Although researchers have been experimenting with gene and cell therapies for decades, these therapies finally started to reach patients in 2017. These breakthroughs have unbelievable potential to not only treat rare forms of disease, but to actually cure patients. But as more of these types of therapies come to market and the treatable patient population expands, they have the ability to break the payer system, which is not set up to handle these costly, single treatment cures. That means restricting access to patients, which is unacceptable but is unfortunately already happening with the second approved CAR T-Cell therapy for cancer, which currently has a 200 person waitlist because most payers have not confirmed they’ll reimburse for it.
However, the latest news around the third gene therapy to be approved in the U.S., Spark’s Luxturna, which treats a rare form of inherited blindness, gives us reason to be hopeful. As reported by Endpoints News, Stat, and others, Spark’s pricing team has come up with a mix of rebates and proposed staggered payment models that are meant to overcome some of the barriers payers have in covering the one-time costs of the therapy. This means patients will have access to Luxturna, and Spark will be rewarded for successful treatment of the drug.
Spark’s pro-active efforts in announcing various creative reimbursement strategies with payers on the same day they announce the price of the drug is groundbreaking. Although the current payer system is not set up to handle one-time, curative treatments, payers like Harvard Pilgrim are willing to stand up and do what’s right for patients. They’re embracing the opportunity to work directly with manufacturers to find solutions, whether that’s value based partnerships or drug financing plans, that work – for patients, for payers, for manufacturers, and for the health care system.
I believe Spark’s willingness to pursue alternative payment models from the very beginning will be, and should be, a model for the coming wave of new gene therapies so that patients can access these life-changing medicines. When payers and manufacturers work together to benefit patients, the possibilities to change the course of disease are endless.
With 30 million people in the U.S. living with a rare disease, there is still much work to be done in treating and curing these patients. Don’t miss MassBio’s forum and Signature Event for Rare Disease Day, taking place February 28th, to learn more about the unmet needs of this population and how gene and cell therapies offer new hope to these patients.
– Bob Coughlin, President & CEO of MassBio