Case Study

of a first-in
class breakthrough

The Question

Orphan therapies face common challenges: limited data on disease natural history, healthcare resource needs, and quality of life impact for patients.

Furthermore, orphan therapies often encounter challenges with novel endpoints designed specifically for investigational studies and high anticipated prices. A client faced the added complexity of being the first company to receive FDA approval for a first-in-class gene therapy. Our client wanted to know: how should we demonstrate the value of a therapy that’s in a class of its own?

The Approach

Medicus balanced methodological rigor and creativity to demonstrate value for this innovative therapy.

We worked closely with our client and external key opinion leaders to develop new health states that accurately reflected the impact of this degenerative disease. We then collaborated with health economic experts in the UK to quantify health-utility estimates for these patients and map to their health states. We assisted our client’s engagement with ICER, including responses to ICER’s scope and model plan, and also published a letter in Value in Health rebutting ICER’s final model. Finally, we supported our client’s marketing team with creating value messages based on our models.

The Results

Our health-economic modeling and evidence generation helped our client demonstrate the cost-effectiveness of this first-in-class gene therapy

We supported our client during their engagement with ICER, enabling them to mount a detailed response to ICER’s model and recommendation. We also provided tangible materials to help our client continue to demonstrate the value of this therapy, including a flexible cost-utility model that was adapted for ex-US health technology assessments, detailed rebuttals for payer objections, and an in-depth explanation of ICER’s findings.

Long And

Of it

Our client had a first-in-class therapy that needed innovative health economics research to match.

We led interdisciplinary efforts to develop novel, tailored approaches to demonstrate its value and justify the market access and pricing strategy.