Dr. Mehmet Oz, head of the Centers for Medicare & Medicaid Services, visited Cleveland on Tuesday to explain how new federal guidelines would ease approval of gene therapy treatments for people with rare diseases.
The two-pronged approach, details of which were first unveiled in February, would speed the approval process of gene therapies and make it easier for Medicaid patients to access resulting care, said Oz, who was joined during the visit to Cleveland Clinic by Acting FDA Commissioner Kyle Diamantas.
Diamantas said the draft FDA guidelines would allow institutions such as Cleveland Clinic to bring new products to market under an existing Biological License Application without having to go through the usual process of submitting new clinical data.
“It’s going to ... streamline the process economically and also make those therapies quicker for patients,” said Diamantas.
The Centers for Medicare & Medicaid Services said the agency plans to expand current partnerships with drug makers treating conditions such as sickle cell disease to cover treatments for other rare disorders, said Deputy Administrator Abe Sutton, who was also present at Tuesday's event.
“We’ll then offer those terms to states and let states opt in instead of negotiating one-by-one with other manufacturers,” he said.
Oz said the initiative would allow states that have a relatively small number of patients with rare diseases the chance to get better pricing from drug manufacturers.
“We get everyone a fair deal, with the innovation not being curtailed, so that we can continue to build better solutions,” Oz said.
Cleveland Clinic has led several research initiatives in using new gene therapies to treat inherited blood disorders such as sickle cell anemia and thalassemia.
Patients and families have long been frustrated by the slow approvals process for new rare-disease treatments, which can cost millions of dollars per patient.
Diamantas said only around 5% of the more than 1,000 rare diseases affecting Americans have an FDA-approved treatment.
