Citations

Abstract Rationale In October 2014, the antifibrotic medications pirfenidone and nintedanib became the first medications approved by the U.S. Food and Drug Administration for use in patients with idiopathic pulmonary fibrosis (IPF). Since approval, there has been no nonregistry analysis of the real-world adoption of these medications in everyday clinical practice. Objectives To evaluate the adoption, persistence, and out-of-pocket (OOP) costs of pirfenidone and nintedanib since their approval in the United States in 2014. Methods A retrospective cohort analysis was performed by identifying privately insured and Medicare Advantage beneficiaries with IPF. We then split the patients into three cohorts: those who were untreated and those who filled a prescription for either pirfenidone or nintedanib between October 1, 2014, and July 31, 2019. The primary outcome was adoption of the medications. Secondary outcomes included medication persistence and prescription drug costs. Results A total of 10,996 patients with IPF were identified in the data set. A minority of patients (26.4%) with IPF identified in the cohort had started either medication since approval in 2014, with the adoption of both medications being comparable at around 13.2%. Those receiving the medications were younger (72 vs. 73.9 yr; P < 0.0001) and healthier (3.9 vs. 4.9 comorbidities; P < 0.0001) than those not receiving treatment. Men were significantly more likely to receive treatment than woman (30.0% vs. 21.9%; P < 0.0001). Among treated patients, 42.8% discontinued the medications during the study period. Patients’ OOP expenses per month were high for both drugs (mean, $397.51 for nintedanib; mean, $394.49 for pirfenidone). Conclusions The adoption of both the antifibrotic medications in the United States in everyday practice has been low since approval and may be associated with the high OOP cost.

Abstract The Accreditation Council for Graduate Medical Education (ACGME) Milestones are a systematic assessment framework for medical trainees within the six core competencies of practice. Their use by internal medicine subspecialties, including semiannual reports to the ACGME, was mandated beginning in 2014. The Milestones, which were based on specific, observable behaviors, improved upon the prior subjective, global comparisons of each fellow with an “average” fellow in his or her field and served the goals of competency-based medical education. However, the original set of Milestones has proven challenging to apply and interpret. Part of the challenge stems from the use of identical Milestones across all medicine subspecialties, which led to unclear relevance of the patient care and medical knowledge domains to the practice of pulmonary and critical care. This also precluded their use for individualized feedback or development of a learning plan for fellows. In addition, verbose behavioral descriptors, which were designed to provide specificity, ultimately led to rater fatigue among assessors and clinical competency committees. Therefore, the ACGME convened committees for each of the medical subspecialties to revise the original Milestones in an effort to improve subspecialty relevance, minimize educational jargon, and simplify the current iteration. New patient care and medical knowledge Milestones were created to be subspecialty specific and improve utility. The remaining four Milestones were developed as a common set of shorter Milestones, harmonized across specialties. For pulmonary, critical care, and combined fellowship programs, the resulting Milestones 2.0 aims to simplify the use, implementation, and interpretation of this framework for program directors, trainees, and society.

Abstract Rationale Since their approval, there has been no real-world or randomized trial evidence evaluating the effect of the antifibrotic medications pirfenidone and nintedanib on clinically important outcomes such as mortality and hospitalizations. Objectives To evaluate the clinical effectiveness of the antifibrotic medications pirfenidone and nintedanib in patients with idiopathic pulmonary fibrosis. Methods Using a large U.S. insurance database, we identified 8,098 patients with idiopathic pulmonary fibrosis between October 1, 2014 and March 1, 2018. A one-to-one propensity score–matched cohort was created to compare patients treated with antifibrotic medications (n = 1,255) with those not on treatment (n = 1,255). The primary outcome was all-cause mortality. The secondary outcome was acute hospitalizations. Subgroup analyses were performed to evaluate mortality differences by drug. Measurements and Main Results The use of antifibrotic medications was associated with a decreased risk of all-cause mortality (hazard ratio [HR], 0.77; 95% confidence interval [CI], 0.62–0.98; P value = 0.034). However, this association was present only through the first 2 years of treatment. There was also a decrease in acute hospitalizations in the treated cohort (HR, 0.70; 95% CI, 0.61–0.80; P value <0.001). There was no significant difference in all-cause mortality between patients receiving pirfenidone and those on nintedanib (HR, 1.14; 95% CI, 0.79–1.65; P = 0.471). Conclusions Among patients with idiopathic pulmonary fibrosis, antifibrotic agents may be associated with a lower risk of all-cause mortality and hospitalization compared with no treatment. Future research should test the hypothesis that these treatments reduce early, but not long-term, mortality as demonstrated in our study.