Challenges and Opportunities in the Development of Medical Therapies for Pediatric Populations and the Role of Extrapolation

A Clinical Trial to Maintain Glycemic Control in Youth with Type 2 Diabetes

TODAY Study Group (2012) · New England Journal of Medicine · doi:10.1056/nejmoa1109333 · Cited by 978 · see citing works

Learning versus confirming in clinical drug development*

Sheiner, L. B. (1997) · Clinical Pharmacology & Therapeutics · doi:10.1016/s0009-9236(97)90160-0 · Cited by 633 · see citing works

Approval summary for imatinib mesylate capsules in the treatment of chronic myelogenous leukemia.

Cohen, M. H., Williams, G., Johnson, J. R., Duan, J. Z., Gobburu, J. V. S., Rahman, A., Benson, K. A., Leighton, J. K., Kim, S. K., Wood, R., et al. (2002) · PubMed · Cited by 465 · see citing works

Off-Label Use of Drugs in Children

Neville, K. A., Frattarelli, D. A. C., Galinkin, J. L., Green, T. P., Johnson, T. D., MMM, Paul, I. M., & van den Anker, J. N. (2014) · PEDIATRICS · doi:10.1542/peds.2013-4060 · Cited by 393 · see citing works

Youth-Onset Type 2 Diabetes Consensus Report: Current Status, Challenges, and Priorities

Nadeau, K. J., Anderson, B. J., Berg, E. G., Chiang, J. L., Chou, H., Copeland, K. C., Hannon, T. S., Huang, T. T.-K., Lynch, J. L., Powell, J., et al. (2016) · Diabetes Care · doi:10.2337/dc16-1066 · Cited by 381 · see citing works

Good Practices in Model‐Informed Drug Discovery and Development: Practice, Application, and Documentation

Workgroup, E. M., Marshall, S., Burghaus, R., Cosson, V, Cheung, S., Chenel, M., DellaPasqua, O., Frey, N., Hamrén, B., Harnisch, L., et al. (2016) · CPT: Pharmacometrics & Systems Pharmacology · doi:10.1002/psp4.12049 · Cited by 373 · see citing works

Extrapolation of Adult Data and Other Data in Pediatric Drug-Development Programs

Dunne, J., Rodriguez, W. J., Murphy, M. D., Beasley, B. N., Burckart, G. J., Filie, J. D., Lewis, L. L., Sachs, H. C., Sheridan, P. H., Starke, P., et al. (2011) · Pediatrics · doi:10.1542/peds.2010-3487 · Cited by 318 · see citing works

Effects of metformin, metformin plus rosiglitazone, and metformin plus lifestyle on insulin sensitivity and β-cell function in TODAY

TODAY Study Group (2013) · Diabetes Care · doi:10.2337/dc12-2393 · Cited by 288 · see citing works

Induction and Maintenance Therapy With Infliximab for Children With Moderate to Severe Ulcerative Colitis

Hyams, J. S., Damaraju, L. V., BLANK, M. A., Johanns, J., Guzzo, C. A., Winter, H. S., Kugathasan, S., Cohen, S. A., Markowitz, J. F., Escher, J. C., et al. (2011) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2011.11.026 · Cited by 287 · see citing works

Model-Based Drug Development: A Rational Approach to Efficiently Accelerate Drug Development

Milligan, P. A., Brown, M. J., Marchant, B., Martin, S. W., van der Graaf, P. H., Benson, N., Nucci, G., Nichols, D. J., Boyd, R. A., Mandema, J. W., et al. (2013) · Clinical Pharmacology & Therapeutics · doi:10.1038/clpt.2013.54 · Cited by 238 · see citing works

Extrapolation of Adult Data and Other Data in Pediatric Drug-Development Programs

(2011) · PEDIATRICS · doi:10.1542/peds.2010-3487d · Cited by 224 · see citing works

Pediatric chronic myeloid leukemia is a unique disease that requires a different approach

