C-Path Launches Clinical Trial Simulator for Duchenne Muscular Dystrophy Research
Critical Path Institute’s (C-Path) Duchenne Regulatory Science Consortium (D-RSC) is excited to announce the launch of...
In 2008, C-Path embarked on a visionary journey, setting the stage for innovation in model-informed drug development (MIDD). A pivotal turning point was reached in 2015 when we significantly expanded our capabilities. In 2019, we cemented our commitment by formalizing the Quantitative Medicine Program.
A major challenge in drug development clinical trials is the proper handling of variability and uncertainty. Without a proper statistical approach, drug development risks including more patients than necessary, resulting in excessive costs and resource requirements, or worse, risks including too few patients, resulting in failed trials. Currently available solutions to handle variability and uncertainty are either nonexistent, inefficient, or not readily accessible. Efficient, publicly available, targeted, and regulatory-grade quantitative solutions are needed to address these specific unmet needs in drug development for various disease areas.
The work of the Quantitative Medicine (QuantMed) Program at Critical Path Institute (C-Path) focuses on the development of advanced, regulatory grade, data analysis (quantitative) solutions to accelerate development. Successes include the transformation of drug development paradigms in areas including (but not limited to) Parkinson’s, Alzheimer’s, Type 1 diabetes, tuberculosis, and a wide array of rare diseases. QuantMed leverages knowledge from a network of experts in industry, academia, nonprofit, and regulatory sciences combined with integrated data from multiple sources to develop actionable solutions that combine clinical pharmacology, statistics, mechanistic modeling, artificial intelligence, pharmacometrics, and digital health technology (or remote health technology) data.
The solutions developed by QuantMed are often formally reviewed by regulatory agencies and endorsed for specific applications in drug development. Because QuantMed’s philosophy is one of open science, solutions developed are publicly available as open-source platforms. The QuantMed Program is highly collaborative and advances the development of novel treatments for patients with unmet medical needs.
Ahamadi M, Conrado DJ, Macha S, Sinha V, Stone J, Burton J, Nicholas T, Gallagher J, Dexter D, Bani M, Boroojerdi B, Smit H, Weidemann J, Chen C, Yang M, Maciuca R, Lawson R, Burn D, Marek K, Venuto C, Stafford B, Akalu M, Stephenson D, Romero K; Critical Path for Parkinson’s (CPP) Consortium. Development of a disease progression model for leucine-rich repeat kinase 2 in Parkinson’s disease to inform clinical trial designs. Clin Pharmacol Ther. 2019 Sep 23. [Epub ahead of print]
Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K; Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC). Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy. J Pharmacokinet Pharmacodyn. 2019 May 24. [Epub ahead of print]
Johnson K, Gomez A, Burton J, White D, Chakravarty A, Schmid A, Bottino D. Directional inconsistency between Response Evaluation Criteria in Solid Tumors (RECIST) time to progression and response speed and depth. Eur J Cancer. 2019 Mar;109:196-203.
Mulberg AE, Bucci-Rechtweg C, Giuliano J, Jacoby D, Johnson FK, Liu Q, Marsden D, McGoohan S, Nelson R, Patel N, Romero K, Sinha V, Sitaraman S, Spaltro J, Kessler V. Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders. Orphanet J Rare Dis. 2019 Feb 8;14(1):36.
Romero K, Conrado D, Burton J, Nicholas T, Sinha V, Macha S, Ahamadi M, Cedarbaum J, Seibyl J, Marek K, Basseches P, Hill D, Somer E, Gallagher J, Dexter DT, Roach A, Stephenson D; Critical Path for Parkinson’s (CPP) Consortium; Parkinson’s Progression Markers Initiative (PPMI). Molecular Neuroimaging of the Dopamine Transporter as a Patient Enrichment Biomarker for Clinical Trials for Early Parkinson’s Disease. Clin Transl Sci. 2019 May;12(3):240-246.
Soul JS, Pressler R, Allen M, Boylan G, Rabe H, Portman R, Hardy P, Zohar S, Romero K, Tseng B, Bhatt-Mehta V, Hahn C, Denne S, Auvin S, Vinks A, Lantos J, Marlow N, Davis JM; International Neonatal Consortium. Recommendations for the design of therapeutic trials for neonatal seizures. Pediatr Res. 2019;85(7):943-954.
Stegall MD, Troy Somerville K, Everly MJ, Mannon RB, Gaber AO, First MR, Agashivala N, Perez V, Newell KA, Morris RE, Sudan D, Romero K, Eremenco S, Mattera M, Spear N, Porter AC, O’Doherty I. The importance of drug safety and tolerability in the development of new immunosuppressive therapy for transplant recipients: The Transplant Therapeutics Consortium’s position statement. Am J Transplant. 2019 Mar;19(3):625-632.
Stephenson D, Hill D, Cedarbaum JM, Tome M, Vamvakas S, Romero K, Conrado D J, Dexter DT, Seibyl J, Jennings D, Nicholas T, Matthews D, Xie Z, Imam S, Maguire P, Russell D, Gordon MF, Stebbins GT, Somer E, Gallagher J, Roach A, Basseches P, Grosset D, Marek K; Critical Path for Parkinson’s Consortium. The Qualification of an Enrichment Biomarker for Clinical Trials Targeting Early Stages of Parkinson’s Disease. J Parkinsons Dis. 2019;9(4):825.
Stephenson D, Hill D, Cedarbaum JM, Tome M, Vamvakas S, Romero K, Conrado DJ, Dexter DT, Seibyl J, Jennings D, Nicholas T, Matthews D, Xie Z, Imam S, Maguire P, Russell D, Gordon MF, Stebbins GT, Somer E, Gallagher J, Roach A, Basseches P, Grosset D, Marek K; Critical Path for Parkinson’s Consortium. The Qualification of an Enrichment Biomarker for Clinical Trials Targeting Early Stages of Parkinson’s Disease. J Parkinsons Dis. 2019;9(3):553-563.
Woosley RD, Romero K, Heise CW, Gallo T, Tate J, Woosley RL. Summary of Torsades de Pointes (TdP) Reports Associated with Intravenous Drug Formulations Containing the Preservative Chlorobutanol. Drug Saf. 2019 Jul;42(7):907-913.
Klaus Romero, MD, MS,
Chief Executive Officer, Chief Science Officer
Shu Chin Ma, PhD, MSc, M. Phil, EMBA,
Vice President, Model-informed Drug Development and Quantitative Medicine
Jagdeep Podichetty, PhD
Senior Director of Predictive Analytics
Yi Zhang, PhD
Director of Pharmacometrics
Sakshi Sardar, PhD
Senior Director, Digital and Precision Medicine
Kimberly Collins, PhD
Senior Quantitative Medicine Scientist, Pharmacometrics
Luke Kosinski, PhD
Scientific Director, Regulatory Strategy
Nicholas Henscheid, MS, PhD
Quantitative Medicine Scientist
Zihan Cui, PhD
Senior Quantitative Medicine Developer
Lauren Quinlan
Quantitative Medicine Developer II
Wes Anderson
Quantitative Medicine Scientist
Ruby Abrams, PhD
Quantitative Medicine Scientist, Digital and Precision Medicine
Grace Lee, PhD
Quantitative Medicine Scientist, Digital and Precision Medicine
Rachel Xu, MS
Quantitative Medicine Developer
Francisco Morales, PhD
Quantitative Medicine Scientist
Christine Miller
Senior Project Manager
Grace Erhart
Project Manager
Bri Sullivan
Project Coordinator II