More than 30,000 Americans are challenged to breathe daily by a condition that doesn’t get the attention it deserves: cystic fibrosis.
A Rare and Complex Condition
A rare genetic condition, cystic fibrosis affects the lungs and digestive system as well as other organs. It leads to thick, sticky mucus buildup, which makes it difficult to breathe and increases the risk of lung infection.
Patients living with cystic fibrosis work hard to stay healthy, and many have their daily routines dictated by treatment sessions. Specialized care can slow disease progression, helping many people with cystic fibrosis to live longer.
The Need for Continued Breakthroughs
Scientific advancements have transformed cystic fibrosis care, with newer therapies providing greater relief from symptoms and restoring quality of life. However, removing insurance barriers like step therapy, prior authorization and high cost sharing for specialty tiered medications can improve access to currently available options.
All treatments are not effective for all patients, highlighting the continued need for investment in new therapies that may be formidable against one or more of the disease’s variations. Expanding participation in clinical trials will provide more patients with the opportunity to be involved in this process and can ease access to the newest breakthrough treatments.
Advocacy, Awareness Critical for Rare Conditions
Patients and advocacy organizations play an important role in advancing access and addressing gaps in care.
The Cystic Fibrosis Engagement Network’s Health Care Bill of Rights, for example, highlights the essential needs of those living with the condition, from robust insurance coverage to policies that encourage innovation. This is particularly important for conditions like cystic fibrosis, which is considered an “orphan disease” because it effects less than 200,000 people.
Increasing awareness through observances like Rare Disease Week, which starts February 24, are also critical to keeping the patient experience top of mind. The observance is an opportunity to continue raising awareness about cystic fibrosis and the over 7,000 rare diseases and the need for innovation and access for every patient.
