Pediatric Medicines: Global Markets
REPORT SCOPE:
This new report on pediatric medicine will provide a brief overview on the current status of the industry and recent developments. It presents the changing environment in terms of new legislation and implementation of global pediatric plans. The report analyzes market trends and identifies key therapeutic and geographical challenges and rising opportunities in the developed and developing world. Finally, the report provides insights on how all stakeholders have an essential interest in prioritizing pediatric drug development targets and improving access to safe and effective medicines to children across the globe.
REPORT INCLUDES:
This new report on pediatric medicine will provide a brief overview on the current status of the industry and recent developments. It presents the changing environment in terms of new legislation and implementation of global pediatric plans. The report analyzes market trends and identifies key therapeutic and geographical challenges and rising opportunities in the developed and developing world. Finally, the report provides insights on how all stakeholders have an essential interest in prioritizing pediatric drug development targets and improving access to safe and effective medicines to children across the globe.
REPORT INCLUDES:
- 55 tables
- An overview of the global markets for pediatric medicines within the pharmaceutical industry
- Analyses of the global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024
- Discussion on market drivers, off-label use, novel formulations and areas of unmet clinical need and emerging countries
- Market analysis for therapeutic fields including neonates and paediatrics childhood cancers, CNS disorders, infectious diseases, immune inflammatory diseases, metabolic diseases, respiratory conditions, vaccinations
- Regulatory environment and incentives for pediatric development
- Profiles of major companies of the industry, including Abbott Laboratories, GlaxoSmithKline, Novartis AG, Sanofi and Vertex Pharmaceuticals Inc.
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 BCC Research 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
Introduction
Study Goals and Objectives
Reasons for Doing This Study
Scope of the Report
Intended Audience
Methodology and Information Sources
Analyst's Credentials
Related BCC Research 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 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
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 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
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
