Launching a treatment for inflammatory diseases can be a make-or-break endeavor. A robust evidence base and a compelling value story are crucial to success, but current challenges and unexpected bumps in the road will require creative solutions.

Here we describe three case study examples of common issues faced in the inflammatory disease space and how these challenges were overcome using novel solutions, ultimately leading sponsors to submission success.

The crowded marketplace

In this first example, a manufacturer was gearing up to launch a new treatment promising best-in-class efficacy. However, the market was saturated with existing options, from low-cost biosimilars to novel treatments with strong clinical results. Comparative efficacy evidence was crucial, but few head-to-head trials were available. The manufacturer’s trial was a placebo-controlled randomized controlled trial (RCT), making it reliant on indirect treatment comparisons (ITCs). Payers had previously criticized the uncertainty of ITC results due to differences in trial designs and outcomes.

The Cytel team carried out an in-depth evidence review and gap analysis, highlighting the key clinical evidence, ITC methodologies, and payer critiques from previous health technology assessment (HTA) submissions. At feasibility assessment, Cytel presented a comprehensive evaluation of various approaches and a plan for robust base case and scenario analyses to inform comparative efficacy estimates and the cost-effectiveness assessment.

This meticulous approach ensured the most robust approaches were applied throughout, streamlining the project and ensuring efficient delivery of results, without the need to revisit, revise, or re-analyze evidence.

The rare disease indication

In our next example, a manufacturer was developing global value materials, including early cost-effectiveness evidence, for a novel therapy in a rare disease with limited treatment options. The clinical guidelines were new and based on scant evidence, and treatment approaches varied widely across countries and even clinics. There were no registries for long-term data and a few small cohort studies formed the entire evidence base. Clinical trials were inconsistent, lacking a unified outcome measure.

The Cytel team started with a targeted review of the existing literature, followed by a gap analysis to determine the evidence base and evidence needs in key areas including epidemiology, disease natural history, treatment pathway, and clinical evidence. We conducted interviews with key opinion leaders (KOLs) to uncover additional evidence sources and elicit expectations for crucial data points.

Combining the available evidence, we built a flexible cost-effectiveness model that could adapt as the evidence base grew. We used probabilistic sampling to address gaps in the patchy evidence base, enabling us to generate economic forecasts. This model helped pinpoint key drivers of cost-effectiveness, prioritize evidence-generation activities, and perform exploratory analyses to understand the potential value landscape under various scenarios.

Leveraging real-world evidence

For our final example, a manufacturer was preparing to launch an intervention in a new, rare condition. Although there were no approved interventions for this indication, many physicians still prescribed off-label comparators.

In order to understand the indication, its epidemiology, natural history, and treatment landscape, Cytel undertook an early value assessment. This comprehensive assessment comprised targeted and systematic reviews to identify the evidence base, including the epidemiological, economic, healthcare resource use, and clinical efficacy and effectiveness specific to this indication.

To understand the comparative efficacy, few RCTs were available in this rare condition. Given the off-label use of several comparators, Cytel included a combination of RCTs, single-arm clinical trials, and retrospective or prospective observational studies to inform their indirect treatment comparison. Due to the heterogeneity in trial designs, careful attention to treatment duration, population descriptions, and outcome definitions were recorded and aligned before model development.

The comprehensive early value assessment and flexible yet robust approach to understanding the comparative treatment landscape gave the client the optimal view of their asset within the market.

Key takeaways

Successfully launching a treatment for inflammatory diseases requires navigating a complex and competitive landscape. These case studies illustrate some of the diverse challenges sponsors face in the inflammatory disease space and how Cytel uses creative solutions to overcome them. Tailored strategies, innovative methodologies, and a thorough approach ensure robust evidence and compelling value stories, paving the way for a successful launch.

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