Specialty Pharmacy for Cystic Fibrosis Improves Access to Medication

The Nationwide Children’s Hospital Cystic Fibrosis (CF) Program cares for a large population of patients of all ages with cystic fibrosis.  The program has participated in many recent clinical trials, including one that led to the FDA approval of Orkambi® (lumacaftor/ivacaftor), a CF transmembrane conductance regulator modulator.

“Across the country, getting people on Orkambi has been challenging,” says Karen McCoy, MD, chief of Pulmonary Medicine at Nationwide Children’s. “Upon FDA approval, we identified about 200 patients who were eligible for the drug. We currently, even with all of our interventions, only have about 80 patients taking it.”

According to Dr. McCoy, some of the barriers to adopting Orkambi® include side effects, limited benefit and difficulty in navigating the specialty pharmacy setting. The final barrier is a challenge associated with many CF drugs.

Specialty pharmacies are pharmacies that predominately obtain and dispense medications that are relatively expensive and/or have highly-specific monitoring requirements. Many CF medications are expensive, and Orkambi® is no exception at approximately $270,000 a year.

To increase patient access to Orkambi, Dr. McCoy and her team worked with the Department of Pharmacy at Nationwide Children’s to create the CF Specialty Pharmacy.

“In order to achieve a high level of organization and be mindful of our responsibility to guard against unwarranted health care costs, we determined a workable process for getting more patients on Orkambi,” Dr. McCoy explains. “That included an in-house specialty pharmacy to aid in getting patients their medication.”

Emily Middleton, PharmD, is the Advanced Patient Care Pharmacist in the CF Specialty Pharmacy.  Well integrated into the CF care team, she works to help patients access the drugs they need, provides thorough patient education, and closely monitors patients for adverse reactions and proper administration.

“With the success of distributing Orkambi through the CF Specialty Pharmacy, we quickly expanded it to the rest of our CF medications,” says Dr. McCoy, who is also professor of Pediatrics at The Ohio State University College of Medicine. “Now, many patients with CF are able to get their medications the same day at their clinic appointment.”

For most patients with CF, getting all of their prescriptions filled is a monthly ordeal that results in many phone calls and some level of frustration and aggravation, according to Dr. McCoy.

Many patients with CF need to use three to five pharmacies to get all of their medications, explains Dr. Middleton. Not all specialty pharmacies carry the medications these patients need, and insurance rules vary by plan and by medication. Depending on the insurer, it could be dramatically more affordable to use multiple pharmacies.

“The Nationwide Children’s Specialty Pharmacy is the one of the few pharmacies in the United States that has access to all CF medication,” says Dr. Middleton. “We are working with insurers to get more contracts in place so that we can truly be the only specialty pharmacy our patients with CF need.”

Even when an insurer won’t allow the prescription to be filled at Nationwide Children’s CF Specialty Pharmacy, Dr. Middleton and her team offer themselves as a resource to families.

“We know how to navigate the system,” Dr. Middleton says. “I have spent hours on the phone with other specialty pharmacists advocating for my patients and educating others about billing and insurance issues.”

“The most rewarding part of this work is to help patients get the medications they need when they need them,” she concludes.

Dr. McCoy and her team in the clinic are monitoring the impact the specialty pharmacy is making on patients.

“We have early signs of increased adherence, and anecdotal evidence of reduced frustration and difficulty for families to get the medications they need,” says Dr. McCoy. “As we move forward, I expect to find that the specialty pharmacy leads to a quantifiable improvement on the outcomes for some patients.”