Overcoming Barriers to Treatment for Patients With Cystic Fibrosis

Patients with cystic fibrosis face a lifetime of time-consuming treatment. There are new breakthrough therapies that not only treat symptoms of the disease, but also the underlying causes, which represents a source of hope for patients. However, barriers to treatment still stand in the way, according to a new white paper from the Cystic Fibrosis Engagement Network.

The high cost of managing cystic fibrosis means that nearly half (45%) of patients depend on Medicaid, which covers doctor visits and FDA-approved medications. However, the white paper points out, coverage varies by state.

Some states involve medical experts to develop criteria that will determine what Medicaid will cover. Other states do not consult with stakeholders or medical professionals when determining coverage. A lack of communication between patients and experts and policymakers “can result in policies that are outdates and inconsistent with current practices,” according to the white paper. “Such a model hurts patients.”

In addition to barriers in Medicaid, patients with cystic fibrosis often face prior authorization issues. Obtaining prior authorization can be as simple as submitting documentation to as difficult and time-consuming as getting tests and treatments done before getting approved.

Another barrier is step therapy. Patients must show that they have already tried and failed on the insurer-preferred treatment before they can get the prescribed medication.

“This process, also known as ‘fail first,’ can be aggravating for doctors and patients in any situation,” the authors of the white paper wrote. “But in the case of cystic fibrosis, delaying treatment can have long-term or permanent effects.”

There is some movement at the state and federal levels to address the barriers patients face and level the playing field. Congress passed the Expanding and Promoting Expertise in Review of Rare Treatments Act, which helps expedite approval of rare disease drugs and mandates that experts are involved in the FDA’s approval process. States have begun addressing step therapy and prior authorization through legislation requiring insurers' criteria for implementing these barriers are not principally focused on cost control. Instead, the implementation of step therapy and prior authorization should be based on science-based guidelines.

“Tackling complex and rare diseases like cystic fibrosis requires cooperation,” the white paper concludes. “Pharmaceutical companies, insurance providers, and state Medicaid systems need to work with patients, families, and physicians to find commonsense solutions that prioritize the health of cystic fibrosis patients—and encourage continued treatment breakthroughs for this and other rare diseases.”