Introduction: The Access Paradox in Cell & Gene Therapy
Regulatory approval is a critical milestone, but it is not synonymous with patient access. Globally, developers are increasingly encountering situations where approved therapies see limited uptake due to operational complexity, insufficient data tailored to the heterogeneity of Health Technology Assessment (HTA) requirements across markets, and broader system readiness constraints. Cell and Gene Therapies (CGTS) place unique demands on healthcare infrastructure. They require specialized manufacturing, complex logistics, highly trained clinical sites, and long-term patient follow-up. These requirements sit uncomfortably within systems historically designed for chronic, repeat dose pharmaceuticals. The result is a gap between scientific innovation and real-world delivery which must be closed to realise the most value.
This access gap is rarely the result of a single failure point. More often, it reflects limited early-stage consideration of the end-to-end patient journey, pricing strategy, and downstream commercialization requirements. These decisions compound over time and become visible only when a therapy reaches the market. This article examines how embedding access thinking from the outset can reduce risk, align stakeholder expectations, and help ensure that scientific breakthroughs translate into real-world patient benefit, not regulatory success alone.Â
The Reality Check: Where CGT Access Works – and Where It Breaks DownÂ
CGT development is progressing within a diverse global regulatory environment, particularly in regions that leverage accelerated or reliance-based pathways. Mechanisms such as the FDA’s RMAT designation, the EMA’s PRIME scheme, and reliance on FDA approvals across parts of MENA have improved speed to regulatory decision-making for selected assets. In these contexts, regulators have demonstrated pragmatism in addressing high unmet need.
However, this flexibility is not applied uniformly, and challenges emerge when moving from initial approval to broader, multi-market access. While agencies such as the FDA and EMA may share overarching benefit–risk principles, alignment on endpoints, comparators, and long-term follow-up expectations are not guaranteed. A cross-agency study analysis showed that only an estimated 20% of clinical data packages submitted to both were identical[1], highlighting the degree to which developers must tailor evidence generation across jurisdictions. In Europe, this complexity is further amplified by the separation between regulatory approval and health technology assessment, where bodies such as NICE introduce additional scrutiny around clinical relevance and value demonstration.
Manufacturing and quality requirements represent one of the most consistent points of friction across regions. Chemistry, manufacturing, and controls (CMC) considerations frequently determine both regulatory timelines and future scalability. For CGTs, where processes are often individualized or highly specialized, comparability standards, and lifecycle planning requirements can delay not only approval, but sustained access post-launch.
Trial Design as an Access Decision
Trial design is one of the earliest and most consequential access decisions for a therapy, yet its downstream access consequences are often underestimated, particularly when trial design decisions are made without upstream input from market access, commercial, and real-world evidence teams. Early alignment between clinical endpoints, regulatory expectations, and anticipated payer and HTA evidence needs are critical to ensuring not only approval, but timely and sustainable patient access.
Design decisions made to optimise for speed, such as accelerated approval pathways or surrogate endpoints, can enable earlier market entry; however, if not balanced against durability, long-term outcomes, and real-world relevance, they risk undermining downstream reimbursement, site readiness, and adoption in clinical practice. Without front-end cross-functional input, trials designed narrowly to achieve regulatory approval, often in a specific geography, may therefore struggle post approval when the evidence generated does not meet payer requirements or fails to reflect real-world care pathways.
Embedding access considerations into trial design, from endpoint selection through to site strategy and evidence generation planning, helps ensure that regulatory success translates into real‑world access without avoidable delays or barriers.
Early Engagement as a Success Factor
Early and structured engagement with regulators remains one of the most effective ways to mitigate access risk. When scientific advice is sought early, it provides clarity on what regulators are likely to consider acceptable, including how endpoints are defined and justified, how trial populations and comparators are framed, and whether the proposed evidence package will withstand scrutiny beyond initial approval. This early alignment allows development teams to make informed choices before designs are finalised, avoiding course corrections later that can slow access and fragment launch timelines.
Engagement with clinical sites and healthcare systems is just as important. Cell and gene therapies place demands on infrastructure and clinical practice that differ markedly from conventional medicines. Without a clear understanding of how sites will manage these requirements in practice, development plans risk becoming disconnected from real-world delivery. Assessing readiness early supports more realistic timelines and helps avoid access delays that emerge only after approval, when system constraints become visible.
Maximising the opportunity for Patient Access after first marketing authorization
Mechanisms exist to enable the supply of products that are not licensed in the patient’s country in response to an unmet clinical need. Typically initiated by healthcare professionals, requests received by biotech and pharmaceutical companies can be serviced to meet the needs of these patients.
Framed correctly, supply through so-called Expanded Access or Named Patient routes can support a coherent introduction of a product into a market through to routine use. Although requests are received in an unsolicited manner, proactively accounting for these mechanisms within commercialization readiness planning can provide significant benefits. These include providing early user experience to the product, mobilization of supply chains into new territories and establishment of qualified treatment centres ahead of commercialization.
Although formal reimbursement is not commonly available, it is possible to provide access either on a charged for or free of charge basis. It is also possible to provide access through these mechanisms into countries where there is limited or no intention to commercialise, which has a profound impact on the scope of patients it is possible to treat.
Drawing on our own experience, Uniphar has supplied products into more than 50 countries, supporting access for over 600 patients and delivering a transformative impact for both developers and patient treatment journeys.
Conclusion: Designing for Access Is Designing for Patients
For cell and gene therapies, access is not a final hurdle, it is a cumulative outcome of decisions made across development, regulation, manufacturing, and delivery. Therapies that succeed are not necessarily those that move fastest, but those built to withstand the operational and economic realities of modern healthcare systems. By embedding access strategy from the outset, organizations can reduce risk, and ensure that scientific breakthroughs translate into meaningful, real-world impact.
Ultimately, designing for access is inseparable from designing for patients. And in advanced therapies, getting those details right is what determines whether innovation truly delivers on its promise.