ESPN 51th Annual Meeting

ESPN 2018


 
OVERCOMING BARRIERS TO THERAPEUTIC DEVELOPMENT IN CHILDREN WITH CKD OPTIMIZATION OF PEDIATRIC CHRONIC KIDNEY DISEASE DRUG TRIALS: THE KHI INITIATIVE
WILLIAM SCHNAPER 1 PAM DUQUETTE 2 DEBBIE GIPSON 3 TERESA VU LEWIS 4 ALICIA NEU 2 MICHELLE RHEAULT 5 FRANZ SCHAEFER 6 MELISSA WEST 8 JOHAN VANDE WALLE 7 STUART GOLDSTEIN 9

1- NORTHWESTERN UNIVERSITY
2- JOHN HOPKINS UNIVERSITY
3- UNIVERSITY OF MICHIGAN
4- UNIVERSITY OF OKLAHOMA
5- UNIVERSITY OF MINNESOTA
6- UNIVERSITAT HEIDELBERG
7- GHENT UNIVERSITY
8- ASN
9- UNIVERSITY CINCINNATI
 
Introduction:

Clinical trials in children, with special emphasis on CKD, remain orphaned despite the FDA and EMA regulations, where only few of the PIP’s resulted in appropriate labelling in children with CKD. The kidney health initiative (KHI), identifies the existing pitfalls

Material and methods:

1)    Recognize the legal remit and regulatory framework for pediatric study plans in US and Europe. Outline the current avenues of collaboration and communication between the regulatory agencies in US and Europe regarding pediatric plans 2) Explore avenues for harmonization of study designs (including endpoints) and timelines for pediatric plans across US and European regulatory agencies. 3) Develop a mechanism to prioritize drugs and drug classes for trials in children with CKD through multi-stakeholder guidance and input, considering the following prioritization factors: a) Unmet patient needs (e.g., new drug class, new mechanism of action) b) Finite research resources in terms of number of patients affected and sites, in context of numerous pediatric commitments c) Regulatory landscape for studies in children with CKD d) Timelines and priority metrics

Results:

 Development of a strategic plan : 

a) Optimize planning of pediatric drug trials by Enabling feasibility assessment in terms of the available patient populations through data sharing and access to CKD pediatric registries. Consultation on design and protocols by a panel of pediatric nephrologists, trialists, patient representatives early in the design process . b) Enhance the likelihood of successful trial execution through assessment of the capacity of various pediatric renal clinical trial organizations  c)Identify key considerations for a balanced assessment of perceived benefits and risks (including toxicity), through a collaboration with experts (including toxicologists, clinical trialists from other pediatric disciplines that deal with toxicology risk vs. benefit, patients and caregivers).

 

Conclusions:

The KHI initiative stresses the need for a more rationalized planning and performance of pediatric clinical trials to furfill the clinical needs of our CKD children