Spark Therapeutics’ FDA approved gene therapy, Luxturna, has a price tag – $850,000 (U.S.). Or $425,00 per eye for a rare type of blindness.

The therapy is approved for persons with confirmed biallelic RPE65 mutation-associated retinal dystrophy. In the United States, there are only 1000 to 2000 people who have this rare, genetic form of blindness. Luxturna replaces the mutated RPE65 gene in these patients with a normal copy of the RPE65 gene that can restore vision loss.

While the $850,000 price tag may seem outrageous, you should keep in mind 2 things. First, it is not just a treatment but almost a cure. It will improve sight to these people. Second, it should only require one injection in each eye and the patient is done. Compare that to the standard enzyme replacement therapies that can cost $150,000 – $400,000 a year for the rest of a person’s life. In other words, $850,000 is a bargain.

Right now, Luxturna is only approved in the United States but eventually, Health Canada will review the drug and if approved, the Canadian Agency for Drugs and Technologies in Health (CADTH) will determine if the gene therapy is eligible for reimbursement.

And when those reviews occur, a key question will be – who will those review panels listen to?  Will there be testimonies by patients, caregivers, scientists, and clinicians familiar with confirmed biallelic RPE65 mutation-associated retinal dystrophy? And if so, will those testimonies provide the reviewers with a complete understanding of the complexities of that rare condition and how it should best be managed?

The Foundation Fighting Blindness is a well organized foundation that has been helping blind Canadians for years. And let’s hope they are doing what they can right now to line up key opinion leaders familiar with biallelic RPE65 mutation-associated retinal dystrophy to present to those agencies when Luxturna is under review.

And when a treatment for your particular disease becomes available for review in Canada, will your organization be ready to provide expert opinions?  If you need assistance on interviewing those experts, please contact us. Providing expert opinions about a treatment is what we do best. Let us help you make that first and only impression with the regulators a good one.