Spark Therapeutics reveals payment options for its one-time treatment of a rare retinal disease

A US-based gene therapy company, Spark Therapeutics, has announced a programme of improved access to its one-time gene therapy treatment, LUXTURNA, which is indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.

The treatment, which was approved by the FDA in December, is currently priced at $425,000 per eye — a reduction on what the investors had originally anticipated as being around $1 million per patient (for both eyes). The new payer programmes announced are aimed at improving patient access and would fall into three categories — an outcomes-based rebate, an innovative contracting model and a proposal to CMS.

At the moment, Harvard Pilgrim and affiliates of Express Scripts have agreed in principle to participate in these programmes with further discussions underway to include additional commercial insurers.

“To help ensure eligible patients have access to LUXTURNA, we are striving to bring the same level of innovation applied in development to the delivery of, and access to, this product,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “We believe that access to therapy is a shared responsibility among Spark Therapeutics, payers, health benefit providers, physicians and treatment centres. We have been working with stakeholders across the health care sector to help ensure that appropriate patients have access to a product that challenges all of the current conventions of how patients are treated, how products are delivered and how payments are handled.”

The payment options, agreed in principle between Spark Therapeutics and Harvard Pilgrim, include making LUXTURNA available under the outcomes-based rebate arrangement and the innovative contracting model that aims to reduce risk and financial burden for payers and treatment centres. Additionally, the company has reached an agreement in principle with affiliates of Express Scripts to enable the innovative contracting model.

According to a story published by The Guardian this treatment may become available on the NHS if given the green light by the European Medicines Agency (EMA). It has already been submitted for approval with the agency.

LUXTURNA is a gene-therapy indicated for the inherited retinal disease (IRD), biallelic RPE65 mutation-associated retinal dystrophy. This rare IRD, in nearly every case, will lead to complete blindness and there are currently no pharmacological treatments options for it. Spark Therapeutics gene therapy should only be administered to patients who have viable retinal cells as determined by their treating physicians.