At a Glance As they advance through clinical trials, gene therapies are coming closer and closer to reality for patients with retinal diseases

But with approaching treatments come approaching costs, and gene therapies can cost as much as US$1 million for a single treatment

Especially in disorders with small patient populations, like many ocular diseases, the price per dose of a new therapy must be high – which can prevent patients from accessing it

I propose a model of annuity payments for gene and stem cell therapies that would charge based on both the duration of treatment and the degree of efficacy

Gene therapy looks like it will transform the treatment of a number of ophthalmic diseases in the near future. Clinical trials in a number of retinal diseases, including Leber congenital amaurosis (LCA), the wet form of AMD, Stargardt disease/ Stargardt’s macular dystrophy (SMD), and Usher Syndrome 1b have shown promising results, with gene therapy providing some vision improvement for most who received it, without causing significant safety concerns. Of course, some questions about gene therapy still remain – will it deliver Ricki Lewis’ holy grail of “the forever fix” (1), or something less? And, with the first Western world approval of a gene therapy treatment – Glybera (alipogene tiparvovec) for lipoprotein lipase deficiency (LPLD) – having been gained, will ophthalmic therapies be next?

But these aren’t the only questions being asked. As ophthalmic gene therapy approaches marketing approval, likely within the next 2–3 years, one of the most significant concerns is cost. Glybera treatment may cost as much as $1.6 million per patient (2) but the extent of its worldwide market numbers in the hundreds – this is whole orders of magnitude smaller than the potential market size of the ophthalmic diseases currently under investigation. Nobody expects any of the proposed ophthalmic treatments to cost that much, as most of the disorders to be treated have much larger patient populations, but that unanswered question remains. What will ocular gene therapy (and, perhaps, stem cell treatment) cost? And how will patients and the medical community pay for it?