Contextualizing Gene Therapy Educational Resources For Hemophilia Patients In Brazil - Translation Process And Preliminary Qualitative Patient Preferences Findings

Sep 29, 2026

Uploaded by Aria

This work addresses culturally attuned health-education needs for hemophilia gene therapy decision-making within a health technology assessment context. A mixed-methods contextualization approach translated the PAVING educational tool into Brazilian Portuguese, followed by expert review and pilot testing with people with hemophilia. Results from a virtual presentation to 12 participants showed minor local adaptations, mobile-phone usability revisions, and measured preferences across ranked treatment attributes, alongside generally positive willingness to receive gene therapy.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages1

Reading Time3 min

AccessFree to download

Introduction

Methods

Results

Conclusions

PP29 Contextualizing Gene Therapy Educational Resources For Hemophilia Patients In Brazil: Translation Process And Preliminary Qualitative Patient Preferences Findings Ana Dantas, Joana Balardin, Carla Fabrine Carvalho, Mariana Alves, Rafael Feitoza, Tania Maria Tania Maria Onzi Pietrobelli, Gabriela Yamaguti-Hayakawa and Daniela Pachito ([daniela.pachito@pfizer.com](daniela.pachito@pfizer.com)) Introduction: Decision-making in the context of health technology assessment (HTA) of gene therapies for hemophilia needs tobe tailored toward patients’ preferences. To promote equitable and patient-centered decision-making, it is crucial to provide patients with culturally attuned educational materials to ensure comprehension of key aspects of gene therapy so they can make legitimate and informed treatment decisions based on their preferences. Methods: In this mixed methods study, we used a contextualization approach to translate the educational tool from the PAVING study (Patient Preferences to Assess Value IN Gene Therapies in Hemophilia) into Brazilian Portuguese. Translated versions were reviewed by a hematologist, a patient association representative, and two industry professionals. The tool was subsequently piloted with two people with hemophilia (PWH) . After revisions, the tool was virtually presented to 12 PWH to transfer knowledge in preparation for the interview about their willingness to receive gene therapy. They were also required to perform the attribute-ranking exercise from the PAVING study. Results: The translation process led to minor adaptations required for local context. The original version designed for computer use was adapted for use on a mobile phone. Pilot testing with PWH resulted in minor changes to optimize clickstreams. Ten participants indicated some baseline awareness of gene therapy for hemophilia (good n=2; reasonable n=3; bad or very bad n=5) . Participants indicated a general willingness to receive gene therapy (very willing n=7; willing n=2; neutral n=3) . The six top-ranked treatment attributes were“effect on factor level,” “dose frequency,” “impact on daily life,”“probability that prophylaxis can be stopped after treatment,” and“effect on annual bleeding rate.” Conclusions: These findings may contribute to further developments of the design and contextualization of hemophilia educational resources of gene therapy. The preliminary results on patient preferences will also inform future quantitative preference studies to be considered in the decision-making related to individual care and to reimbursement and coverage decisions in health systems, thus incorporating the patient perspective in HTA processes. PP30 A Costing Template For The Implementation Of A Public And Patient Advisory Board For Health Research In Dementia Paola Vasquez ([paolandreavasquezv@gmail.com](paolandreavasquezv@gmail.com)), Daniel X Bailey and Paul Prudon Introduction: Patient and public involvement (PPI) is becoming increasingly embedded in research. While financial constraints are cited as barriers to PPI, there is a lack of understanding of its financial affordability and factors influencing costs. This work aimed to:(i) clarify PPI board costs; (ii) find cost-saving opportunities; and (iii) provide a cost template for others to implement a PPI board. Methods: A cost analysis was conducted to identify the financial capital and staffing hours required to establish and maintain a PPI board. This analysis involved the following steps: (i) identifying the stages ofthe project; (ii) outlining the activities associated with each stage; (iii) establishing the time frame for each stage and its activities;(iv) identifying the necessary positions; and (v) calculating the associated costs. Results: The board’s design, implementation, one-year operation, and evaluation cost AUD150,229 (EUR84,076.41) (43% for design, 21% for implementation, 19% for operation, and 17% for evaluation) . Although the distribution of

FAQs

Why was culturally attuned educational material needed for hemophilia gene therapy decisions?

To support equitable, patient-centered decision-making by improving comprehension of key gene therapy aspects so patients can make legitimate and informed treatment decisions based on their preferences.

How was the educational tool translated and prepared for Brazilian Portuguese use?

The PAVING tool was translated into Brazilian Portuguese, reviewed by a hematologist, a patient association representative, and industry professionals, then piloted with people with hemophilia and revised before virtual presentation.

What did participants most value about treatment attributes?

The highest-ranked attributes included effect on factor level, dose frequency, impact on daily life, the probability prophylaxis can be stopped after treatment, and effect on annual bleeding rate.

Sep 29, 2026

Uploaded by Aria