TY - JOUR
T1 - Clinical trial considerations in sickle cell disease
T2 - patient-reported outcomes, data elements, and the stakeholder engagement framework
AU - Badawy, Sherif M.
N1 - Funding Information:
Projects are organized by level of funding support from low to high.
Funding Information:
Table 3. Examples of SCD projects funded by the PCORI
Funding Information:
Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways. First, consensus recommendations for evidencebased SCD end points have been developed as a result of a collaborative effort from 7 panels of patients, clinicians, and researchers established through a partnership between the American Society of Hematology (ASH) and the FDA.16,17 Second, the ASH Research Collaborative SCD Clinical Trials Network has focused on building partnerships with the SCD community and stakeholders, establishing collaboration across SCD centers, streamlining clinical trial operations with a single institutional review board approval, and facilitating data sharing with a centralized data repository through the ASH Research Collaborative Data Hub.18Third, the PhenX (Phenotypes and eXposures) project has been funded by different sources, including the National Human Genome Research Institute, the National Institute on Drug Abuse, the Offce of Behavioral and Social Sciences Research, the National Institute of Mental Health, the National Heart, Lung, and Blood Institute (NHLBI), the National Institute on Minority Health and Health Disparities, the National Cancer Institute, and the Tobacco Regulatory Science Program of the National Institutes of Health (NIH). In SCD, PhenX efforts have focused on selecting highquality SCDrelated outcome measures to be included in the Toolkit (consensus measures; www.phenxtoolkit .org), guided by the Sickle Cell Disease Research and Scientifc Panel.19 Finally, the Cure Sickle Cell Initiative (CureSCi), led by the NHLBI, has centered on innovating genetic therapies, nurturing a collaborative, PCR environment, and establishing data standards for SCD clinical trials.20 In particular, interest in curative therapies in the SCD community has been growing as evidence has emerged and continues to materialize from ongoing clinical trials.
Publisher Copyright:
© 2021 by The American Society of Hematology
PY - 2021/12/10
Y1 - 2021/12/10
N2 - Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.
AB - Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.
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U2 - 10.1182/hematology.2021000252
DO - 10.1182/hematology.2021000252
M3 - Article
C2 - 34889394
AN - SCOPUS:85122487213
SN - 1520-4391
VL - 2021
SP - 196
EP - 205
JO - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
JF - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
IS - 1
ER -