Q & A
1- Don't children's medicines already exist?
Yes, however, many essential medicines do not exist in child-size dosage forms or there may not be enough information about the medicines' efficacy and safety. Currently, many medicines for priority diseases are not developed for children; and if they are they are not reaching the children who need them most. For children with chronic conditions, such as HIV/AIDS, where several medicines must be taken daily, the fixed-dose combination approach - several medicines in one pill - is best. However, the few existing paediatric fixed dose combinations developed for children are generally three times more expensive than the adult dosage form.
2- Why is it important?
Over 9 million children under five years old die each year, more than half of these deaths are due to illnesses and diseases which could be treated with safe essential paediatric medicines manufactured to suit the age, physical condition and body weight of the child taking them.
Children's bodies differ from adults and metabolize medicines differently. Therefore they need different dosage forms. Similar differences exist between children of different ages, weights, and physical conditions. However, many medicines for priority diseases have only been developed for and studied in adults, leading to a lack of suitable treatment for children.
There is little knowledge about the effects certain medicines can have on children. Apart from dose, children's medicines need to be in a format that is palatable to children. Small children have trouble swallowing big tablets whereas flexible oral dosage forms, such as dispersible tablets are much easier.
3- Why are children's medicines only being addressed now?
2007 marked the 30th anniversary of the Model List of Essential Medicines. Over time, children's medicines have been included in this list but there has not been a comprehensive review of what children's medicines are available, what medicines are needed, and what needs to be done to address the gaps.
Reducing child mortality and treating children affected by major diseases are global priorities expressed in the Millennium Development Goals four and six. In May 2007, the World Health Assembly passed resolution WHA60.20 "Better Medicines for Children" setting goals and calling for action by Member States, WHO and its partners to address the global need for safe, effective and accessible children's medicines.
To ensure greater access to children's medicines, there needs to be work done across sectors. While many stakeholders are taking individual steps towards ensuring improved access -- regulatory and legislative bodies are introducing changes, governments are providing more funds towards these efforts -- there is an opportunity for global collaboration to really make a difference.
4- What are the priority medicine areas?
The priority areas are HIV/AIDS, malaria, pneumonia, tuberculosis and diarrhoea, which are together responsible for over 50% of under-five deaths. This initiative will aim to ensure that appropriate medicines and dosage forms are available and accessible to treat these diseases.
5- What are WHO's specific targets?
WHO, together with partners, has identified research, development, regulatory, legislative, and supply gaps that need to be addressed to ensure that children receive the right medicine in the right dose at the right time. Throughout the five year campaign WHO, together with partners, will release new efforts to meet specific targets, raise awareness, and galvanize stakeholder action.
Within the next five years and with the help of all stakeholders, the campaign aims to tackle the priority research gaps, where medicines do not exist or safety and efficacy are not known, for medicines for second-line treatment of TB (for TB resistant to existing medicines); medicines for TB/HIV co-infection; and medicines for neglected diseases - schistosomiasis, filariasis, and soil transmitted worms.
The campaign aims to further tackle development gaps where medicines, research or knowledge exist but medicines require development or adaptation, for four quality assured Fixed Dose Combinations (FDC) for malaria; four FDCs for HIV/AIDS; three FDCs for tuberculosis; and antibiotics for neonatal infections.
Regarding the access gaps, where medicines exist but are not reaching those who need them, the campaign plans to address problems related to pain medication, oral rehydration salts with zinc to treat diarrhoeal diseases, child specific antibiotics for pneumonia, and asthma medication.
6- How will WHO achieve these goals?
WHO will galvanize action from stakeholders (legislation/regulation/policy from governments, research from academia and private sector, production from industry, funding from donors); provide industry with key information on public health needs; advise countries on issues of quality, safety and efficacy, and supply management, and provide the healthcare community with information on dosage and treatment guidelines.
Specific examples of action include: working with partners for additional and independent studies on the development of paediatric medicines and their efficacy and safety; providing better information on child specific medicines to prescribers, pharmacists and health workers; exploring ways of fast-tracking the regulation of quality paediatric formulations; establishing systems for ensuring that health facilities have adequate stocks of essential medicines and clinical consumables for children.
Where possible, clinical trials in children should be encouraged. In the absence of clinical trials, WHO and the committed stakeholders will identify safety and efficacy guidelines for which essential medicines may be used in paediatric dosage forms. In addition, WHO will assist in monitoring the use of unlicensed, off-label and unsafe medicines for children.
7- What progress has been made since the launch of 'make medicines child size'?
The 'make medicines child size' campaign has been endorsed by industry through the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA); civil society through Save the Children and Medicines Sans Frontières, Caritas Internationalis and others; as well as professional associations, the National Institutes of Health, EMEA and UNICEF.
The IFPMA has launched a special paediatric task force building industry’s pediatric medicine and vaccine development and access activities, and working to address specific concerns highlighted by WHO and other organizations. These include identifying appropriate dosage forms and strengths of medicines for children (starting with HIV/AIDS, tuberculosis, malaria and chronic diseases), encouraging R&D of appropriate medicines for diseases that specifically affect children, and ensuring high quality and ethical standards in clinical trials involving children, including informed consent.
The first Model List of essential Medicines for children developed in 2007 by a WHO Subcommittee on Selection and Use of Essential Medicines was revised in 2008 to include missing essential medicines for children using evidence-based clinical guidelines.
The International Conference of Drug Regulatory Authorities, held a two day pre-conference in September 2008 to discuss the regulation of medicines for children, including clinical trials, streamlining registration procedures and guidance for prescribing medicines for children.
The Expert Committee on Pharmaceutical Specifications is drafting a guidance document on the development of paediatric medicines that will be a resource for industry.