With the advent of each New Year, my retina clinics become overwhelmed with a whirl of paperwork.  On an annual basis, starting each January, I reconsent my patients who need intravitreous injections for these procedures and also reevaluate the need for them to sign consents for benefits investigation, in case their insurance coverage for a specific medication has changed.  The flurry of new consent forms reaches its height in January and February as patients cycle through for their first visits of the year, and then quiets down gradually.

Although the annual paperwork slows down my clinic flow, I find it provides a valuable opportunity to have an in-depth discussion with each patient and review the pros and cons of each treatment option.  We discuss the various intravitreous anti-VEGF agents and steroid formulations.  Results from comparative effectiveness studies such as DRCR.net Protocol T, CATT, and SCORE2 are often relevant to these conversations.  Whereas some diseases seem to respond equally well to lower- vs higher-cost anti-VEGF alternatives, others do not.  For patients with diabetic macular edema in particular, the increased efficacy of aflibercept over bevacizumab in eyes with moderate or more severe vision loss must be weighed in the context of the financial cost and accessibility of each drug to individual patients.

But how do we and our patients decide what price to put on each line or letter of potential vision?  In my experience, the decision-making process can vary dramatically from patient to patient.  Whereas many of my patients would prefer not to incur any additional costs for their ocular treatments, I’ve had rare patients who are willing to pay the full out-of-pocket price for medications such as aflibercept and ranibizumab because of potential real or perceived benefits from using these drugs.  Fortunately, patient access programs are often available to ameliorate anti-VEGF drug–associated costs for patients who are underinsured or uninsured.

Headlines were made in January of this year when Spark Pharmaceuticals set the price for a single dose of their gene therapy drug, voretigene neparvovec, at $425 000 per eye, or $850 000 per patient.  This medication thereby gained the distinction of not only being the first gene therapy approved by the United States Food and Drug Administration for an inherited disease (biallelic RPE65-mediated inherited retinal disease), but also the most expensive medication sold in the United States.  Although the price tag seems extraordinarily high at first glance, it is within the range of other treatments for similarly rare diseases.  Furthermore, patients should theoretically only require a single dose administered to each eye  to receive long-term and potentially substantial visual benefit.  Still, the high unit cost per dose will necessitate creative solutions to ensure adequate patient coverage and drug accessibility.  Spark has announced options to help patients manage the exorbitant cost of this drug, including rebates for drug ineffectiveness, a partnership to allow patients to directly buy the drug from a pharmacy benefits manager, and the future possibility of multiyear payment plans.

As newer, more effective therapies are developed for indications across the range of retinal disease, medication prices will inevitably trend upwards.  In the face of burgeoning public health care costs, it will be critical for us as ophthalmologists to continue to perform well-designed comparative studies to best understand differences and similarities in outcomes between treatment alternatives.  Analyses that address the cost-effectiveness of specific treatments can help guide our approaches, but specific treatment decisions must be made with sensitivity to individual patient circumstances and preferences.

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