Picture a patient in Texas with a rare blood cancer. Before they can get a cell therapy that could save their life, doctors need to collect their immune cells first. For years, that’s meant travel: specialized collection centers are few and far between, and patients have had to come to them, often at real cost and real delay.
Together with Kite Pharma, a Gilead company, (BioBridge Global) BBG Advanced Therapies decided that didn’t have to be true anymore. The new Mobile Leukapheresis Center brings that first step directly to patients across Texas instead of asking patients to come to it.
That’s a small-sounding fix to a big problem, and it’s the kind of move worth celebrating, not just noting in passing.
Here’s what I keep coming back to. We’ve become extraordinary at building these therapies. Especially over the last two decades. Cell and gene therapy has changed what’s possible in medicine: editing genes, reprogramming immune cells, treating cancers and inherited disorders that used to have no real answer.¹ But building a therapy and getting it to the person who needs it are two different problems, and right now we’re much better at the first one.
Meaning, access clearly isn’t accelerating at the same pace.
Most conversations about the future of this field start with manufacturing. Centralized or decentralized. Hospital-based or point-of-care. People want to debate which model wins. I don’t think that’s the right question. Honestly, it doesn’t matter who wins. What matters is whether a specific patient, in a specific place, can get the therapy they need in time. Centralized manufacturing, decentralized, point-of-care, individualized protocols, even the in vivo therapies now emerging: every one of these models will have a role. None of them needs to be the winner.
Patients don’t care how or where their therapy was made. They care whether they get it.
And, if they get it, in the background, that looks like a healthcare system capable of supporting the right model for the right patient at the right time – (for a price that’s sustainable). Meaning, access is shaped by three interconnected realities: Place, Knowledge, and Affordability.
What actually gets in the way
A therapy can exist and still be out of reach for the person who needs it. Three things tend to be the reason, and none of them are about science.
Where a patient lives shouldn’t decide whether they get a life-changing treatment, but it often does. Patients still travel hundreds of miles to reach one of a handful of specialized centers. As more people become eligible for these therapies, that model stops holding up.
Coordination is the part people underestimate. These therapies only work if referring physicians, hospitals, manufacturers, labs, and caregivers are all moving together. That seems like it takes as much operational precision as it does scientific breakthroughs.
Cost goes beyond the price tag on the therapy itself. Travel. Lodging. Missed work. A caregiver who must put their own life on hold. Those costs can end treatment just as effectively as a denied approval.²
The BBG and Kite partnership is a demonstration of taking one of these three problems seriously and solving it, not for visibility, but because a patient was stuck. That’s the model. More of the industry should be doing exactly this.
Impact means who gets treated, not just what gets built
The science has brought us to an inflection point. The real measure of impact isn’t what we’ve engineered. It’s who gets to benefit from it.
That means building delivery models around the patient, instead of retrofitting ones built for traditional medicine. It means partnerships that remove a step for the patient instead of adding one. And it means closing the distance between a scientific breakthrough and the person who’s been waiting on it. Use knowledge to streamline, not continue, or even worse, add unnecessary complexities.
Access has to include the ability to pay for it
If we get this right, patients won’t remember whether their therapy was made centrally or locally, or which two companies worked together behind the scenes. They’ll remember that when they needed something that could save their life, they could get it and they could afford to get it.
Access without a way to pay for it isn’t really access. A patient who can reach the therapy but can’t afford it, and a patient who can afford it but can’t reach it, end up in the exact same place: untreated.
The inconvenient truth is that full story goes beyond the patient, but it still pulls on the patient and it contributes to the total patient experience.
Affordability extends well beyond the price of the therapy itself. For many patients and families, the financial burden begins long before treatment. Travel to distant centers, temporary lodging, meals, transportation, time away from work, and the requirement for a dedicated caregiver can create tens of thousands of dollars in out-of-pocket costs. Studies have documented the significant financial toxicity associated with CAR-T therapy, with travel, lost wages, and caregiver responsibilities becoming major contributors to the overall burden.
But affordability is also a challenge for the healthcare organizations delivering these therapies. Hospitals and treatment centers often assume substantial financial risk, purchasing therapies that can exceed $1 million before reimbursement is complete. In many payment models, reimbursement may depend on clinical milestones achieved months after treatment, requiring providers to carry significant working-capital exposure while simultaneously investing in the specialized personnel, infrastructure, and intensive monitoring these therapies require. For many community hospitals, this financial uncertainty becomes another barrier to offering treatment at all.
If we want advanced therapies to become truly accessible, affordability must be engineered into the system, not only for the patient receiving treatment, but for every organization responsible for delivering it.
What’s next?
As we look ahead, I believe three challenges will determine how successfully advanced therapies reach the patients who need them.
First, we must redesign healthcare delivery models that were built for traditional medicines, not personalized therapies requiring coordination across multiple organizations.
Second, we must simplify partnerships. Scientific innovation increasingly depends on operational collaboration among hospitals, manufacturers, technology providers, and healthcare systems.
Third, we must reduce the impact of geography. Patients should not face unnecessary barriers simply because they live outside a major academic medical center.
It is now our responsibility to ensure that care evolves.
If your organization is working on any part of this puzzle, whether that’s manufacturing, reimbursement, or logistics, I’d like to hear from you. For real impact, this is a problem we solve together, or not at all.
References
- American Society of Gene & Cell Therapy (ASGCT) & Citeline. Gene, Cell & RNA Therapy Landscape Report (latest edition).
- Alliance for Regenerative Medicine (ARM). Annual Report: State of the Cell & Gene Therapy Sector (latest edition).