Science & Research
Resources for You
PUBLICATIONS: 2006- 2013
From the Office of Pediatric Therapeutics and FDA
A. PEER REVIEWED ARTICLES PUBLISHED:
Published (N=47) Pending (N=6)
1. Systematic Review of Alternate Endpoints and Clinical Outcome Assessments in Pediatric Ulcerative Colitis Registration Trials.
Sun H, Lee JJ, Papadopoulos EJ, Lee CS, Nelson RM, Sachs HC, Rodriguez WJ, Mulberg AE.
J Pediatr Gastroenterol Nutr 2013 Oct 10 [Epub ahead of print] http://www.accessdata.fda.gov/scripts/publications/search_result_record.cfm?id=48038
2. *Failed Pediatric Trials of Acute Migraine Treatments: Lessons Learned From a Systematic Review of Trial Data Submitted to the Food and Drug Administration (FDA). Haihao Sun, MD, PhD, Eric Bastings, MD, Jean Temeck, MD, P. Brian Smith, MD MPH MHS, Angela Men, MD, Veneeta Tandon, PhD, Dianne Murphy, MD; William Rodriguez, MD, PhD
JAMA Pediatrics March 2013; 167 (3): 243-249
3. The Impact of the Pediatric Written Request Process on Drug Development in Childhood Cancer. Kristen M.Snyder, MD, Gregory Reaman, MD, Debbie Avant, RPh, Richard Pazdur, MD
Pediatric Blood & Cancer (Published online Jan 13, 2013)
In Progress for 2013
1. Analysis of method to capture adverse events in children in tertiary care pediatric hospitals. McMahon AW, Abrams B, Wharton GT, MS, Weinel P, Damilano CP, DeLeon D, Lieu P, Yen L, Taketomo C, Jackson B, Snyder J, Thornton PS, Sood B, Doe E, Baker R, Rodriguez W, Murphy D.
2. What We Have Learned from Pediatric Studies: Progress and Economic Overview. Gerold T. Wharton, BA; Dianne Murphy, MD, John Goldsmith, PhD, Debbie Avant, RPh, William J. Rodriguez, MD, PhD, Eric L. Eisenstein, DBA
3. *Literature Review Assessing the Impact of Supplementing Infant Formula with Long Chain Polyunsaturated Fatty Acids. Haihao Sun, MD, PhD, Wiley Chambers, MD, P. Brian Smith, MD, MPH MHS, L. Corbin Downey, MD, William Rodriguez, MD, PhD, Dianne Murphy, MD
4. Drug Labeling and Exposure in Neonates. Matthew M. Laughon, MD, Debbie Avant, RPh, Nidhi Tripathi, MD, Christoph Hornik, MD, Michael Cohen-Wolkowiez, MD, Reese Clark, MD, P. Brian Smith, MD, William Rodriguez, MD, PhD
(Accepted and to be published by JAMA Pediatrics online Dec 9, 2013)
5. Steps towards Harmonization for Clinical Development of Medicines in Pediatric Ulcerative Colitis--a Global Scientific Discussion.
Part 1: Efficacy Endpoints and Disease Outcome Assessments
Haihao Sun, MD, PhD, Richard Vesely, MD, Jan Taminiau,MD, Peter Szitanyi, MD,Maria Isaac, MD, Agnes Klein, MD, Shinobu Uzu, Donna Griebel, MD, Andrew E. Mulberg, MD on behalf of the International Inflammatory Bowel Disease (i-IBD) Working Group
(Provisionally accepted by JPGN)
6. Steps towards Harmonization for Clinical Development of Medicines in Pediatric Ulcerative Colitis --- Global Scientific Discussion.
Part 2: Data Extrapolation, Trial Design, and Pharmacokinetics
Haihao Sun, MD, PhD, Richard Vesely, MD, Robert M Nelson, MD, PhD, Jan Taminiau, MD, Peter Szitanyi, MD, Maria Isaac, MD, Agnes Klein, MD, Shinobu Uzu, Donna Griebel, MD, Andrew E. Mulberg, MD on behalf of the international Inflammatory Bowel disease (i-IBD) Working Group
(Provisionally accepted by JPGN)
1. The Globalization of Pediatric Clinical Trials. Julia Dunne, MD, Dianne Murphy, MD, William Rodriguez, MD, PhD
Pediatrics December 2012; 130 (6): e1583 -e1591 (Published online November 5,2012)
2. Pediatric Use of Insulin Pump Technology: A Retrospective Study of Adverse events in Children ages 1-12 Years. Judith U. Cope, MD, MPH, Joy H. Samuels-Reid, MD, Audrey E. Morrison, RN, BSN.