Hijiya, N., Schultz, K. R., Metzler, M., Millot, F., & Suttorp, M. (2015) · Blood · doi:10.1182/blood-2015-06-648667 · Cited by 214 · see citing works

Anti-TNF-α Agents in the Treatment of Immune-Mediated Inflammatory Diseases: Mechanisms of Action and Pitfalls

Silva, L. C., Ortigosa, L. C. M., & Benard, G. (2010) · Immunotherapy · doi:10.2217/imt.10.67 · Cited by 214 · see citing works

The utility of modeling and simulation in drug development and regulatory review

Huang, S.-M., Abernethy, D. R., Wang, Y., Zhao, P., & Zineh, I. (2013) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.23570 · Cited by 206 · see citing works

Copy Number Variations in the Survival Motor Neuron Genes: Implications for Spinal Muscular Atrophy and Other Neurodegenerative Diseases

Butchbach, M. E. R. (2016) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2016.00007 · Cited by 193 · see citing works

Paediatric drug development: The impact of evolving regulations

Turner, M. A., Catapano, M., Hirschfeld, S., & Giaquinto, C. (2014) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2014.02.003 · Cited by 167 · see citing works

Improving the outcome for children with cancer: Development of targeted new agents

Adamson, P. C. (2015) · CA A Cancer Journal for Clinicians · doi:10.3322/caac.21273 · Cited by 143 · see citing works

Manipulation of drugs to achieve the required dose is intrinsic to paediatric practice but is not supported by guidelines or evidence

Richey, R. H., Shah, U. U., Peak, M., Craig, J. V., Ford, J. L., Barker, C. E., Nunn, A. J., & Turner, M. A. (2013) · BMC Pediatrics · doi:10.1186/1471-2431-13-81 · Cited by 140 · see citing works

Clarification on Precision Criteria to Derive Sample Size When Designing Pediatric Pharmacokinetic Studies

Wang, Y., Jadhav, P. R., Lala, M., & Gobburu, J. V. S. (2011) · The Journal of Clinical Pharmacology · doi:10.1177/0091270011422812 · Cited by 116 · see citing works

Adolescent Dosing and Labeling Since the Food and Drug Administration Amendments Act of 2007

Momper, J. D., Mulugeta, Y. A., Green, D. J., Karesh, A., Krudys, K. M., Sachs, H. C., Yao, L. P., & Burckart, G. J. (2013) · Archives of Pediatrics and Adolescent Medicine · doi:10.1001/jamapediatrics.2013.465 · Cited by 114 · see citing works

Safety, dosing, and pharmaceutical quality for studies that evaluate medicinal products (including biological products) in neonates

Ward, R. M., Benjamin, D. K., Barrett, J. S., Allegaert, K., Portman, R. J., Davis, J. M., & Turner, M. A. (2016) · Pediatric Research · doi:10.1038/pr.2016.221 · Cited by 104 · see citing works

Pediatric Analgesic Clinical Trial Designs, Measures, and Extrapolation: Report of an FDA Scientific Workshop

Berde, C. B., Walco, G. A., Krane, E. J., Anand, K. J. S., Aranda, J. V., Craig, K. D., Dampier, C., Finkel, J. C., Grabois, M., Johnston, C. C., et al. (2012) · PEDIATRICS · doi:10.1542/peds.2010-3591 · Cited by 99 · see citing works

Modeling and simulation in pediatric drug therapy: Application of pharmacometrics to define the right dose for children

Vinks, A. A. T. M. M., Emoto, C., & Fukuda, T. (2015) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.169 · Cited by 98 · see citing works

Concepts and Challenges in Quantitative Pharmacology and Model-Based Drug Development

Zhang, L., Pfister, M., & Meibohm, B. (2008) · The AAPS Journal · doi:10.1208/s12248-008-9062-3 · Cited by 93 · see citing works

Guidelines for Pediatric Cancer Centers

Hematology/Oncology, S. O. (2004) · PEDIATRICS · doi:10.1542/peds.113.6.1833 · Cited by 89 · see citing works

Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation

Gamalo, M., Savić, J., Basu, C., Zhao, X., Gopalakrishnan, M., Gao, A., Song, G., Baygani, S., Thompson, L., Xia, H. A., et al. (2017) · Pharmaceutical Statistics · doi:10.1002/pst.1807 · Cited by 89 · see citing works

Pediatric Medicine Development: An Overview and Comparison of Regulatory Processes in the European Union and United States

Penkov, D., Tomasi, P. A., Eichler, I., Murphy, D., Yao, L. P., & Temeck, J. W. (2017) · Therapeutic Innovation & Regulatory Science · doi:10.1177/2168479017696265 · Cited by 87 · see citing works

Early phase clinical trials of anticancer agents in children and adolescents — an ITCC perspective

Moreno, L., Pearson, A. D. J., Paolettí, X., Jiménez, I., Geoerger, B., Kearns, P. R., Zwaan, C. M., Doz, F., Baruchel, A., Vormoor, J. H., et al. (2017) · Nature Reviews Clinical Oncology · doi:10.1038/nrclinonc.2017.59 · Cited by 80 · see citing works

Efficacy of antiepileptic drugs in adults predicts efficacy in children

Pellock, J. M., Carman, W. J., Hoffman, V., Daniels, T., Morris, D. L., & D’Cruz, O. (2012) · Neurology · doi:10.1212/wnl.0b013e31826d5ec0 · Cited by 79 · see citing works

Effects of Metformin, Metformin Plus Rosiglitazone, and Metformin Plus Lifestyle on Insulin Sensitivity and [Beta]-Cell Function in TODAY: TODAY STUDY GROUP

Arslanian, S. A., Pyle, L. L., Payan, M., Bacha, F. F., Caprio, S., Haymond, M. W., Levitsky, L. L., Goland, R., White, N. H., & Sm, W. (2013) · Diabetes Care · Cited by 75 · see citing works

Extrapolation of Efficacy in Pediatric Drug Development and Evidence-based Medicine: Progress and Lessons Learned

Sun, H., Temeck, J. W., Chambers, W., Perkins, G., Bonnel, R., & Murphy, D. (2018) · Therapeutic Innovation & Regulatory Science · doi:10.1177/2168479017725558 · Cited by 70 · see citing works

Model‐Based Drug Development Survey Finds Pharmacometrics Impacting Decision Making in the Pharmaceutical Industry

Stone, J. A., Banfield, C. R., Pfister, M., Tannenbaum, S. J., Allerheiligen, S., Wetherington, J. D., Krishna, R., & Grasela, D. M. (2010) · The Journal of Clinical Pharmacology · doi:10.1177/0091270010377628 · Cited by 50 · see citing works

The Integration of Allometry and Virtual Populations to Predict Clearance and Clearance Variability in Pediatric Populations over the Age of 6 Years

Edginton, A. N., Shah, B., Sevestre, M., & Momper, J. D. (2013) · Clinical Pharmacokinetics · doi:10.1007/s40262-013-0065-6 · Cited by 47 · see citing works

The Need for Rigorous Evidence on Medication Use in Preterm Infants

Davis, J. M., Connor, E. M., & Wood, A. J. J. (2012) · JAMA · doi:10.1001/jama.2012.12883 · Cited by 44 · see citing works

Single‐ and Multiple‐Dose Pharmacokinetics of Pioglitazone in Adolescents With Type 2 Diabetes

Christensen, M. L., Meibohm, B., Capparelli, E. V., Velasquez‐Mieyer, P., Burghen, G. A., & Tamborlane, W. V. (2005) · The Journal of Clinical Pharmacology · doi:10.1177/0091270005279578 · Cited by 42 · see citing works

Antiepileptic Drug Development in Children

Chiron, C., Dulac, O., & Pons, G. (2008) · Drugs · doi:10.2165/00003495-200868010-00002 · Cited by 41 · see citing works

