its expected utility for the design of industry-sponsored studies, and its limitations.