Critical Success Factors for Commercializing Cell & Gene Therapies (4): Planning for a Challenging Reimbursement Process by Solving for the Whole System

In our previous installment, we explored how manufacturers need to map and shape the referral pathway to ensure patient momentum toward treatment. In this post, we address what happens when a referred patient arrives at an authorized treatment center (ATC) and the ATC encounters one of the most challenging (and underestimated) barriers in the entire cell / gene therapy (CGT) journey: reimbursement.

CGT reimbursement is not a harder version of specialty drug reimbursement. It’s very different, with distinct economic, operational, and contractual dimensions that conventional pharmaceutical launch playbooks are poorly equipped to handle.

The Payer Problem – and Why It’s Only Half the Story

Payer skepticism about CGTs is well documented. With price tags that can reach seven figures for a single administration, payers face an immediate, concentrated financial exposure that is unlike anything in the traditional drug benefit. Without long-term real-world durability data, that exposure feels particularly difficult to underwrite. Prior authorization requirements are stringent, appeals are common, and coverage decisions can vary dramatically across payer types, geographies, and lines of business.

Most manufacturers understand this dimension of the challenge and invest accordingly in payer engagement, health economics data, and coverage strategies. Of course, securing payer coverage is necessary. However, it’s not sufficient, and manufacturers often under-appreciate that fact. The ATC sitting between the payer and the patient faces its own formidable set of reimbursement challenges. If those aren’t solved, access breaks down regardless of what the coverage policy says on paper.

Solve for the ATC, Not Just the Payer

Treatment centers administering CGTs carry substantial financial and administrative burden. They must document patient eligibility in detail, negotiate case rates with individual payers, manage prior authorization submissions and appeals, and in many cases absorb significant financial risk during the gap between product acquisition and reimbursement. For a therapy that may cost millions of dollars per patient, that exposure is not trivial, especially when multiple patients at the same ATC receive a CGT in a short timeframe. Even a willing center may hesitate to move forward with a case.

A key dimension of this burden is the single case rate agreement, which is the predominant contracting mechanism between ATCs and payers for CGT administration. These agreements evolved from legacy transplant reimbursement dynamics, where case-by-case negotiation was manageable given relatively low procedure volumes. However, as CAR-T and other CGT volumes have scaled, the requirement to negotiate individual case rates for each patient has imposed significant administrative workload on both payers and providers, creating a bottleneck that can delay or even deter treatment.

Manufacturers that recognize this dynamic and actively work to reduce the ATC’s burden are far more likely to achieve consistent, scalable access. This means more than providing reimbursement hotlines and coding guides. It means helping ATCs identify and engage the right contacts at specific payers, supporting them through the case rate negotiation process, providing financial bridging mechanisms that reduce exposure during reimbursement delays, and building dedicated field-based reimbursement support that functions as a genuine operational partner to the site. The goal is to make it financially and administratively viable for an ATC to treat CGT patients consistently, not just possible in the best-case scenario.

Address Durability Skepticism Head-On

One of the most persistent reimbursement challenges for CGTs is the durability question. Payers are being asked to make large, upfront coverage commitments for therapies whose long-term effectiveness, by the time of approval, is supported by clinical trial data that may span only a few years. The concern is straightforward: what if the effect wanes? What if patients ultimately require retreatment or return to standard of care?

This skepticism cannot be dismissed or argued away: it must be addressed directly and proactively. That means entering the market with the strongest possible evidence package. This can include long-term follow-up data where it exists (or a well-documented plan and commitment to collect long-term follow-up data), robust health economic modeling that accounts for durability uncertainty, and a clear narrative about what the clinical trial data does and does not show. Critically, the evidence story does not end at launch. Post-launch real-world evidence (RWE) generation by manufacturers, combined with outcomes reported through established registries such as CIBMTR, can strengthen the durability narrative and provide payers with the ongoing reassurance they need to maintain and expand coverage over time.

It also means being prepared to engage in creative contracting structures. Outcomes-based or value-based contracts (VBCs), which tie a portion of payment to demonstrated clinical outcomes over time, are increasingly relevant in CGT reimbursement. While VBCs are operationally complex—requiring data-sharing infrastructure, agreed outcome definitions, and long-term administrative commitment from both parties—they can provide payers with the risk mitigation they need to commit to coverage. Manufacturers that have laid the groundwork for VBC engagement before launch, rather than scrambling to construct them under coverage pressure, are in a meaningfully stronger position.

Navigate the “One-Time Use” Complexity

Perhaps the most structurally novel aspect of CGT reimbursement is the one-time nature of the therapy itself. Pharmaceutical reimbursement systems, such as coding structures, fee schedules, formulary frameworks, and rebate models were designed around chronic therapies with ongoing utilization. A single administration that may deliver years of clinical benefit does not fit neatly into any of those constructs.

This mismatch creates practical challenges at every level. Coding and billing for CGT administration often requires navigating a patchwork of existing codes that were not designed for this purpose or pursuing new coding designations that take time to establish. Budget impact models that spread cost over time require payers to adopt accounting frameworks they may not have in place. Rebate and discount structures that are standard in specialty pharma may be irrelevant or counterproductive when there is no repeat purchasing.

Manufacturers must therefore invest early in working through these structural issues. That requires engaging with coding bodies, educating payer medical and pharmacy directors on CGT-specific frameworks, and developing contracting templates that reflect the economic reality of one-time therapies rather than forcing them into chronic-drug conventions.

The Bottom Line

CGT reimbursement rewards manufacturers that approach it as a systems problem rather than a coverage problem. Payer engagement is essential, but it is only one dimension of a challenge that also encompasses ATC financial viability, durability evidence strategy, and the structural mismatch between one-time therapies and the reimbursement infrastructure built to handle chronic ones. Companies that solve for the whole system are the ones that turn coverage policies into actual patient access.

Next in the series: Success Factor 5 – Supplemental Evidence Generation, Including Long-Term Follow-Up.