Researchers at Columbia University propose that respiratory-related hospitalization rates are an important “patient-centered” outcome measure that should be considered in clinical trials assessing treatment effectiveness in patients with idiopathic pulmonary fibrosis (IPF), according to a recent report.
The study, titled “Patient-Centered Outcomes in Idiopathic Pulmonary Fibrosis Clinical Trials,” was published in the American Journal of Respiratory and Critical Care Medicine.
In 2008, Japan approved the first treatment for IPF, Esbriet (pirfenidone, developed by Genentech). Esbriet wouldn’t be approved in the United States until October 2014, the same month and year the U.S. Food and Drug Administration (FDA) approved another approved treatment for IPF — Ofev (nintedanib; developed by Boehringer Ingelheim).
Supported by an extensive clinical trial program, these two drugs have been demonstrated to slow disease progression.
The identification of outcome measures for the evaluation of a treatment’s effectiveness is a major issue in any clinical trial.
Forced vital capacity (FVC; a measure of lung function) has become the primary endpoint used and recommended by the FDA for the evaluation of a therapy’s efficacy in clinical trials for IPF patients. Although FVC is linked to disease progression, it does not reflect the patient’s overall status.
“A pharmacologically induced reduction in the rate of FVC decline has no tangible benefit from the patient’s perspective,” the researchers wrote.
A recent study demonstrated that a more patient-centered analysis can help improve assessments of effectiveness through a better sense of patients’ reality. The study showed that Esbriet was associated with a lower risk of respiratory-related hospitalization after one year.
These findings suggested that a suitable treatment can result in reduced rate of respiratory hospitalizations. In addition, adding respiratory hospitalization as a secondary outcome in clinical trials can provide a patient-centered and clinically relevant outcome for IPF patients.
“We have come a long way in ten years. Patients are increasingly engaged in decision making about the role of IPF therapy in their lives,” the researchers wrote. It is important, then, to have patient-centered outcomes and clinically meaningful measures that can be easily perceived by patients.
“While it seems that FVC will remain the primary endpoint for many Phase 3 trials in the near future, confirmation of the usefulness of a respiratory hospitalization endpoint in these trials could influence the decision to use patient-centered primary endpoints in IPF trials,” the team concluded.
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