J Diabetes Sci Technol September 2012; 6(5):1053-1059 **
3. Trends of Outpatient Prescription Drug Utilization in U.S. Children, 2002 - 2010. Grace Chai, Pharm D, Laura Governale, Pharm D, MBA, Ann W. McMahon, MD, MS, Judy Staffa, PhD, R Ph, Dianne Murphy, MD
Pediatrics, 2012 June 18 [Epub ahead of print]
4. Pediatric Devices and Adverse Events from A to Z: Understanding the Benefits and Risks. Joy Samuels-Reid, MD, Brenda Lawrence, MD, Courtney Millin, PhD, Judith Cope, MD, MPH
Expert Review of Medical Devices, June 2012; 9 (3): 275-282 **
5. A capacity-based approach for addressing ancillary care needs: implications for research in resource limited settings. Bright Pl, Nelson RM
J Med Ethics. 2012 May 5.[Epub ahead of print] **
6. Research Letter: Pediatric Information in Drug Product Labeling. Aaron N. Sachs, BS, Debbie Avant, RPh, Catherine S. Lee, DrPH, William Rodriguez, MD, PhD, M. Dianne Murphy, MD
JAMA May 2012; 307(18): 1914-1915
7. Factors Related to Voluntary Parental Decision-Making in Pediatric Oncology. Victoria A. Miller, PhD, Robert M. Nelson, MD, PhD
Pediatrics April 2012; 129: 903-909
8. Is there an objective way to compare research risks? Rossi J, Nelson RM
J Med Ethics. 2012 Feb 25. [Epub ahead of print] **
9. What Orphan Drug Act has done lately for children with rare diseases: A 10-Year Analysis. Chandana Thorat, BTech, Kui Xu, MD, PhD, Scott Freeman, PhD, Renan Bonnel, PharmD, MPH, Francesca Joseph, MD, M Phillips, PhD, DSc, Menfo Imoisili, MD, MPH
Pediatrics Feb 2012/peds.2011-1798
1. An Analysis of the Effect of Age on Risks of Long-Acting Beta-2 Agonists. Ann W. McMahon, MD, MS, Mark S. Levenson, PhD, Bradley W. McEvoy, DrPH, Andrew D. Mosholder, MD, MPH, and Dianne Murphy, MD
Pediatrics October 2011/peds.2010-1720
2. Extrapolating of Adult and Other Data in Pediatric Drug Development Programs. Julia Dunne, William Rodriguez, Dianne Murphy, Nhi Beasley, Gilbert Burckhart, Jane Filie, Linda Lewis, Hari Sachs, Philip Sheridan, Peter Starke and Lynne Yao
Pediatrics October 2011; peds.2010-3487
1. Globalization Facilitates Pediatric Drug Development in the 21st Century. Julia Dunne, Lala Margaryants, M. Dianne Murphy, Ann M. Myers, Debbie Avant and William J. Rodriguez
Drug Information Journal 2010 : 44(06)
2. Letter to the Editor: The Globalization of Pediatric Trials: Should We be Worried? Julia Dunne, Dianne Murphy, Gerold Wharton
Pediatrics October 6, 2010
3. Emergency Department (ED) Visits for Medical Device-Associated Adverse Events Among Children in United States. Wang C, Hefflin B, Cope JU, Gross T, Ritchie MB, Qi Y, Chu J.
Pediatrics Aug 2010; 126: 247-259
4. Ethical and Regulatory Considerations for the Inclusion of Adolescents in HIV Biomedical Prevention Research. Nelson, Robert M MD, PhD; Lewis, Linda L MD; Struble, Kimberly PharmD; Wood, Susan F PhD
J Acquir Immune Defic Syndr July 2010;54:S18–S24 **
5. Nine key functions for a human subjects protection program for community-engaged research: points to consider. Ross LF, Loup A, Nelson RM, Botkin JR, Kost R, Smith GR, Gehlert S.
