Essential medicines for children

Background Information: Child-specific medicines

A global priority

Reducing child mortality and treating children affected by major diseases are global priorities expressed in the Millennium Development Goals (Goals Four and Six).

A pre-condition to achieve these goals is increased production and availability of essential medicines for children. At present, many medicines for priority diseases are not developed for children; and when they are they are not reaching the children who need them most.

Children metabolize medicines differently from adults. They therefore need different dosage forms. Differences also exist between children of different ages, body weight and physical conditions. Child-specific medicines are those manufactured to suit the age, physical condition and body weight of the child taking them.

Apart from dosage, child specific medicines need to be in a format that is palatable to children. Small children have trouble swallowing big tablets but can tolerate oral solution or syrups. 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.

Research and development gaps

There is little knowledge about the effects certain medicines can have on children. This is partly due to the fact that fewer clinical trials are conducted in children than in adults.

The ethical approach to conducting clinical trials is to obtain signed informed consent from volunteers before they take part in a trial. Obtaining informed consent from a child - particularly in the younger age bracket - is clearly difficult. Insufficient clinical trials for paediatric medicines in turn lead to information gaps related to quality and safety. Those gaps deter pharmaceutical companies from researching and developing child-friendly medicines and generics companies from producing them at lower cost.

In addition, children are generally a silent sector of the population, relying on others to speak for them - they do not vote, they do not buy and generally do not have a public voice in society. Although they would benefit from more pharmaceutical research and development, they do not have the means to demand it.

Distribution problems

Child specific medicines already developed and available often do not reach the children who need them most. For instance, diarrhoea, which can easily be treated with oral rehydration salts plus zinc, still kills 1.9 million under-fives every year. Oral rehydration salts are easy to use, easy to manufacture and relatively inexpensive. Due to years of advocacy campaigns, they are widely available in developing countries. Zinc, on the other hand, is not easily found in areas with a high incidence of diarrhoeal disease. Oral rehydration solution with zinc is more effective than without it.

There may be numerous, poverty-related causes for the fact that children are still dying of diarrhoeal diseases, but one obvious reason is that the full remedy - rehydration salts plus zinc - is not made available to them.

The target diseases

WHO estimates that of all the child killers, five conditions in particular demand immediate action:
Pneumonia and other acute lower respiratory infections

An estimated 20% of all deaths in children under-five are due to acute lower respiratory infections, representing the single most important cause of infant mortality worldwide. Pneumonia alone causes approximately 2 million deaths every year which could be prevented with proper access to child-specific medicines.

HIV/AIDS

Although contributing to only about 3% of all annual deaths in children under-five, paediatric HIV is a growing public health challenge. Every day, an estimated 1150 children become infected.

Malaria

An estimated 1 million children die every year due to malaria infection and 40% of the world's children live in malaria-endemic countries. In Africa, a child dies of malaria every 30 seconds. Although malaria is a priority illness and has been the subject of numerous global conferences and calls to action, the issues of access to and development of child specific treatment need to be further addressed.

Diarrhoeal diseases

An estimated 1.9 million children under five die each year from diarrhoea and related complications. This amounts to 18% of all under-five deaths and means that more than 5000 children die every day as a result of diarrhoeal diseases, which can be treated easily and effectively.

Tuberculosis

About 1.1 million (12%) of the 8.8 million new tuberculosis cases in 2005 occurred in children under 14 years of age.

Lymphatic filariasis and schistosomiasis

Although not major killers of children, the neglected tropical diseases filariasis and shistiosomiasis are also prioritized by WHO because of gaps in either the development or accessibility of medicines.

WHO estimates that 330 million children under 15 years of age currently require chemotherapy to prevent Lymphatic Filariasis in endemic areas (Asia, Pacific, Africa and South America). Another 125 million children in the same age category require preventive treatment for schistosomiasis. The necessary medicines exist in paediatric form but are hardly available or accessible to the targeted populations.

WHO action

Recognizing the lack of child specific medicines, the Member States of WHO passed a resolution on "Better Medicines for Children" during the 2007 World Health Assembly, the Organization's annual meeting.

The resolution mandates WHO to explore ways to promote more research and development into paediatric medicines and to improve knowledge on the quality, effectiveness and safety of these medicines.

To address this, WHO created an Expert Sub-Committee to develop an essential medicines list for children. The list, finalized and published in December 2007, consists of 206 medicines, including anti-AIDS treatment, vaccines, anaesthetics, hormones, vitamins and minerals.

The list should serve as a reference for countries to develop national lists according to their specific public health priorities, and is the beginning of a longer process to ensure that child specific medicines are developed and delivered to the intended patient group.

More needs to be done to identify research gaps in children's medicines and problems related to supply and distribution. WHO is beginning to address these issues with UN partners, in particular UNICEF.

The initial objective of WHO's work in this area is to tackle HIV/AIDS, malaria, tuberculosis, pneumonia and diarrhoeal diseases, which account for over 50% of under-five mortality.

More specifically, WHO will work towards the following objectives:

• Increase additional and independent studies on the development of paediatric medicines and their efficacy and safety;

• Provide better information on child specific medicines to prescribers, pharmacists and health workers; • Explore ways of fast-tracking the regulation of quality paediatric formulations;

• Establish systems for ensuring that health facilities have adequate stocks of essential medicines and clinical consumables for children;

• In the absence of clinical trials, identify safety and efficacy indications for which essential medicines may be used in paediatric formulations;

• Improve infrastructure and equipment to store liquid paediatric formulations which are less stable than solid dosages;

• Monitor the use of unlicensed, off-label and unsafe medicines for children.

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