Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

the Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC)

Research output: Contribution to journalArticle

1 Citation (Scopus)

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.

Original languageEnglish (US)
JournalJournal of Pharmacokinetics and Pharmacodynamics
DOIs
StatePublished - Jan 1 2019

Fingerprint

Duchenne Muscular Dystrophy
Clinical Protocols
Rare Diseases
Uncertainty
Clinical Trials
Pharmaceutical Preparations
Information Storage and Retrieval
Statistical Models
United States Food and Drug Administration
Therapeutics
Databases
Population

Keywords

  • Drug development tools
  • Duchenne muscular dystrophy consortium (D-RSC)
  • Model-informed drug development
  • Rare diseases
  • Regulatory endorsement

ASJC Scopus subject areas

  • Pharmacology

Cite this

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy. / the Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC).

In: Journal of Pharmacokinetics and Pharmacodynamics, 01.01.2019.

Research output: Contribution to journalArticle

@article{c361a69d565a4f4ca94d93b6346dc6f1,
title = "Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy",
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.",
keywords = "Drug development tools, Duchenne muscular dystrophy consortium (D-RSC), Model-informed drug development, Rare diseases, Regulatory endorsement",
author = "{the Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC)} and Conrado, {Daniela J.} and Jane Larkindale and Alexander Berg and Micki Hill and Jackson Burton and Abrams, {Keith R.} and Abresch, {Richard T.} and Abby Bronson and Douglass Chapman and Michael Crowther and Tina Duong and Heather Gordish-Dressman and Lutz Harnisch and Erik Henricson and Sarah Kim and McDonald, {Craig M} and Stephan Schmidt and Camille Vong and Xiaoxing Wang and Wong, {Brenda L.} and Florence Yong and Klaus Romero and V. Vishwanathan and S. Chidambaranathan and {Douglas Biggar}, W. and McAdam, {Laura C.} and Mah, {Jean K.} and Mar Tulinius and Avital Cnaan and Morgenroth, {Lauren P.} and Robert Leshner and Carolina Tesi-Rocha and Mathula Thangarajh and Andrew Kornberg and Monique Ryan and Yoram Nevo and Alberto Dubrovsky and Clemens, {Paula R.} and Hoda Abdel-Hamid and Connolly, {Anne M.} and Alan Pestronk and Jean Teasley and Bertorini, {Tulio E.} and Richard Webster and Hanna Kolski and Nancy Kuntz and Sherilyn Driscoll and Bodensteiner, {John B.} and Ksenija Gorni and Henricson, {Erik K}",
year = "2019",
month = "1",
day = "1",
doi = "10.1007/s10928-019-09642-7",
language = "English (US)",
journal = "Journal of Pharmacokinetics and Pharmacodynamics",
issn = "1567-567X",
publisher = "Springer New York",

}

TY - JOUR

T1 - Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy

AU - the Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC)

AU - Conrado, Daniela J.

AU - Larkindale, Jane

AU - Berg, Alexander

AU - Hill, Micki

AU - Burton, Jackson

AU - Abrams, Keith R.

AU - Abresch, Richard T.

AU - Bronson, Abby

AU - Chapman, Douglass

AU - Crowther, Michael

AU - Duong, Tina

AU - Gordish-Dressman, Heather

AU - Harnisch, Lutz

AU - Henricson, Erik

AU - Kim, Sarah

AU - McDonald, Craig M

AU - Schmidt, Stephan

AU - Vong, Camille

AU - Wang, Xiaoxing

AU - Wong, Brenda L.

AU - Yong, Florence

AU - Romero, Klaus

AU - Vishwanathan, V.

AU - Chidambaranathan, S.

AU - Douglas Biggar, W.

AU - McAdam, Laura C.

AU - Mah, Jean K.

AU - Tulinius, Mar

AU - Cnaan, Avital

AU - Morgenroth, Lauren P.

AU - Leshner, Robert

AU - Tesi-Rocha, Carolina

AU - Thangarajh, Mathula

AU - Kornberg, Andrew

AU - Ryan, Monique

AU - Nevo, Yoram

AU - Dubrovsky, Alberto

AU - Clemens, Paula R.

AU - Abdel-Hamid, Hoda

AU - Connolly, Anne M.

AU - Pestronk, Alan

AU - Teasley, Jean

AU - Bertorini, Tulio E.

AU - Webster, Richard

AU - Kolski, Hanna

AU - Kuntz, Nancy

AU - Driscoll, Sherilyn

AU - Bodensteiner, John B.

AU - Gorni, Ksenija

AU - Henricson, Erik K

PY - 2019/1/1

Y1 - 2019/1/1

N2 - 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.

AB - 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.

KW - Drug development tools

KW - Duchenne muscular dystrophy consortium (D-RSC)

KW - Model-informed drug development

KW - Rare diseases

KW - Regulatory endorsement

UR - http://www.scopus.com/inward/record.url?scp=85066886701&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85066886701&partnerID=8YFLogxK

U2 - 10.1007/s10928-019-09642-7

DO - 10.1007/s10928-019-09642-7

M3 - Article

C2 - 31127458

AN - SCOPUS:85066886701

JO - Journal of Pharmacokinetics and Pharmacodynamics

JF - Journal of Pharmacokinetics and Pharmacodynamics

SN - 1567-567X

ER -