Will Shrank, MD, in December 2025 unveiled a new plan for managing the high costs of an expected future wave of gene-based treatments as many drugmakers seek to more broadly apply the kind of innovation seen recently in cancer and sickle cell disease.

Shrank is the chief executive and co-founder of Aradigm, a company formed to create a new health benefits platform for cell and gene therapies. Aradigm said it has been seeking advice from 15 of the largest US employers as well as insurers about how to create a system for pooling and capping the expected financial risk that comes with more cell and gene therapies.

Aradigm’s efforts represent a new direction in managing the high costs of cell and gene therapies, but it’s far from the only example of innovation being tried in this field.

And that’s a good thing, according to Shrank, whose previous work includes key posts at the Innovation Center of the Centers for Medicare and Medicaid Services (CMS), Harvard Medical School, and the healthcare giants Humana and CVS.

“This is not a problem that any individual employer or insurer can solve on their own. We really need collective action. You really need to pool resources. We really need to create a sort of a new kind of platform that asks everybody in the ecosystem to come to the table and create some alignment, to actually make sure that these therapies are sustainable,” Shrank told Medscape Medical News.

“There’s not going to be some simple single silver-bullet solution on this,” Shrank added.

The US healthcare system already is struggling to manage issues of cost and access for about two dozen approved cell and gene therapies, including the chimeric antigen receptor (CAR) T treatments used for cancer.

James Chambers, PhD, MPharm, MSc, of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, Boston, and coauthors in September published an analysis in JAMA of current coverage policies in commercial health plans for 25 cell and gene therapies approved in the US.

They found insurers rarely outright denied use for approved indications, although they did impose some restrictions, such as implementing step therapy.

Gene and cell therapies can cost in the range of $500,000 to $3 million, creating a high one-time cost for insurers for patients who may then switch to a competitor. Chambers and his coauthors said the analysis shows the challenges that insurers face.

“It’s not that the payers are trying to be profiteering in any way,” Chambers said. “It’s maybe a microcosm of the broader challenges that payers face, given this sort of wonderful technology that we have being developed. We need to find a way to make sure patients have appropriate access.”

Some researchers have proposed using a single-payer approach for covering these treatments.

In addition, the high costs of cell and gene therapies are triggering an increased focus on outcomes. The Trump administration has opted to continue a Medicaid test program for covering new gene-based sickle cell disease treatments that the Biden administration initially proposed.

CMS in July announced that 33 states, along with the District of Columbia and Puerto Rico, will participate in the Cell and Gene Therapy Access Model.

Under this agreement, CMS negotiated outcomes-based agreements for gene therapies for people with sickle cell disease with Vertex Pharmaceuticals for exagamglogene autotemcel (Casgevy) and bluebird bio (now called Genetix Biotherapeutics) for lovotibeglogene autotemcel or lovo-cel (Lyfgenia).

Cell-Based Therapies for Autoimmune Diseases

More cell and gene therapies are expected to reach the market within a few years, with many products in testing aimed at autoimmune diseases. For example, Elena Schiopu, MD, the director of Clinical Trials for the Division of Rheumatology at the Medical College of Georgia at Augusta University, Augusta, Georgia, is involved with six CAR T-cell-based clinical trials for patients with myositis, scleroderma, systemic lupus erythematosus (SLE) and lupus nephritis, and antineutrophil cytoplasmic antibody-associated vasculitis.

“I want to call it a revolution, but to me it’s more like an explosion right now, a big bang, right?” Schiopu said. “That means a great deal of excitement but a little bit of confusion as well.”

In the past, advances in rheumatology tended to arrive in a more sequential fashion, she said.

For example, the FDA approved etanercept as the first monoclonal antibody for rheumatoid arthritis in 1998, and then adalimumab followed in 2002. Rheumatologists had time to learn, adapt, and incorporate new modalities in their clinical practices.

Bristol Myers Squibb, a leader in CAR T-cell treatments for hematology, is seeking to apply this approach to several autoimmune disorders, including SLE. It describes its CD19 NEX-T platform as a potential treatment to address the “underlying cause of multiple severe/refractory autoimmune diseases by inducing immune reset.”

Data presented at American College of Rheumatology (ACR) Convergence 2025 Annual Meeting on CD19 NEX-T included results for patients with SLE with active, severe organ involvement and highly refractory to prior treatment.

