Scaling Cell Therapy in Europe: Why the Biotech Act Must Keep Pace with Innovation

Some medical advances treat a disease. A rare few can end it. CAR T-cell therapy belongs to the second kind. Some patients who received a single CAR T-cell infusion for blood cancers ten years ago now have no evidence of disease — no ongoing treatment, no relapse. That is a different category of medicine, and Europe helped build it.

Pioneering treatments, powerful returns

The science of CAR T-cell therapy has advanced rapidly. The field is now moving into earlier treatment settings and beyond blood cancers entirely — into autoimmune disease. Patients living with conditions such as lupus and multiple sclerosis face a lifetime of treatment with no end in sight. CAR T-cell therapy has the potential to change that with a single infusion. This is a fundamental shift in what healthcare can deliver.

When one treatment can do the work of a lifetime of interventions, the economics of care are transformed. A therapy that returns a patient to their family, their work, their life is a return on investment, paid back in reduced welfare costs, workforce productivity released, hospital capacity freed, and years of life enjoyed.

The science is ready. The systems are not.

I lead the Cell Therapy Organization at Bristol Myers Squibb. We are the only company with two approved CAR T-cell therapies against two distinct targets, and  more than 19,000 patients globally have been treated with a BMS cell therapy product. We are building toward providing treatment for  five times more patients by 2030. 

Whether Europe will be at the centre of this transformation comes down to choices:  about how these medicines are assessed, manufactured, regulated, and delivered. Those choices are exactly what the Biotech Act gives co-legislators the opportunity to get right.

Three interconnected shifts are needed to allow health systems in Europe to deliver this next wave of treatment to patients. 

First, recognising the true value of these medicines is recognized by payers. 

A one-time, potentially curative therapy cannot be judged by a single year’s budget impact — yet that is precisely what most national reimbursement frameworks do. For many CAR T-cell therapies, randomised controlled trials are neither feasible nor ethical. The EMA has accepted this, granting authorisation on single-arm data. Most national Health Technology Assessment (HTA) bodies have not followed, and the result is a therapy that clears the regulatory bar and then stalls at the reimbursement door.

Closing that gap requires two things: HTA frameworks that assess Advanced Therapy Medicinal Product (ATMP) on the long-term value they create — for patients, for workforce participation, for healthcare systems — and the real-world evidence to support those assessments. Today, that evidence sits trapped in fragmented national registries that cannot communicate with each other, meaning each country relearns the same lessons independently. Interoperable registries, built on common standards and the European Health Data Space, can be the foundation of every reimbursement decision that follows. The Biotech Act is the right vehicle to incentivize common registry standards and anchor EU-level guidance on what constitutes robust ATMP evidence.

Second, the way regulators handle manufacturing enhancements for these therapies needs to reflect how they actually work.

An ATMP is not a conventional pill. It is an individualized therapy shaped by the process that takes patient cells from apheresis, through manufacturing and supply chain, to infusion. That process is integral to the ultimate therapy, and it will continue to evolve and improve. Yet today, even a routine process change can trigger a lengthy regulatory submission, regardless of how minor that change may be.

Other regulatory systems, have addressed this. Submissions are tiered to match the significance of the change, where minor updates are filed quickly, and  intensive review is reserved for changes that genuinely warrant it.

The EU needs more flexibility. That means a framework proportionate to risk: faster handling of routine manufacturing variations, adaptive GMO-related exemptions, and early and agile EMA scientific guidance so manufacturers are not navigating uncertainty alone. The Biotech Act should establish that framework explicitly, tailored to ATMPs rather than borrowed from conventional biologics. 

Third, the places and people that deliver them must be strenghtened to ensure access at scale. 

Delivering these therapies at scale will require a broader network of treatment-ready hospitals, and enough trained staff to administer them beyond oncology and haematology. 

Targeted investment to qualify more treatment centres and expand fast-track professional training would allow more patients to receive treatment closer to home. The Biotech Act can enable this funding of strategic infrastructure  and co-legislators should ensure it does.

Now it falls to co-legislators

The Biotech Act is the first time Europe has treated biotechnology as what it is: strategic infrastructure, no less than energy or defence. But the real opportunity now lies with the European Parliament and the Council. As co-legislators, they have the mandate and the tools to go further and to ensure the Act funds ATMP-ready infrastructure across Member States, incentivizes the interoperable data architecture the science demands, and tailors its regulatory provisions to how CAR T-cell therapies are made and delivered.

I have seen what happens when policy and science move together — and I have seen what CAR T-cell therapy does for patients. Europe has every reason to lead in this next era of medicine — and the Biotech Act is the opportunity to make sure patients will benefit.