Remaining Challenges in Childhood Cancer and Newer Targeted Therapeutics

Smith, M. A., & Reaman, G. H. (2014) · Pediatric Clinics of North America · doi:10.1016/j.pcl.2014.09.018 · Cited by 41 · see citing works

Expanding Treatment Options for Youth With Type 2 Diabetes: Current Problems and Proposed Solutions

Tamborlane, W. V., Haymond, M. W., Dunger, D. B., Shankar, R. R., Gubitosi‐Klug, R., Bethin, K. E., Karres, J., Tomasi, P. A., Libman, I. M., Hale, P. H., et al. (2016) · Diabetes Care · doi:10.2337/dc15-1649 · Cited by 39 · see citing works

Modeling and Simulation as a Tool to Bridge Efficacy and Safety Data in Special Populations

Harnisch, L. O., Shepard, T., Pons, G., & Pasqua, O. E. D. (2013) · CPT Pharmacometrics & Systems Pharmacology · doi:10.1038/psp.2013.6 · Cited by 37 · see citing works

Regulation (EC) No 1901/2006 on medicinal products for paediatric use & clinical research in vulnerable populations

Lehmann, B. (2008) · Child and Adolescent Psychiatry and Mental Health · doi:10.1186/1753-2000-2-37 · Cited by 35 · see citing works

Use of Modeling and Simulation in the Design and Conduct of Pediatric Clinical Trials and the Optimization of Individualized Dosing Regimens

Stockmann, C. R., Barrett, J. S., Roberts, J., & Sherwin, C. M. T. (2015) · CPT Pharmacometrics & Systems Pharmacology · doi:10.1002/psp4.12038 · Cited by 34 · see citing works

Immediate Rescue Designs in Pediatric Analgesic Trials

Kossowsky, J., Donado, C., & Berde, C. B. (2014) · Anesthesiology · doi:10.1097/aln.0000000000000445 · Cited by 32 · see citing works

Joining Forces: A Call for Greater Collaboration to Study New Medicines in Children and Adolescents With Type 2 Diabetes

Karres, J., Pratt, V., Guettier, J., Temeck, J. W., Tamborlane, W. V., Dunger, D. B., Bejnariu, C., de Beaufort, C. E., & Tomasi, P. A. (2014) · Diabetes Care · doi:10.2337/dc14-0494 · Cited by 30 · see citing works

The development of antiepileptic drugs for children report from the NIH workshop, Bethesda, Maryland, February 17–18, 1994

Sheridan, P. H., & Jacobs, M. P. (1996) · Epilepsy Research · doi:10.1016/0920-1211(95)00043-7 · Cited by 29 · see citing works

Model‐Based Drug Development: Strengths, Weaknesses, Opportunities, and Threats for Broad Application of Pharmacometrics in Drug Development

Wetherington, J. D., Pfister, M., Banfield, C. R., Stone, J. A., Krishna, R., Allerheiligen, S., & Grasela, D. M. (2010) · The Journal of Clinical Pharmacology · doi:10.1177/0091270010377629 · Cited by 28 · see citing works

Modeling and simulation for medical product development and evaluation: highlights from the FDA-C-Path-ISOP 2013 workshop

Romero, K., Sinha, V. P., Allerheiligen, S. R. B., Danhof, M., Pinheiro, J. C., Kruhlak, N. L., Wang, Y., Wang, S.-J., Sauer, J. M., Marier, J., et al. (2014) · Journal of Pharmacokinetics and Pharmacodynamics · doi:10.1007/s10928-014-9390-0 · Cited by 18 · see citing works

Type 2 Diabetes in Pediatrics and Adults: Thoughts from a Clinical Pharmacology Perspective

Vaidyanathan, J., Choe, S. Y., & Sahajwalla, C. G. (2012) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.23085 · Cited by 18 · see citing works

