Addressing Barriers to Access for Today’s Cell and Gene Therapies

The FDA approved more than 25 new cell and gene therapies in the past four years alone, with several more approvals expected in 2026, bringing the total number of approved products in the U.S. to more than 50.¹ However, more than a year after approval, many of these therapies continue to face significant barriers to broad patient access. As more therapies move from development into commercialization, the industry’s challenge is ensuring patients can access them.

Chris Learn, Senior Vice President, Head, Cell and Gene Therapy and Early Phase at Parexel, has helped guide more than 20 gene therapy programs through development. Chris maintains that many of the barriers to scale are established years before approval. Decisions around trial design, regulatory strategy, and manufacturing planning ultimately determine whether a therapy reaches patients broadly or remains confined to a handful of specialized sites.

Because many cell and gene therapies are designed as one-time interventions, sponsors have little room for error. Programs built without a clear understanding of how therapies will be manufactured and delivered risk limiting access long after approval. Chris explores these issues and shares insight on how regulatory decisions made early in development can shape patient access years later.

Contract Pharma: Why are commercialization challenges often created long before a therapy reaches the market?

Chris Learn: In cell and gene therapy, the decisions that shape commercialization happen during development, years before launch. The choices a sponsor makes early about manufacturing, trial design and where studies run tend to set the ceiling on how many patients a therapy can eventually reach. The stakes are unusually high here because many of these therapies are designed as one-time interventions, so there is very little room for error.

Manufacturing is a good example. When it is left loosely defined, sponsors risk negative regulatory feedback at early engagement meetings such as INTERACT, and they lose months they cannot get back. FDA leaders have been direct on this, encouraging sponsors to define their manufacturing earlier so a program can move cleanly through development. I have seen scientifically elegant therapies stall simply because the process behind them was never built to scale. Getting the fundamentals right early is what protects the commercial opportunity later.

Contract Pharma: How do regulatory, clinical and manufacturing decisions influence long-term patient access?

Chris Learn: These three decisions compound on one another. On the regulatory side, aligning early on the fastest defensible path to approval, and on which agencies are involved, shapes how quickly and how broadly a therapy becomes available. 

On the clinical side, protocol design largely determines who can participate in the first place. When trials sit at a handful of academic medical centers, the resulting evidence reflects a narrow slice of the patient population. 

And on the manufacturing side, a heavy reliance on centralized production keeps a therapy tethered to a small number of specialized centers. This is why point-of-care and more distributed models are getting so much attention, because they begin to loosen that constraint. I think of access as the sum of these choices. When they are made in sequence and with intent, a therapy can reach the community. When they are made in isolation, access tends to stay with the few sites already equipped to deliver.

Contract Pharma: What separates therapies that scale successfully from those that remain limited to a small number of treatment centers?

Chris Learn: In my experience, the therapies that scale are designed from the start to be delivered well beyond the academic medical center. Practically, that means writing protocols that sites can actually execute. We have seen how complex protocols and heavy logistics leave healthcare teams under-resourced, fatigued and burned out, particularly at the academic centers carrying the most cell and gene trials. Scaling depends on relieving that pressure through real site collaboration. That includes locking down key workflows by site consensus, running feasibility assessments of the Schedule of Events, and agreeing early on which endpoints are primary and which are simply nice to have. 

It also means bringing regional and community hospitals into the work and giving them the support to succeed. When only a small percentage of eligible patients enroll at saturated academic centers, casting a wider net helps the sponsor’s enrollment and reaches more of the patients who stand to benefit. Therapies tend to stay stuck when that operational burden is never engineered out.

Contract Pharma: Why is cross-functional planning becoming increasingly important as the cell and gene therapy field matures?

Chris Learn: As the field matures, the hardest problems are less about the science and more about how the functions work together. A program today has to connect preclinical design, regulatory strategy, clinical operations, manufacturing and market access as one continuous effort. When any one of those functions optimizes on its own, I find the handoffs are where value tends to leak out. That is especially true given the financial pressure many developers are under, where a sponsor’s capital can run short months before its next data readout, and there is very little room to redo work. 

What I always come back to is that no sponsor closes this gap alone.  When you are shaken to your core, you tend to go back to fundamentals and process, and process is really what good cross-functional planning captures. Aligning these functions early, and keeping them aligned, is how sponsors get to quality and speed affordably. I think that coordination is fast becoming the thing that separates a therapy that reaches patients from one that never does.

References:

1. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

Chris Learn, Ph.D., PMP, is Franchise Head of Cell & Gene Therapy and Early Phase at Parexel. He brings more than 20 years of experience in clinical trial execution and team management. Before joining Parexel, Chris held clinical development leadership roles at Istari Oncology, IQVIA and ICON. He has supported the registrational development, agency submission and regulatory approval of several notable therapies, including Imbruvica, Yondelis and Keytruda, as well as Yescarta following its initial market authorization. Chris has also overseen outsourced global trial operations for the clinical development of CAR-T assets under review by the U.S. Food and Drug Administration (FDA).