When Orphan Disease Value Was Difficult to Demonstrate, We Built a Defensible Narrative.

CASE STUDY | PAYER COMMUNICATIONS

When Orphan Disease Value Was Difficult to Demonstrate, We Built a Defensible Narrative.

THE CHALLENGE

Demonstrating Value in a Small, Complex Rare Disease Population

A pharmaceutical company sought to shape the scientific and value story for a novel orphan disease product in a rare genetic disorder of copper transport and metabolism.

With a small patient population of only approximately 3,000 diagnosed individuals in the United States, it was challenging to demonstrate the economic burden of disease and the potential value and cost impact of the treatment to payers.

THE BREAKTHROUGH

Translating Limited and Fragmented Data into a Defensible Value Narrative

Using our Breakthrough Process, powered by 3D Science, BGBx identified that the challenge was not a lack of data, but the need to integrate and translate fragmented scientific evidence into a cohesive and credible value story.

The strategy centered on building a unified scientific and value foundation, leveraging both published data and real-world insights to clearly articulate disease burden, treatment impact, and economic considerations for payer decision-making.

The Solution

Scientific platform development to synthesize available evidence
Centers of Excellence data integration to demonstrate real-world relevance
Payer value proposition development to communicate disease burden and treatment impact
AMCP dossier development to support payer evaluation and reimbursement decision-making

Perspective

“By integrating fragmented evidence and Centers of Excellence data into a single, defensible narrative, we gave payers the clarity needed to evaluate value in a complex, low-prevalence disease.”

— BGBx Market Access Lead

THE RESULTS

Established a Defensible Value Narrative to Support Coverage and Reimbursement Decisions

The integrated scientific and value platform enabled a clear, structured narrative to support payer evaluation and reimbursement discussions. Scientific and value evidence were consolidated into a single platform, Centers of Excellence data extended the narrative beyond published evidence, and comprehensive materials were developed to support payer review.

~3,000
Diagnosed Patients

Rare disease population context

COE
Data Integration

Real-world evidence support

AMCP
Dossier Developed

Coverage discussion support

Let’s Build Your Next Market Access Breakthrough

At BGBx, we develop data-driven scientific and value platforms that integrate real-world insights, helping translate complex evidence into clear, credible narratives that support access, reimbursement, and real-world impact.

All