Background

Generalized Myasthenia Gravis (gMG) is a rare, chronic autoimmune neuromuscular disorder that leads to muscle weakness and significant impairment in daily activities. The condition arises due to autoantibodies disrupting communication between nerves and muscles, leading to fluctuating muscle fatigue that can impact mobility, breathing, and overall quality of life. While recent advances in targeted therapies have improved treatment outcomes, challenges remain in addressing variability in response, long-term disease management, and accessibility to novel treatments across different healthcare systems.

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Challenge

The client sought to position a novel therapy for gMG within a highly specialised and evolving treatment landscape. A primary challenge was demonstrating the therapy’s clinical and economic value amidst the availability of alternative targeted treatments and established off-label options. Regulatory agencies and health technology assessment (HTA) bodies required rigorous indirect treatment comparisons and cost-effectiveness models to assess the therapy’s place within the existing treatment paradigm, particularly in patients with treatment-resistant disease.

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Approach

A global advisory board was convened, consisting of 8 leading health economists, HTA experts, and clinical specialists in IBD. The process included:​​

• A pre-advisory board webinar providing an overview of the therapy’s Phase 3 clinical trial results

• Two half-day discussions focused on market access strategy, clinical differentiation, and economic modeling approaches

• Analysis of patient-relevant endpoints and their incorporation into cost-effectiveness models

• Expert review of indirect treatment comparison methodologies to assess positioning relative to existing therapies

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Outcomes

The advisory board discussions provided valuable insights into the challenges and opportunities for positioning the therapy within the competitive IBD treatment landscape. Experts highlighted key considerations related to clinical differentiation, economic modelling, and HTA agencies' expectations in light of evolving market dynamics.

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The client gained a deeper understanding of the factors that would be most relevant to patients, prescribers, and HTA agencies in different markets, enabling them to refine their approach to evidence generation, economic modelling, and payer engagement, and to strengthen their global market access strategy.