Meeting the care coordination needs of complex therapies for rare neuromuscular/neuro-genetic disorders: the development of a complex drug program.


Clinical Management

Poster Number: 13


Taylor Schwab, RN, Children's Hospital Colorado, Kaitlin Haug, RN, Christine Caneva, BS, Children's Hospital Colorado, Jennifer Coffman, DNP, CPNP-AP, Stefanie Leonard, MSN, RN, Children's Hospital Colorado, Melissa Gibbons, MS, CGC, University of Colorado/Children's Hospital Colorado, Scott Demarest, MD, Julie Parson, MD, Children's Hospital Colorado, Aurora, CO, 80045 USA

Authors: Taylor Schwab, RN, Kaitlin Haug, RN, Christine Caneva, BS, Jennifer Coffman, DNP, CPNP-AP, Stefanie Leonard, MSN, RN, Melissa Gibbons, MS, CGC, Scott Demarest, MD, Julie Parsons, MD

Background: Since 2016, the FDA has approved three novel therapies for Spinal Muscular Atrophy (SMA), four for Duchenne Muscular Dystrophy (DMD), and others for previously untreatable rare neuro-genetic conditions. The coordination for therapies requiring implanted ports, strict handling precautions, and/or routine intrathecal injections, all with unprecedented costs, is both clinically and administratively complex. Considerations such as subspecialty consultation, patient education and compliance, supply procurement, insurance authorization, scheduling, and follow up monitoring require specific expertise.

Objective: Create a team of doctors, advanced practice providers, nurses, and administrative professionals dedicated to ensuring patients with rare neuromuscular and neuro-genetic conditions have timely access to high cost, complex therapies with minimal complications.

Methods: In 2016, the complex drug team of two lead physicians, one registered nurse, and one administrative professional was assembled. The program has navigated the onboarding of new therapies and increasing patient counts by creating standardized clinical pathways, order templates, expectation agreements with families, consistent clinical-team training, financial team liaisons to assist with single case payor agreements, predictable outreach with manufacturers, and connections with necessary departments and subspecialties to ensure all care is delivered in a timely manner. Guidelines have been developed to determine if a medication is appropriate for the complex drugs program.

Results: This specialized program has successfully treated 50 patients with nusinersen, 12 patients with onasemnogene abeparvovec, 14 patients with risdiplam, 4 patients with cerliponase alfa for CLN2 Battens disease, and 8 patients with exon skipping therapies for DMD. SMA patients receive therapy, on average, 26 days after initial newborn screening identification. Of 540 nusinersen doses, only 6 (1.1%) experienced complications beyond a post-LP headache and over 96% have received doses within their treatment window.

Conclusion: A specialized clinical and administrative team is beneficial in ensuring timely treatment with complex, novel therapies with minimal complications.