J Empir Res Hum Res Ethics. Mar 2010;5(1):33-47 **
6. The challenges of collaboration for academic and community partners in a research partnership: points to consider. Ross LF, Loup A, Nelson RM, Botkin JR, Kost R, Smith GR, Gehlert, S.
J Empir Res Hum Res Ethics. Mar 2010;5(1):19-31 **
7. Human subjects protections in community-engaged research: a research ethics framework. Ross LF, Loup A, Nelson RM, Botkin JR, Kost R, Smith GR, Gehlert S.
J Empir Res Hum Res Ethics. Mar 2010;5(1):5-17 **
8. *Cough in Pediatric Patients Receiving Angiotensin-Converting Enzyme Inhibitor Therapy or Angiotensin Receptor Blocker Therapy in Randomized Controlled Trials. Baker-Smith CM, Benjamin DK Jr, Califf RM, Murphy MD, Li JS, Smith PB.
Clin Pharmacol Ther. 2010 Feb 3
9. Assessment of parental decision-making in neonatal cardiac research: a pilot study. Nathan AT, Hoehn KS, Ittenbach RF, Gaynor JW, Nicolson S, Wernovsky G, Nelson RM.
J Med Ethics. Feb 2010;36(2):106-110 **
1. Safety and Transparency of Pediatric Drug Trials. Daniel K. Benjamin Jr, MD, PhD, MPH; P. Brian Smith, MD, MHS; M. Jessica M. Sun, MD; M. Dianne Murphy, MD; Debbie Avant, RPh; Lisa Mathis, MD; William Rodriguez, MD; Robert M. Califf, MD; Jennifer S. Li, MD, MHS
Arch Pediatr Adolesc Med. December 2009;163(12):1080-1086 **
2. ACR Appropriateness Criteria on Developmental Dysplasia of the Hip—Child Expert Panel on Pediatric Imaging. Boaz K. Karmazyn, MD; Richard Gunderman, MD, PhD; Brian D. Coley, MD; Ellen R. Blatt, MD; Dorothy Bulas, MD; Lynn Fordham, MD, Daniel J. Podberesky, MD; Jeffrey Scott Prince, MD; Charles Paidas, MD; William Rodriguez, MD.
Journal of the American College of Radiology (JACR), Volume 6 No. 8, August 2009, pgs 551-557 **
3. Drug Therapy In Pediatrics: A Developing Field. Lisa Mathis, MD and William Rodriguez, MD.
Dermatologic Therapy Journal, Volume 22, p. 257-261, May-June 2009 **
4. Device Safety and Effectiveness in the Pediatric Population: A US FDA Perspective. Joy Samuels-Reid, Judith U. Cope, and Audrey E. Morrison
Expert Rev Med. Devices 6(2), 131-135, March 2009 **
5. Parental perception of time and decision-making in neonatal research. Hoehn KS, Nathan A, White LE, Ittenbach RF, Reynolds WW, Gaynor JW, Wernovsky G, Nicolson S, Nelson RM.
J Perinatol. 2009 Jul;29(7):508-11 **
6. Challenges in measuring a new construct: perception of voluntariness for research and treatment decision making. Miller VA, Reynolds WW, Ittenbach RF, Luce MF, Beauchamp TL, Nelson RM.
