OP33 The Voice Of Patients And Caregivers - Social Impact Of Rare Diseases In Health Technology Assessment In Ecuador - Study report

Sep 29, 2026

Rare diseases create major barriers to timely treatment and fair health policy, yet patients and caregivers often lack representation in health technology assessment (HTA) in Ecuador. This study amplifies their voices to understand lived experiences and to build inclusive, more equitable HTA methodologies. Using a mixed-methods design, it integrates interviews, surveys, and observation with input from patient organizations, combining thematic coding with quantitative assessment of access, emotional impact, and socioeconomic burden.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages1

Reading Time3 min

AccessFree to download

Introduction

Methods

Results

Conclusions

OP33 The Voice Of Patients And Caregivers: Social Impact Of Rare Diseases In Health Technology Assessment In Ecuador Veronica Simbaña ([veronicasguzman@hotmail.es](veronicasguzman@hotmail.es)) Introduction: Rare diseases, including Gaucher disease and spinal muscular atrophy, impose significant challenges in access to treatments and equitable healthcare policies. In Ecuador, patients and caregivers lack representation in health technology assessment (HTA) processes. This study aimed to amplify their voices, exploring their experiences to develop inclusive methodologies that promote equity and transparency in healthcare decisionmaking. Methods: This study employed a mixed-methods approach combining qualitative and quantitative data comprising perspectives of patients with Gaucher disease and spinal muscular atrophy (as well as the perspectives of their caregivers), in-depth interviews, surveys, and observation. Collaboration occurred with patient organizations. Qualitative analysis focused on thematic coding of experiences, while quantitative surveys assessed access to treatment and socioeconomic burdens. Data triangulation ensured robust findings. Results: Preliminary results indicated significant challenges in equitable access to treatments for rare diseases in Ecuador. Patients and caregivers reported high emotional and financial burdens, with limited systemic support. Early findings suggested disparities in treatment availability and awareness between urban and rural settings. Engagement workshops demonstrated a strong willingness among stakeholders to participate in developing inclusive HTA processes. Conclusions: Preliminary findings highlighted the urgent need for inclusive HTA processes in Ecuador. Amplifying patient and caregiver voices can improve equity in rare disease management and inform policy changes. Future research will focus on refining methodologies for participatory HTA and addressing systemic gaps in access to treatment, with emphasis on rural and vulnerable populations. OP34 Methodological Approach For Synthesizing Public Consultations To Inform Decision-Making In Health Technology Incorporation in Brazil: A Delphi study Viviane Karoline da Silva Carvalho (vivi_unb@hotmail. com), Jorge Otavio Maia Barreto and Everton Nunes da Silva Introduction: Public consultations (PCs) are widely used by health technology assessment (HTA) agencies, but there is no consensus on methodological procedures for analyzing PCs. We propose a method for analyzing and synthesizing PCs, offering an agile approach, systematic and transparent steps, reproducible aspects, and reliable analyses. The aim of this study was to validate the method. Methods: The Delphi technique was used to validate the proposed method in terms of face (applicability and relevance) and content validity (analysis results) . The setting included the HTA processes conducted by the National Committee for Health Technology Incorporation (CONITEC) . A group of 20 Brazilian experts was invited to participate, each of whom had experience in at least oneof the following areas: HTA, social participation, IRaMuTeQ software, or qualitative research. Consensus was defined as at least 70 percent with “agree” or “strongly agree” responses or an interquartile range of one or less. Comments were summarized and categorized thematically. A pilot study was also conducted. Results: Fifteen experts participated in the face validity assessment and 14 in the content validity assessment. Both validations achieved over 80 percent consensus in the first round. In face validity, five methodological steps were validated, including corpus analysis, corpus preparation and organization, data mining using IRaMuTeQ software, systematization and interpretation, and synthesis of findings. In content validity, the results were deemed adequate, providing sufficient information to understand PCs. Although this method was used in the Brazilian HTA setting, it can be adapted

FAQs

What problem does the study address in Ecuador's HTA process?

Patients and caregivers are not adequately represented in HTA processes for rare diseases, limiting inclusive and equitable decision-making.

How was the study conducted?

It used a mixed-methods approach combining interviews, surveys, and observation, drawing perspectives from patients with Gaucher disease and spinal muscular atrophy and from their caregivers, with collaboration from patient organizations.

What did preliminary results suggest about access to treatments?

They indicated significant challenges in equitable access, with high emotional and financial burdens and limited systemic support, including disparities between urban and rural settings.

Sep 29, 2026

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