Pediatric drug development programs for type 2 diabetes: A review

Christensen, M. L., Franklin, B. E., Momper, J. D., & Reed, M. D. (2015) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.497 · Cited by 14 · see citing works

Esomeprazole FDA Approval in Children With GERD

Earp, J. C., Mehrotra, N., Peters, K. E., Fiorentino, R. P., Griebel, D. J., Lee, S. C., Mulberg, A. E., Rohss, K. M., Sandström, M., Taylor, A. C., et al. (2016) · Journal of Pediatric Gastroenterology and Nutrition · doi:10.1097/mpg.0000000000001467 · Cited by 14 · see citing works

The treatment of pediatric Philadelphia positive (Ph+) leukemias in the imatinib era

Burke, M. J., Willert, J. R., Desai, S. J., & KADOTA, R. P. (2009) · Pediatric Blood & Cancer · doi:10.1002/pbc.22172 · Cited by 12 · see citing works

Adverse Event Detection and Labeling in Pediatric Drug Development: Antiretroviral Drugs

Momper, J. D., Chang, Y., Jackson, M. S., Schuette, P., Seo, S. K., Younis, I. R., Abernethy, D. R., Yao, L. P., Capparelli, E. V., & Burckart, G. J. (2015) · Therapeutic Innovation & Regulatory Science · doi:10.1177/2168479014565471 · Cited by 12 · see citing works

Extrapolation in the development of paediatric medicines: examples from approvals for biological treatments for paediatric chronic immune-mediated inflammatory diseases

Stefanska, A. M., Distlerová, D., Musaus, J., Olski, T. M., Dunder, K., Salmonson, T., Mentzer, D., Müller‐Berghaus, J., Hemmings, R., & Veselý, R. (2017) · Archives of Disease in Childhood · doi:10.1136/archdischild-2016-312259 · Cited by 12 · see citing works

Pediatric News

(2016) · The Journal of Pediatric Pharmacology and Therapeutics · doi:10.5863/1551-6776-21.1.98 · Cited by 8 · see citing works

Hematopoietic stem cell transplantation and immunotherapy for pediatric acute myeloid leukemia: an open challenge

Lucchini, G., & Bader, P. (2014) · Expert Review of Hematology · doi:10.1586/17474086.2014.878644 · Cited by 6 · see citing works

Stratification, Hypothesis Testing, and Clinical Trial Simulation in Pediatric Drug Development

McMahon, A. W., Watt, K., Wang, J., Green, D. J., Tiwari, R. C., & Burckart, G. J. (2016) · Therapeutic Innovation & Regulatory Science · doi:10.1177/2168479016651661 · Cited by 5 · see citing works

Physiologically-Based and Population Pharmacokinetic Modeling and Simulation to Support Dose Selection and Study Design for Eslicarbazepine Acetate (ESL) Adjunctive Therapy in Infants with Partial Onset Seizures (POS) (P3.244)

Sunkaraneni, S., Ludwig, E. A., Bihorel, S., Morris, D., Hopkins, S. C., Galluppi, G. R., Fiedler‐Kelly, J., & Blum, D. E. (2017) · Neurology · doi:10.1212/wnl.88.16_supplement.p3.244 · Cited by 1 · see citing works

Modeling and Simulation Strategy to Support Eslicarbazepine Acetate (ESL) Pediatric Dose Selection in the Treatment of Partial Onset Seizures Based on Matching Adult Exposures (P3.246)

Sunkaraneni, S., Bihorel, S., Hopkins, S. C., Fiedler‐Kelly, J., Ludwig, E. A., Galluppi, G. R., & Blum, D. E. (2017) · Neurology · doi:10.1212/wnl.88.16_supplement.p3.246 · Cited by 1 · see citing works

Applications, Challenges, and Outlook for PBPK Modeling and Simulation: A Regulatory, Industrial and Academic Perspective

Lin, W., Chen, Y., Unadkat, J. D., Zhang, X., Wu, D., & Heimbach, T. (2022) · Pharmaceutical Research · doi:10.1007/s11095-022-03274-2 · Cited by 137 · see citing works

