@article{c2d1dfea8f5344f7b12ffea61b66aeef,
title = "End points for sickle cell disease clinical trials: Patient-reported outcomes, pain, and the brain",
abstract = "To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.",
author = "Farrell, {Ann T.} and Julie Panepinto and Carroll, {C. Patrick} and Darbari, {Deepika S.} and Desai, {Ankit A.} and King, {Allison A.} and Adams, {Robert J.} and Barber, {Tabitha D.} and Brandow, {Amanda M.} and DeBaun, {Michael R.} and Donahue, {Manus J.} and Kalpna Gupta and Hankins, {Jane S.} and Michelle Kameka and Kirkham, {Fenella J.} and Harvey Luksenburg and Shirley Miller and Oneal, {Patricia Ann} and Rees, {David C.} and Rosanna Setse and Sheehan, {Vivien A.} and John Strouse and Stucky, {Cheryl L.} and Werner, {Ellen M.} and Wood, {John C.} and Zempsky, {William T.}",
note = "Funding Information: This article summarizes topics addressed at the US Food and Drug Administration (FDA)–American Society of Hematology (ASH) Sickle Cell Disease Clinical Endpoints Workshop. ASH and FDA engaged the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points. This work, plus >30 preparatory calls with the panels and engaging discussions at the workshop, contributed to the development of 2 articles that present the findings of the panels. The Contribution section details how the authors were involved in the development of the actual manuscripts. Kathi E. Hanna, the contracted science writer, provided summaries based on discussions at the workshop and initial summaries submitted by panels; prepared drafts of the manuscripts; managed review of the papers; and prepared the manuscript for submission. The authors acknowledge Peter Marks (Center for Biologics Evaluation and Research, FDA) and 2018 ASH President Alexis A. Thompson (Ann & Robert H. Lurie Children{\textquoteright}s Hospital of Chicago, Feinberg School of Medicine, Northwestern University) for their support and involvement with the 2018 FDA-ASH Sickle Cell Disease Clinical Endpoints Workshop. ASH received support from the Doris Duke Charitable Foundation (DDCF) and the ASH Foundation to cover meeting expenses. The views of the authors represent their own and should not be interpreted to reflect the official policy of the US FDA. Publisher Copyright: {\textcopyright} 2019 American Society of Hematology. All rights reserved.",
year = "2019",
doi = "10.1182/bloodadvances.2019000882",
language = "English (US)",
volume = "3",
pages = "3982--4001",
journal = "Blood Advances",
issn = "2473-9529",
publisher = "American Society of Hematology",
number = "23",
}