Experts Highlight Obstacles in Delivering New CF Therapies

Care for cystic fibrosis (CF) has improved substantially in recent decades, but there are still challenges ahead, particularly in ensuring that all patients have access to the best available care, according to a new study.

The Lancet, a renowned medical journal, commissioned nearly 40 experts to write a paper to explore the current state of care for people with CF, and to highlight obstacles that need to be overcome. The paper, “The future of cystic fibrosis care: a global perspective,” was published in the journal The Lancet Respiratory Medicine.

The experts’ most substantial recommendation was that CFTR modulators — a class of therapies that work to directly correct the aberrant protein function that causes CF — need to be made available to everyone who could benefit from them.

“Up to now treatment has always centred on the downstream consequences and symptoms such as mucus-clearing drugs and treatments, antibiotics and anti-inflammatories,” Scott Bell, MD, said in a press release. Bell is a researcher at the QIMR Berghofer Medical Research Institute in Australia, and co-author of the study.

“However, recent advances in CFTR modulator therapies to address the basic defects of the CF gene have been phenomenal, making targeted, gene-specific drugs a reality,” Bell said. “These CFTR modulator therapies have the potential to revolutionise the care of CF … they could effectively stop the disease from becoming fatal for as many as 80% of patients.”

These advances in treatment have been accompanied by advances in genetic testing and newborn screening. In addition to helping identify people with CF who don’t exhibit classical symptoms, these technologies have highlighted the breadth of those affected by CF. Once thought to be a disease that predominantly affected Caucasian people, it is now known to occur at similar frequencies among people with ancestry in Southeast Asia and Africa.

Despite these advances, obstacles remain in the delivery of treatment to those who need it, particularly with the cost of new therapeutics.

“CFTR modulators approved for use to date are highly expensive, which has prompted questions about the affordability of new treatments, and served to emphasise the considerable gap in health outcomes for patients with cystic fibrosis between high-income countries, and low-income and middle-income countries,” the researchers wrote.

This highlights the need to ensure that optimal care is provided in places that currently may not have the required resources and/or infrastructures. For instance, according to the team, China is thought to have the largest number of undiagnosed people with CF in the world, but currently there are few treatment options available in the country, and expertise in treatment is lacking.

Properly addressing these challenges will require the cooperation of lay organizations, governments, and pharmaceutical companies, the experts said.

And, as treatment continues to improve and patients live longer, healthcare providers will need to find better care strategies and patient monitoring, in order to minimize the burden of the disease for patients and their families.

“Put simply, if we don’t collaborate and bring to fruition our blueprint for care, hundreds of thousands of patients around the world could be at risk of missing out on the best available treatment,” Bell concluded.

The data from this research paper were presented recently at the European Respiratory Society Congress in Madrid, Spain.