Best Practices to Maximize the Use and Reuse of Quantitative and Systems Pharmacology Models: Recommendations From the United Kingdom Quantitative and Systems Pharmacology Network

Cucurull‐Sanchez, L., Chappell, M. J., Chelliah, V., Amy Cheung, S. Y., Derks, G., Penney, M., Phipps, A., Malik‐Sheriff, R. S., Timmis, J., Tindall, M. J., et al. (2019) · CPT: Pharmacometrics & Systems Pharmacology · doi:10.1002/psp4.12381 · Cited by 61 · see citing works

Commentary on the EMA R eflection P aper on the use of extrapolation in the development of medicines for paediatrics

Ollivier, C., Thomson, A., Manolis, E., Blake, K. V., Karlsson, K. E., Knibbe, C. A. J., Pons, G., & Hemmings, R. (2019) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.13883 · Cited by 42 · see citing works

Pediatric Drug Development: Outlook for Science‐Based Innovation

Green, D. J., Zineh, I., & Burckart, G. J. (2018) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.1001 · Cited by 31 · see citing works

Dose rationale and pharmacokinetics of dexmedetomidine in mechanically ventilated new-borns: impact of design optimisation

van Dijkman, S. C., Cock, P. A. J. G. D., Smets, K. J., Decaluwe, W., Smits, A., Allegaert, K., Walle, J. V., Paepe, P. D., & Pasqua, O. E. D. (2019) · European Journal of Clinical Pharmacology · doi:10.1007/s00228-019-02708-y · Cited by 23 · see citing works

Extrapolation of Efficacy and Dose Selection in Pediatrics: A Case Example of Atypical Antipsychotics in Adolescents With Schizophrenia and Bipolar I Disorder

Kalaria, S. N., Farchione, T. R., Uppoor, R. S., Mehta, M., Wang, Y., & Zhu, H. (2021) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.1836 · Cited by 18 · see citing works

Accelerating Drug Development in Pediatric Oncology With the Clinical Pharmacology Storehouse

Shebley, M., Menon, R. M., Gibbs, J. P., Dave, N., Kim, S. Y., & Marroum, P. J. (2018) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.1359 · Cited by 16 · see citing works

A Model‐Based Framework to Inform the Dose Selection and Study Design of Emicizumab for Pediatric Patients With Hemophilia A

Yoneyama, K., Schmitt, C., Chang, T. Y., Dhalluin, C., Nagami, S., Petry, C., & Levy, G. G. (2021) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.1968 · Cited by 13 · see citing works

Pediatric Extrapolation in Type 2 Diabetes: Future Implications of a Workshop

Barrett, J. S., Bucci-Rechtweg, C. M., Cheung, S. Y. A., Gamalo, M., Haertter, S., Karres, J., Marquard, J., Mulugeta, Y. A., Ollivier, C., Strougo, A., et al. (2020) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.1805 · Cited by 13 · see citing works

Dosing Common Medications in Hospitalized Pediatric Patients with Obesity: A Review

Ameer, B., & Weintraub, M. A. (2020) · Obesity · doi:10.1002/oby.22739 · Cited by 12 · see citing works

A Quantitative Justification of Similarity in Placebo Response Between Adults and Adolescents With Acute Exacerbation of Schizophrenia in Clinical Trials

Kalaria, S. N., Zhu, H., Farchione, T. R., Mathis, M. V., Gopalakrishnan, M., Uppoor, R. S., Mehta, M., & Younis, I. R. (2019) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.1501 · Cited by 10 · see citing works

The Influence of Haemostatic System Maturation on the Dose–Response Relationship of Unfractionated Heparin

Derbalah, A., Duffull, S. B., Moynihan, K. M., & Al-Sallami, H. S. (2020) · Clinical Pharmacokinetics · doi:10.1007/s40262-020-00949-0 · Cited by 9 · see citing works

