In recent years, the introduction of modulator therapies that impact the cystic fibrosis transmembrane conductance regulator (CFTR) have altered the patient journey for people living with cystic fibrosis.

Whereas previous treatments addressed symptoms, CFTR modulators aim to fix the malfunctioning protein made by the CFTR gene. This malfunctioning protein leads to mucus buildup and infections due to an inability to regulate the flow of water and chloride in cells that line the lungs, digestive tract and other organs.

Not every person living with CF is currently able to use a CFTR modulator, whether due to their mutation type, age, drug interactions or a bevy of other factors. And the impact of these therapies can differ wildly depending on the individual. However, CFTR modulators, including the fourth and newest combination therapy approved in October 2019, offer relief and hope that enhance the quality of life and increase the life expectancy of many people living with CF.

A year-over-year analysis of Health Union’s first two Cystic Fibrosis In America syndicated research surveys – the 2019 survey finished fielding a few months before the most recent modulator was approved, and the second fielded in mid-2020 – uncovers important changing positive aspects of living with CF. This includes increased usage of CFTR modulators, confidence with treatment and optimism for the future. Importantly, findings are consistent whether reported by patients themselves or by a caregiver (e.g., of younger patients). 

In 2019, with three CFTR modulators approved for use, nearly half of respondents – both patients and caregivers – reported current CFTR modulator usage. Following the approval of the fourth modulator, that number increased to 68% in 2020.

In addition, the community’s outlook, on the whole, has improved significantly in just one year. Nearly two-thirds of 2020 patient respondents said they feel their CF is under control with their current treatment plan, representing a significant increase from the 41% who answered the same way in 2019.

Fewer Symptoms and Less Reliance on Other Treatments

Over the course of a year, patients and caregivers have reported less symptoms. In terms of respiratory symptoms, 2020 respondents were less likely than 2019 respondents to say that, over the previous month, they had experienced frequent lung infections (17% vs. 33%), frequent cough expelling thick mucus (33% vs. 63%) and wheezing or shortness of breath (37% vs. 58%). From a digestive and gastrointestinal standpoint, they were also less likely to experience bowel disturbances (18% vs. 24%) and poor weight gain (22% vs. 35%)

Treating the various CF symptoms also typically requires a cadre of specific treatments. Potentially related to fewer symptoms, this year’s respondents were also less likely than last year’s to say a host of other treatments were being used to treat CF. Specifically, they were less likely to be currently using oral (40% vs. 51%), inhaled (20% vs. 30%) and IV antibiotics (6% vs. 18%), as well as albuterol (74% vs. 81%) and inhaled corticosteroids (39% vs. 45%).

Although these responses could be due to a number of factors, including possibly fewer infections resulting from less public exposure during the COVID-19 pandemic, these numbers suggest CFTR modulator usage may contribute to less symptoms and less reliance on commonplace treatments.

Whether it’s due to less symptoms, fewer treatments needed or a combination of factors, 2020 patient respondents were more likely to have confidence in their ability to manage and complete their daily treatments (64% vs. 53%). And compared to just a third of 2019 patient respondents, 55% in 2020 said they feel optimistic about the long-term success of their treatment plan.

Future Outlook Continues to Improve

All of these changes – in symptoms and perspectives – point to an overall improvement in quality of life for the cystic fibrosis community. Chief among these perspective shifts is how people living with the condition view their future. Compared to 2019, when 56% of patient respondents said they were concerned about their future due to CF, that number dropped to 43% in 2020.

The introduction of more CFTR modulators, as well as a slew of other treatments and approaches to care, has contributed to an increase in estimated life expectancy. Based on data from 2018 (before the most recent CFTR modulator was approved), the Cystic Fibrosis Foundation estimated that people with CF who were recently born would live into their 40s.

With that estimate likely to increase, it makes sense that nearly two-thirds of patient respondents from this year’s Cystic Fibrosis In America survey said the increase in life expectancy for people with CF has given them hope, compared to just over half in 2019.

The 2nd Annual Cystic Fibrosis In America survey, which fielded from March 31 to July 28, 2020, featured responses from 404 patients and 230 current caregivers. The inaugural 2019 survey fielded from April 30 to Sept. 10, 2019, and it featured responses from 329 patients and 162 current caregivers.

For healthcare providers and pharma marketers alike, understanding the impact of CFTR modulators on the lives of people impacted by cystic fibrosis – specifically the need, due to a more positive future outlook, to navigate aspects of the patient journey that might not have been considered – can help improve healthcare experiences for these patients.

Engagement on Health Union’s growing portfolio of online health communities combined with large scale patient-reported data from its syndicated In America surveys can offer unique insight and understanding into nuances of the patient journey experienced by different groups of patients. Learn more about Health Union’s custom media, marketing research and clinical services that can help you create smarter, more effective solutions for patients and caregivers.