# Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient‐Centered Approach

## TL;DR

This study explores how patients with lysosomal storage diseases view their treatments, revealing differences that reflect unmet medical needs and the potential for more patient-centered care.

## Contribution

The study applies the Beliefs in Medicine Questionnaire to lysosomal storage diseases, revealing variability in treatment beliefs across patient groups.

## Key findings

- Gaucher disease patients mostly viewed their treatment as necessary with low concern.
- Fabry disease patients showed varied beliefs, with females more skeptical and males more ambivalent.
- Mucopolysaccharidosis III patients were indifferent, reflecting low perceived necessity and concern.

## Abstract

Despite life‐long pharmacotherapy for many people affected by lysosomal storage diseases, no data are available on their beliefs about their treatments. Therapeutic options range from disease‐specific, with varying levels of effectiveness, to purely supportive. This spectrum is illustrated by the three diseases Gaucher disease type 1 (effective disease‐specific therapies), Fabry disease (disease‐specific therapies with variable effectiveness), and mucopolysaccharidosis type III A/B (supportive care only). Employing the Necessity‐Concerns Framework of the Beliefs in Medicine Questionnaire, we investigated intra‐ and intergroup variability in adults with Gaucher disease type 1, Fabry disease, and parents of children with mucopolysaccharidosis type III A/B. Participants rated necessity and concern items on a Likert scale, leading to categorization as accepting, skeptical, indifferent, or ambivalent. Self‐reported demographic, disease‐, and therapy‐related data were also obtained. Eighty‐one surveys were completed. Gaucher disease respondents (n = 15) were overwhelmingly categorized as accepting (high necessity, low concern). Female Fabry disease respondents (n = 43) were almost equally distributed over all categories except accepting. Male Fabry disease respondents (n = 16) were mostly ambivalent or accepting, indicating overall high necessity scores but varying concern. All mucopolysaccharidosis type III participants (n = 7) were categorized as indifferent (low necessity, low concern). The Beliefs in Medicine Questionnaire emerged as a valuable and feasibly employable tool for individual and group assessments in these populations. It reveals differences in beliefs aligned with current unmet medical needs. Expansion of this approach is warranted to optimize personalized counseling on therapeutic choices and to align drug development with the needs and beliefs of potential recipients.