Pediatric oncology drug development and dosage optimization

Cheung, S. Y. A., Hay, J. L., Lin, Y.-W., de Greef, R., & Bullock, J. M. (2024) · Frontiers in Oncology · doi:10.3389/fonc.2023.1235947 · Cited by 9 · see citing works

Role of Clinical Pharmacology in Diversity and Inclusion in Global Drug Development: Current Practices and Industry Perspectives: White Paper

Sawant‐Basak, A., Urva, S., Mukker, J. K., Haertter, S., Mariano, D., Parasrampuria, D. A., Goteti, K., Singh, R. S. P., Chiney, M. S., Liao, M. Z., et al. (2024) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.3350 · Cited by 8 · see citing works

Advancing research and development of anti-infectives for children with a focus on antiretroviral therapy: A clinical development perspective

Buchanan, A. M., Bekker, A., Chandasana, H., DeMasi, R. A., Lulic, Z., Ernest, T. B., Brothers, C. H., Min, S., Ruel, T., & Tan, L. (2024) · International Journal of Antimicrobial Agents · doi:10.1016/j.ijantimicag.2024.107306 · Cited by 8 · see citing works

Industry Perspective on Using MIDD for Pediatric Studies Requiring Integration of Ontogeny

Corriol‐Rohou, S., & Cheung, S. Y. A. (2019) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.1495 · Cited by 7 · see citing works

Pharmacokinetic evaluation of vigabatrin dose for the treatment of refractory focal seizures in children using adult and pediatric data

Rodrigues, C., Chiron, C., Ounissi, M., Dulac, O., Gaillard, S., Nabbout, R., & Jullien, V. (2019) · Epilepsy Research · doi:10.1016/j.eplepsyres.2019.01.002 · Cited by 7 · see citing works

Evaluation of non-linear-mixed-effect modeling to reduce the sample sizes of pediatric trials in type 2 diabetes mellitus

Rigaux, C., & Sébastien, B. (2020) · Journal of Pharmacokinetics and Pharmacodynamics · doi:10.1007/s10928-019-09668-x · Cited by 6 · see citing works

Pregabalin Population Pharmacokinetic and Exposure‐Response Analyses for Focal Onset Seizures in Children (4–16 years) and Adults, to Support Dose Recommendations in Children

Chan, P. L. S., Marshall, S. F., McFadyen, L., & Liu, J. (2020) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.2132 · Cited by 6 · see citing works

Risk assessment of therapeutic agents under consideration to treat COVID‐19 in paediatric patients and pregnant women

Barrett, J. S. (2020) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.14630 · Cited by 5 · see citing works

Harnessing formulation and clinical pharmacology knowledge for efficient pediatric drug development: Overview and discussions from M-CERSI pediatric formulation workshop 2019

Khong, Y. M., Liu, J., Cook, J., Purohit, V. S., Thompson, K. C., Mehrotra, S., Cheung, S. Y. A., Hay, J. L., Fletcher, E. P., Wang, J., et al. (2021) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2021.04.010 · Cited by 5 · see citing works

Asking More of Our EHR Systems to Improve Outcomes for Pediatric Patients

Barrett, J. S. (2020) · Frontiers in Pharmacology · doi:10.3389/fphar.2020.00253 · Cited by 2 · see citing works

Pediatric Extrapolation Approach for U.S. Food and Drug Administration Approval of Brexpiprazole in Patients Aged 13 to 17 Years with Schizophrenia

Zhang, H., Liu, J., Sharma, V. L., Zhuang, L., Horn, P., Uppoor, R. S., Mehta, M., & Zhu, H. (2024) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.2429 · Cited by 2 · see citing works

Trends in clinical pharmacology as represented by international and specialized journals

Колбин, А. С., Radaeva, К. S., Motrinchuk, A. S., & Размиковна, С. И. (2024) · Kachestvennaya Klinicheskaya Praktika = Good Clinical Practice · doi:10.37489/2588-0519-2024-2-33-42 · Cited by 2 · see citing works

