Design of a “Lean” Case Report Form for Heart Failure Therapeutic Development

Psotka, Mitchell A.; Fiuzat, Mona; Carson, Peter E.; Kao, David; Cerkvenik, Jeffrey; Schaber, Daniel E.; Verta, Patrick; Kazmierski, Robert; Shinnar, Meir; Stockbridge, Norman; Unger, Ellis F.; Zuckerman, Bram; Butler, Javed; Felker, G. Michael; Konstam, Marvin A.; Lindenfeld, JoAnn; Solomon, Scott D.; Teerlink, John R.; O’Connor, Christopher M.; Abraham, William T.

doi:10.1016/j.jchf.2019.07.001

Cited by 8 publications

(4 citation statements)

References 41 publications

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“…8 The HFC has expended considerable effort to define a standardized minimal data set CRF, with participants from academia, industry, and regulatory agencies, as well as patients. 11 These instruments may be supplemented by modules, where additional types of data, such as echocardiographic measurements are defined in a standardized manner. Another potential advance is to move rapidly toward electronic data entry across all trials and sites.…”

Section: Discussionmentioning

confidence: 99%

Barriers to Clinical Trial Efficiency and Patient Access: The Heart Failure Collaboratory Industry Survey

Sinha

Psotka

Fiuzat

et al. 2020

Preprint

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Aims: Clinical trial inefficiency and lack of patient access to novel therapies have been identified as key barriers to successful heart failure innovation. The Heart Failure Collaboratory (HFC) is a consortium developed to identify and address barriers to bringing drugs and devices to market. The HFC performed an electronic cross-sectional survey of key leaders within industry to collate and interpret a sample of viewpoints on these challenges. Methods and Results: From August to September 2018, self-administered survey data were electronically collected from industry partners. Group comparisons were made via Fisher's Exact or chi-square test. Respondents most commonly rated the United States Food and Drug Administration (44.2%), Health Canada (39.5%) and European Medicines Agency (32.6%) as efficient. Respondents rated the top 3 areas with the greatest opportunity for regulatory agencies to improve efficiency: 'improve usefulness of agency feedback' (48.8%); 'improved timeliness of agency responses' (41.9%); and 'pre-specification of required magnitude of clinical effect' and 'limit excessive data requirements' (32.6%). Respondents rated items of 'excessive clinical site staff workload' (55.8%), 'overly complex case report forms' (51.2%) and 'data input errors' (39.5%) as the top 3 factors influencing data quality. Respondents rated items of 'onerous prior authorization requirements' (51.2%), 'availability of decision-making rationale' (46.5%) and 'rationality of access barriers created by payers' (44.2%) as the top 3 impediments to improving patient access to approved therapies. Conclusion: This broadly distributed survey of industry respondents identified multiple specific barriers to heart failure clinical trial efficiency and patient access, which should help direct efforts toward improvement.

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Section: Discussionmentioning

confidence: 99%

Barriers to Clinical Trial Efficiency and Patient Access: The Heart Failure Collaboratory Industry Survey

Sinha

Psotka

Fiuzat

et al. 2020

Preprint

Self Cite

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“…The lean CRF eliminates non-critical elements, which reduced the number of elements from 176 in the original CRF's to 75 elements. This freely available tool aims to standardize data collection in HF clinical trials and to reduce the cost and burden to run the trials ( 23 ).…”

Section: Clinical Trial Designmentioning

confidence: 99%

Rethinking heart failure clinical trials: the heart failure collaboratory

Alkalbani,

Psotka

2024

Front. Cardiovasc. Med.

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The Heart Failure Collaboratory (HFC) is a consortium of stakeholders in the heart failure (HF) community that aims to improve the infrastructure of clinical research to promote development of novel therapies for patients. Since its launch in 2018, HFC has implemented several solutions to tackle obstacles in HF clinical research including training programs to increase the number of clinicians skilled in conducting clinical trials, novel study designs, and advocacy for a diverse and inclusive HF research ecosystem. We highlight some of the HFC successes since its establishment.

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“…Demographic characteristics, most frequently age, have been associated with variable safety and efficacy in response to therapeutic interventions 43 . The expanded racial categorization from the United States National Institutes of Health data standard and US FDA guidance balances specificity with appropriate data entry workload 44–46 . Definitions for age, sex, gender, race, and ethnicity are presented in Table 5 , though it is acknowledged that governmental prohibitions in some regions may limit collection of these data.…”

Section: Heart Failure Definitionsmentioning

confidence: 99%

Standardized definitions for evaluation of heart failure therapies: scientific expert panel from the Heart Failure Collaboratory and Academic Research Consortium

Abraham

Psotka

Fiuzat

et al. 2020

European J of Heart Fail

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The Heart Failure Academic Research Consortium is a partnership between the Heart Failure Collaboratory (HFC) and Academic Research Consortium (ARC), comprised of leading heart failure (HF) academic research investigators, patients, United States (US) Food and Drug Administration representatives, and industry members from the US and Europe. A series of meetings were convened to establish definitions and key concepts for the evaluation of HF therapies including optimal medical and device background therapy, clinical trial design elements and statistical concepts, and study endpoints. This manuscript summarizes the expert panel discussions as consensus recommendations focused on populations and endpoint definitions; it is not exhaustive or restrictive, but designed to stimulate HF clinical trial innovation.

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Design of a “Lean” Case Report Form for Heart Failure Therapeutic Development

Cited by 8 publications

References 41 publications

Barriers to Clinical Trial Efficiency and Patient Access: The Heart Failure Collaboratory Industry Survey

Barriers to Clinical Trial Efficiency and Patient Access: The Heart Failure Collaboratory Industry Survey

Rethinking heart failure clinical trials: the heart failure collaboratory

Standardized definitions for evaluation of heart failure therapies: scientific expert panel from the Heart Failure Collaboratory and Academic Research Consortium

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