Proactive Education Accelerated Patient Access to a Life-Changing Eye Treatment

Too often, the journey from research and development to market is long for new medical treatments. Even amid the ongoing COVID-19 pandemic, we have observed challenges in distributing FDA-approved vaccines to states and communities quickly and effectively.

For patients with Thyroid Eye Disease (TED), the journey to realize a new therapy for the disease was unique because there wasn’t a single FDA-approved treatment option beyond cosmetic surgery before last year. But today, we can reflect on the lessons and work that allowed patients with TED to access the first and only FDA-approved treatment soon after it came to market. The speed at which the first approved treatment for TED was delivered to patients across the country – in the midst of the COVID-19 pandemic – demonstrates the value in educating industry, providers, and stakeholders across the healthcare system prior to FDA approval to pave a smooth pathway to patients. Through proactive education and engagement, we can ensure patients with TED and other complex medical conditions receive the therapies they need without delay between discovery, approval, and delivery.

TED is a serious, vision-threatening autoimmune disease that causes muscles and fatty tissues behind the eye to become inflamed, resulting in serious eye pain, double vision, light sensitivity, and even blindness. Effective management of TED requires early diagnosis and targeted treatment during a very narrow window of time, as patients will only respond to medication during the initial “active” phase of the disease. Once TED progression stops and becomes chronic, treatment options are limited to complex cosmetic surgeries with serious potential complications.

Prior to 2020, the U.S. Food and Drug Administration (FDA) had not approved a single treatment for TED. Before then, patients were prescribed steroids and immunosuppressant drugs that failed to reach the root of the problem, instead providing stopgap solutions to some of the symptoms, while presenting a range of debilitating side effects, including glaucoma, diabetes, bone thinning, and stroke. But on January 21, 2020, FDA approved the first and only treatment for TED, revolutionizing the way we treat and manage the rare disease.

In an historic vote, the FDA Advisory Committee unanimously recommended the approval of Teprotumumab (TEPEZZA) for the treatment of TED based on a comprehensive review of the treatment’s safety and efficacy data. Teprotumumab works by blocking the receptor that causes the immune system in TED patients to mistakenly attack the muscle and fat tissues behind the eyes, reducing eye bulging and double vision.

But FDA approval of Teprotumumab was only the first step in bringing the treatment to patients. Providers, patients, and advocates were acutely aware of the roadblocks and hurdles that have delayed patient access to other first-in-class treatments post-commercialization. To ensure that TED patients were able to access Teprotumumab soon after FDA approval, it was imperative that we apply lessons learned from other medical breakthroughs. Unlike other disease areas, TED represented an opportunity for the National Infusion Center Association (NICA) to work with drug maker Horizon Therapeutics to address those hurdles proactively.

Seven months prior to FDA approval of TEPEZZA, NICA and its infusion provider members leveraged lessons learned from previous post-commercialization challenges to provide a roadmap for approaching commercialization and patient access for an under-treated patient population. Together with Horizon, NICA demonstrated why outpatient infusion centers represent an efficient and affordable setting for office-based infusion of Teprotumumab, built relationships between prescribers and infusion centers to streamline the referral process, and educated payers to ensure that initial claims were not denied as investigational.

Ultimately, the foundational efforts to educate patient, prescriber, and provider stakeholders across the TED care continuum successfully reduced the time between FDA approval of TEPEZZA and patient access to the treatment. Patients received the first infusions of TEPEZZA in NICA’s member infusion centers shortly after the FDA announced approval, and those patients quickly began experiencing the life-changing benefits of the drug.

Rodney, a TED patient who experienced significant bulging of his eyes that prevented him from being able to drive or work due to reduced vision, received the first infusion of TEPEZZA provided in a private practice on February 12, 2020. After receiving his second TEPEZZA infusion in March 2020, Rodney described significant improvements, including reduced eye pressure and watering, reduced double vision, and improved eye alignment. “If this drug was not approved, they were talking about surgeries,” Rodney wrote. “They said they would have to go in and shave the bone. It would have been really bad. This drug was approved at the perfect time.”  Instead, because he received the right treatment at the right time, Rodney recovered sufficient sight to be able to drive and work again.

Rodney’s journey emphasizes the power of patient access to the right treatment at the right time – and is just one example. We have long heard powerful stories from patients about how TED has negatively impacted their vision and enjoyment of many aspects of life that we take for granted: sleep, daily function, the ability to work, the ability to drive, and the ability to lead a life that’s not defined by a medical condition.

For patients like Rodney, the importance of eliminating hurdles to accessing TEPEZZA or any new drug that comes to market cannot be overstated.

Many Americans were eager to put the challenges of 2020 behind and embrace 2021. But, in the midst of a pandemic – and during Thyroid Awareness Month this January – it is important to reflect on the breakneck speed at which we were able to change the course of TED for patients after drug discovery and why that happened. 

Patients long waited for an FDA-approved treatment for TED. Thanks to medical innovation and proactive outreach and education, we were able to bring a treatment to patients without unnecessary delays. Amidst the COVID-19 pandemic, and the rapid pace at which new treatments are coming to patients with rare and complex diseases, immediate and efficient access is changing the outlook for patients sooner than ever before.

Brian Nyquist is President and CEO of the National Infusion Center Association, a nonprofit trade association that serves as the national voice for non-hospital, community-based infusion providers that constitute a safe, more efficient, and more cost-effective alternative to hospital care settings for provider-administered therapies, like TEPEZZA and the monoclonal antibody therapies being used to treat high-risk COVID-positive patients under Emergency Use Authorization.