Gene therapy offers a paradigm shift in the management of Duchenne Muscular Dystrophy (DMD), yet access remains constrained by systemic barriers including insurance denials, high treatment costs, and social determinants of health (SDOH). This presentation examines the impact of interdisciplinary collaboration among hospital social workers, specialty pharmacies, and manufacturer patient services teams in mitigating these challenges.
Social workers provide emotional support and counseling, address psychosocial needs and assess barriers to care, while Specialty pharmacies contribute expertise in therapy logistics, insurance navigation, and medication management. Manufacturer patient services teams play a pivotal role in facilitating prior authorizations, coordinating benefits, and providing financial assistance programs. Through integrated efforts—such as unified communication systems, joint patient education, and structured escalation protocols—these stakeholders collectively reduce treatment delays, improve adherence, and enhance patient and family satisfaction.
A case study illustrates successful collaboration between social workers, specialty pharmacies, and patient services which resulted in overturning an initial coverage denial for gene therapy, demonstrating how coordinated advocacy and resource alignment can overcome systemic barriers. Recommendations include implementing cross-training programs for social workers on genetic therapies and advancing policy initiatives to expand rare disease coverage.
By fostering robust partnerships and prioritizing patient-centered care, healthcare teams can transform access to life-changing therapies for children with DMD. This collaborative model not only addresses clinical and non-clinical barriers but also establishes a framework for equitable care delivery in rare diseases.