J Empir Res Hum Res Ethics. 2009 Sep;4(3):21-31. **
1. Amoxicillin For Post-Exposure Inhalational Anthrax (Peia) In Pediatrics: Rationale For Dosing Recommendations. J. J. Alexander MD, MPH.; P. M. Colangelo, PharmD, PhD.; C. K. Cooper MD; R. Roberts MD; W. J. Rodriguez MD; Mary D. Murphy MD
Pediatric Infectious Disease Journal, Volume 27, No. 11, November 2008
2. *The economic returns of pediatric clinical trials of antihypertensive drugs. Carissa M. Baker-Smith, MD, MS, Daniel K. Benjamin Jr, MD, PhD, Henry G. Grabowski, PhD, Elizabeth D. Reid, BA, Barry Mangum, PharmD, John V. Goldsmith, PhD, M. Dianne Murphy, MD, Rex Edwards, BS, Eric L. Eisenstein, DBA, Jessica Sun, MD, Robert M. Califf, MD, and Jennifer S. Li, MD, MHS
American Heart Journal Volume 156, Issue 4, October 2008, Pages 682-688
3. Pediatric Drug Surveillance and the Food and Drug Administration's Adverse Events Reporting System: An Overview of Reports, 2003-2007. Rosemary Johann-Liang MD, Jo Wyeth Pharm D, Min Chen MS, MPH, and Judith Cope MD, MPH
Pharmacoepidemiology and Drug Safety (2008), November, DOI:10.1002/pds.1679
4. *Racial Difference in Blood Pressure Response to Angiotensin Converting Enzyme Inhibitors in Children: A Meta Analysis. Jennifer S. Li MD MHS, Carissa M. Baker-Smith MD, P. Brian Smith MD, Vic Hasselblad PhD, M. Dianne Murphy MD, Robert M. Califf MD, Daniel K. Benjamin Jr. MD PhD
Clinical Pharmacology and Therapeutics, September 2008, Vol. 84, Issue 3, p.315-319
5. *Safety Monitoring of Drugs Receiving Pediatric Marketing Exclusivity. Smith PB, Benjamin DK Jr, Murphy MD, Johann-Liang R, Iyasu S, Gould B, Califf RM, Li JS,
Pediatrics, Sept. 2008, Vol. 122, Issue 3. e 628-633
6. Adolescent Use of Insulin and Patient Controlled Analgesia Pump Technology: A 10-year FDA Retrospective Study of Adverse Events. Judith U. Cope, MD, MPH1, Audrey E. Morrison, RN, BScN1, and Joy Samuels-Reid, MD, FAAP
Pediatrics 2008; e1133-e1138, Sept. 2008
7.*Racial differences in blood pressure response to angiotensin-converting enzyme inhibitors in children: a meta-analysis. Li JS, Baker-Smith CM, Smith PB, Hasselblad V, Murphy MD, Califf RM, Benjamin DK Jr.
Clin Pharmacol Ther. 2008 Sep;84(3):315-9. Epub 2008 Jun 11. Review. Erratum in: Clin Pharmacol Ther. 2008 Nov;84(5):636
8. *Pediatric Antihypertensive Trial Failures: Analysis of Endpoints and Dose Range. Daniel K. Benjamin, Jr., MD, PhD, MPH; P. Brian Smith, MD; Pravin Jadhav, PhD; Jogarao V. Gobburu, PhD; M. Dianne Murphy, MD; Victor Hasselblad, PhD; Carissa Baker-Smith, MD; Robert M. Califf, MD; and Jennifer S. Li, MD, MHS
Hypertension 2008; 51; 834-840, March 10, 2008
9. Improving Pediatric Dosing Through Pediatric Initiatives – What We Have Learned. William Rodriguez, M.D., Ph.D.; Arzu Selen, Ph.D.; Debbie Avant, R.Ph.; Chandra Chaurasia, Ph.D.; Terrie Crescenzi, R.Ph.; Gerlie Gieser, Ph.D.; Jennifer Di Giacinto, Pharm.D.; Shiew-Mei Huang, Ph.D.; Peter Lee, Ph.D.; Lisa Mathis, M.D.; Dianne Murphy, M.D.; Shirley Murphy, M.D.; Rosemary Roberts, M.D.; Hari Cheryl Sachs, M.D.; Sandra Suarez, Ph.D.; Veneeta Tandon, Ph.D.; and Ramana S. Uppoor, Ph.D.
Pediatrics Vol. 121, Number 3, March 2008
10. *Safety of Placebo Controls in Pediatric Hypertension Trials. Brian Smith MD MHS, Jennifer Li MD MHS, Dianne Murphy MD, Robert M. Califf and Daniel K Benjamin MD PhD MPH
Hypertension 2008; 51; 829-833, February 19, 2008
11. “Vertebroplasty and Kyphoplasty Are Associated with an Increased Risk of Secondary Vertebral Compression Fractures: A Population-based Cohort Study.” Mudano AS, Bian J, Cope JU, Curtis JR, Gross TP, Allison JJ,Kim Y, Briggs D, Melton , Juan Xi J, Saag KG.
Osteoporosis International 2008 Sep 17
12. Parent-child roles in decision-making about medical research. Ethics & Behavior (Special Issue: Ethical Issues in Intervention Research with Children and Adolescents).Miller VM, Reynolds WW and Nelson RM.
