What ICER’s Draft Sickle Cell Disease Report Means for CGT Pricing

Recently, the Institute for Clinical and Economic Review (ICER) published its draft evidence report for sickle cell disease. The report assesses initial cost-effectiveness thresholds for two potential gene therapies against existing standard of care.  

Despite ICER not having authority over the final list prices set by manufacturers, payers increasingly take these assessments into consideration when determining coverage, reimbursement and access. Media often picks up on final ICER assessments, which can influence how stakeholders feel about the value of a product prior to approval. While initial media coverage has been minimal, Endpoints and Fierce Pharma noted that both gene therapies offer promise of a potential cure, adding to pricing speculation in the lead up to price disclosure.  

While the final assessment won’t be released until July, ICER’s value assessment of two gene therapy drug candidates for sickle cell disease provides some additional signals about how ICER will value gene therapies going forward. This is significant since we’re on the precipice of a wave of gene therapies entering the market. The science is exciting, but the multimillion-dollar price tags raise questions from health assessors like ICER and payers. 

A closer look – What does the ICER report signal for future gene therapies? 

The drug pricing watchdog group reaffirmed that an appetite remains for high-priced therapies that demonstrate incredible efficacy. However, there are several notable trends that will be critical for cell and gene therapy manufacturers moving forward:  

• Health equity - The importance of working across subpopulations
• Broad access - ICER to manufacturers: "lower your prices for the patients who desperately need these therapies"
• Patient voice - Raising the stakes for manufacturers to develop value messaging

To uncover more details about the future of gene therapies, read our latest blog.

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