Breakthrough Treatments Face Reimbursement Hurdles
The recent wave of FDA approvals for groundbreaking gene therapies has sparked intense debate about pricing models and equitable access to these potentially curative treatments. With therapies like Pfizer's Beqvez for hemophilia B and PTC Therapeutics' Kebilidi for AADC deficiency entering the market, healthcare systems worldwide are grappling with how to afford treatments that can cost millions per patient while ensuring those who need them most can actually receive them.
The Growing Access Gap
According to a comprehensive 2025 ISPOR review of the gene therapy access landscape, payers are becoming increasingly skeptical and implementing more restrictive management strategies as high-cost therapies enter the market. The study analyzed policies from 7 national managed care organizations and 13 regional plans covering over 200 million lives, revealing that ultra-high-cost therapies face significantly more restrictions than lower-priced alternatives.
'We're seeing payers require patients to match clinical trial inclusion and exclusion criteria almost exactly in prior authorizations,' explains Dr. Sarah Chen, a healthcare policy analyst who contributed to the ISPOR study. 'Sometimes they're delaying or denying coverage altogether, creating significant barriers for patients who could benefit from these treatments.'
Innovative Payment Models Emerge
To address these challenges, manufacturers and payers are developing innovative contracting approaches. Outcomes-based agreements (OBAs) have emerged as a promising solution, linking pricing and reimbursement to clinical endpoints. According to recent analysis, therapies with OBAs experience less restrictive management, and products that have been on the market longer face fewer restrictions due to established contracting strategies.
The CMS Cell and Gene Therapy Access Model represents a significant government-led initiative to improve access for Medicaid beneficiaries. As of July 2025, 33 states plus Washington D.C. and Puerto Rico are participating in this voluntary model, which focuses initially on sickle cell disease treatments and involves outcomes-based agreements between states and manufacturers to lower costs and tie payments to patient outcomes.
Manufacturing and Distribution Challenges
The 2025 Advanced Therapies Report highlights that despite rapid expansion in the field—with over 22 FDA-approved therapies currently available and projections of 200+ approvals treating 100,000+ patients by 2030—significant challenges persist. Only 20-40% of eligible patients get referred for these treatments, with insurance verification cited as the top enrollment barrier by 77% of providers.
'The logistical coordination required for these therapies is immense,' says Michael Rodriguez, a pharmacist specializing in advanced therapies. 'From cold chain requirements to specialized administration centers, we're building entirely new healthcare delivery systems around these treatments.'
Global Disparities in Access
The access debate extends beyond U.S. borders. While Europe has approved groundbreaking treatments like Vertex's Casgevy—the first gene editing therapy for blood disorders—and countries like China and India have approved domestic CAR-T products, significant disparities exist in global access. The high costs of these therapies make them largely inaccessible in lower-income countries, raising ethical questions about equitable distribution of medical breakthroughs.
'We're at a critical juncture where we need to balance innovation with accessibility,' states Dr. Elena Martinez, director of a nonprofit focused on rare disease treatments. 'These therapies have the potential to cure diseases that were previously untreatable, but if only the wealthiest patients can access them, we've failed in our mission as a healthcare community.'
The Path Forward
Experts agree that several strategies will be crucial for improving access to gene therapies. Multi-year payment contracts, site-of-care contracting, and value-based approaches are gaining traction. The ICER white paper on paying for gene therapy emphasizes the need for collaborative solutions involving manufacturers, payers, providers, and patients.
As the field continues to evolve with advancements in genetic engineering techniques and improved delivery systems, the conversation around pricing and access will only intensify. With projections indicating hundreds of new approvals in the coming years, healthcare systems must develop sustainable models that ensure these life-changing treatments reach all patients who need them, regardless of their economic circumstances or geographic location.