PCORI has identified the need for large studies that look at real-life questions faced by diverse patients, caregivers, and clinicians. To address this need, PCORI launched the Pragmatic Clinical Studies initiative in 2014. Pragmatic clinical studies allow for larger-scale studies with longer timelines to compare the benefits and harms of two or more approaches known to be effective for preventing, diagnosing, treating, or managing a disease or symptom. They focus on everyday care for a wide range of patients. This research project is one of the studies PCORI awarded as part of this program.

This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.

What is the research about?

Chronic obstructive pulmonary disease, or COPD, is a lung problem that makes it hard to breathe. It often gets worse over time. Symptoms of COPD include shortness of breath, wheezing, chest tightness, and lack of energy. COPD affects at least 15 million Americans and is the third leading cause of death in the United States. COPD often affects people with low incomes and people who are elderly or part of a minority group. When COPD symptoms become worse than usual, they can disrupt patients’ lives for days, weeks, or longer. These serious episodes of COPD lead to about 700,000 hospitalizations in the United States each year.

Studies have shown that two drugs help reduce severe COPD episodes. Roflumilast is an anti-inflammatory medicine that can prevent serious COPD episodes and reduce hospital stays. Azithromycin is an antibiotic medicine used to treat serious COPD episodes and to reduce their frequency when taken long term. However, no studies have compared the two medicines to each other to see which works better for which patients.

The research team is running a large study to compare long-term roflumilast use with long-term azithromycin use. The team wants to learn how well these medicines work in preventing serious COPD episodes and understand the benefits and harms of long-term use.

Who can this research help?

People with COPD and their doctors can use information from this study to make decisions about which medicine to use to help prevent serious COPD episodes. People with COPD who may need stronger treatment because they have more frequent episodes and hospital stays may also find the information from this study helpful.

What is the research team doing?

The research team is recruiting up to 3,200 adults who were hospitalized for COPD in the past year. The team is assigning patients to one of two groups of 1,600 patients by chance. One group receives roflumilast. The other group receives azithromycin. The research team is asking patients in both groups to take their medicine for at least six months and up to three years. Patients in the study are not required to attend in-person follow-up visits or have lab work. Participants can report on COPD symptoms, medicine side effects, and overall well-being through the study’s web portal, or choose telephone follow-up from the research team. The team is collecting information at three and six months after the patient joins the study, and then every six months. The research team is looking at patients’ electronic health records to learn how long-term usage of the medicines compares in

• Helping patients avoid another hospital stay up to 36 months after they were released
• Reducing the likelihood of patients dying for any reason during the study
• Treating COPD in patients who smoke
• Treating COPD in patients who have quit smoking
• Helping patients maintain their physical, mental, and social well-being
• The research team is also comparing the side effects of each medicine.

People with COPD are helping design consent forms and other materials for the study so that they are easier for patients to read and use. Doctors and clinic staff provide advice on how to make it easier for patients to take part in the study. The research team is working with patient advisors to design the study and interpret the findings.

Research methods at a glance