the offset of other concrete medical costs that never come to bear because a patient is no longer ill? Or should the calculation also include more abstract “societal” costs and benefits, such as people’s quality of life or productivity because they are able to rejoin the workforce?
Gene therapy developers should see warning signs in the ongoing response that insurers are having to Biogen’s (NASDAQ: [[ticker:BIIB]]) recently approved spinal muscular atrophy drug nusinersen (Spinraza), said Roger Longman, CEO of drug reimbursement specialist Real Endpoints. Insurers have limited access to the drug despite the FDA’s green light to use the drug for all SMA patients and the lack of any alternative treatments. They’re seizing on the fact that there isn’t clinical evidence of the drug’s value for a portion of SMA patients, because Biogen’s key trials that led to FDA approval only tested the drug in some, not all types of SMA patients. The takeaway, Longman said, is that “different audiences will have different values for the same therapy.”
Bring that back to Spark, then: Even if the FDA approves voretigene for all IRD patients with RPE65 mutations, for instance, could insurers draw a narrow reimbursement circle because Spark’s trials have only enrolled LCA2 patients, and not others?
Marrazzo feels Spark has a good case to make. In voretigene’s Phase 3 trial, 93 percent of patientshave gained back some amount of visual function, and the treatment has been proving more and more durable—in January, Spark released data showing effects lasting four years in some patients. “You multiply that out, and then you have a value point,” Marrazzo told Xconomy in January. A value of what, though? It’s one thing to calculate that a cure for, say, hepatitis C will save the healthcare system money per cured patient, based on the years or decades of treatments, hospital visits, and more that have been avoided. It’s another to say that people who regain vision are less expensive to take care of. Or, broken down into increments that a for-profit, shareholder-driven insurance company can understand: What is the yearly value of such a benefit? What is the length of the benefit Spark can promise? That’s tougher to calculate than a readily tangible impact on an insurer’s budget, like the yearly cost of drugs a hemophilia patient needs to prevent dangerous bleeds.
It’s critical, Marrazzo said this week, to hammer out those coverage details, no matter how unconventional they are, before voretigene gets to market: “What I don’t want to do is have an approval and then have patients waiting many, many years because of trying to get the perfect model.”