Novartis, which sells a CAR T-cell therapy for use in cancer, also presented data at ACR 2025 on its experimental drug, YTB323 (rapcabtagene autoleucel), which is being tested for both hematology uses and for SLE.

Also seeking to develop new CAR T-cell therapies for autoimmune diseases are smaller companies like Cabaletta Bio. The FDA has indicated a strong interest in this field, with several companies announcing that the agency granted regenerative medicine advanced therapy designation for their products, indicating the agency sees them as potentially filling unmet needs in therapy.

At this time, patients with autoimmune diseases such as SLE often adhere to difficult medication regimens, sometimes involving a dozen pills a day. These regimens also can increase people’s risk for infection, said Elizabeth Volkmann, MD, MS, a rheumatologist and the director of the Scleroderma Center at the David Geffen School of Medicine at the University of California Los Angeles.

There’s understandable excitement about “this promise of resetting the immune system so that you wouldn’t have to take these therapies in the long run,” Volkmann said.

“But there are a lot of unanswered questions,” she added. “We don’t know what the long-term safety is of doing this. We don’t know whether in the future, someone will have to go back on their immunosuppressive therapies or how long will remission be sustained.”

The likely costs involved also concerned Volkmann.

“These are tremendously expensive protocols to execute,” she said. “So even if this is something that is really revolutionary in the treatment of autoimmune disease, would it be something that’s accessible to all patients?”

Cheaper Off-the-Shelf Therapies?

Rheumatologists like Volkmann are watching with great interest as companies attempt approaches that might bring down the costs of cell and gene therapies.

Several companies are trying to develop off-the-shelf versions, reducing costs now incurred in creating individualized CAR T-cell treatments. For example, San Diego-based Fate Therapeutics intends to use an induced pluripotent stem cells platform to produce off-the-shelf treatments, using a master bank of cells, which could be genetically engineered and indefinitely expanded in culture.

Fate’s experimental drug, FT819, is now in phase 1 testing. In April, the FDA gave the regenerative medicine advanced therapy designation to FT819 for SLE.

“We are targeting a path to getting an approved product over the next couple of years if everything falls our way,” Victor Hong, MPH, MBA, vice president of corporate development and finance for Fate Therapeutics, told Medscape Medical News. “We’re probably not on insurers’ radar yet but hopefully will be soon.”

Before joining Fate Therapeutics in 2021, Hong worked at Bristol Myers Squibb as vice president for hematology strategy, where he worked on commercialization strategies, including launch and reimbursement for CAR T-cell therapies, idecabtagene vicleucel (Abecma) and lisocabtagene maraleucel (Breyanzi).

The first CAR T-cell products such as lisocabtagene and idecabtagene are derived from patients’ own T cells, leading to a complex and complicated manufacturing of the treatment.

In contrast, Fate Therapeutics intends to use the induced pluripotent stem cells platform to produce off-the-shelf treatments, using a master bank of cells, which could be genetically engineered and indefinitely expanded in culture.

“We can scale this up,” Hong said. “So our cost of goods is around $3000 per dose, which is multiples lower than cost of goods for currently available CAR T drugs.”

Shrank reported receiving personal fees from Andreessen Horowitz (also known as a16z, an investment firm that invests in many technologies, including artificial intelligence and related companies) and Humana; equity from RxCap, Nest, UpDoc, Thrive Health Tech, and AnewHealth; personal fees from Walgreens Boots Alliance and Weight Watchers; and nonfinancial support from the National Committee for Quality Assurance outside the submitted work.

Schiopu reported receiving research funds from Boehringer Ingelheim, Bristol Myers Squibb, Johnson & Johnson, Novartis, Amgen, Priovant, EMD Serono, and Eli Lilly, and consulting fees from Novartis.

Volkmann reported received consulting fees and research grants from Boehringer Ingelheim, GSK, AbbVie, and Bristol Myers Squibb. She also reported receiving research funding from the National Institutes of Health and the Kadmon, Horizon, Prometheus, aTyr Pharma, and AstraZeneca.

Kerry Dooley Young is a freelance journalist based in Washington, DC. She has covered medical research and healthcare policy for more than 20 years.