Dank an Frau Tschabitscher für die Präsentation und Frau Budin für die Grafiken

Transcription

1 Dank an Frau Tschabitscher für die Präsentation und Frau Budin für die Grafiken

2 Paediatric Regulation First experiences of a national competent authority Thanks to Mrs Tschabitscher for the slides and Mrs Budin for the figures

3 Off-label use & ADR 10,0 zugelassene AM nicht zugelassene 6,0 % ADR 5,0 3,9 3,4 1,4 0,0 Stationärer Bereich Ambulanter Bereich Turner, Acta Paediatr 1999 Horen, Br J Clin Pharm

4 Objectives of the Paediatric Regulation Improve health of children by increasing - high quality, ethical research into medicines for children - availability of authorised medicines for children - information on medicines Without - unnecessary studies in children - delaying authorisation for adults 4

5 PDCO Composition: Chair: Daniel Brasseur (BE) Vice-chair: Gerard Pons (FR) 5 Members + alternates of CHMP 24 Members + alternates appointed by MS 3 Members + alternates representing health professionals 3 Members + alternates representing patient associations EMEA Paediatric Team: Head: Agnes Saint Raymond 10 Paediatric Coordinators 5

6 PDCO To evaluate whether proposed plan - ensures generation of relevant data - significant therapeutic benefit - fulfil a therapeutic need of the paediatric population NOT to assess results of already performed studies will be done at time of MAA 6

7 Paediatric Investigation Plan (PIP) Basis for development and authorisation of a medicinal product for all paediatric population subsets Includes details of the timing and the measures proposed to demonstrate: - Quality -Safety - Efficacy To be agreed upon and/or amended by the PDCO Is binding on company and NCA 7

8 First experiences Overview of PIP / w aiver applications number (Aug-Dec) 2008 (Jan-Apr) Total number of PIP/w aiver Article 7 Article 8 Article 30 8

9 First experiences Total num ber of validated PIP/w aiver applications August 2007-April 2008 EU - Austria (Rapporteur / Peer Review) Anzahl Gesamt EU Peer 9 Rapp 8 AT 9

10 First experiences Areas covered by PIPs/w aiver applications Juli 2007-April 2008 Infectious desease 9% Neurology 8% Cardiovascular diseases 11% Oncology 14% Endocrinology gynaecology fertility metabolism 20% Pneumology- Gastroenterology Allergology 6% Hepatology 5% Uro-Nephrologie 4% Diagnostics 2% Anaesthesiology 1% Ophthalmology 1% Haematology Haemostaseology 4% Dermatology 1% Nutrition 1% Pain 1% Vaccines 6% Immunology Rheumatology Transplantation 6% Psychiatry 4% 10

11 Other activities Regulation includes a number of additional measures to: - Define paediatric needs (survey) - Inventory of use - Increase transparency (e.g. EudraCT) - Increase availability of paediatric information (submission of studies, EudraCT) - High quality paediatric research (7th Research Framework Programme, EC) - Avoid unnecessary studies (EudraCT, Network) 11

12 Other activities Free scientific advice for paediatric studies Comments on GL / AT topic leader - Haemophilia GLs (supported by DE) - GL on Antiarrhythmics - GL on Teratment of Pulmonary Hypertension (sup. PL+DE) - GL on Alcohol Dependence Comments on GL / AT supports - Guidance fixed combination in the treatment of Hypertension (topic leader: NO) 12

13 Inventory of use (Art. 42) Pädiatrieprojekt AGES PharmMed: survey on all existing uses of medicinal products in the paediatric population of Austria (current status of authorised, off-label and unlicensed use) 13

14 Objectives Investigation and evaluation of the use: 1. Out of hospital: a) Only available on prescription and reimbursed MPs b) Selected OTCs Data sources: a) Austrian Social Security Institutions: HVB, ASVG 2 Kassen, BVA, SVA Gewerbliche Wirtschaft, SVA Bauern, VA Eisenbahn und Bergbau b) IMS-Health 2. In hospital: Data from selected Hospital pharmacies (AKH Wien, AKH Linz, BH SR Linz, Universitätsklinik Innsbruck, Klinikum KS Wels, LKH Feldkirch, LKH Oberwart) 3. NCA database & Industry Currently 72% (of ) MP assessed 41% have a paediatric indication 14

15 Thanks to Mrs Tschabitscher for the slides and Mrs Budin for the figures 15

16 Backup slides 16

17 Other activities European Paediatric Network: -Link together existing networks, investigators and centres with paediatric expertise - Build up competences at an European level - Avoid duplication of studies - Implementation strategy objectives, organisation and structure, action programme, priorities, resource allocation, and monitoring of the outcomes and performance of the network - Establish links to Ethic Committees, the PDCO, industry and other stakeholders - AT: ÖGKJ submitted a project-manual to BMGFJ 17

18 Paediatric Symbol Art. 32: all MPs with granted paediatric indication shall get a symbol on the label; PDCO shall recommend symbol EC should have made symbol public by 26 January 2008 BUT: PDCO decided against a symbol (vote: 4+/18-), because identified risks would outweigh the benefits EC : Publication of this announcement serves to inform stakeholders that on the basis of the recommendation, the European Commission is at present not in a position to select a symbol and the provisions of Article 32 of the Paediatric Regulation cannot therefore be implemented. 18