Global pricing and access strategic issues assessment for rare diseases gene therapy portfolio

April 2022
Genetic research, conceptual image


Our client is developing gene therapies for different rare disease indications, some with effective treatments available and others with only symptom management options. Our client wanted to further develop and refine their overall pricing and access strategy approach for gene therapies given the evolving dynamics of the market.


CRA’s rare disease consultants conducted an assessment across key geographies (US, Europe, Japan, Canada) to understand the impact of different issues including the external policy environment, reference pricing, timely access, and evolution of the cell & gene therapy landscape. We analyzed the evolving policy landscape and feasibility of different pricing methodologies using secondary research and analogue analysis. We examined the potential risks for gene therapy pricing and access associated with reference pricing with implications for launch sequencing. We generated additional insights through primary research with payers and policymakers, pressure testing the feasibility, requirements, and impact of gene therapy pricing approaches including the appetite for alternative payment models. Based on all the gathered insights, we developed key pricing and access opportunities and risks for the gene therapy assets exploring various pricing scenarios across different geographic regions to determine what would be the best approach to meet the client objectives.

Client Impact

CRA’s team of life sciences consultants provided implications and recommendations for the gene therapy portfolio and to inform the global pricing and access strategy, which were refined through a series of global client team meetings. We delivered recommendations on optimization and risk mitigation for gene therapy pricing strategy implementation and the likely impact of the changing policy environment and competitive landscape in key markets.

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