TY - JOUR
T1 - Meaningful Endpoints for Idiopathic Pulmonary Fibrosis (IPF) Clinical Trials
T2 - Emphasis on 'Feels, Functions, Survives'. Report of a Collaborative Discussion in a Symposium with Direct Engagement from Representatives of Patients, Investigators, the National Institutes of Health, a Patient Advocacy Organization, and a Regulatory Agency
AU - Raghu, Ganesh
AU - Ghazipura, Marya
AU - Fleming, Thomas R.
AU - Aronson, Kerri I.
AU - Behr, Jürgen
AU - Brown, Kevin K.
AU - Flaherty, Kevin R.
AU - Kazerooni, Ella A.
AU - Maher, Toby M.
AU - Richeldi, Luca
AU - Lasky, Joseph A.
AU - Swigris, Jeffrey J.
AU - Busch, Robert
AU - Garrard, Lili
AU - Ahn, Dong Hyun
AU - Li, Ji
AU - Puthawala, Khalid
AU - Rodal, Gabriela
AU - Seymour, Sally
AU - Weir, Nargues
AU - Danoff, Sonye K.
AU - Ettinger, Neil
AU - Goldin, Jonathan
AU - Glassberg, Marilyn K.
AU - Kawano-Dourado, Leticia
AU - Khalil, Nasreen
AU - Lancaster, Lisa
AU - Lynch, David A.
AU - Mageto, Yolanda
AU - Noth, Imre
AU - Shore, Jessica E.
AU - Wijsenbeek, Marlies
AU - Brown, Robert
AU - Grogan, Daniel
AU - Ivey, Dorothy
AU - Golinska, Patrycja
AU - Karimi-Shah, Banu
AU - Martinez, Fernando J.
N1 - Publisher Copyright:
Copyright © 2024 by the American Thoracic Society.
PY - 2024/3/15
Y1 - 2024/3/15
N2 - Background: Idiopathic pulmonary fibrosis (IPF) carries significant mortality and unpredictable progression, with limited therapeutic options. Designing trials with patient-meaningful endpoints, enhancing the reliability and interpretability of results, and streamlining the regulatory approval process are of critical importance to advancing clinical care in IPF. Methods: A landmark in-person symposium in June 2023 assembled 43 participants from the US and internationally, including patients with IPF, investigators, and regulatory representatives, to discuss the immediate future of IPF clinical trial endpoints. Patient advocates were central to discussions, which evaluated endpoints according to regulatory standards and the FDA's 'feels, functions, survives' criteria. Results: Three themes emerged: 1) consensus on endpoints mirroring the lived experiences of patients with IPF; 2) consideration of replacing forced vital capacity (FVC) as the primary endpoint, potentially by composite endpoints that include 'feels, functions, survives' measures or FVC as components; 3) support for simplified, user-friendly patient-reported outcomes (PROs) as either components of primary composite endpoints or key secondary endpoints, supplemented by functional tests as secondary endpoints and novel biomarkers as supportive measures (FDA Guidance for Industry (Multiple Endpoints in Clinical Trials) available at: https://www.fda.gov/media/162416/download). Conclusions: This report, detailing the proceedings of this pivotal symposium, suggests a potential turning point in designing future IPF clinical trials more attuned to outcomes meaningful to patients, and documents the collective agreement across multidisciplinary stakeholders on the importance of anchoring IPF trial endpoints on real patient experiences-namely, how they feel, function, and survive. There is considerable optimism that clinical care in IPF will progress through trials focused on patient-centric insights, ultimately guiding transformative treatment strategies to enhance patients' quality of life and survival.
AB - Background: Idiopathic pulmonary fibrosis (IPF) carries significant mortality and unpredictable progression, with limited therapeutic options. Designing trials with patient-meaningful endpoints, enhancing the reliability and interpretability of results, and streamlining the regulatory approval process are of critical importance to advancing clinical care in IPF. Methods: A landmark in-person symposium in June 2023 assembled 43 participants from the US and internationally, including patients with IPF, investigators, and regulatory representatives, to discuss the immediate future of IPF clinical trial endpoints. Patient advocates were central to discussions, which evaluated endpoints according to regulatory standards and the FDA's 'feels, functions, survives' criteria. Results: Three themes emerged: 1) consensus on endpoints mirroring the lived experiences of patients with IPF; 2) consideration of replacing forced vital capacity (FVC) as the primary endpoint, potentially by composite endpoints that include 'feels, functions, survives' measures or FVC as components; 3) support for simplified, user-friendly patient-reported outcomes (PROs) as either components of primary composite endpoints or key secondary endpoints, supplemented by functional tests as secondary endpoints and novel biomarkers as supportive measures (FDA Guidance for Industry (Multiple Endpoints in Clinical Trials) available at: https://www.fda.gov/media/162416/download). Conclusions: This report, detailing the proceedings of this pivotal symposium, suggests a potential turning point in designing future IPF clinical trials more attuned to outcomes meaningful to patients, and documents the collective agreement across multidisciplinary stakeholders on the importance of anchoring IPF trial endpoints on real patient experiences-namely, how they feel, function, and survive. There is considerable optimism that clinical care in IPF will progress through trials focused on patient-centric insights, ultimately guiding transformative treatment strategies to enhance patients' quality of life and survival.
UR - https://www.scopus.com/pages/publications/85185911364
U2 - 10.1164/rccm.202312-2213SO
DO - 10.1164/rccm.202312-2213SO
M3 - Conference article
C2 - 38174955
AN - SCOPUS:85185911364
SN - 1073-449X
VL - 209
SP - 647
EP - 669
JO - American Journal of Respiratory and Critical Care Medicine
JF - American Journal of Respiratory and Critical Care Medicine
IS - 6
ER -