ABS1255-PARE INSIGHTS FROM THE ADULT DERMATOMYOSITIS EL-PFDD: CAPTURING PATIENT PERSPECTIVES TO IMPROVE GLOBAL MYOSITIS CARE

Abstract

Background:

Adult Dermatomyositis (DM) is a rare, chronic autoimmune disease characterized by muscle weakness, debilitating skin rashes, and systemic involvement, including interstitial lung disease and increased cancer risk. Limited FDA-approved therapies highlight significant unmet medical needs. To address these challenges, Myositis Support and Understanding (MSU) and The Myositis Association (TMA) convened an Externally Led Patient-Focused Drug Development (EL-PFDD) meeting in June 2024 to amplify patient and caregiver voices in therapeutic development.

Objectives:

[1] To capture the lived experiences, treatment challenges, and unmet needs of patients with adult DM. [2] To highlight patient-reported priorities for future therapeutic innovation. [3] To integrate these insights into regulatory, clinical, and international therapeutic strategies that focus on the patient to develop new drugs.

Methods:

• 1Participant Testimonies:

• ○Ten carefully selected patients with varied forms and severities of DM shared their personal testimonies during the meeting. These narratives captured the diversity of patient experiences.

• 2Panel Discussions:

• ○The testimonies were followed by moderated virtual panel discussions, led by a moderator. These sessions facilitated deeper exploration of the themes raised during the presentations and allowed for real-time engagement from the audience.

• 3Thematic Structure:

• ○The morning session focused on the symptoms and daily impact of DM. Patients shared insights into how the disease affects physical functionality, emotional health, social relationships, and quality of life.
• ○The afternoon session concentrated on current treatment approaches, clinical trial participation, and unmet needs. Topics included the limitations of existing therapies, barriers to clinical trials, and patient hopes for future treatment innovations.

• 4Surveys and Polling:

• ○A pre-meeting survey completed by 200 DM patients and caregivers captured detailed qualitative and quantitative data on symptom prevalence, treatment challenges, and therapy preferences.
• ○Live polling during the meeting enabled immediate feedback, allowing organizers to gauge the audience's priorities dynamically.

• 5Written Submissions:

• ○An open call for written comments, accepted before and after the meeting, enabled broader community participation. These submissions brought diverse perspectives from patients unable to attend the live session.

Results:

• 1Symptom Impact: Key burdens include relentless fatigue, muscle weakness, and skin-related pain, leading to disrupted daily functioning and careers. A multiplicity of skin symptoms was reported with varied severity. Involvement of organs, specially the lungs, were severe, often acute with high morbidity. Participants indicated that pain was often ignored. These findings underscored profound mental health impacts, including anxiety, depression, and social isolation.
• 2Treatment Gaps: Despite the use of corticosteroids, immunosuppressants, and intravenous immunoglobulin (IVIG), many patients reported suboptimal symptom control, significant side effects, and administration challenges. The landscape of approved therapies is starkly limited, with only one DM-specific treatment receiving FDA approval.
• 3Unmet Needs: Patients expressed an urgent need for more effective, disease-modifying treatments that address the root causes of DM rather than merely alleviating symptoms. Key desires included:

• -Personalized therapies: Tailored treatments capable of addressing the highly variable symptoms of DM, such as skin rashes, muscle weakness, calcinosis, and organ involvement.
• -Improved delivery mechanisms: Patients highlighted the preference for oral medications or simpler, less invasive delivery methods to reduce the physical and emotional toll associated with current regimens such as IVIG.
• -Minimization of side effects: There is a strong desire for therapies with reduced toxicity and fewer adverse effects, particularly for long-term management.

• 4Clinical Trial Barriers: Several barriers were identified by the participants:

• -Limited research site locations: Often located far away from patient's homes, making on-site visits difficult.
• -Restrictive exclusion Criteria: Patients often found themselves excluded due to specific symptom profiles, or pre-existing conditions. This has been referred to as the "Goldilocks Trial Dilemma," where patients' muscle/skin severity is deemed "too mild" or "too severe" to meet trial eligibility criteria.
• -Placebo risks: Being placed in a placebo group during clinical trials was a significant concern. Many feared that discontinuation of standard treatments or trial-related delays could lead to disease flares.

Conclusion:

The EL-PFDD findings provided a robust framework for addressing therapeutic gaps in adult DM management. Incorporating patient-reported priorities can catalyze innovation in international therapeutic development and clinical trial design. Future collaborative efforts should prioritize the alignment of patient-centric insights with global research and regulatory objectives. The Findings will be published in the Voice of the Patient Report as formal summary of the meeting.

REFERENCES:

[1] Voice of the Patient Report: Adult Dermatomyositis EL-PFDD,Myositis Support and Understanding (MSU), The Myositis Association (TMA), 2025 online at: https://dermatomyositis-el-pfdd.org. [2] U.S. Food and Drug Administration (FDA). Patient-Focused Drug Development: Collecting Comprehensive and Representative Input Guidance for Industry, FDA Staff, and Other Stakeholders. 2020. https://shorturl.at/3HaUs.

Acknowledgements:

We sincerely thank the patients who courageously shared their personal testimonies, the patient panelists who contributed invaluable insights, and the broader myositis community for their active participation in the meeting. Their voices and experiences are the foundation of this work and continue to drive progress toward improved care and treatment for myositis worldwide.

Disclosure of Interests:

Lynn Wilson EL-PFDD meeting was made possible with grants from Amgen, CSL Behring, J&J, Priovant, Argenx, Galapagos, Benita Moyers: None declared, Karen Cheng K.C. is an employee of Sobi since 1 Oct 2024, working on projects unrelated to this manuscript, Paula Eichenbrenner: None declared, Linda Kobert: None declared, Martha Arnold: None declared, Manuel Lubinus Vertex, Pfizer, Roche, Merck. © The Authors 2025. This abstract is an open access article published in Annals of Rheumatic Diseases under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). Neither EULAR nor the publisher make any representation as to the accuracy of the content. The authors are solely responsible for the content in their abstract including accuracy of the facts, statements, results, conclusion, citing resources etc.