This past quarter saw two new gene therapies: Novartis AG subsidiary AveXis, Inc.'s Zolgensma (onasemnogene abeparvovec-xioi) received FDA approval May 24 for the treatment of spinal muscular atrophy, and bluebird bio's Zynteglo (autologous CD34+ cells encoding βA-T87Q- globin gene) received conditional marketing authorization from the European Commission for transfusion-dependent beta thalassemia.
While only a handful of therapies in the broader regenerative medicine/advanced therapy (RM/AT) space are available globally, a new report shows that is likely to change, as there are more than 1,000 products in the pipeline, AIS Health reported.
The Alliance for Regenerative Medicine published the report, titled Quarterly Global Regenerative Medicine Sector Report: Q2 2019, on Aug. 1. It shows there are 1,069 clinical trials using specific RM/AT technologies, which include gene therapy, gene-modified cell therapy, cell therapy and tissue engineering. Ninety-four of those products are in Phase III trials.
While Zolgensma's $2.125 million price for a one-time infusion makes it the costliest drug on the planet, many other newer RM/AT products are certainly not cheap. Though many manufacturers are offering various reimbursement schemes, including rebates if a therapy doesn't work, other outcomes-based deals and multiyear pay-over-time payment options, experts note that many barriers exist in the execution of these strategies.
"In principle, spreading the cost over a five-year period and putting the cost installments at risk based upon efficacy is a good approach," says Winston Wong, Pharm.D., president of W-Squared Group, about Zolgensma. "However, the devil is in the details. Do we have the systems in place that have the capability to administer a five-year contract?"
According to Wong, "From the manufacturer perspective, a value-based contract implies that no payment would be made if the patient relapses or passes on. Systems are not in place to have the ability to track the patient once therapy is administered."