Pediatric Medicines: Global Markets

Chapter 1 Introduction to Pediatric Medicine

• Introduction
• Study Goals and Objectives
• Reasons for Doing This Study
• Scope of the Report
• Intended Audience
• Methodology and Information Sources
• Analyst's Credentials
• Related Reports

Chapter 2 Summary and Highlights

Chapter 3 Pediatric Market Dynamics

• Addressable Pediatric Population
• Off-Label Drug Use
• Areas of Unmet Clinical Need
• Market Resistors and Drivers

Chapter 4 Regulation of Pediatric Medicine

• The U.S.
• The E.U.
• Paediatric Committee
• Rewards and Incentives
• Procedural Guidelines
• Japan
• Australia
• Canada
• Switzerland
• The Impact of New Pediatric Legislation
• Alignment Between the FDA and EMA

Chapter 5 Challenges of Developing Pediatric Medicine

• Access to Pediatric Medicines
• Pediatric Clinical Trials
• Trial Designs
• Ethical Considerations
• Recruitment and Retention
• Age-Appropriate Formulations
• Product Approval
• The U.S.
• The E.U.
• Japan
• Pricing and Reimbursement
• The U.S.
• The E.U.
• Japan

Chapter 6 Opportunities for Pediatric Medicine

• Therapeutic Areas for Pediatric Drug Development
• Leading Therapeutics Categories
• Allergy and Respiratory Conditions
• Anti-Infectives
• Antidiabetes Therapies
• Central Nervous System, Neurology and Mental Health
• Oncology
• Orphan Drug and Rare Diseases
• Other Pediatric Therapies
• Vaccines

Chapter 7 Future Outlook for Pediatric Medicine

• Market Trends and Analysis
• Regional Trends and Analysis
• The U.S.
• Europe
• Japan
• Rest of World (ROW)
• Overall Conclusions

Chapter 8 Company Profiles

• Leading Pharmaceutical Companies
• Abbott Laboratories
• Astellas Pharma Inc.
• Astrazeneca Plc
• Bristol-Myers Squibb Co.
• Eli Lilly & Co.
• Forest Laboratories
• Gilead Sciences
• Glaxosmithkline Plc
• Hoffmann La Roche
• Merck & Co.
• Novartis Ag
• Pfizer
• Sanofi
• Shire Plc
• Sumitomo Dainippon Pharma Co.
• Teva Pharmaceuticals
• UCB
• Leading Biotechnology Companies
• Avexis
• Biomarin Pharmaceuticals
• Biogen Inc.
• Gw Pharmaceuticals
• Jacobus Pharmaceuticals
• Proqr Therapeutics Nv
• Regeneron Pharmaceuticals Inc.
• Stemline Therapeutics Inc.
• Vertex Pharmaceuticals Inc.
• Leading Pediatric Networks
• International Networks
• National Networks
• Medicines For Clinical Research Network - U.K.
• Medicines For Clinical Research Network - The Netherlands
• Finnish Investigators Network For Paediatric Medicines - Finland
• Pediatric Trials Network - U.S.

Chapter 9 Appendix: Acronyms

Chapter 10 References

List of Tables
Summary Table: Global Pediatric Market, Through 2024
Table 1: Distributions of the World Population, by Age Group, 2018, 2025 and 2050
Table 2: Population Between 0 and 14 Years of Age, by Region, 2018
Table 3: Categories of Off-Label Use
Table 4: Examples of Common Off-Label Use in Children
Table 5: Key Areas of Unmet Pediatric Need To be Cited by the EMA, 2013
Table 6: Global Pediatric Market, by Region, Through 2024
Table 7: Comparison of the Main Feature of the U.S. Pediatric Incentive Programs, 2019
Table 8: Classification of Pediatric Age Categories in Europe and the U.S.
Table 9: Procedural Guidelines for Pediatric Drug Development for Europe and the U.S.
Table 10: Scientific Guidelines for Pediatric Drug Development for Europe and the U.S.
Table 11: Current Pediatric Legislative/Regulatory Initiatives
Table 12: Key Global Pediatric Initiatives
Table 13: Leading Causes of Death in Postneonatal Children, Risk Factors and Response, WHO 2019
Table 14: Priority Medicines for Children Under Five Years of Age
Table 15: Priority Medicines for Child Health and Survival in Need of R&D
Table 16: Top 10 Countries Ranked by Study Participation and Patient Enrollment for Studies on Drugs, 2007-2010
Table 17: Top 10 Countries Ranked by Study Participation and Patient Enrollment for Studies on Vaccines, 2007-2010
Table 18: U.S. Pediatric Networks
Table 19: Criteria for Reimbursement in Countries in the WHO European Region, 2017
Table 20: FDA Recently Approved Pediatric Drugs
Table 21: Pediatric Drugs Approved for Respiratory Conditions Through Pediatric Regulatory Pathways
Table 22: Global Sales of Top Five Pediatric Drugs Approved for Allergies and Respiratory Conditions, 2018
Table 23: Global Sales of Top Five Pediatric Drugs Approved for Allergies and Respiratory Conditions, by Region, 2018
Table 24: Global Sales of Top Five Pediatric Drugs Approved for Allergies and Respiratory Conditions, 2018
Table 25: Pediatric Drugs Approved for Infectious Diseases Through Pediatric Regulatory Pathways, 2018
Table 26: Sales of Anti-Infective Pediatric Drugs, 2018
Table 27: Global Sales of Top Five Pediatric Drugs Approved for Infectious Diseases, by Region, 2018
Table 28: Global Sales of Top Five Pediatric Drugs Approved for Infectious Diseases, 2018
Table 29: Leading Pediatric Antidiabetes Therapies, 2018
Table 30: Global Sales of Pediatric Drugs Approved for the Treatment of Diabetes by Route, 2018
Table 31: Global Sales of Pediatric Drugs Approved for the Treatment of Diabetes, by Region, 2018
Table 32: Global Sales of Top Five Pediatric Antidiabetes Drugs, 2018
Table 33: Pediatric Drugs Approved for the Treatment of CNS, Neurology and Mental Health, 2018
Table 34: Global Sales of Pediatric Drugs Approved for CNS, Neurology and Mental Health, 2018
Table 35: Global Sales of Pediatric Drugs Approved for CNS, Neurology and Mental Health, by Region, 2018
Table 36: Global Sales of Top Five Pediatric Drugs Approved for the Treatment of CNS, Neurology and Mental Health, 2018
Table 37: Pediatric Drugs approved for the Treatment of Cancer, 2018
Table 38: Global Sales of Pediatric Drugs Approved in Oncology, by Region, 2018
Table 39: Global Sales of Top Five Pediatric Oncology Drugs, 2018
Table 40: Approved Pediatric Orphan Drugs in the U.S., 2006-2018
Table 41: Pediatric Orphan Drugs in Clinical Development by Orphan Disease, Company, Product and Phase, 2019
Table 42: Global Sales of Pediatric Drugs Approved in Orphan Conditions, by Therapy Area, 2018
Table 43: Global Sales of Pediatric Drugs Approved in Orphan Conditions, by Region, 2018
Table 44: Global Sales of Top Five Pediatric Drugs Approved in Orphan Conditions, 2018
Table 45: Global Sales of Pediatric Drugs Approved for Other Conditions, by Region, 2018
Table 46: Leading Pediatric and Adolescent Vaccines Globally, 2018
Table 47: Global Sales of Vaccines, by Age/Indication, 2018
Table 48: Global Sales of Pediatric Vaccines, by Region, 2018
Table 49: Global Sales of Top Five Pediatric/Adolescent Vaccines, 2018
Table 50: Global Sales of Pediatric Drugs, by Therapeutic Area, Through 2024
Table 51: U.S. Sales of Pediatric Drugs, by Therapeutic Area, Through 2024
Table 52: E.U. Sales of Pediatric Drugs, by Therapeutic Area, Through 2024
Table 53: Japanese Sales of Pediatric Drugs, by Therapeutic Area, Through 2024
Table 54: Rest of World Sales of Pediatric Drugs, by Therapeutic Area, Through 2024

