AMD Awareness Month: For drugmakers, value is the name of the game
February is AMD/Low Vision Awareness Month, and a perfect time to think about the state of ophthalmologic therapeutics. In a market driven by new metrics and rigorous guidelines set forth by payers, value is now the name of the game.
Implementing pay-for-performance pricing parameters and strict prior authorization processes is an approach that payers are using to curtail costs and limit reimbursement. One thing is clear, however: In areas where there is a clear unmet medical need or room for substantial improvement, payers are much more likely to pay for newly approved innovative drugs—especially when there are millions of people involved.
Ophthalmology is a vast reservoir of unmet medical needs
Ophthalmology is one example of this phenomenon. "There is still an enormous amount of unmet need in ophthalmology," said Nouhad Husseini, Vice President of Business Development at Regeneron. "There have been very few companies making significant investments in this therapeutic area."
Eylea, which is used to treat age-related macular degeneration (AMD and diabetic macular edema (DME), happens to be the “most successful product in the history of ophthalmology,” according to Husseini, who was a panelist at an ophthalmology-focused panel at BIO CEO in New York City last week.
Yet, despite the fact that Eylea is the market leader in the $15 billion anti-VEGF market, Husseini recognizes the need for more patient-friendly treatment options.
Finding new targets
"The anti-VEGF approach is great in that it has changed how AMD and DME are treated," said Husseini. "However, there continues to be an unmet medical need associated with treatment burden. Roughly 15 million Americans have AMD. When these patients are treated, they have to come in for injections into their eyes every month or two. Clearly, there is need for improvement in how anti-VEGF treatments are delivered.
"The other unmet need is finding new mechanisms that could replace anti-VEGF therapy and require fewer treatments."
Towards that end, Regeneron is exploring new targets for treating AMD, with a focus on ANG2 and PGEF. Husseini explained, "We are looking for robust clinical data so that we can make a determination. The question being: ‘Is this really an advance in the standard of care?’ We have a lot of shots on goal."
A new MOA in glaucoma?
Glaucoma affects more than three million Americans. And Inotek, a MA-based biotech company, is moving into late-stage trials with a first-in-class drug (trabodenosan) which, like the currently available treatments—beta-blockers and prostaglandins—is designed to lower intraocular pressure (IOP).
Available treatments have been around for almost 40 years. Although they are efficacious at the beginning of therapy, their IOP-lowering efficacy declines over time.
"Trabodenosan has a brand new mechanism of action (MOA) for treatment of glaucoma," said Inotek CEO David Southwell. "Trabodenosan is a very selective adenosine-1 receptor agonist, which has already been proven safe and tolerable in phase 2 trials.
"In addition, efficacy data from these trials showed a substantial additive effect in terms of lowering IOP when trabodenosan was added to latanoprost."
Additive therapy can advance the standard of care
Given the fact that half of patients who are treated with a first-line agent for glaucoma end up needing a second-line agent as well, Inotek’s drug represents an opportunity to fill a longstanding medical need.
Southwell also pointed out that compliance with eyedrop-based regimens for glaucoma is only 25% to 50%—meaning that up to half of patients don’t follow the treatment regimen—a factor that contribute to the poor outcomes associated with glaucoma and one of the reasons that it continues to be a leading cause of blindness in the U.S.
"There are some side effects associated with current treatments, including redness," he said. "Our goal is to develop a drug without side effects or a novel delivery system. We think that our side effects with trabodenosan compare well with placebo. The FDA is being pretty tough with eye drop therapies right now, but we have reason to believe that trabodenosan offers a strong possibility of FDA approvability.”
Broadening the R&D pool
Regeneron and Inotek are two companies focused on advancing the standard-of-care in ophthalmology, and there are others (including Shire, which has been attempting to gain approval for liftegrast for treatment of dry eye disease.
Regardless, there is plenty of room in ophthalmology for other companies to jump into the drug-development fray. In fact, there are currently no approved treatments for uveitis and dry AMD.
Gary Eakin, PhD, Vice President of Scientific Affairs at the BrightFocus Foundation, emphasized the need for development of new drug-delivery technologies to improve compliance and outcomes.
"If we develop new delivery technologies, then we can identify new druggable targets, including back-of-the-eye targets where there has been almost no progress," he said.
In the final analysis, although the paradigm for getting payers to cover newly approved drugs has shifted towards a more stringent approach, drugs that fill an unmet need in the ophthalmology space and are approvable are also likely to be reimbursable. It all comes down to value.