Ethics & Behavior, 1532-7019, Volume 18, Issue 2, 2008, Pages 161 – 181
13. The Children's Hospital of Philadelphia's experience with donation after cardiac death. Naim MY, Hoehn KS, Hasz RD, White LS, Helfaer MA, Nelson RM.
Crit Care Med. 2008 Jun;36(6):1729-33
14. Physicians' contact with families after the death of pediatric patients: a survey of pediatric critical care practitioners' beliefs and self-reported practices.. Borasino S, Morrison W, Silberman J, Nelson RM, Feudtner C.
Pediatrics. 2008 Dec ;122(6):e1174-8
1. *Economic Return of Clinical Trials Performed Under the Pediatric Exclusivity Program. Jennifer S. Li, MD, MHS, Eric L. Eisenstein, DBA, Henry G. Grabowski, PhD, Elizabeth D. Reid,, Barry Mangum, PharmD, Kevin A.. Schulman, MD, John V. Goldsmith, PhD, M. Dianne Murphy, MD Robert M. Califf, MD, Daniel K. Benjamin, Jr, MD, PhD
JAMA, February 7, 2007-Vol 297, No. 5
1. *Peer-Reviewed Publication of Clinical Trials Completed for Pediatric Exclusivity. Daniel K. Benjamin, Jr, MD, PhD,Philip Brian Smith, MD,M. Dianne Murphy, MD, Rosemary Roberts, MD, Lisa Mathis, MD, Debbie Avant, RPh, Robert M. Califf, MD, Jennifer S. Li, MD, MHS.
JAMA, September 13, 2006—Vol. 296, No. 10
2. More Appropriate Clinical Trial Design for Drug Labels. McCune S, Mathis L, Cochetto D, Ball S and Rodriguez W.
Drug Information Journal April 2006; 40:185-195
3. *Criteria Supporting the Study of Drugs in the Newborn. Ward R, Benitz E, Benjamin D. Blackmon L., Giacoia G.,Huddack M., Lasky T., Rodriguez W and Selen A.
Clinical Therapeutics September 2006; 28:1385-1398
B) BOOK or BOOK CHAPTERS:
Published (N=10) Pending (N=1)
1. Pharmacovigilance in Pediatrics
Dianne Murphy MD, Judith Cope MD, MPH, Solomon Iyasu MD, MPH. in Pharmacovigilance, 3nd Edition, Editors: R.D. Mann and Andrews.
Accepted by Wiley & Sons in 2012 (pending)
1. Pediatric Drug Development: Concepts and Applications. 2nd Edition. Editors: Andrew E. Mulberg, Dianne Murphy, Julia Dunne, Lisa L. Mathis. Published by Wiley & Sons, August 2013
2. Extrapolation. Dunne J, Rodriguez W, and Murphy D in Pediatric Drug Development: Concepts and Applications. 2nd Edition. Editors: Andrew E. Mulberg, Dianne Murphy, Julia Dunne, Lisa L. Mathis. Published by Wiley & Sons, August 2013
3. Challenges Evaluating the Safety of Pediatric Therapeutic Agents in Trials and Post-Marketing Studies. McMahon AW, Feudtner C, Murphy D in Pediatric Drug Development: Concepts and Applications. 2nd Edition. Editors: Andrew E. Mulberg, Dianne Murphy, Julia Dunne, Lisa L. Mathis. Published by Wiley & Sons, August 2013
4. Infection Control in the Child Care Center and Preschool. "Amebiasis" (p.91-94), "Cryptosporidia" (p.119-122), " Escherichia coli (Diarrhea)" (p. 138-142), and "Yersinia" (p.138-142), by Jantausch Barbara and Rodriguez William, edited by Dr. Leigh B. Grossman.
ISBN: 978-1-936287-64-2, Paperback, 351 pages, Demos Medical Publishing, February 2012
5. Pharmacovigilance in Pediatrics. Solomon Iyasu, MD, MPH and Dianne Murphy, MD in Pharmacovigilance, 2nd Edition, Editors: R.D. Mann and Andrews.
Published by Wiley & Sons, January 2007
6. Pediatric Medical Device Use. Judith Cope, MD, MPH and Thomas Gross, MD, MPH.
Chapter 15: Medical Device Epidemiology and Surveillance. Pgs 203-218.
Editors:Brown SL, Bright RA, Tavris DR.
