Pediatric Investigation Plans (PIP) in the EU
The European Medicines Agency plays an important role in the development of medicines for children. In the past, many medicines authorized in Europe were not studied adequately or authorized in children. This caused difficulties for prescribers and pharmacists treating children, as well as for their patients and careers.
To help to solve this problem, the European Union's Paediatric Regulation gave the EMA new responsibilities in 2007. These responsibilities allowed the EMA to stimulate research into the uses of medicines in children and to lead to their authorization in all ages.
The Paediatric Regulation's main aim is to improve the health of children in Europe without subjecting children to unnecessary trials, or delaying the authorisation of medicinal products for use in adults.
The Regulation came into force on 26 January 2007. Its main impact was the establishment of the Paediatric Committee (PDCO), which is responsible for coordinating the EMA's work on medicines for children. The Committee's main role is to determine the studies that companies must carry out on children as part of paediatric investigation plans. The PDCO replaced the Agency's previous Paediatric Working Group.
Paediatric development has now become a more integral part of the overall development of medicinal products in the EU, with the Regulation working as a major catalyst to improve the situation for young patients.
Several incentives for the development of medicines for children are available in the EU and in Member States:
- Medicines that have been authorized across the EU with the results of PIP studies included in the product information are eligible for an extension of their patent protection by six months. This is the case even when the studies' results are negative;
- For orphan medicines, the incentive is an additional two years of market exclusivity;
- Scientific advice and protocol assistance at the Agency are free of charge for questions relating to the development of medicines for children;
- Medicines developed specifically for children that are already authorized but are not protected by a patent or supplementary protection certificate, can apply for a paediatric-use marketing authorization (PUMA). If a PUMA is granted, the product will benefit from 10 years of market protection as an incentive.
The EMA maintains a database of studies carried out in children that were completed before the Paediatric Regulation came into force in 2007. The database contains the details of each study, including its title, its aims, the medicines studied and the ages of the patients included.
The EMA works closely with its international partners on medicines for children:
- It has agreed to share information with the United States Food and Drug Administration (FDA) on scientific and ethical issues related to the development of medicines for children, to foster global development plans;
- It participated in the launch of the World Health Organization (WHO) initiative make medicines child size;
- It is a member of the Paediatric Medicines Regulators' Network (PmRN). This is a network of national medicines regulatory authorities set up by the WHO in 2010, which promotes the quality and availability of medicines for children.
Why should you Attend: Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union.
Designation as an orphan medicinal product in the EU has important advantages. Sponsors who obtain orphan designation benefit from a number of incentives, including protocol assistance, a type of scientific advice specific for designated orphan medicines, fee waivers or reductions for the regulatory procedures or a 10 year market exclusivity once the medicine is on the market. Therefore, it is important for companies to know the process for designation.
In this Webinar the process for obtaining designation is explained in detail. There are many factors to consider when preparing an application, and taking those into account will ensure a smooth process. The main factors to consider are:
- Definition of a medical condition
- Dealing with subsets
- How to show significant benefit
Areas Covered in the Session:
- Paediatric Legislation
- The PDCO
- The COMP
- PIP's
- Timing of the PIP
- Incentives
- Application procedure
- Results of the legislation
Who Will Benefit:
- Regulatory Affairs personnel
- Senior management
- Project Managers
- Medical writers
- CRAs and CRCs
- QA / Compliance personnel
- Investigators
- Clinical Research Scientists
- QA / QC Auditors and Staff
- Consultants
He is currently Director Regulatory Affairs at CATS Consultants. Until March 2004 he has been Head of the Oncology Group within Global Regulatory Affairs at Bayer AG, Wuppertal, Germany, and Bayer Corporation, West Haven, CT, USA. Between 2001 and 2003 he was Director of Regulatory Affairs at Micromet AG, a biotech company in Munich, Germany. Prior to joining Micromet he has worked during four years as a Project Manager for Oncology Projects at the European Medicines Agency in London, United Kingdom
He joined the European Medicines Agency from Novartis AG, Basel, Switzerland, where he was Regulatory Affairs Project Manager in the Oncology group in 1996 and 1997. Before 1996 he was Head of Drug Regulatory Affairs for six years at Ciba-Geigy in the Netherlands, and has worked as Manager Regulatory Affairs at Glaxo, also in the Netherlands.