Multinational life sciences manufacturer
To assess the value of rare disease medicines, regulators, payers, and providers often use traditional value frameworks that quantify value in terms of measurable clinical trial endpoints, which omit qualitative and functional outcomes that may be more important to patients and their caregivers. A life sciences manufacturer sought to foster more holistic and patient-centric understanding of the total disease burden of MG by generating qualitative and quantitative data on how MG impacts the lives of patients and caregivers and how these two groups rank those effects in terms of importance.
We designed a mixed-methods study to generate a comprehensive catalog of MG impacts on patients and their caregivers, as well as to rank impacts by relevance, severity, and frequency.
The study began with structured interviews with two cohorts, one of patients and one of caregivers. We elicited information on disease impacts, including qualitative lifestyle impacts not included in traditional value assessments, and identified several impact domains omitted from existing MG literature. We then developed surveys to ask patients and caregivers how they would rank these impact domains (which we divided into more specific impact elements) in terms of relevance, severity, and frequency. The rankings enabled us to develop a weighted algorithm to determine how patients and caregivers prioritize the MG impacts identified (e.g., patients ranked financial and occupational impact domains higher than domains more traditionally measured in clinical trials and considered in product value assessments).
We then designed a validation study to test the weighted algorithm on a larger cohort of approximately 300 patients and 150 caregivers and to quantify the financial costs of the impact domains and elements.
This study generated quantitative data on qualitative aspects of patient care that traditional value assessment frameworks do not capture. These data can help the client demonstrate to regulators, payers, and providers the patient-centric value of its product, as measured by metrics that patients with MG and their caregivers prioritize.
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