Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children

Cheng, Katharine; Mahler, Fenna; Lutsar, Irja; Escalera, Begonya Nafria; Breitenstein, Stefanie; Vassal, Gilles; Claverol, Joana; Palacio, Nuria Noel; Portman, Ron; Pope, Gavin; Bakker, M.; Geest, Tessa van der; Turner, Mark A.; Wildt, Saskia N. de

doi:10.1111/cts.13459

“…c4c has set up a European multidisciplinary advice service that aids in the planning of innovative and feasible pediatric trials 19 . c4c created a database with over 400 clinical and innovative methodology experts, divided over 24 expert groups based on their expertise.…”

Section: Methodsmentioning

confidence: 99%

“…c4c has set up a European multidisciplinary advice service that aids in the planning of innovative and feasible pediatric trials. 19 c4c created a database with over 400 clinical and innovative methodology experts, divided over 24 expert groups based on their expertise. Based on a scoping interview between EU‐PEARL and c4c, the best suited experts for the requested advice were selected in collaboration with the leads of the relevant expert groups.…”

Section: Methodsmentioning

confidence: 99%

Optimizing expert and patient input in pediatric trial design: Lessons learned and recommendations from a collaboration between conect4children and European Patient‐CEntric ClinicAl TRial PLatforms

Dhaenens

¹

,

Mahler

²

,

Batchelor

³

et al. 2023

Clinical Translational Sci

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0

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Advice from multiple stakeholders is required to design the optimal pediatric clinical trial. We present recommendations for acquiring advice from trial experts and patients/caregivers, derived from advice meetings that were performed through a collaboration of the Collaborative Network for European Clinical Trials for Children (c4c) and the European Patient‐CEntric ClinicAl TRial PLatforms (EU‐PEARL). Three advice meetings were performed: (1) an advice meeting for clinical and methodology experts, (2) an advice meeting for patients/caregivers, and (3) a combined meeting with both experts and patients/caregivers. Trial experts were recruited from c4c database. Patients/caregivers were recruited through a patient organization. Participants were asked to provide input on a trial protocol, including endpoints, outcomes, and the assessment schedule. Ten experts, 10 patients, and 13 caregivers participated. The advice meetings resulted in modification of eligibility criteria and outcome measures. We have provided recommendations for the most effective meeting type per protocol topic. Topics with limited options for patient input were most efficiently discussed in expert advice meetings. Other topics benefit from patient/caregiver input, either through a combined meeting with experts or a patients/caregivers‐only advice meeting. Some topics, such as endpoints and outcome measures, are suitable for all meeting types. Combined sessions profit from synergy between experts and patients/caregivers, balancing input on protocol scientific feasibility and acceptability. Both experts and patients/caregivers provided critical input on the presented protocol. The combined meeting was the most effective methodology for most protocol topics. The presented methodology can be used effectively to acquire expert and patient feedback.

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“…While working with existing data to improve therapy development now, RDCA-DAP is also working towards establishing standards for regulatory-ready natural history data collection over time, to further improve the rare disease drug development ecosystem.It is hoped that RDCA-DAP can serve as a model for patient-focused drug development incorporating the voice of the patient but managing both regulatory interest and PhRMA engagement. The role of patients and parents has been clearly articulated by others[18,19] and the evolution of the platform is clearly planned on additional and customized engagement with patients and parents.…”

mentioning

confidence: 99%

The future of Rare Disease Drug development: the Rare Disease Cures Accelerator Data Analytics Platform (RDCA-DAP)

Barrett

¹

,

Betourne

²

,

Walls

³

et al. 2023

Preprint

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Rare disease drug development is wrought with challenges not the least of which is access to the limited data currently available throughout the rare disease ecosystem where sharing of the available data is not guaranteed. Most pharmaceutical sponsors seeking to develop agents to treat rare diseases will initiate data landscaping efforts to identify various data sources that might be informative with respect to disease prevalence, patient selection and identification, disease progression and any data projecting likelihood of patient response to therapy including any genetic data. Such data are often difficult to come by for highly prevalent, mainstream disease populations let alone for the 8000 rare disease that make up the pooled patient population of rare disease patients. The future of rare disease drug development will hopefully rely on increased data sharing and collaboration among the entire rare disease ecosystem. One path to achieving this outcome has been the development of the rare disease cures accelerator, data analytics platform (RDCA-DAP) funded by the US FDA and operationalized by the Critical Path Institute. FDA intentions were clearly focused on improving the quality of rare disease regulatory applications by sponsors seeking to develop treatment options for various rare disease populations. As this initiative moves into its second year of operations it is envisioned that the increased connectivity to new and diverse data streams and tools will result in solutions that benefit the entire rare disease ecosystem and that the platform becomes a Collaboratory for engagement of this ecosystem that also includes patients and caregivers.

show abstract

The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)

Barrett

¹

,

Betourne

²

,

Walls

³

et al. 2023

J Pharmacokinet Pharmacodyn

View full text Add to dashboard Cite

Rare disease drug development is wrought with challenges not the least of which is access to the limited data currently available throughout the rare disease ecosystem where sharing of the available data is not guaranteed. Most pharmaceutical sponsors seeking to develop agents to treat rare diseases will initiate data landscaping efforts to identify various data sources that might be informative with respect to disease prevalence, patient selection and identification, disease progression and any data projecting likelihood of patient response to therapy including any genetic data. Such data are often difficult to come by for highly prevalent, mainstream disease populations let alone for the 8000 rare disease that make up the pooled patient population of rare disease patients. The future of rare disease drug development will hopefully rely on increased data sharing and collaboration among the entire rare disease ecosystem. One path to achieving this outcome has been the development of the rare disease cures accelerator, data analytics platform (RDCA-DAP) funded by the US FDA and operationalized by the Critical Path Institute. FDA intentions were clearly focused on improving the quality of rare disease regulatory applications by sponsors seeking to develop treatment options for various rare disease populations. As this initiative moves into its second year of operations it is envisioned that the increased connectivity to new and diverse data streams and tools will result in solutions that benefit the entire rare disease ecosystem and that the platform becomes a Collaboratory for engagement of this ecosystem that also includes patients and caregivers.

show abstract

Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children

Cited by 7 publications

References 6 publications

Optimizing expert and patient input in pediatric trial design: Lessons learned and recommendations from a collaboration between conect4children and European Patient‐CEntric ClinicAl TRial PLatforms

Optimizing expert and patient input in pediatric trial design: Lessons learned and recommendations from a collaboration between conect4children and European Patient‐CEntric ClinicAl TRial PLatforms

The future of Rare Disease Drug development: the Rare Disease Cures Accelerator Data Analytics Platform (RDCA-DAP)

The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP)

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