In this excerpt from our on-demand webinar, “Navigating the cell and gene therapy marker access landscape” , we explore four common access challenges that have been observed in the cell and gene therapy space:

1. Demonstrating long-term benefit and managing data collection uncertainties
2. Managing complexities around small patient pools
3. Affordability of “one-off” high-cost treatments paid over a short duration
4. Supporting value differentiation against highly competitive pipelines.

Demonstrating the long-term benefit of cell and gene therapies and managing data collection uncertainties

As shown on this timeline, past regulatory and payer approvals of CGTs have been based on small, open-label, single-arm, non-comparative trials. These trials have relied on historical controls and intermediate endpoints as surrogates for survival outcomes.

Thus, due to the lack of long-term comparative evidence at the time of evaluation, the magnitude of incremental clinical benefit has been regarded by payers as uncertain.

Likewise, payers and clinicians have also voiced long-term concerns around safety and impact on HRQL, particularly for gene therapies that target pediatric patients. Even the generalizability of trials has been questioned by some payers if trials have not been multicentred or trial sites or comparators have not been representative of local clinical practice.

To manage this, coverage resolutions for many CGTs have been conditional to future data submissions. Payers have asked for re-assessments when mature, long-term, clinical data, and realworld evidence required to establish curative potential are available.

Managing the complexities around small patient pools for cell and gene therapies

As precision medicines, CGTs target highly selective patient populations. For gene therapies, due to the ultra-rare nature of certain genetic diseases, the small patient pools may result in challenges around patient recruitment for clinical studies.

In addition, the identification and quantification of eligible patients in practice can be challenging. The diagnostic process can be complex particularly for gene therapies given the lack of awareness among non-specialized clinicians, which may lead to delayed or misdiagnosis. Limited availability and access to specialists and genetic testing can also result in patient withdrawals.

Certain CGTs, especially CAR-Ts, are currently positioned as the last resort for heavily pre-treated patients. In such cases, the heterogeneity in prior treatments can increase uncertainties around patient eligibility and raise further concerns about the generalizability of the trials to clinical practice.

Affordability of ‘one-off’ high-cost cell and gene therapies paid over a short duration

CGTs as one-time treatments are associated with high upfront costs, and benefits will be accrued over a longer period. Given that long-term treatment effect can be uncertain at present, it is challenging for manufacturers to demonstrate cost-effectiveness and budget impact to payers.

Particularly for gene therapies, even though certain targeted genetic diseases are rare, treatment acquisition cost can exceed millions of dollars. For example, the recent FDA-approved gene therapy Hemgenix for hemophilia B costs US$3.5 million per dose and became the world’s most expensive treatment. Costly novel treatments can be out of reach for patients and balancing timely access with sustainable funding remains a key challenge for stakeholders.

To mitigate risks associated with high costs and uncertainties around long-term effectiveness, there is an urgent need for payers and manufacturers to collaborate on developing innovative and more affordable pricing mechanisms.

Supporting value differentiation of cell and gene therapies in highly competitive pipelines

Research in CGTs has grown considerably, and oncology and rare diseases remain the top areas of development in the pipeline. Over the next decade, CGTs will face competition from not only innovative small-molecule and protein-based therapies, but also similar CGTs—potentially with the same mechanism of action.

Given that cell therapies are moving into earlier lines of treatments with larger eligible patient populations, and competitions among gene therapies are increasing, the perception of unmet need in these TAs is expected to evolve. In addition, gene therapies and cell therapies are likely to compete with each other as treatment pathways evolve. Achieving a high price potential will become more challenging in the future.

As competition increases, payers’ expectations on comparative clinical evidence to justify higher price will also evolve. Therefore, optimal positioning of new treatments relative to current and future competitors will become critical to support evidence generation and value differentiation based on payer archetypes.

Navigating the cell and gene therapy market access landscape

These insights were taken from our free webinar “Navigating the cell and gene therapy marker access landscape” which is now available to watch on-demand.

The webinar explores the cell and gene therapy landscape, highlighting key market access challenges, and concludes with considerations for optimizing patient access. This includes:

• A summary of the evolving treatment landscape in cell and gene therapies
• An overview of the key challenges to overcome for achieving optimal access, including:
  – Demonstrating long-term benefit and managing data collection uncertainties
  – Managing complexities around small patient pools
  – Affordability of “one-off” high-cost treatments paid over a short duration
  – Supporting value differentiation against highly competitive pipelines
• Practical considerations on overcoming market access challenges and key barriers for cell and gene therapies.

Supporting your policy, access, value, and evidence needs for cell and gene therapies

The strategic importance of policy, access, value, and evidence for cell and gene therapies is increasing, and the need for an integrated 360 approach is becoming ever clearer. Our dedicated experts can partner with you to explore current trends and challenges and help you understand how you can optimize access for your assets.