Federal health officials gathered at Cleveland Clinic to lay out plans for speeding up approval of gene therapies and reshaping how Medicaid pays for them, a shift that could matter for patients facing treatments that cost millions of dollars per patient. Dr. Mehmet Oz, who leads the Centers for Medicare & Medicaid Services, visited Cleveland to explain federal guidelines easing gene therapy approval for rare diseases.
Oz was joined by Cleveland Clinic CEO Dr. Tomislav Mihaljevic, acting FDA Commissioner Kyle Diamantas and CMS Deputy Administrator Abe Sutton, according to Ideastream. The venue was no accident: Cleveland Clinic has led research initiatives using gene therapies for inherited blood disorders including sickle cell anemia and thalassemia, putting it at the center of a national conversation about how to get these treatments to patients faster and cheaper.
What the New Federal Guidance Would Change
At the heart of the announcement is draft guidance from the Food and Drug Administration aimed at speeding up development of gene therapies that rely on genome editing. When finalized, the guidance would outline how sponsors can use publicly available information and established platform knowledge — including chemistry, manufacturing and controls data, nonclinical study results and clinical information — to streamline regulatory submissions, according to the FDA.
A related FDA recommendation describes leveraging prior chemistry, manufacturing and controls knowledge, along with nonclinical and clinical data, as a way to expedite product development. The agency says this approach to using prior knowledge may be particularly helpful in certain gene therapy development efforts, per the FDA's guidance document on the topic.
Per Ideastream's reporting on the Cleveland event, Diamantas said the guidelines would allow institutions such as Cleveland Clinic to bring new products to market under an existing biological license application without submitting new clinical data, and that the approach would streamline the process economically and get therapies to patients more quickly.
Medicaid's New Approach to Paying for Pricey Treatments
The policy push isn't limited to approvals. Sutton said CMS would offer treatment terms to states and allow them to opt in, rather than forcing each state to negotiate separately with drug manufacturers, the Ideastream report states. That builds on a Medicaid payment model already in motion: CMS has been negotiating the cost of sickle cell gene therapies with drugmakers on behalf of state Medicaid programs and holding companies accountable for treatment outcomes, according to NPR.
Under that arrangement, participating states receive discounts and rebates from drugmakers if the treatments don't work as promised, NPR reported. Thirty-three states, the District of Columbia and Puerto Rico have signed agreements to participate in the Cell and Gene Therapy Access Model, according to the Centers for Medicare & Medicaid Services, and the program could run for as long as 11 years.
Oz said the initiative would give states with relatively few rare-disease patients access to better drug-maker pricing, and that federal guidelines would speed gene-therapy approvals while making the resulting care easier for Medicaid patients to access, per the account relayed by Ideastream. He characterized the approach as one that would give everyone a fair deal while preserving innovation.
Why Access Still Lags Behind Approval
The gap between approval and access remains wide. There are two FDA-approved sickle cell gene therapies on the market, one costing $2.2 million per patient and the other $3.1 million, not counting the extended hospital stay the treatment requires, according to NPR. Patients often face a fragmented and uneven healthcare landscape where treatment costs, a complex treatment process, provider availability and access to specialized treatment centers vary widely depending on where they live, CNBC reported.
The entire therapy process can take up to a year to complete, and the conditioning process often causes permanent infertility or sterility, per the same CNBC account. The FDA approved Casgevy and Lyfgenia in 2023 as the first two gene therapies for patients with severe forms of sickle cell disease, but uptake has been slow: by the end of 2025, only 64 patients worldwide had received Casgevy infusions, while another 147 had undergone their first cell collection for the treatment, according to maker Vertex Pharmaceuticals' full-year earnings report cited by CNBC.
Cleveland Clinic has its own stake in this research. The health system has enrolled patients in a multicenter trial of EDIT-301, a single-dose gene-editing cell therapy for sickle cell disease, according to a Cleveland Clinic newsroom announcement. That study initially planned to enroll 40 adult patients ages 18 to 50 with severe sickle cell disease, with potential expansion to adolescents.
A Broader Shift in FDA Posture
Industry trackers have noted a broader shift underway at the FDA. The agency issued 79 regulatory decisions in the first half of 2026, down from 85 during the same period in 2025, even as novel therapy approvals rose from 19 to 26, according to BioSpace. BioSpace described the agency as taking a seemingly more open stance on rare disease therapies under Diamantas, though not every application has cleared the bar — the FDA rejected REGENXBIO's RGX-121 Hunter syndrome gene therapy in February, citing uncertainty about enrollment criteria, external controls and a surrogate endpoint.
Whether the sickle cell payment model becomes a broader template remains an open question, though NPR has reported that if it works, it will probably lead to similar arrangements for other costly therapies, particularly those treating rare diseases. For now, the Cleveland Clinic event signals that federal regulators and payers are trying to tackle both halves of the problem — getting treatments approved and making sure patients can actually afford and access them — at the same time.
