C-Path ePRO Consortium and PRO Consortium Announce COVID-19 Risk Assessment and Mitigation Strategies

Recommendations provided for sponsors and eCOA providers to facilitate the continued collection of PRO data in clinical trials.

 

TUCSON, Ariz., April 29, 2020 — In collaboration with Critical Path Institute’s (C-Path) Patient-Reported Outcome (PRO) Consortium, the Electronic Patient-Reported Outcome (ePRO) Consortium announces “Coronavirus Disease 2019 (COVID-19): Risk Assessment and Mitigation Strategies for the Collection of Patient-Reported Outcome Data through Clinical Sites.” The presentation focuses on the current challenges of capturing PRO data originally intended to be collected electronically (i.e., ePRO) from study participants during in-person visits to clinical trial sites. Recommended risk assessment and mitigation strategies are provided for consideration by trial sponsors and electronic clinical outcome assessment (eCOA) providers to facilitate the continued collection of PRO data in clinical trials.

Due to concerns surrounding COVID-19, many patients are either unable or unwilling to travel to sites for scheduled visits or sites have had to close due to social distancing measures. Representatives of ePRO Consortium and PRO Consortium member firms were invited by C-Path to collaborate on the development of recommendations aimed at lessening the impact of the disruption on PRO data collection at clinical trial sites. Over a one-month period, member representatives participated in a series of teleconferences to discuss and debate approaches to assessing the situation and effectively responding to it. The objective of the resulting presentation is to provide recommended risk assessment and mitigation strategies for consideration by sponsors and eCOA providers to facilitate the continued collection of PRO data in clinical trials. Topics include regulatory considerations, licensing considerations when altering the mode of administration of a PRO measure, and reporting protocol changes to the institutional review board.

“These mitigation strategies have led us to rethink traditional clinical trial data collection approaches and to recognize that technology, which has become increasingly important in clinical research, is available to support decentralized trials now and in the future,” said Paul O’Donohoe, M.Sc., Scientific Lead, eCOA and Mobile Health at Medidata Solutions and Industry Vice Director of the ePRO Consortium.

“COVID-19 has impacted clinical trials in significant ways, and C-Path is proud to have supported the crucial conversations among eCOA providers and clinical trial sponsors that led to the roll out of strategies aimed to facilitate the continued collection of PRO data in clinical trials,” said Sonya Eremenco, M.A., Acting Director, ePRO Consortium. “These strategies reflect the collective effort of both consortia to work together for the greater good, ensuring patient safety in these uncertain times.”

*Funding for this press release was made possible, in part, by the Food and Drug Administration through grant U18 FD 005320. Views expressed here do not necessarily reflect the official policies of the Department of Health and Human Services nor does any mention of an organization imply endorsement by the United States Government.

 


 

About C-Path’s Electronic Patient-Reported Outcome Consortium

The Electronic Patient-Reported Outcome (ePRO) Consortium was established by Critical Path Institute in 2011. Along with C-Path, the members of the ePRO Consortium are firms that provide electronic data collection technologies and services for capturing patient-reported outcome (PRO) and other clinical outcome assessment (COA) data in clinical trials. The mission of the ePRO Consortium is to advance the science of clinical trial endpoint assessment by collaboratively supporting and conducting research, designing and delivering educational opportunities, and developing and disseminating best practice recommendations for electronic collection of clinical outcome data.

 

About C-Path’s Patient-Reported Outcome Consortium

The Patient-Reported Outcome (PRO) Consortium was formed in 2008 by Critical Path Institute in cooperation with the US Food and Drug Administration’s Center for Drug Evaluation and Research and the pharmaceutical industry. The mission of the PRO Consortium is to establish and maintain a collaborative framework with appropriate stakeholders for the qualification of PRO measures and other clinical outcome assessment (COA) tools that will be publicly available for use in clinical trials where COA-based endpoints are used to support product labeling claims.

 

About Critical Path Institute

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

 

Contact:

Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Receives FDA Grant to Establish Rare Disease Clinical Outcome Assessment Consortium

 

 

TUCSON, Ariz., March 19, 2020 — The U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has funded a cooperative agreement to establish a Rare Disease Clinical Outcome Assessment (COA) Consortium. The grant (U01FD006882)* was awarded to the Critical Path Institute (C-Path) with the National Organization for Rare Disorders (NORD) as a sub-awardee. The first step taken toward the establishment of the new consortium has been the creation of the Rare Disease Subcommittee within C-Path’s Patient-Reported Outcome (PRO) Consortium. The PRO Consortium will serve as an incubator for the maturation of a pre-competitive, multi-stakeholder consortium within C-Path’s COA Program.

The PRO Consortium’s Rare Disease Subcommittee includes representatives from C-Path, NORD, FDA, the Patient-Centered Outcomes Research Institute, the National Center for Advancing Translational Sciences, and biopharmaceutical firms within the PRO Consortium that are developing treatments for rare diseases. In addition, plans are underway to enable rare disease-focused biotech firms not currently members of the PRO Consortium to be included in the strategic planning for the new consortium. Once established, the Rare Disease COA Consortium’s activities will be aimed at accelerating the development of new medical products intended to safely and effectively treat people with rare diseases by creating and curating a resource of information on publicly available COAs identified as potentially fit-for-purpose endpoint measures in treatment trials for rare diseases. The premise is that existing COAs may be able to be used or modified for use across multiple diseases sharing common characteristics.

