Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Drug Discovery: Overview01:26

Drug Discovery: Overview

7.2K
Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
7.2K
Preclinical Development: Overview01:28

Preclinical Development: Overview

4.0K
Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
4.0K
Clinical Trials: Overview01:11

Clinical Trials: Overview

2.5K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
2.5K
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

386
Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
386
Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

658
Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
658
EPS and iPS Cells in Disease Research01:21

EPS and iPS Cells in Disease Research

2.7K
Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
2.7K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Beyond Impossibility: Reimagining Clinical Evidence for Children with Ultra-Rare Diseases.

Journal of clinical pharmacology·2026
Same author

Invisible No More: How Redefining Rare Diseases Can Advance Health Equity.

Journal of clinical pharmacology·2026
Same author

Population Pharmacokinetics and Exposure-Response Model-Based Bayesian Extrapolation of FVC-Based Efficacy Endpoints From Adults to Pediatric Patients Receiving Nintedanib.

CPT: pharmacometrics & systems pharmacology·2025
Same author

Improvements in Data Quality Can Boost Efficiency and Reduce Development Costs: A Pharmacometric CRO's Perspective.

The AAPS journal·2025
Same author

Has Pharmacologic Contextualization Become a Lost Art? How Can We Revive the Integrator Pharmacologist?

Journal of clinical pharmacology·2025
Same author

Exposure-Efficacy Meta-Model of Nintedanib in Adult Patients With Chronic Fibrosing Interstitial Lung Diseases.

CPT: pharmacometrics & systems pharmacology·2025

Related Experiment Video

Updated: May 13, 2025

In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
08:04

In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing

Published on: May 11, 2021

2.7K

Innovations and Best Practices for Therapeutic Development in Pediatric Rare Diseases: A Model-Informed Drug

Rajesh Krishna1, Satyendra Suryawanshi2, Juliane Rascher3

  • 1Certara Drug Development Solutions, Certara USA, Inc., Radnor, Pennsylvania, USA.

Clinical Pharmacology and Therapeutics
|April 16, 2025
PubMed
Summary

Model-informed drug development (MIDD) enhances therapeutic development for pediatric rare diseases by enabling efficient, data-driven decisions. This approach optimizes trials and personalizes treatments for children with rare conditions.

More Related Videos

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery
12:40

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery

Published on: May 19, 2018

10.2K
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
05:10

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System

Published on: December 11, 2016

9.2K

Related Experiment Videos

Last Updated: May 13, 2025

In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
08:04

In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing

Published on: May 11, 2021

2.7K
Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery
12:40

Using Human Induced Pluripotent Stem Cell-derived Hepatocyte-like Cells for Drug Discovery

Published on: May 19, 2018

10.2K
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
05:10

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System

Published on: December 11, 2016

9.2K

Area of Science:

  • Pharmacology and Therapeutics
  • Rare Diseases
  • Pediatric Medicine

Background:

  • Pediatric rare disease drug development faces challenges due to small, heterogeneous patient populations and limited treatment options.
  • Existing challenges are compounded by differences between adult and pediatric disease presentations.
  • Regulatory guidance (ICH E11(R1)) emphasizes modeling and simulation to address knowledge gaps and uncertainties.

Purpose of the Study:

  • To critically examine the role of model-informed drug development (MIDD) in advancing therapeutic strategies for pediatric rare diseases.
  • To explore the integration of biomarkers, statistical innovations, and best practices in modeling and simulation.
  • To highlight the potential of digital biomarkers, patient-reported outcomes, and quality of life measures.

Main Methods:

  • Review and analysis of existing literature and regulatory guidance on MIDD in pediatric rare diseases.
  • Examination of the application of modeling and simulation techniques for extrapolation and decision-making.
  • Discussion of the integration of novel data sources like digital biomarkers and patient-reported outcomes.

Main Results:

  • MIDD offers a powerful solution for efficient, data-driven decision-making in pediatric rare disease drug development.
  • MIDD facilitates extrapolation of adult treatment responses to pediatric populations, aiding target viability and dose selection.
  • The integration of advanced methodologies can reduce the need for large, costly trials while ensuring relevant clinical endpoints.

Conclusions:

  • MIDD is crucial for overcoming challenges in pediatric rare disease therapeutic development.
  • Integrating digital biomarkers, patient-reported outcomes, and quality of life methodologies will drive personalized, patient-centered care.
  • These advancements promise a significant shift towards more effective and tailored therapies for this vulnerable population.