Why Horizon Pharma Focuses on Rare Patients

Tim Walbert’s own medical experiences have shaped how he has built Horizon Pharma

In 2008, Horizon Pharma had just one investigational medicine. Duexis was being studied in arthritis and osteoarthritis patients to reduce pain and inflammation and to decrease the risk of developing stomach ulcers. At that time, stomach ulcers caused by chronic pain medication led to one hundred thousand hospitalizations and sixteen thousand deaths in the United States annually. By adding a protective agent for the gastrointestinal tract, Duexis reduced the rate of ulcers by 50 percent in clinical trials.

Since then, Horizon has experienced exponential growth. The company has added ten medicines, is growing by two hundred new employees each year, and had projected sales for 2016 of approximately $1.5 billion. Six of the company’s new medications address rare or “orphan” diseases—those that affect fewer than two hundred thousand people in the United States. Krystexxa is a medication for refractory, chronic gout, which was acquired through Crealta Pharmaceuticals. Two other medications are related to cystic fibrosis: Quinsair, which is used to manage specific chronic pulmonary infections due to pseudomonas aeruginosa in adults, and Procysbi, which is used to treat nephropathic cystinosis (which affects only about five hundred US patients). Both were acquired through Horizon’s purchase of Raptor Pharmaceutical Corp. The other products are Actimmune—which is used for chronic granulomatous disease and severe, malignant osteopetrosis —and Ravicti and Buphenyl , which are both used for urea cycle disorders.

A Ray of Hope

Krystexxa is emblematic of Horizon Pharma’s focus on rare diseases. It is the first and only FDA-approved medicine for refractory, chronic gout—a type of arthritis that occurs when uric acid built up in the blood remains high and inflammation persists even after treatment with conventional therapies. The medication is intended for adults who have tried or cannot take oral gout medications and still have high uric acid levels and signs and symptoms of gout. Refractory, chronic gout impacts approximately 40,000–50,000 people in the United States, which is only about three percent of all gout patients.

Focusing on medications for such rare diseases might seem like an unusual business strategy because they target such small populations and present limited commercial resources. However, it was for those very reasons that Horizon chairman, president, and CEO Tim Walbert chose to pursue the “orphan” space. Not only did he lead the team at Abbott Labs that launched Humira—the first fully human monoclonal antibody drug approved by the US Food and Drug Administration—but he also has several autoimmune diseases.

“As a patient myself, my passion is in the rare disease space, and I built a company around that focus,” Walbert says. “I know the value of innovation and the difference it can make in someone’s life. I’ve benefited personally from some of the very breakthroughs I’ve helped bring to market.”

He admits that, with the cost to develop new medications for smaller patient populations being in excess of $1 billion, developing investment for new products is challenging. However, by centering Horizon around conditions and patients that typically get very little attention, Walbert says Horizon is able to make a difference for patients who have no other treatment options.

Although Horizon has been able to create strategic advantages through the synergies between its six rare medications, its success has been built differently than companies producing “blockbuster” drugs. Horizon’s markets and associated communities are much smaller, so they lend themselves to developing much closer, more intimate relationships between the company, patients, their families, clinicians, and advocacy groups.

To help foster those relationships, Horizon invites patients and their physicians to speak to the entire staff each month. The company also brings in representatives from advocacy groups to provide educational seminars on their activities and areas of expertise.

“Direct engagement with patients, physicians, and advocates helps us understand what they go through and what their lives look like from every angle,” Walbert explains. “It ensures that everyone is reminded of why we come to work every day.”

Horizon also frequently participates in outside activities. Employees volunteered for various legs of the Friedreich’s Ataxia Research Alliance’s national rideATAXIA fundraiser, and the company regularly partners with groups such as the Arthritis Foundation, the Immune Deficiency Foundation, and the Gout & Uric Acid Education Society.

In addition to advocacy and charitable efforts, Horizon is also very active in providing financial support to the communities it serves. With the cost for rare medications ranging from $50,000 to $300,000 per year per patient, Walbert and Horizon work with partners to create reimbursement support that reduces patients’ out-of-pocket costs as much as possible—often to zero. The company also keeps copays for Horizon’s conventional drugs to no more than ten dollars.

“Direct engagement with patients, physicians, and advocates helps us understand what they go through and what their lives look like from every angle. It ensures that everyone is reminded of why we come to work every day.”

With all the media attention devoted to drug costs, Walbert says that it’s important to understand that the headlines have focused on gross prices, not the final cost to patients. He also says that a huge proportion of the higher costs are covered by discounts and rebates that go to payers and pharmacy benefits managers.

“Horizon’s increases last year were 3 percent or below for conventional medicines and about 5 percent for rare diseases,” Walbert says. “Our focus is always on doing the right thing for patients and covering the copay to make sure they get the medicines they need at the lowest possible cost or nothing at all.”

A direct result of those efforts is the company’s four-year-old HorizonCares program, which offers eligible patients that are having difficulty paying for their prescriptions a range of support services, copay assistance, and access to medicines for free or at reduced prices. In 2015, the company also provided more than $1 billion in additional patient support.

In a healthcare environment that continues to experience ongoing changes and is likely to see more contentious exchanges about its structure, Walbert believes the greatest contribution he, Horizon, and its employees can make is to stay focused on patient solutions.

“With so much divisiveness and misinformation, all we can control is getting our medicines to patients at the lowest possible costs,” he says. “If the media or
decision makers misunderstand that, we still stand by doing the right things for the right reasons.” AHL