Cost: The Final Frontier

The signs on both these frontiers of medicine are promising, and although their introduction has been “only 5–10 years away” for as long as I can remember, this time many of the problems that had stymied development appear to have been overcome. We have even witnessed the first European regulatory approvals for a gene therapy-based treatment: in October 2012, the European Medicines Agency approved alipogene tiparvovec (Glybera), a gene therapy that compensates for lipoprotein lipase deficiency.

Ironically, once functional gene and stem cell therapies are approved, they may still fall at the biggest, and final, hurdle: affordability. In some cases, a single application of gene therapy might confer a cure. It’s a cure that has taken decades of eye-wateringly expensive research, development and clinical evaluation to make. All that has to be paid for. In alipogene tiparvovec’s case, where the market is small –  just a few hundred patients worldwide  – the per-patient treatment charges may be pitched as high as US$1.6 million/ €1.25 million.

The market size for most of the ophthalmic diseases currently under investigation for stem cell and gene therapies is orders of magnitude greater so per-patient costs might be expected to be orders of magnitude lower, right? Wrong! Economists tell us that the value of a product is simply what people are prepared to pay for it. The question is, is there a sweet-spot? Will there be a price for gene and cell treatments that will be affordable to those who manage healthcare budgets; that will satisfy the companies that have finally realized the promise of a new category of medicines; and that will be palatable to the general public, who will rightly pore over all aspects of these new therapeutics? Getting these negotiations right is one of the major factors Jim Taylor alludes to when he describes the challenge facing global health care as “the perfect storm”.

Vision is precious as its impairment is profoundly disabling; the value of an effective treatment or cure to patients – and to society – is great. What impact this will have upon the price on these agents once available remains to be seen. Now is the time for ophthalmologists to start thinking about this. What would you be prepared to spend on a novel gene therapy agent to improve your sight? To restore sight from blindness? What should our health insurers, whether private or public, be prepared to pay?