Survey Reveals Unmet Needs in Treatment for Rare Nerve Disorder

CSL Behring

KING OF PRUSSIA, PA — A recent survey sponsored by CSL Behring has illuminated the treatment preferences and concerns of patients living with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare autoimmune disorder damaging the peripheral nerves’ protective myelin sheath. The findings reveal a significant gap between patient preferences and the current standard of care, underscoring the need for more patient-centric treatment options.

The survey, conducted by Harris Poll, included 100 patients diagnosed with CIDP and 100 neurologists/neuromuscular specialists who regularly treat at least one CIDP patient. It found that while the majority of patients expressed interest in exploring convenient treatment options (82%) and valued at-home administration (72%), less than half reported discussing potential new treatments or switching treatments with their physician (47% and 43% respectively).

CIDP symptoms can progressively worsen, leading to substantial activity limitations. Patients’ priorities for treatment included efficacy (95%), minimal side effects (97%), condition maintenance (96%), at-home administration (72%), and flexible dosing options (70%). However, fewer than half of the surveyed patients felt very or somewhat knowledgeable about how different CIDP treatment options work (40%) or are administered (45%).

Dr. Jeffrey Allen, MD, Associate Professor of Neurology at the University of Minnesota Medical School, emphasized the importance of shared decision-making in treating rare diseases like CIDP. Regular discussions about treatment options, including self-administered treatments at home, are crucial for patients who may find travel to an infusion center challenging or burdensome.

CSL Behring’s Hizentra® is the only self-administered subcutaneous immunoglobulin (SCIg) approved for maintaining CIDP therapy to prevent neuromuscular disability and impairment relapse. Administered under the skin instead of intravenously, Hizentra allows patients to self-manage their treatment at home, a significant advantage for those with venous access issues.

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Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring, reiterated the company’s commitment to addressing unmet needs in rare disease treatment. He highlighted CSL Behring’s efforts to improve the treatment experience by offering Hizentra in various prefilled syringe sizes, including a soon-to-be-available 10g size.

The survey findings underscore the need for patient-centric treatment approaches in managing CIDP. The insights gained could guide biotech firms in developing treatments that align more closely with patient preferences, potentially improving adherence and outcomes. As such, these results could be of significant interest to investors tracking developments in the treatment of rare diseases.

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