**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Agnes Farrugia, Amyloidosis Alliance, Marseille, France

**Main Body of the Abstract**
Background: Early diagnosis is a key determinant of patient outcomes in amyloidosis. Yet, as with many adult-onset rare diseases (AO-RDs), diagnostic delays are common, often driven by low healthcare professional (HCP) awareness and misattribution of symptoms to ageing. The COLLABORATE initiative convened a global multistakeholder expert group to understand challenges and opportunities in AO-RD.
Objective: To present diagnosis-related challenges and opportunities in AO-RD from COLLABORATE.
Materials & Methods: Three challenge areas were identified at the first-of-its-kind, multistakeholder COLLABORATE I summit in 2022: diagnosis, access, and care coordination. Three working groups then developed solutions for each. At COLLABORATE II in 2023, 45 patient advocates and 11 HCPs (18 with amyloidosis expertise) from 31 countries voted on the significance of challenges in their nation. 29 participants completed a survey providing policy insights from represented nations. Outcomes from both summits informed the development of opportunities for change.
Results: COLLABORATE II attendees overwhelmingly voted that limited HCP/patient awareness, low health-related patient self-worth, ageism, and stigma contribute to delayed AO-RD diagnosis. Limited HCP expertise and training represent a particular unmet need in amyloidosis. Opportunities include awareness campaigns targeting HCPs, medical students, and the public, integration of AO-RDs into national policies, and a patient rights charter. Working group outputs include a self-advocation toolkit, multistakeholder collaboration guidance, and an amyloidosis-specific HCP checklist.
Summary & Conclusions: Timely diagnosis is critical to survival and quality of life in amyloidosis. The COLLABORATE initiative identified key challenges and opportunities for diagnosis in AO-RDs, including amyloidosis.
Support & Funding: Pfizer sponsored COLLABORATE.



**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Agnes Farrugia, Amyloidosis Alliance, Marseille, France

**Main Body of the Abstract**
**Background:** Early diagnosis is a key determinant of patient outcomes in amyloidosis. Yet, as with many adult-onset rare diseases (AO-RDs), diagnostic delays are common, often driven by low healthcare professional (HCP) awareness and misattribution of symptoms to ageing. The COLLABORATE initiative convened a global multistakeholder expert group to understand challenges and opportunities in AO-RD.
**Objective:** To present diagnosis-related challenges and opportunities in AO-RD from COLLABORATE.
**Materials & Methods:** Three challenge areas were identified at the first-of-its-kind, multistakeholder COLLABORATE I summit in 2022: diagnosis, access, and care coordination. Three working groups then developed solutions for each. At COLLABORATE II in 2023, 45 patient advocates and 11 HCPs (18 with amyloidosis expertise) from 31 countries voted on the significance of challenges in their nation. 29 participants completed a survey providing policy insights from represented nations. Outcomes from both summits informed the development of opportunities for change.
**Results:** COLLABORATE II attendees overwhelmingly voted that limited HCP/patient awareness, low health-related patient self-worth, ageism, and stigma contribute to delayed AO-RD diagnosis. Limited HCP expertise and training represent a particular unmet need in amyloidosis. Opportunities include awareness campaigns targeting HCPs, medical students, and the public, integration of AO-RDs into national policies, and a patient rights charter. Working group outputs include a self-advocation toolkit, multistakeholder collaboration guidance, and an amyloidosis-specific HCP checklist.
**Summary & Conclusions:** Timely diagnosis is critical to survival and quality of life in amyloidosis. The COLLABORATE initiative identified key challenges and opportunities for diagnosis in AO-RDs, including amyloidosis.
**Support & Funding:** Pfizer sponsored COLLABORATE.



Multistakeholder perspectives on challenges and solutions in the diagnosis of adult-onset rare disease: the amyloidosis paradigm PB72 (#232)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
This program will provide a comprehensive review of state-of-the-art education in systemic amyloidosis: basic science, pathophysiology, diagnosis, current and emerging treatments. Scientific presentations will include competitive oral and poster abstracts. The rapid evolution of scientific discovery and expanding treatment options has created tremendous interest in the field and this meeting will fill and important gap in educating scientists and clinicians.