Case Studies

Pricing & Market Access Strategy Case Study

U.S. TPP testing and pricing & market access assessment for an ultra-rare disease asset

The Situation

A multi-national pharmaceutical company was planning their Ph3 trial for an ultra-rare disease asset
They wanted to test and further refine their TPP with U.S. physicians and payers, and understand implications for market access in the U.S., as well as what the pricing potential could look like

The Ask

Test the Ph3 TPP with U.S. physicians and payers, focusing on clinical relevance of the endpoints in the indication, magnitude of effect needed, and impact on utilization and access
Assess pricing potential for the asset, given the ultra-rare disease space and limited standard of care

The Actions

1

TPP Testing and In-Depth Interviews

Gathered feedback from U.S. physicians and payers on the TPP in the context of current standard of care and precedent set by other ultra-rare disease drugs
Conducted van Westendorp price testing for the asset with payers
Created a payer weighted lives chart based on payer-reported restrictions at different list price points

2

Analog Analysis

Identified 7 analog drugs and several key metrics impacting price flexibility in the ultra-rare disease space
Assessed analog drugs along these parameters in relation to the client’s asset
Determined the analogs’ price window to help inform pricing potential for the client’s asset

The Impact

Provided physician and payer perspectives on the Ph3 TPP, including implications for trial design

Outlined what potential payer management and restrictions for the product could look like, and listed action steps to help optimize access

Recommended a list price range for the product to optimize value within the guardrails of the rare disease space

Bluestar BioAdvisors, LLC

521 Fifth Avenue, 25th Floor
New York, NY USA 10175

©2018–2025 Bluestar BioAdvisors, LLC. All rights reserved.