ESPN 51th Annual Meeting

ESPN 2018


 
OVERCOMING BARRIERS TO THERAPEUTIC DEVELOPMENT IN CHILDREN WITH CKD: A KHI INITIATIVE
William Schnaper 1 Pam Duquette 2 Teresa VU Lewis 4 Alicia Neu 2 Michelle Rheault 3 Debbie Gipson 10 Katrin Uhlig 8 Franz Schaefer 5 Stuart Goldstein 9 Melissa West 7 Johan Vande Walle 11

1- Northwestern university
2- John Hopkins University
3- University of Minnesota
4- University of Oklahoma
5- Universitat Heidelberg
6- Northwestern university
7- ASN
8- Keryx Biopharmaceuticals
9- University Cincinnati
10- University of Michigan
11- safepedrug Ugent
 
Introduction:

 

 A Kidney Health Initiative (KHI) workgroup on ‘Overcoming Barriers to Drug Development in Children with CKD’ identified the need for more concerted and rational planning and conduct of clinical trials in children with CKD to address EMA and FDA regulatory requirement. 

Material and methods:

 An Implementation Committee was charged to establish an organizational entity that will help optimize planning of therapeutic trials in children with CKD. Its charter addresses aims, guiding principles, composition of a standing Leadership Committee, committee operations, and funding.

Results:

 The aims of the new entity are to a) Enable feasibility assessment of available patient populations through data sharing and access to CKD pediatric registries b) Facilitate assessment of the capacity of various pediatric kidney clinical trial organizations c) Assist with identification of expertise that can provide consultation on study planning.   To manage potential conflicts of interest, this entity needs to adhere to guiding principles regarding transparency, information sharing, international participation and collaboration, and financial independence, while respecting the rights and intellectual property of principal investigators, sponsors, and trial networks, and ensuring the confidentiality of proprietary information and protected patient information. The Leadership Committee will be comprised of representatives from pediatric collaborative trial networks, patient/family support groups and subject matter experts without industry sponsors. Operationally, industry sponsors will be able to submit queries and requests that will be reviewed, triaged, and addressed by the Leadership Committee. The Implementation Committee will draft a finance model, sustainability plan, and identify a permanent administrative home with a commitment to transparency, equity, and economy.  In the interim, KHI will continue to support this entity.

Conclusions:

 : This new entity will seek collaboration with other stakeholders to better support therapeutic development in children with CKD.