Cell and gene therapies are approved — so why aren’t they reaching patients?
New industry report warns access systems — not science — are now the primary bottleneck
A new report from Phacilitate argues that the biggest threat to cell and gene therapy (CGT) expansion is no longer scientific feasibility — it is access infrastructure.
Capturing expert insights from Dr. Ronit Slotky, Director of the Cell Therapy Manufacturing Facility at Hackensack University Medical Center; Craig Martin, CEO and Founder of Orphan Therapeutics Accelerator; and Kimberley Barnes, President of Phacilitate, the report From Breakthrough to Bottleneck – Why Cell and Gene Therapies Still Struggle at Market Access examines the clinical and commercial realities that explain why many CGTs still struggle to reach patients. It explores the three most pressing dimensions shaping CGT access today: point-of-care (PoC) manufacturing, alternative funding models and PoC finance, and the evolution of health technology assessment (HTA) frameworks.
“Cell and gene therapies aren’t stalling because the science isn’t working — they’re stalling because access expectations haven’t caught up with reality,” said Kimberley Barnes, President of Phacilitate. “High upfront costs, hospital readiness gaps, and HTA frameworks that weren’t built for one-time curative treatments are creating bottlenecks that the industry needs to confront head-on.”
Key insights from the report include:
- Hospital readiness is a critical bottleneck — and one that is often underestimated by developers. Point-of-care manufacturing is promising, but only realistic today for a small number of indications. Most advanced therapies are still delivered only in centers of excellence because they require specialist storage, trained staff and complex logistics. Early decisions about product design have irreversible consequences for patient access.
- Creative funding models are starting to unlock momentum for stalled programs. Nonprofit commercialization, tax-incentivized program transfers and flexible reimbursement structures offer viable alternatives for therapies that cannot progress through traditional routes — benefiting developers, patients and the broader ecosystem. The future landscape will favor lean, adaptable models built around realistic deployment conditions.
- HTA must evolve as CGT expands beyond rare diseases. As CGTs expand into larger patient populations, a more holistic, value‑based HTA approach is needed. Measures such as quality-adjusted life years (QALYs) often fail to capture the full impact of treatment, or non‑treatment, on patients, families, and health systems. A more holistic, value-based approach is needed that reflects societal benefit, economic productivity and lived patient reality. Lowering manufacturing costs, shortening hospital stays and reducing toxicity to enable outpatient delivery are critical levers to success.
What “access-ready” means in 2026
The report also sets out what “access-ready” looks like in practice for 2026, identifying earlier clinical engagement, greater transparency on true costs, and the shift toward off-the-shelf and in vivo modalities as the factors that will accelerate CGT success.
“Being access-ready in 2026 means building for the real world from day one — engaging treatment centers early, designing therapies that can be delivered outside of centers of excellence, and having honest conversations with payers about what these treatments actually cost to develop and deliver,” added Barnes. “The next big shift in advanced therapies is cultural: developers, hospitals and payers working together before late-stage surprises force their hand.”
The report, ‘From breakthrough to bottleneck – why cell and gene therapies still struggle at market access’, is available to download here.
This article is a press release submitted to Life Science Daily News and does not represent the editorial views or opinions of Life Science Daily News. It has been published as received from the submitting organisation.