Seeking Similarities Rather Than Differences With Adults to Aid in Therapeutic Advancement for Children

Becker, M. L., & Kraft, W. K. (2021) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.2285 · Cited by 2 · see citing works

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

Hay, J. L., Manon, A., Stephens, H., Cheung, S. Y. A., de Keizer, B., & Beek, E. T. T. (2025) · British Journal of Clinical Pharmacology · doi:10.1002/bcp.70418 · Cited by 1 · see citing works

Supporting Prospective Pregnancy Trials via Modeling and Simulation: Lessons From the Past and Recommendations for the Future

Cheung, S. Y. A., & Barrett, J. S. (2023) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.2284 · Cited by 1 · see citing works

A paradigm shift

Rose, K. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-823888-2.00005-3

Breaking Down Barriers to Effective Patient Care

O’Donnell, P. H. (2018) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.1026

Challenges at the interface of science, drug development, and drug approval beyond specific disease areas

Rose, K. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-823888-2.00032-6

Introduction: specific disease areas

Rose, K. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-823888-2.00021-1

What do young patients really need?

Rose, K. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-823888-2.00023-5

Introduction

Rose, K. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-823888-2.00030-2

Neonatology

Rose, K. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-823888-2.00020-x

Global Statistics and Data Sciences (GSDS), BeiGene USA, Fulton, MD

Ye, J., Pan, H., Reaman, G. H., Roychoudhury, S., Lu, C. C., Renfro, L. A., Ji, Y., Liu, R., Yuan, Y., & Zhang, W. (2024) · Medical Research Archives · doi:10.18103/mra.v12i1.5088

Global Statistics and Data Sciences (GSDS), BeiGene USA, Fulton, MD

Ye, J., Pan, H., Reaman, G. H., Roychoudhury, S., Lu, C. C., Renfro, L. A., Ji, Y., Liu, R., Yuan, Y., & Zhang, W. (2024) · Medical Research Archives · doi:10.18103/mra.v11i11.5088

Editorial comment on “Benralizumab in children with severe eosinophilic asthma: Pharmacokinetics and long‐term safety ( TATE study)”

Cabrera‐Perez, J. S., Eigenmann, P., & Akenroye, A. T. (2024) · Pediatric Allergy and Immunology · doi:10.1111/pai.14128

Nirmatrelvir/Ritonavir for the Treatment of COVID-19 in Children Aged 6 Years and Older

Gerhart, J. G., Leister-Tebbe, H. K., Chan, P. L., McComsey, G. A., Carey, W., Shi, H., Yan, J., Leaney, J. L., Singh, N., Baniecki, M. L., et al. (2026) · PEDIATRICS · doi:10.1542/peds.2025-073999

Pediatric Extrapolation

Ye, J., & Nie, L. (2024) · ICSA book series in statistics · doi:10.1007/978-3-031-60843-8_7

Extrapolation as a Default Strategy in Pediatric Drug Development

Gamalo, M., Bucci-Rechtweg, C. M., Nelson, R. M., Vanh, L., Porcalla, A., Thackray, H. M., Cooner, F., Cutler, L., Friend, D., & Portman, R. (2022) · Therapeutic Innovation & Regulatory Science · doi:10.1007/s43441-021-00367-9 · Cited by 22 · see citing works

Quantitative system pharmacology as a legitimate approach to examine extrapolation strategies used to support pediatric drug development

Azer, K., & Barrett, J. S. (2022) · CPT Pharmacometrics & Systems Pharmacology · doi:10.1002/psp4.12801 · Cited by 13 · see citing works

Bridging the gap

Wadsworth, I. (2018) · Lancaster EPrints (Lancaster University) · doi:10.17635/lancaster/thesis/348

Developmental Pharmacodynamics and Modeling in Pediatric Drug Development

Conklin, L. S., Hoffman, E. P., & van den Anker, J. N. (2019) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.1482 · Cited by 27 · see citing works