Two events in London this week and last testified to a more propitious climate for the development of medicines for children in the UK and Europe.
One was the first meeting of the European Medicines Agency’s (EMEA) new Paediatric Committee (PDCO) since the EU’s paediatric regulation came into force in January. The other was the opening of the UK’s first unit devoted solely to paediatric clinical research at St Mary’s Hospital in London.
The PDCO’s main responsibility will be delivering opinions on the development of medicines for use in children. It will assess the content of paediatric investigation plans (PIPs) and adopt opinions on them, including applications for waivers and deferrals. It will also, on request, evaluate data generated in accordance with agreed PIPs and adopt opinions on the quality, safety or efficacy of any medicine for use in the paediatric population.
Among the committee’s other tasks will be providing advice on any question relating to paediatric medicines and establishing and regularly updating an inventory of paediatric medicinal product needs.
Topics covered at the PDCO’s inaugural meeting on July 4-5 included the scientific and procedural aspects of assessing PIPs; the EMEA’s implementation strategy for an EU-wide paediatric research network; a recommendation to the European Commission on selecting a symbol for medicines with a paediatric indication; criteria for a survey of existing drug use in the EU’s paediatric population; and funding provided by the EU’s Seventh Framework Programme for studies of off-patent medicines.
Six-months' extra protection
The regulation on medicinal products for paediatric use offers a six-month extension to a product’s supplementary protection certificate in return for meeting the requirements of an agreed PIP. It also establishes a new type of marketing approval, the Paediatric Use Marketing Authorisation (PUMA), which allows 10 years of data protection for innovative uses of off-patent products. Supporting measures include plans to create as an EU-wide network of paediatric investigators and trial centres.
When it is fully resourced, the PDCO will include five members of the EMEA’s Committee for Medicinal Products for Human Use (CHMP), appointed by the CHMP itself; one member from each of the EU member states, other than those already represented through the CHMP appointees; three members representing associations of healthcare professionals; and three representing patient associations.
So far, under the acting chairmanship of Daniel Brasseur, former chair of the Paediatric Working Party, all but those members representing healthcare professional and patient associations have been selected. These latter, who will be appointed by the Commission after consultation with the European Parliament, are the subject of an ongoing public call for expressions of interest. The deadline for applications is August 31, 2001. A permanent chair for the PDCO will be elected at the committee’s meeting in September.
Paediatric Research Unit
The newly opened Paediatric Research Unit at St Mary’s Hospital in London is based next to the hospital’s paediatric wards in Paddington and will be run by researchers from St Mary’s and its university partner, Imperial College London. The £650,000 unit includes two outpatient consulting rooms and two ensuite inpatient rooms, as well as a waiting and play area, a lung function investigation room, a laboratory, a treatment room and offices for staff.
Among the areas slated for research at the unit are paediatric allergies, sleep disturbance, new treatments for acute or chronic chest disorders and neuromuscular diseases, infectious diseases and immunisation, and preventing complications of sickle cell disease. The first studies will look at allergy prevention in high-risk infants using prebiotics; a unique treatment for the hitherto incurable neuromuscular condition Duchenne dystrophy; use of a helium oxygen mixture in intensive care and to treat acute bronchiolitis; and the impact of various allergic problems on sleep and daytime behaviour.
Speaking at the opening, Professor John Warner, chair in paediatrics and head of the Department of Paediatrics at Imperial College, as well as consultant paediatrician at St Mary’s Hospital, said researchers should be designing therapies specifically for children, rather than scaling down treatments developed for adults.
“To create the best therapies for children we need to include them in our research,” he commented. “A lot of paediatricians’ work doesn’t have much of a scientific evidence base and we prescribe drugs by extrapolating from what we know about adult bodies. We have a desperate need to understand precisely how children's bodies work so that we can custom-design therapies for them and their problems.”