Abstract
PurposeUnder a societal perspective, disease and treatment attributes that the general public deem important should be considered within value frameworks. The objective was to investigate how members of the general public value attributes beyond health gains and healthcare system expenditures; and better understand their perspectives regarding the importance of attributes typically characterizing rare genetic diseases like Duchenne muscular dystrophy (DMD).MethodsQualitative interviews were conducted to elicit feedback on the importance of disease and treatment attributes from general public participants from three US cities. Participants ranked attributes (scale, 1–10) in terms of importance for future research, reported their rationale for ranking, and provided feedback specific to rare diseases. Interview transcripts were coded using NVivo for thematic analysis.ResultsThe 33 participants (median age, 51 years; 48.5% male) ranked disease severity (mean [median] ranking, 8.7 [9.0]), treatment availability (8.7 [9.0]), and impact on life expectancy (8.4 [9.0]), as most important. The impact on the family, need for equity, and intrinsic value of life were frequently provided rationales. While rare disease as an attribute received a relatively low ranking (6.1 [7.0]), 88% of participants prioritized disease profiles including attributes of severity, health related quality of life (HRQoL) impact, limited lifespan and young age at onset.ConclusionAttributes including disease severity, impact on life expectancy and HRQoL, and treatment availability were all highly important to members of the general public. These findings support the growing evidence regarding the importance of expanding value assessments to include attributes considered important from a societal perspective.
Highlights
Conventional value assessment frameworks, tools developed by health economic and health professional organizations to guide priorities in healthcare provision, Szabo et al Journal of Patient-Reported Outcomes (2022) 6:9 relative to the standard of care, and incremental costs per quality-adjusted life years (QALYs) are compared to established benchmarks to assess treatment value [3]
The median age was 51 (26–77) years, 49% were male, 33% lived with children under the age of 18 years, 24% were afflicted or had a family member afflicted by a rare disease, and 30% said they were not personally affected but were familiar with rare diseases
This study provided insight into how members of the general public viewed the cumulative impact of different levels of, or combinations of, disease and treatment attributes
Summary
Conventional value assessment frameworks, tools developed by health economic and health professional organizations to guide priorities in healthcare provision, Szabo et al Journal of Patient-Reported Outcomes (2022) 6:9 relative to the standard of care, and incremental costs per QALY are compared to established benchmarks to assess treatment value [3]. In 2018, an International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Special Task Force Report identified and defined novel elements of value that have, to date, been largely unexamined within value assessment frameworks; most of these components would be relevant under a broader societal, rather than narrower health care perspective of valuation [1] These novel elements included the insurance value of new treatments, considering both physical and financial risk protection; the value of hope, which considers the impact of individual risk preferences on decision making; scientific spillover, where a new technology can benefit future innovation; and the impact of the severity of disease [1]. These and other attributes (such as disease rarity, young age at disease onset or the benefit of “buying time”) [4], may be pertinent for life-limiting, progressive and irreversible diseases with a high disease burden and poor prognosis such as Duchenne muscular dystrophy (DMD); diseases for which the impact is felt not by the patient alone, and their families and potentially other members of society [5]
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