Orphan drugs aren’t always reaching the patients who need them, and an advocacy group known as the Cystic Fibrosis Engagement Network has a few ideas as to why. In a new white paper, the network details what’s standing in the way of patient access – and how addressing bureaucratic barriers could help the 30,000 Americans with this rare disease.
Entitled, the white paper pinpoints two key barriers to treatment:
1. Medicaid barriers. Due to the expense of managing the disease, many patients with cystic fibrosis – about 45 percent – depend upon Medicaid. Criteria and processes for determining prescription drug coverage can vary by state, where Drug Utilization Reviews and Pharmacy and Therapeutics committees often decide which patients get access to certain drugs. Officials do not necessarily communicate with actual patients or confer with experts, leading to decisions that can be “outdated and inconsistent with current practices.”
The paper describes a 2014 incident in Arkansas where Medicaid officials demanded a 12-month waiting period and required patients to show proof that their condition was worsening before they could receive the prescribed drug. “Arkansas ultimately covered the treatment,” the paper continues, “but only after a lengthy court process during which all the plaintiffs were hospitalized for cystic fibrosis-related issues at least once.”
2. Prior authorization requirements. The Arkansas example also points to the paper’s second access concern: prior authorization requirements.The technique requires patients and their families to appeal to the health plan for permission to access the medication their physician prescribed. Insurers may require paperwork, tests, or proof that a patient has failed on a less expensive therapy before they approve the requested drug.
The delay can be painful, even damaging, for patients with the progressive disease.
These barriers can block vital access to advanced and long-awaited treatments. For decades, patients were able only to treat the symptoms of the disease, using mucus-thinning drugs and vests that vibrate the chest cavity to help clear mucus buildup from the lungs and vital organs. In recent years, however, new treatments have been approved to target some of the genetic mutations that cause cystic fibrosis. For the patients with specific mutations, these treatments can significantly improve lung function.
For the sake of these patients, the paper urges, policymakers, advocates, patients and health care providers should work toward commonsense solutions that improve access to cystic fibrosis medications.
To learn more, read “Making Treatment Accessible to Cystic Fibrosis Patients.”