Journal article
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy
Journal of pharmacokinetics and pharmacodynamics, Vol.46(5), pp.441-455
10/2019
DOI: 10.1007/s10928-019-09642-7
PMID: 31127458
Abstract
Drug development for rare diseases is challenged by small populations and limited data. This makes development of clinical trial protocols difficult and contributes to the uncertainty around whether or not a potential therapy is efficacious. The use of data standards to aggregate data from multiple sources, and the use of such integrated databases to develop statistical models can inform protocol development and reduce the risks in developing new therapies. Achieving regulatory endorsement of such models through defined pathways at the US Food and Drug Administration and European Medicines Authority allows such tools to be used by the drug development community for defined contexts of use without further need for discussion of the underlying model(s). The Duchenne Regulatory Science Consortium (D-RSC) has brought together multiple stakeholders to develop a clinical trial simulation tool for Duchenne muscular dystrophy using such an approach. Here we describe the work of D-RSC as an example of how such an approach may be effective at reducing uncertainty in drug development for rare diseases, and thus bringing effective therapies to patients faster.
Details
- Title: Subtitle
- Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy
- Creators
- Daniela J Conrado - Critical Path Institute, 1730 E. River Road, Tucson, AZ, 85705, USA. DConrado@c-path.orgJane Larkindale - Critical Path Institute, 1730 E. River Road, Tucson, AZ, 85705, USAAlexander Berg - Critical Path Institute, 1730 E. River Road, Tucson, AZ, 85705, USAMicki Hill - University of Leicester, Leicester, England, UKJackson Burton - Critical Path Institute, 1730 E. River Road, Tucson, AZ, 85705, USAKeith R Abrams - University of Leicester, Leicester, England, UKRichard T Abresch - Critical Path Institute, 1730 E. River Road, Tucson, AZ, 85705, USAAbby Bronson - Parent Project Muscular Dystrophy, Hackensack, NJ, USADouglass Chapman - Pfizer, Groton, Groton, CT, USAMichael Crowther - University of Leicester, Leicester, England, UKTina Duong - Stanford University, Stanford, CA, USAHeather Gordish-Dressman - Children's National Medical Center, Washington, D.C., USALutz Harnisch - Pfizer, Groton, Groton, CT, USAErik Henricson - University of California, Davis, CA, USASarah Kim - Center for Pharmacometrics and Systems Pharmacology, Department of Pharmaceutics, University of Florida, Orlando, FL, USACraig M McDonald - University of California, Davis, CA, USAStephan Schmidt - Center for Pharmacometrics and Systems Pharmacology, Department of Pharmaceutics, University of Florida, Orlando, FL, USACamille Vong - Pfizer, Groton, Groton, CT, USAXiaoxing Wang - Pfizer, Groton, Groton, CT, USABrenda L Wong - University of Massachusetts Medical School, Boston, MA, USAFlorence Yong - Pfizer, Groton, Groton, CT, USAKlaus Romero - Critical Path Institute, 1730 E. River Road, Tucson, AZ, 85705, USADuchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC)
- Contributors
- Katherine D Mathews (Contributor) - University of Iowa, Stead Family Department of Pediatrics
- Resource Type
- Journal article
- Publication Details
- Journal of pharmacokinetics and pharmacodynamics, Vol.46(5), pp.441-455
- Publisher
- United States
- DOI
- 10.1007/s10928-019-09642-7
- PMID
- 31127458
- ISSN
- 1567-567X
- eISSN
- 1573-8744
- Language
- English
- Date published
- 10/2019
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984070664502771
Metrics
15 Record Views