Along with planning the membership, governance and organizational structure of the new consortium, the Rare Disease Subcommittee has launched a multi-pronged effort aimed at tackling challenges in the assessment of clinical benefit in rare disease treatment trials. The first pilot project under this initiative is to identify COAs that can be used in children to assess activities of daily living, which is a meaningful aspect of life impacted by many rare diseases. This will be the first in a series of reviews to identify COAs aimed at symptom and functional domains that reflect important aspects of patients’ lives that are impacted by rare diseases. Concurrently, the Rare Disease Subcommittee has initiated a second pilot project that involves a literature review to explore ways in which researchers have handled heterogeneity in clinical trials including an examination of the advantages and disadvantages of the approaches to personalizing endpoints. Development of best practice recommendations for assessing clinical benefit in rare disease trials will be subsequently explored.

*Funding for this press release was made possible, in part, by the Food and Drug Administration through grant U01FD006882. Views expressed here do not necessarily reflect the official policies of the Department of Health and Human Services nor does any mention of an organization imply endorsement by the United States Government.

 


 

 

About Patient-Reported Outcome Consortium

The Patient-Reported Outcome (PRO) Consortium was formed in 2008 by Critical Path Institute in cooperation with the US Food and Drug Administration’s Center for Drug Evaluation and Research and the pharmaceutical industry. The mission of the PRO Consortium is to establish and maintain a collaborative framework with appropriate stakeholders for the qualification of PRO measures and other clinical outcome assessment (COA) tools that will be publicly available for use in clinical trials where COA-based endpoints are used to support product labeling claims.

 

About Critical Path Institute

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

 

Contact:

Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Receives Qualification from FDA for the Asthma Daytime Symptom Diary (ADSD) and the Asthma Nighttime Symptom Diary (ANSD)

 

 

TUCSON, Ariz., April 9, 2019 — Critical Path Institute’s (C-Path) Patient-Reported Outcome (PRO) Consortium announces U.S. Food and Drug Administration (FDA) qualification of two new clinical outcome assessment tools: the Asthma Daytime Symptom Diary (ADSD) and the Asthma Nighttime Symptom Diary (ANSD). The qualification of the ADSD and the ANSD represents a major milestone for the PRO Consortium and specifically for the Asthma Working Group. It is the culmination of a multi-year collaboration between FDA’s Center for Drug Evaluation and Research (CDER) and the PRO Consortium.

The ADSD and the ANSD are each 6-item, PRO measures developed to document the core symptoms of asthma in adults and adolescents with a clinical diagnosis of mild to severe persistent asthma.

“Asthma symptoms have a substantial impact on patients, including limiting the ability to participate in daily activities and disrupting sleep. The qualification of the ADSD and the ANSD is a significant advance in our ability to document the patient’s experience of asthma symptoms in clinical trials,” stated Linda Nelsen, MHS, Senior Director and Head, Patient Centered Outcomes, Value Evidence and Outcomes, at GlaxoSmithKline and co-chair of the PRO Consortium’s Asthma Working Group.

“The development of the ADSD and ANSD represents a close collaboration of clinicians, measurement experts, and regulators focused on developing a robust measure of asthma symptoms,” Nelsen added. “Most importantly, the ADSD and ANSD were developed with extensive involvement of people living with asthma to clearly define the experience of asthma symptoms and the relative importance of those symptoms from their perspective. Thus, the ADSD and ANSD bring the patient’s voice into clinical trials in a way that allows the quantification of important concepts of patient experience in treatment.”

Sonya Eremenco, MA, Associate Director of the PRO Consortium, said, “Sponsors of asthma treatment trials have been using a variety of asthma symptom measures over the years, but we now have a standard set of symptom measures created via a multi-stakeholder, precompetitive collaboration that can be used in asthma drug development programs. This is a significant step toward providing enhanced consistency and comparability in the evaluation of the patient-focused clinical benefit from new asthma drugs.”

CDER has determined that the ADSD and ANSD have demonstrated adequate evidence of content validity and cross-sectional measurement properties (i.e., internal consistency reliability, test-retest reliability, convergent validity and known-groups validity) to measure symptoms of asthma in adolescents and adults.

Drug developers seeking to use the ADSD or ANSD as a primary or secondary endpoint measure in confirmatory studies should discuss plans with the appropriate CDER review division. It is recommended to use the ANSD with the ADSD to better characterize the drug effect and understand the measurement properties of each instrument. Further evaluation is needed on the instruments’ longitudinal properties and the interpretation of clinically meaningful within-patient change in score.

The Qualification Statement can be found on the FDA’s Drug Development Tools Qualified Clinical Outcome Assessments (COA) web page.

Further information about the ADSD and the ANSD, and how to access these measures, is available by contacting procadmin@c-path.org.


 

About C-Path

C-Path (Critical Path Institute) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include over 1,500 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path is headquartered in Tucson, Arizona, with additional staff in multiple remote locations. For more information, visit www.c-path.org.

 

About C-Path’s Patient-Reported Outcome (PRO) Consortium

The Patient-Reported Outcome (PRO) Consortium was formed in 2008 by Critical Path Institute in cooperation with the US Food and Drug Administration’s Center for Drug Evaluation and Research and the pharmaceutical industry. The mission of the PRO Consortium is to establish and maintain a collaborative framework with appropriate stakeholders for the qualification of PRO measures and other clinical outcome assessment (COA) tools that will be publicly available for use in clinical trials where COA-based endpoints are used to support product labeling claims.

 

Contact:

Kissy Black
615.298.1144
kissyblack@lotosnile.com