Will Shrank: The Trillion-Dollar Question Facing CGT Access

Will Shrank, MD, co-founder and CEO of Aradigm, sees one of the most consequential tensions in modern medicine playing out in real time: a pipeline of potentially curative therapies arriving faster than the systems built to pay for and deliver them can adapt.

The numbers underscore the urgency. More than 4,200 cell, gene, and RNA therapies are currently in development — ranging from preclinical through pre-registration — with gene therapies alone accounting for nearly half the total pipeline, according to the American Society of Gene and Cell Therapy and Citeline's Q4 2024 landscape report.¹ Analysts expect the market to expand sharply in the years ahead, as the global cell and gene therapy market, valued at roughly $8.94 billion in 2025, is projected to approach $47 billion by 2035.² And the therapies already on the market are not cheap: FDA-approved CGTs range in cost from $373,000 to $4.25 million per treatment.³ As curative therapies expand from ultra-rare indications into conditions affecting millions of eligible patients, such as type 1 diabetes, multiple sclerosis and wet macular degeneration, the financial pressure on payers, providers, and manufacturers will only intensify.

The problem, as Shrank sees it, is not confined to any single part of the system. Payers struggle to absorb the unpredictability of high-cost, one-time claims. Providers face reimbursement uncertainty that makes it difficult to invest in the infrastructure needed to deliver these therapies. And manufacturers have watched breakthrough products stumble at launch, not because of clinical shortcomings, but because the ecosystem was not ready to receive them. With 61 advanced therapies currently approved in the US and that number projected to nearly triple by 2030, the pressure on every link in the access chain will only grow.

Shrank argues the next three to five years are a defining window — one that will require every stakeholder in the ecosystem to give a little in order to gain a lot. In the following Q&A, he breaks down where the system is straining, who bears the greatest burden, and what structural changes could determine whether these therapies reach the patients who need them.

Access all three parts of Shrank’s video interview with PC:

1. Asembia AXS26: How the Next 3-5 Years Will Define Specialty Pharma
2. Asembia AXS26: How Drug Innovation Could Strain Access Systems
3. How Reimbursement Uncertainty Delays Access to Rare Disease Therapies

PC: What are the key access opportunities and challenges over the next 3-5 years?

Shrank: Just to set the context, this is a moment where we have therapies that are extraordinary, totally transforming the lives of patients who can benefit. They're also unbelievably expensive, the cost is up front, and the value is accrued over a lifetime. This is the moment where the highly fragmented way that we pay for and deliver care in the US breaks down as a way to deliver the highest value, highest quality, and best experience care to patients. These next three to five years, we've got to figure this out. This is a remarkable moment, because if we don't figure out how to get cures, how to ultimately address the underlying problems of disease now, we could miss a really big opportunity to leverage this incredible science to really change people's lives.

There’s not a simple, single silver bullet here. There's not one thing that's wrong. It's a whole ecosystem problem, and everybody's struggling. Payers are struggling. They don't really know how to anticipate or deal with the unpredictability, the volatility, the spikiness of these costs, and if you're an employer, it can really be sort of an existential challenge. providers are struggling in a setting where they've got to make investments to be able to deliver this care sustainably, and they don't know a lot about the amount of volume they're going to get. They don't know what the reimbursement's are going to look like. The business model isn't really crystal clear going into that investment, and manufacturers, we've seen, have come out with incredible products that have had rather unimpressive launches, not because of the quality of the product, but because of the receptivity of the marketplace and the ecosystem. This is the moment where you kind of have to take a step back. These next three to five years are going to rely on our ability as an industry and as an ecosystem to come to the table together and figure out how we solve some of these bigger problems. Everybody's got to give a little bit, and we'll get a lot.

How could the scale of drug innovation strain traditional access models?

This is the trillion-dollar question. So what happens when there's a cure for type one diabetes, wet macular degeneration, multiple sclerosis – conditions with over a million eligible members, and if these therapies cost over a million dollars? That creates a perturbation to the financing system. The healthcare financing system can't react to that. This is the moment that we have to be more proactive and come up with much more thoughtful, progressive approaches at how to deal with the fact that these therapies aren't just incredible and really expensive, but they may not be that rare. When the cure for hepatitus C came out it created an incredible amount of energy, because there was this need and this desire to treat people, but also this recognition that $84,000 a pop when Sovaldi first came out would create a huge budgetary challenge. What happens when we're talking about orders of magnitude more expensive? I think that scale is really around the reservoir of patients that ultimately could be impacted by these therapies.

What hurdles delay specialty and rare disease patients from getting their medicine on time?

There are a lot of hurdles. It's a really complex process. I think one of the biggest and under-appreciated hurdles is the challenge that providers face as true advocates for their patients. Today, providers don't know a lot about how much volume they're going to get. It's not a predictable amount of volume. They generally buy and bill these therapies, so they have to put the money up front to pay for the therapy and get reimbursed later, creating cash flow problems. And they generally get reimbursed on what's called a single case agreement, so they often don't know even what they're going to get paid when that therapy is being planned. That creates incredible unpredictability for providers and it makes it hard to sort of invest up front into a sustainable business model. Being able to have a truly effective practice to deliver these therapies requires investment. So if providers are reticent to make that investment and concerned about how reimbursement's going to work, they play a much different kind of role as advocates for their patients. And I think until we really create a much more predictable and sustainable business model for providers, we're going to see a lot of unevenness in terms of who ultimately gets access to these treatments.

Which US health policy shift will disrupt market access most by 2030?

The FDA has been really progressive around trying to facilitate market access to therapies for rare disease and for cell and gene therapies in particular. They've also been really thoughtful around creating flexibilities around the platforms that are used to develop and manufacture these treatments. Ultimately, it's those kinds of policies that support and influence and reinforce manufacturing that allows us to really lower the cost of goods sold, and it's going to be a massive impact on our ability to sustainably and affordably get these therapies to patients.

References

1. American Society of Gene and Cell Therapy (ASGCT) and Citeline. Gene, Cell, & RNA Therapy Landscape Report Q4 2024. January 2025. https://www.asgct.org/global/documents/asgct-citeline-q4-2024-report.aspx
2. Precedence Research. Cell and Gene Therapy Market Size, Share & Growth Report. 2026. https://www.precedenceresearch.com/cell-and-gene-therapy-market
3. CGTLive. Pros and Cons of Various Reimbursement Models for Cell and Gene Therapies. https://www.cgtlive.com/view/pros-cons-reimbursement-models-cell-gene-therapies
4. Genetic Engineering & Biotechnology News. 2025 Cell & Gene Therapy Reimbursement Outlook. November 2024. https://www.genengnews.com/topics/bioprocessing/2025-cell-gene-therapy-reimbursement-outlook/