Key Takeaways: A Cystic Fibrosis Patient’s Journey

Understanding what a patient with cystic fibrosis (CF) goes through as part of managing his or her disease was the focus of a panel discussion at the 2021 PBMI Annual National Conference held in Orlando earlier this year.

During the panel discussion, moderated by Darren McLane, PharmD, director of clinical programs at AllianceRx Walgreens Pharmacy, participants heard from several vantage points – including a parent, physician, specialty pharmacist and advocacy organization – each of whom impact the journey of a patient with CF. 

Attendees of the session were able to walk away with a better view of the journey of a patient living with CF, and how clinical care teams and patient-advocacy groups provide critical services. 

Some key takeaways from the panel discussion:

The prognosis of CF has changed drastically over time as new treatments have emerged. 
Fifty years ago, people with CF were not reaching elementary school age, whereas today there are more adults with CF than children. The FDA approved the first therapy developed specifically for CF in the 1990s. Today, there is a suite of treatments available for people with CF. In 2012, the FDA approved the first CFTR modulator therapy for people with certain CF mutations. This was the first therapy to address the underlying cause of CF. Since then, three additional, highly effective modulators have been approved. For young children who begin disease-modifying therapy early, there can be profound changes to the course of the disease including the use of treatments that target the symptoms of the disease.

Diagnostic tests help determine genetic causes and offer advance treatments.
According to George (Marty) Solomon, MD, director at the Universitiy of Alabama at Birmingham Adult Cystic Fibrosis Program, clinicians use a combination of sweat chloride testing gene sequencing to determine the genetic mutations causing the patient’s disease and to offer advance treatments. Dr. Solomon sees patients routinely for quarterly check-ins with their multidisciplinary team, sometimes including other specialists such as otolaryngology, gastrointestinal, and endocrinology. He also sees patients as-needed to assess for declines in pulmonary function or other manifestations of the disease.

Specialty pharmacists play a key role in patient care. 
Nishita Hira, PharmD, CSP, clinical program manager at AllianceRx Walgreens Pharmacy, says it’s important to have a dedicated team of individuals who are knowledgeable about CF, the medications used to treat CF, and have the ability to navigate potential insurance challenges. Continuity of care is important – so working closely with the physician team to address changes in therapy or dosing adjustments or to inform of patient-reported issues is key.

Research is underway to continue finding ways to help patients.
There is a concerted effort underway in the CF research community to understand the long-term and real-world impacts of modulators on health status, quality of life, health care resource utilization, and other factors. The Cystic Fibrosis Foundation (CFF) is sponsoring several studies to evaluate the safety and effectiveness of withdrawing certain symptomatic treatments among individuals taking elexacaftor/tezacaftor/ivacaftor. The CFF is also making major investments in gene therapy and understanding and treating infections.

The bottom line: A patient’s healthcare team, along with advocacy groups, must work together to manage treatment and provide the highest quality of life. That means open communication, sharing data, asking questions and seeking solutions, together.

Panelists included:
• George (Marty) Solomon, MD, Director, UAB Adult Cystic Fibrosis Program
• Nishita Hira, PharmD, CSP, Clinical Program Manager, AllianceRx Walgreens Pharmacy
• Olivia Dieni, MHA, Health Systems Innovation and Navigation, Cystic Fibrosis Foundation 
• Mary Sullivan, mother of Betsy, diagnosed with CF at 18 months, now 21 years old

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