1. 'Hot topics' ESSENTIAL MEDICINES FOR CHILDREN Suzanne Hill September 2006

2. What is the problem? n Children are therapeutic orphans ä Lack of appropriate clinical trials ä Lack of licensed medicines ä Lack of formulations ä Lack of information n Initiatives so far concentrated on: ä Developed country regulatory structures (eg FDA, EMEA) ä Developed country drug information (eg BNF- C) ä HIV (eg new public private partnership)

3. For example… n Creation of a Paediatric Committee at EMEA n Requirements and Rewards/Incentives for medicinal products still under patent, orphan drugs and for off-patent medicinal products Paediatric Investigation Plans (PIP) n Other measures

4. Patent-protected products n Obligation to submit results of studies conducted according to agreed Paediatric Investigation Plan (PIP) at time of marketing authorisation, or variation (exclude generics) If not: Invalid application for MA $: 6-month extension of the patent protection (= Supplementary Protection Certificate) n For orphan drugs, + 2 yrs market exclusivity

5. Obvious gaps n Essential medicines list – paediatrics n Medicines for other diseases n Formulations eg fixed dose combinations for malaria, TB n Prescribing information n Market incentives for appropriate drug development n International regulatory standards, including quality n International safety monitoring and post-marketing surveillance

6. Work so far… HIV Professor Tony Nunn, Liverpool

7. Current work with WHO n Paediatric HIV dosing information ä From - mg/kg and mg/m2 ä To - agreed weight bands with mg/m2 assigned to weight bands ä Excel spreadsheets to examine closeness of fit to desired dose limitation of –weight banding and formulation –solids preferred to liquids; half tablet? –avoiding asymmetric 2 x daily dosing ä Present as ‘simple tables’

9. Simple table abacavir

10. What has been done so far…essential medicines n Review of current EML identifying paediatric gaps ä Preliminary applications for isoniazid 50mg, pyrazinamide 150mg, phenobarbitone injection, update on caffeine n Post marketing surveillance proposal n Preliminary survey of countries about paediatric medicines, prices, guidelines, information

11. What has been done so far….UNICEF n Global commitments include the Millennium Development Goals, 'A World Fit For Children', Unite for Children n Surveys in countries about children's access to medicines, both as part of the MICS and annual UNICEF country report n Dialogue with pharmaceutical industry around products required for Child Survival (HIV-AIDS, Malaria, ARI, diarrhoea, multimicronutrients)

12. Plan n Work on a global project to make paediatric medicines a priority: ä Add missing essential formulations to the Model list in 2007; advise on doses ä Update treatment guidelines ä Develop paediatric prescribing information – a formulary ä Develop effective methods for provision of information at the point of care ä Collaborate with regulatory authorities to encourage appropriate drug development and approval processes in all regulatory authorities ä Advocate for the development of paediatric medicines by the industry ä Develop quality standards for paediatric medicines ä Develop a system for enhancing safety monitoring of medicines in children ä Provide guidance on procurement and supply of paediatric medicines