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Test Tubes

Assessment of evidence gaps and support needs for a cystic fibrosis treatment


A medium-sized biotech company was developing a new product for cystic fibrosis indicated for the treatment of multiple disease genotypes, with varying levels of severity.

Our client wanted to conduct an evidence gap analysis and develop a support plan that captured the local needs and learnings from market access experience with the company’s other cystic fibrosis products.


RJW&partners had worked with the client on multiple global value dossiers so was well placed to support the project and provide in-depth therapy area expertise.

“Thank you again for the dedication to excellence you've shown throughout this project.”

Senior Director, HEOR


Our client’s product was being investigated for several genotype groups, with differing levels of disease severity.

The target patient population had some overlap with two of the client’s existing products. This created challenges when determining the relevant comparator for each trial and what evidence would best demonstrate the level of absolute and relative clinical benefit.

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RJW&partners conducted in-depth reviews of clinical trial protocols, and HEOR plans to develop a payer value proposition and evidence summary for the product.

These were then reviewed with Medical and Market Access Directors. The aim was to identify areas of improvement and additional evidence needs, in light of their experience with the client’s other cystic fibrosis products.

We then prepared and facilitated an interactive workshop with affiliates from Europe, Australia, and South America to align on the data gaps and regional support needs.


A critical success factor was ensuring affiliate involvement. Having already successfully navigated two products through local market access hurdles, they had important expert input on evidence needs and therapy area specific pricing and reimbursement challenges.


RJW&partners followed an iterative and collaborative approach that leveraged our expertise and the client’s local market access experience.

Our team clearly summarised and articulated complex trial designs, evidence requirements, and the key challenges affecting the product.


We delivered a detailed evidence gap analysis and support plan for the global market access and HEOR teams, which captured regional support needs and evidence requirements.


Following the project, RJW&partners went on to develop the product’s global value dossier, which was used during the preparation of successful reimbursement dossiers.

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