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By Katie Moses, Principal, Director of Cell and Gene Therapy Strategy and Jen Klarer, Partner, Head of Cell and Gene Therapy

In high-cost categories like cell and gene therapy, clinical trial design has increasingly had implications on commercial access. Since trials are critical to obtaining FDA approval, pharma companies have focused heavily on demonstrating efficacy of clinical trial design without much attention to the potential access implications of trial inclusion and exclusion criteria on patient eligibility for commercial coverage. However, current on-market cell and gene therapies have highlighted the need for proactive consideration of clinical trial design impact on access strategy and additional parallel data generation to protect against potential clinically inappropriate restrictions.

Here’s how clinical trial design impacts market access and why pharma needs to develop clinical trial criteria with access and outcomes-based research in mind before clinical trials start.

Payer use of clinical trial design in management

To ensure eligible patients most likely to respond to treatment are prioritized for high-cost agents, payers often reference clinical trial inclusion and exclusion criteria (i.e., pivotal trials) to develop coverage policies for cell and gene therapies. Once a payer has developed a coverage policy, the criteria can be difficult to remove. To ensure appropriate access, pharma should consider developing pivotal clinical trial criteria with commercial management in mind.

Access implications of clinical trial exclusion criteria

The goal of clinical trial criteria is to ensure controlled efficacy results for products, but this can inadvertently narrow patient populations and access for those products post-approval. For example, clinical trial criteria of “no prior BCMA-targeted therapy” has been relatively prevalent across payer policies for multiple myeloma 5L+ CAR-Ts. Patients who were treated with a BCMA-targeted therapy (e.g., select bispecific products) prior to being evaluated for a CAR-T therapy could become ineligible for receiving CAR-T treatment depending on the payer plan, despite data supporting patient response (e.g., greater than the control following BCMA exposure).

Another example can be found on the gene therapy side. In the pivotal trial for Zynteglo, a gene therapy treatment, clinical trial criteria excluded eligibility for patients that have a known and available HLA-matched family donor. This criterion, which has been widely used in commercial coverage policies, indicates that if a donor is available, the patient must be evaluated for transplant prior to Zynteglo treatment. The donor type specified, such as sibling or family, varies across policies. Although transplant is viewed as a functional cure, transplants come with health risks for both patients (e.g., GvHD) and donors, and patients who undergo transplants experience both clinical complications and economic burden.

Trial design impact on innovative payment model opportunity

Additionally, primarily in the gene therapy space, pharma companies have commonly developed innovative payment models to contract with payers for commercial products that need to be proactively planned during the clinical trial design phase. Innovative payment models have been a hot topic in the space, particularly for therapies that have higher price tags that put more financial strain on payer organizations. For these payment models, pharma must align clinical trial design with feasible payment model structures or risk creating access barriers in the future.

Outcomes-based rebate models are one of the more commonly leveraged innovative payment models but can be challenging as outcomes are sometimes difficult to measure. Consideration of clinical trial endpoints that may be seen in claims processing or have ease of measurement may enhance the likelihood that payers will adopt these payment models and execute on them.

Opportunities for strategic access planning in clinical trial design

While certain criteria may not be able to be excluded from clinical trial design, cell and gene therapy companies may consider proactive planning for strategies or additional clinical studies to obtain substantial data evidence to mitigate access restrictions.

Clinical trial design shapes the landscape of therapy access. Understanding how clinical trials influence commercial product (e.g., payer policies, payment model implementation) is crucial for ensuring broad patient access to innovative therapies. Strategically selecting control criteria, or proactively planning to mitigate potential access risks associated with necessary control criteria, in pivotal clinical trials can avoid clinically inappropriate implications on commercial access.

To mitigate trial design implications on commercial cell and gene therapy product access, pharma companies should:

  • Strategically select control criteria to account for commercial product management and patient eligibility
  • Proactively plan to mitigate potential access risks associated with necessary control criteria in pivotal clinical trials (e.g., additional data generation)
  • Consider feasibility of clinical trial endpoints for commercial product innovative payment model implementation and pull-through

As the field continues to evolve, proactive and thoughtful clinical trial design is key for pharma companies to overcome access challenges and advance patient care.

To learn more about The Dedham Group’s strategic consulting services, contact us.