ATMP development and market access in Europe and the United States: why scientific innovation alone no longer wins
Cell and Gene Therapy Insights 2026; 12(8), 802–812
10.18609/cgti.2026.097
Advanced therapies are changing medicine. Gene therapies can address the root cause of inherited disease. Cell therapies are reshaping oncology and immunology. Regenerative approaches are opening treatment options that were inconceivable only a few years ago. Few sectors combine this level of scientific progress with equally visible commercial friction. Across Europe and the United States, highly innovative therapies have reached regulatory approval, yet commercial uptake has often been slower, narrower, or more difficult than expected. In some cases, products were even withdrawn despite strong science and significant medical need. Several analyzes have shown that these challenges are particularly pronounced for advanced therapies because commercial uncertainty often extends far beyond regulatory approval itself [1–4]. This gap is often misunderstood. Many organizations still treat market access as a late-stage commercial exercise that starts once pivotal data are available. In reality, especially for advanced therapy medicinal products (ATMPs), access is shaped much earlier. It is influenced by how evidence is generated, how uncertainty is managed, how manufacturing is industrialized, and how convincingly long-term value can be translated into a funding decision. Emerging evidence suggests that organizations integrating market access considerations early during development are better positioned to address reimbursement uncertainty and facilitate broader patient access following regulatory approval. Increasing evidence indicates that successful commercialization depends not only on scientific efficacy, but also on how effectively and iteratively patient access and reimbursement planning, evidence generation, durability assessment, manufacturing readiness, and operational delivery evolve in parallel throughout development.This article is a senior executive perspective based on a targeted review of relevant published literature, policy documents, and publicly available industry information, combined with the authors’ experience in ATMP development, market access and commercialization.