Systemic gaps hinder cell and gene therapy adoption, access

Access barriers

Based on a national survey of 104 healthcare professionals, including referring physicians, administering specialists and CGT pharmacists, the report finds that access to CGTs remains limited by restrictive payer policies and insufficient care infrastructure.

Over 80% of respondents reported persistent payer-related coverage issues. Among the top challenges cited were restrictive prior authorization requirements (74%), long approval timelines (66%) and coverage criteria that "exceed the label language" (60%). These obstacles often delay care and, in some cases, prevent patients from accessing treatment altogether.

These delays are compounded by the widespread use of single-case agreements, which were described in the report as adding "significant operational burdens and delays to case processing because demand for CGTs has increased."

"Single-case agreements are still the dominant form of payer-provider contracting for CGT reimbursement," added Jen Klarer, Managing Partner & Head of Cell and Gene Therapy at The Dedham Group, in the report.

The geographic concentration of treatment sites also limits access. For example, most CGTs are currently administered at academic medical centers or large integrated health systems. According to the report, nearly half of the surveyed providers would like to see more treatment centers outside of academic and medical centers to increase equity and reduce travel burdens.

Treatment enrollment itself poses additional obstacles. Providers in the report cited insufficient caregiver support (64%), insurance-related prior authorization denials (57%) and out-of-network designations (52%) as the most common reasons patients fail to initiate CGT.

"If the process is linear, the turnaround is 6–8 business days; however, a concurrent enrollment process can have it completed in 2–3 business days," explained Derek Todd, Director of Sonexus Access and Patient Support at Cardinal Health, in the report.

Operational hurdles

The report revealed that high upfront costs and reimbursement challenges remain a significant obstacle to the adoption of CGTs. Of the providers surveyed, 79% believe that innovative reimbursement strategies, including value-based contracts, are needed to improve access. However, the practical execution of these models is challenging.

"There are a number of questions around who's responsible for data collection, what metrics are [evaluated] and who's going to be actually putting the logistics in place [for tracking the outcomes]," Kevin Niehoff, Director of Market and Financial Insights at IPD Analytics, noted in the report. "Even in the last five years, we've seen a tremendous evolution in how CGTs are discussed and considered. I believe that is only going to continue, especially following the introduction of CMS' CGT Access Model."

The need for robust, long-term outcome tracking also presents infrastructure hurdles. Regulatory bodies often mandate follow-up for up to 15 years post-treatment to track adverse events and long-term efficacy. While this mandate requires substantial effort from both patients and providers, maintaining contact with patients over that time frame is difficult, especially when they live far from treatment centers or change insurance. Nearly one in four patients are lost to follow-up within the first year.

"Behind a CGT program, there's a lot of infrastructure," said Dr. Robert Wolf, Director of Pharmacy at Mayo Clinic, who was in the report. "From the clinical perspective, we have a whole team of individuals who are responsible and accountable for measuring and documenting outcomes."

To supplement traditional data collection, the report highlights the growing role of real-world evidence (RWE).

"RWE can alleviate the burden on the patient; rather, it relies on the ability to amass the appropriate patient data from routine clinical practice," explained Andy Klink, Senior Director of Real-World Evidence, Cardinal Health, in the report. "The less engagement that’s required of the patient outside their routine care, the better."

Strategic imperatives

To unlock the full potential of CGTs, the report emphasizes that biopharma companies and commercialization leaders must do the following:

• Engage payers early in development. Initiating discussions during clinical trials helps align on coverage criteria, data needs, and value frameworks ahead of launch.
• Decentralize treatment access. Decentralizing CGT administration to outpatient and community settings reduces travel burdens and improves equity.
• Invest in scalable digital infrastructure. Tools like eligibility checkers and scheduling platforms can streamline enrollment and reduce patient access delays.

"Biopharma manufacturers preparing for commercialization should consider the complexity of the payer landscape earlier in their product's clinical development [versus more conventional specialty therapies]," said Kathryne Kirk, Director of Commercial Development at Cardinal Health, in the report.

The report cautioned that, "The road ahead will be full of challenges, some already known and others yet to come." Among the top concerns cited by surveyed stakeholders were inadequate payer reimbursement (54%), high acquisition costs and other financial constraints (56%) and limited infrastructure to support delivery (38%).

As the CGT market and adoption increase, sustainable commercialization will depend on more than product innovation. Instead, it will require strategic infrastructure, payer alignment and a commitment to equitable access across geographies and patient populations.

Alivia Kaylor is a scientist and the senior site editor of Pharma Life Sciences.