EU Paediatric Medicines Regulation: Boon or Burden?

Published by: Informa Healthcare

Published: Jan. 1, 2007 - 99 Pages

Table of Contents

CHAPTER 1 INTRODUCTION

1.1 The Paediatric Medicines Regulation

1.2 The need for paediatric testing

1.3 The history of the Regulation

CHAPTER 2 THE PAEDIATRIC REGULATION

2.1 The text of the Regulation

2.2 The Paediatric Committee

2.2.1 Committee composition

2.2.2 Objectives

2.2.3 Safeguards

2.3 Marketing authorisation requirements

2.3.1 General authorisation requirements

2.3.2 Waivers

2.3.3 Paediatric investigation plans

2.3.4 Deferrals

2.3.5 Compliance with the PIP

2.3.6 Procedure

2.3.7 Advice

2.4 Marketing authorisation procedures

2.4.1 Patented products

2.4.2 Off-patent products

2.4.3 Identification

2.4.3.1 Choice of symbol

2.5 Post-authorisation requirements

2.6 Rewards and incentives

2.6.1 Patented products

2.6.2 Orphan products

2.6.3 Off-patent products

2.6.4 Additional funding

2.7 Communication and co-ordination

2.7.1 Clinical trials register

2.7.2 Paediatric therapeutic needs inventory

2.7.3 European network of expertise

2.7.4 Paediatric study reporting requirements

2.8 General and final provisions

2.8.1 Reports and analyses

2.8.2 Annex

2.9 Amending Regulation

2.10 Deadlines

CHAPTER 3 THE EMEA AND THE PAEDIATRIC COMMITTEE

3.1 EMEA funding

3.2 EMEA implementation priorities

3.3 EMEA paediatric activity 1995-2005

3.3.1 Analysis

3.3.2 Conclusions

3.4 Paediatric Expert Group (PEG)

3.4.1 Mandate and objectives

Product-related tasks

Non-product related activities

Activities with external parties

3.4.2 PEG publications

3.4.3 The PEG and the Paediatric Committee

3.5 Paediatric Committee

CHAPTER 4 TEDDY

4.1 Objectives

4.2 Partners

4.3 Activities

4.3.1 Ethics

4.3.1.1 Ethics and pharmacogenetics

4.3.2 Pharmacoepidemiology

4.3.3 Genomics and paediatric pharmacogenetics

4.3.4 Methodology of paediatric clinical trials

4.3.5 Key therapeutic questions in children

4.3.6 Rare diseases

4.3.7 Post-marketing studies

4.3.8 Paediatric drug databases

4.3.9 Gender issues in drug evaluation

CHAPTER 5 US AND EU APROACHES

5.1 The US paediatric initiatives

5.2 Evaluation of the US paediatric initiatives

5.2.1 FDA assessments

5.2.1.1 2001 status report

5.2.1.2 Recent FDA data

5.2.2 Tufts Impact Report

5.2.3 Congressional criticisms

5.2.4 Public Citizen

5.3 Lessons from the US experience

5.4 What the EU has learned from the US experience

CHAPTER 6 OPPORTUNITIES AND COSTS: RAND AND THE EUROPEAN COMMISSION

6.1 R&D-based companies

6.1.1 Costs

6.1.2 Rewards

6.2 Generic companies and SMEs

6.2.1 Costs

6.2.2 Incentives

6.2.3 Orphan drugs

6.2.4 Paediatric study programme

6.3 The EMEA

6.4 Healthcare systems

6.4.1 Costs

6.4.2 Benefits

6.5 Children

6.6 EU research bodies

6.7 Impact outside the EU

CHAPTER 7 OPPORTUNITIES AND COSTS: AN ALTERNATIVE ANALYSIS

7.1 Criticisms of the Rand report

7.2 An alternative calculation of rewards and costs

Calculation 1

Alternative calculation 1

Calculation 2

Alternative calculation 2

Calculation 3

Alternative calculation 3

Calculation 4

Alternative calculation 4

7.3 Impact on R&D-based companies

7.3.1 Costs

7.3.2 Rewards

7.4 Impact on generic companies

7.5 Impact on regulatory agencies

7.5.1 The EMEA

7.5.2 National regulatory authorities

7.6 Impact on healthcare systems

CHAPTER 8 WHAT HAPPENS NEXT

8.1 Requirements for companies

8.1.1 All companies

8.1.2 Companies seeking SPC extensions

8.1.3 Companies discontinuing a paediatric product

8.1.4 Pharmacovigilance

8.2 Approval routes

8.2.1 Centralised approval

8.2.2 Decentralised approval

8.3 Penalties for non-compliance

8.4 Clinical trials

8.5 Databases

8.5.1 EudraPharm

8.5.2 EudraVigilance

8.5.3 EudraCT

8.6 Identifying unmet paediatric needs

8.6.1 Paediatric research needs

8.6.2 Paediatric research priorities for off-patent drugs

CHAPTER 9 CONTROVERSIES AND UNCERTAINTIES

9.1 SPCs - too much?

9.1.1 Political disagreements

9.1.2 Alternatives

9.1.3 Support for six months

9.2 PUMAs and MICE - too little?

9.2.1 Data protection

9.2.2 Formulations and formularies

9.2.3 Paediatric Study Fund

9.2.4 Potential PUMA applicants

CHAPTER 10 CONCLUSION

10.1 What the Regulation should achieve

10.2 What the Regulation may not achieve

10.3 Summary of benefits

APPENDIX A

APPENDIX B

REFERENCES

Abstract

Draft EU regulations on paediatric medicines were approved by the European Parliament on 1 June 2006. It came into force on 26 January 2007.

It is estimated that between 50-90% of medicines used to treat children have never been specifically studied in or approved for the paediatric population. Through a combination of obligations and incentives, the proposed regulation aims to ensure that:

• paediatric medicines are subject to high-quality research and clinical development, while avoiding any unnecessary clinical trials in children;
• the resulting products are duly authorised across the EU;
• there is more information on the use of medicines developed specifically for children and more transparency in paediatric clinical trials.

In particular, the new regulations will provide incentives to develop paediatric forms and doses of medicines currently only approved for adults, the main one being a six-month extension to patent protection for innovator companies that test their products on children.

There will also be a separate paediatric marketing authorisation for off-patent medicines that are developed for children and a special fund to encourage research into paediatric forms of such products. The regulation also sets up a paediatric committee at the European Medicines Agency that will agree paediatric investigation plans with companies and determine research priorities.

The R&D-based industry believes the regulation would allow Europe to reclaim a central role in innovative drug development. It is less popular with the generics community. Last minute amendments, negotiated behind the scenes at the European Parliament, mean that during a five-year transition period, innovator companies have up to 6 months before expiry to submit the data and apply for the extension. The original draft suggested a two-year deadline. This change is likely to dramatically affect the timing of generic entry. It is also likely to pose issues for some Eastern European states whose native pharma industry is largely generic and who, since accession, have opposed any measures which have delayed the entry of generic drugs.

Use this new report to:

• Understand and comply with the new legislation
• Profit from the incentives on offer as a result of the new legislation
• Understand how similar legislation has been applied in the US and assess its impact
• Identify key market opportunities for on and off patent drugs

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