List of Figures
Summary Figure: Global Pediatric Market, 2018-2024
Figure 1: Number of Children Under Age 18 in the U.S. and Percentage of the Total Population 2000-2050
Figure 2: Health Status for Children Under Age 18 Years, by Selected Characteristic, 2018
Figure 3: Adjusted Percentages of Hay Fever, Respiratory Allergies, Food Allergies and Skin Allergies for Children Under Age 18 years, by Age Group, 2018
Figure 4: Number of Orphan Indications Approved in the U.S. since the Orphan Drug Act, 1983-2018
Figure 5: Differences between Adult and Pediatric Drug Design
Figure 6: Aims of the Pediatric Formulations Initiatives
Figure 7: Evolution of Pediatric Legislation in the U.S., 1994-2012
Figure 8: General Principles Guiding Pediatric Drug Development, ICH E11 Guidelines, 2010
Figure 9: Evolution of Pediatric Legislation in Europe
Figure 10: Medical Needs for Pediatric Medicines
Figure 11: Pediatric and Adult Trials as a Percentage of Total Trails, 2010-2018
Figure 12: Medical Needs for Orally Available Pediatric Medicines
Figure 13: FDA Process for Seeking Pediatric Drug Approval
Figure 14: FDA Process for Seeking Pediatric Drug Approval
Figure 15: FDA Process for Expedited Pediatric Drug Approval
Figure 16: Therapeutic Areas for Pediatric Drug Approvals Since the Implementation of Pediatric Regulations
Figure 17: Allergies and Respiratory Conditions for Which Pediatric Medicines Have Been Approved
Figure 18: Number of Medicines in Development for Allergies and Respiratory Diseases
Figure 19: Aniti-Infective Conditions for Which Pediatric Medicines Have Been Approved
Figure 20: Approved Pediatric Antidiabetes Therapies for Type 2 Diabetes
Figure 21: CNS, Neurology and Mental Health Conditions for Which Pediatric Medicines Have Been Approved
Figure 22: Top Five Cancer Sites Associated with Mortality, by Age, 1975-2010
Figure 23: Number of Orphan Drug Designations Applications, Designations and Approved Orphan Products, 1983-2018
Figure 24: Orphan Approvals, by Therapeutic Area, 2018
Figure 25: Orphan Approvals in 5-Year Periods, by Therapeutic Area, 1993-2017
Figure 26: Designated Orphan Medicines for the Treatment of Children and Adults, 2018
Figure 27: EMA Opinions on PIPs and Waivers, 2014-2018
Figure 28: Other Therapeutic Areas of Pediatric Product Approvals
Figure 29: Conditions for Which Vaccines Have Been Approved
Figure 30: Recommended Immunization Schedule for Persons Aged 0 Through 18 Years in the U.S., 2018
Figure 31: Estimated Global Pediatric Product Market Shares, by Therapeutic Area, 2024
Figure 32: Estimated U.S. Pediatric Product Market Shares, by Therapeutic Area, 2024
Figure 33: Estimated E.U. Pediatric Product Market Shares, by Therapeutic Area, 2024
Figure 34: Estimated Japanese Pediatric Product Market Shares, by Therapeutic Area, 2024
Figure 35: Estimated Rest of World Pediatric Product Market Shares, by Therapeutic Area, 2019