OP08 A Comprehensive Collaborative Framework To Enhance Orphan Drug Accessibility For Rare Diseases - Insights From Türkiye

Sep 29, 2026

Uploaded by Theodore

Restricted access to orphan drugs significantly limits outcomes and quality of life for rare disease patients in Türkiye. The study evaluates systemic barriers through stakeholder perspectives, addressing regulatory inefficiencies, economic constraints, and inadequate collaboration among key actors. A qualitative design used focus groups with patients and caregivers and in-depth interviews with healthcare providers, policymakers, industry, and regulators, followed by thematic analysis and grounded theory synthesis. The proposed framework targets centralized regulatory mechanisms, public-private partnerships, and improved communication, supported by stakeholder-validated adaptability.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages1

Reading Time3 min

AccessFree to download

Introduction

Methods

Results

Conclusions

OP07 Shared Learning: An Alternative Model For Patient Capacity Development To Participate In Health Technology Assessment Kris Pierce ([krispconsulting@gmail.com](krispconsulting@gmail.com)), Ann Single, Melanie Funk and Karen Van Gorp Introduction: The Shared Learning model redefines patient engagement in health technology assessment (HTA) by combining immersion, knowledge sharing, and network expansion. Through flexible programs like Patient Voice Initiative (PVI) Patient Partners, itempowers advocates to deepen their understanding of HTA processes and disseminate insights broadly. This innovative, scalable approach ensures patient voices are central to decision-making, fostering informed contributions and addressing the challenges of rapidly evolving health technologies. Methods: The Shared Learning model piloted by the PVI supported three patient advocates through a structured immersion in HTA processes. Advocates engaged directly in HTA discussions, meetings, and consultations, attending the HTAi Annual Meeting 2024 for deeper insights and networking. The model emphasized peer knowledge dissemination through webinars, workshops, and community forums, creating a ripple effect of shared learning. Advocates reflected on their experiences, identifying insights to benefit broader patient networks. This approach combined immersive learning, knowledge-building, and capacity expansion, fostering both vertical engagement with HTA stakeholders and horizontal knowledgesharing within patient communities. Results: The Shared Learning pilot demonstrated success in enhancing patient engagement within HTA. Advocates deepened their understanding of HTA processes through direct participation and shared insights with wider patient networks, fostering a multiplier effect. Peer-to-peer knowledge dissemination expanded patient capacity, strengthening advocacy communities. Advocates utilized their experiences to host webinars, workshops, and community forums, broadening the reach of HTA knowledge. The initiative also improved the ability of patient networks to contribute meaningfully to decision-making processes. These outcomes highlight the model’s potential for creating sustainable, informed, and resilient advocacy communities equipped to address the challenges of rapidly evolving health technologies. Conclusions: The Shared Learning model revolutionized patient engagement in HTA by emphasizing immersion, knowledge sharing, and network expansion. Through flexible programs like PVI Patient Partners, the model deepened engagement while enabling broader dissemination of insights. As health technologies rapidly evolve, this scalable approach ensures patient voices remain central to HTA decision-making, fostering informed, sustainable contributions within patient communities. OP08 A Comprehensive Collaborative Framework To Enhance Orphan Drug Accessibility For Rare Diseases: Insights From Türkiye Birol Tibet ([drbiroltibet@gmail.com](drbiroltibet@gmail.com)), Selma Karabey, Ayse Emel Onal and Ugur Ozbek Introduction: Restricted access to orphan drugs significantly challenges rare disease patients, impairing health outcomes and quality of life. In Türkiye, systemic barriers such as regulatory inefficiencies, economic limitations, and inadequate stakeholder collaboration persist. This study evaluated these issues through stakeholder perspectives, proposing a collaborative framework to improve access, ensure equity, and enhance systemic efficiency in addressing rare disease challenges in Türkiye. Methods: This qualitative study employed semi-structured focus group discussions with 19 patients and caregivers and 13 in-depth interviews with healthcare providers, policymakers, industry representatives, and regulatory bodies. Thematic analysis of 1,996 coded excerpts identified key barriers to orphan drug access, categorized as director indirect impacts. Grounded theory was applied to synthesize these findings into a cohesive framewo

FAQs

What problem does the study address regarding orphan drugs in Türkiye?

The study examines how restricted access to orphan drugs challenges rare disease patients, driven by regulatory inefficiencies, economic limitations, and insufficient stakeholder collaboration.

How was the research conducted and analyzed?

It used semi-structured focus group discussions with patients and caregivers and in-depth interviews with healthcare providers, policymakers, industry representatives, and regulatory bodies, then applied thematic analysis and grounded theory. Data triangulation and iterative stakeholder feedback supported validity and practicality.

What does the proposed framework include to improve orphan drug accessibility?

The framework emphasizes centralized regulatory mechanisms to streamline approvals, public-private partnerships to reduce financial constraints, and enhanced communication to support transparency and accountability. It also defines success metrics for future implementation and continuous improvement.

Sep 29, 2026

Uploaded by Theodore