Optimizing a Clinical Program for an Ultra-Rare Treatment

A biopharmaceutical manufacturer sought to validate its clinical program design and test minimum acceptable and ideal product profiles of an asset in early stages of development with a potential ultra-rare neurodegenerative indication affecting children and adult males. Given the heterogeneity of phenotypes associated with the disease, the client aimed to convey the clinical value of its product to payers, which required prioritizing data collection for endpoints most likely to demonstrate the disease-modifying characteristics of the treatment.

Leveraging our deep expertise in rare diseases and commercialization, we recruited and interviewed a group of payers and key decision makers from the United States and other major markets, such as Germany, Spain, Japan, and the United Kingdom. Our clinical bench aligned with the client’s medical team to develop thorough pre-read materials to familiarize the payers with the characteristics of the ultra-rare indication. We also evaluated the client’s previous research findings, clinical development program materials, clinical study protocols, and minimal and ideal target product profiles and used the insights gained from these evaluations to develop a structured discussion guide for in-depth interviews.

We designed the interview guide to validate the client’s target patient population, optimize its planned clinical program, test its potential pricing bands, and assess its target product profiles. We synthesized insights from these interviews and turned them into recommendations for the client’s executive leadership to use in prioritizing clinical endpoints, validating pricing bands, and refining target product profiles to ensure appropriate reimbursement for the client’s product in key payer markets.

Applying our recommendations, the client has amended its clinical program to ensure that it uses endpoints capable of demonstrating to payers the disease-modifying characteristics of its product.