Navigating cell and gene therapeutic access

NPC paper offers insights for patients and caregivers, with suggestions for industry to optimize and invigorate this field.

The National Pharmaceutical Council released a recent white paper aimed at patients and caregivers who may be having challenges understanding their diseases, accessing therapies or navigating healthcare and reimbursement necessities to receiving cell and gene therapies (CGTs). Most of the therapies developed in the CGT space are for rare diseases. However, the complexities of reimbursement and access span the vast therapeutic area spectrum.

There are currently 43 FDA approved CGTs, dating back to 2017, with the Council’s article predicting 66 new approvals expected by 2032. With this in mind, Brian Sils, NPC Research consultant, said in the article: "This report helps decision-makers better understand what patients are experiencing throughout this process. We can then have an informed conversation about how to help patients reach their full potential through these transformative therapies.”

For industry, the paper delves into stakeholder solutions, specifically calling out new or innovative payment models that could help inform reimbursement, and thus incentivizing the development of CGTs, as follows:

• Annuity-Based Payments – Where a payer pays a fixed price for the therapy at regular installments, spreading the costs out over time instead of paying all at once.
• Outcomes-Based Payments – Where a payer pays a portion of the therapy’s price up front and only pays the remainder if predetermined outcomes are met.
• Outcomes-Based Rebates – Where a payer pays the full price of the therapy up front but will have some amount of that rebated if the therapy does not meet predetermined outcomes.
• Outcomes-Based Annuities – Where a payer pays a fixed price for the therapy at regular installments, but only if the therapy continues to meet its predetermined outcomes.

The report also called out a “key treatment bottleneck” for CGTs, which is based on the limited number and geographic distribution of specialized treatment centers, including Centers of Excellence (COEs). Here, the NPC recommends a “hub-and-spoke” model that links community healthcare practices with COEs or other specialized treatment centers, referencing this article “Strengthening health systems for access to gene therapy in rare genetic disorders.”

To learn more about barriers to market access, as well as rare diseases, please see our upcoming events.

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