Published by Wiley & Sons, Ltd., 2007.
7. Labeling of Pediatric Pain Medications In "Pain in Children: A Practical Guide for Primary Care,” Hari Cheryl Sachs, MD, FAAP, Debbie Avant, R.Ph, and William Rodriguez, MD, PhD, Editor Gary A. Walco, PhD and Kenneth R. Goldshneider, MD
Published by Humana Press, July 2008
8. Additional Protections for Children Enrolled in Clinical Investigations. Pages 81-102 in Pediatric Drug Development: Concepts and Applications, by Robert “Skip” Nelson” edited by Andrew E. Mulberg, Steven Silber, and John N. van den Anker.
ISBN: 978-0-470-16929-2, Hardcover, 683 pages, April 2009, Wiley-Blackwell.
9. United States Paediatric Legislation Impact on Paediatric Studies. Pages 12-14 in Guide to Paediatric Drug Development and Clinical Research, Rodriguez William and Maldonado Sam Editors Klaus Rose and John N. van den Anker
ISBN 978-3-8055-9362-5, Hardcover, 221pages, June 2010, S. Karger AG, Basel
10. Penicillins, Beta-Lactamase Inhibitors, Cephalopsporins, and Other Beta-Lactams, in Neonatal and Pediatric Pharmacology: Therapeutic Principles in Practice, Fourth Edition, by Sumathi Nambiar and William J. Rodriguez edited by Sumner J. Yaffe and Jacob V. Aranda.
Published Nov 1, 2010
1. Letter to the Editor: Why Any Analysis of Infant Formula Effects on Infant Visual Acuity Performed Before the Age of 7 Years is Not Likely Predictive.
Wiley A. Chambers, MD, Dianne Murphy, MD, William Rodriguez, MD, PhD, Hiahao Sun, MD, PhD
Pediatrics. February 11, 2013
2. Time for a focus on pediatric friendly formulations
Mansoor A. Khan, PhD RPh and William Rodriguez, MD PhD
Infectious Diseases in Children. June 2011
3. Children Neglected in Clinical Drug Trials. Perspective by Benjamin Ortiz, MD
Infectious Diseases in Children. 2012 June; 24 (6)
1. Pediatric Therapies: Post-marketing Safety Reviews. Judith U. Cope, MD, MPH, Dianne Murphy, MD, Amy Odegaard, MPH, Pamela Weinel, MS, MBA, RN
Presented on May 5, 2013, Pediatric Academies Society (PAS) meeting in Washington D.C
2. Off-Label Use of Octreotide in Infants. Ann W McMahon, Suzanne Treadway, Pamela Weinel, Cecilia P. Damilano, Phuong Lieu, Lilly Yen, Judith Cope, Marilyn Flack, Suzanne Rich and M.Dianne Murphy
Presented on August 17, 2012 at the Annual Meeting of the International Society of Pharmacoepidemiology (ISPE) in Barcelona, Spain.
3. Review of Alternate Endpoints and Activity Indices Used in the Pediatric Ulcerative Colitis Registration Trials. Haihao Sun, MD PhD, Catherine S Lee, DrPH, Jessica J Lee, MD, Robert M Nelson, MD PhD, Hari C Sachs, MD, William J Rodriguez, MD PhD and Andrew E Mulberg, MD
Presented on April 30, 2012 Pediatric Academic Society (PAS) meeting in Boston.
4. Pediatric Use of Insulin Pump Technology: A Food and Drug Administration Retrospective Study of Adverse Events in Children Ages 1-12 years. Judith U. Cope, MD, MPH, Joy H. Samuels-Reid, MD, Audrey E. Morrison, RN
Presented on April 20-21, 2012 at International Clinical Diabetes and Technology meeting in Los Angeles.
5. Extrapolating Efficacy, Maximizing the Use of Adult & Other Data in Pediatric Drug Development. Julia Dunne, William Rodriguez, Dianne Murphy, Nhi Beasley, Gilbert Burckhart, Jane Filie, Linda Lewis, Hari Sachs, Philip Sheridan, Peter Starke and Lynne Yao
Presented on April 30, 2011, Pediatric Academic Society (PAS) meeting.
Work involving the Duke IPA faculty is identified by an *.
** A copy can be requested from Office of Pediatric Therapeutics.