Framework for Patient Experience Value Elements in Rare Disease: A Case Study Demonstrating the Applicability of Combined Qualitative and Quantitative Methods

Abstract

Background and Objective Several novel methods have been suggested to extend a conventional value assessment to capture a more comprehensive perspective of value from a patient perspective. The objective of this research was to demonstrate a framework for implementing a combined qualitative and quantitative method to elicit and prioritize patient experience value elements in rare diseases. Neuromyelitis optica spectrum disorder was used as a case study. Methods The method for eliciting and prioritizing patient experience value elements involved a three-step process: (1) collecting potential patient experience value elements from existing literature sources followed by deliberation by a multistakeholder research team; (2) a pre-workshop webinar and survey to identify additional patient-reported value elements; and (3) a workshop to discuss, prioritize the value elements using a swing weighting method. Outcomes were prioritized value elements with normalized weights for patients considering a treatment for neuromyelitis optica spectrum disorder. Results A literature review and deliberation resulted in the following initial value elements: ability to reach important personal milestones, patient's financial burden, value of hope/balance or timing of risks and benefits, Uncertainty about long-term benefits and safety of the treatment, Patient empowerment through therapeutic advancement and technology, Caregiver/family's financial burden, patient experience related to treatment regimen, Therapeutic options, and Caregiver/ family's quality of life. Eight patients with neuromyelitis optica spectrum disorder participated in the case study. In the online survey, participants found the nine proposed patient experience value elements both understandable and important with no additions. During the workshop, 'Uncertainty about long-term benefits and safety,' 'Patient experience related to treatment regimen,' and 'Patient's financial burden' were found to be the most important patient experience value elements, with a respective weight of 25%, 19.2%, and 14.4% (out of total 100%). Conclusions This case study provides a framework for eliciting and prioritizing patient experience value elements using direct patient input. Although elements/weights may differ by disease, and even in neuromyelitis optica spectrum disorder, additional research is needed, value frameworks, researchers, and manufacturers can use this practical method to generate patient experience value elements and evaluate their impact on treatment selection.

Navigating the unknown: how to best ‘reflect’ standard of care in indications without a dedicated treatment pathway in health technology assessment submissions

“…Innovative study designs incorporating RWE collection (adaptive study designs, basket trials) or before-and-after study designs focusing on meaningful patient-relevant outcomes may overcome some of the challenges of comparative clinical and economic assessments when there is no established SoC for the targeted condition [ 53 , 54 ]. Existing frameworks such as the use of a meaningful, valid, expedited, and transparent evidence framework [ 55 ], the US FDA's Sentinel Initiative [ 56 ], and value-based frameworks specific for rare and very rare diseases [ 57 ], as well as existing analytical methodologies proposed by international initiatives (such as GetReal), can guide some of the decisions on evaluating clinical estimates between the new technology under assessment and what patients most likely would have experienced in clinical practice if there was SoC (i.e., using weighting approaches) [ 58 ].…”

Development of the Emirates Multi-Criteria Decision Analysis Tool for Orphan Drugs

“…Traditional value assessment frameworks may not capture all the benefits of orphan drugs, as they focus on health gain versus the incremental cost and neglect other benefits, such as response to an unmet medical need without alternative treatment options, or promoting equity [15,16]. Adding these criteria to evaluation frameworks should provide orphan drugs with a fair chance of proving their true societal value [16,17].…”