Dr Peter Folb, University of Cape Town
Dr Piero Olliaro, UNDP/World Bank/WHO
Special Programme for Research and Training
in Tropical Diseases, Geneva

In an ideal world, all countries would have abundant medicines which are safe, effective and of good quality and regulated by a competent national drug regulatory authority respected for its expertise, professionalism, efficiency, independence, and integrity. Policy would be made confidently on the basis of scientific criteria by the national drug regulatory authority, as would the decisions taken by any government-based authority responsible for considering and negotiating the costs of medicines.

Cost controls would be firm but at the same time respectful of the rights, including intellectual property rights, of the pharmaceutical industry. Of the total national expenditure on health - which itself would be competently controlled - a fixed 6-10% would be spent on pharmaceuticals. The health budget would be a little greater than 5% of the gross national product. The development of local industry would be encouraged and appropriately supported, and the national drug regulatory authority would actively encourage and stimulate local enterprise in the production of essential drugs with emphasis on appropriate standards.

There would be a strong focus on the manufacture and rational use of generic medicines, particularly those that are included in the national or WHO Model List of Essential Drugs. There would also be uninterrupted supply and access to essential medicines, including vaccines and birth control measures. New drug development, where it is relevant to the health of the country, would be served by the conduct of clinical trials, approved in advance by the national drug regulatory authority as well as by the institutional review board at those sites where studies are conducted. The health professionals and the general public would be well informed about the medicines they take, and they would have access to independent and authoritative information should they require it. As a result, the public would have confidence in the medicines available to them and, by extension, would be supportive of the health system.

Such an apparently straightforward and reasonable set of circumstances would seem necessary. However, this situation scarcely exists anywhere and certainly not in the developing world. It may be time for an objective evaluation of the reality.

The current situation

World Bank health reports published in 1993 and 1994 covering sub-Saharan Africa showed that more than 85% of funding allocated to acquisition of pharmaceuticals in the region was lost through waste and inefficiency. The reports found that at every level - whether in procurement, distribution, rational and informed use, or patient compliance - any real value which is targetted to advantage the patients themselves is eroded before it reaches the consumer. Donors are working at cross-purposes: and in so doing undermining their own programmes as well as those of others; national policies fail to consider important principles necessary for the conduct of a sound national pharmaceuticals policy; and important partners - including the pharmaceutical industry - work in disharmony.

Following an analysis of the report, it was concluded that a sound pharmaceuticals policy is achievable only if:

• The issues are adequately understood and there is political will to address them.

• International organizations, The World Bank, UNICEF, WHO, and nongovernmental organizations accept a shared responsibility and recognise their considerable potential to contribute to sound drug policies in the countries that they support.

• The national drug regulatory authority is fully supported and is acknowledged as being crucial to the success of these policies.

The role of the national drug regulatory authority

A description has already been provided of the expected role of the national drug regulatory authority. Words such as sound, independent, competent, reasonable, professional and efficient reflect their required characteristics. A budget should be available of 1 US dollar per 1000 dollars of national turnover in pharmaceuticals. The activities of the authority should be independent of any cost control initiatives, but the national drug regulatory authority decisions and advice should inform the latter. All decisions and contributions to policy should be underpinned by good science and a thorough and first-hand appreciation of clinical medicine. The national drug regulatory authority needs to act independently of government and industry in its decision-taking to ensure that the drugs available and the way they are used are suitable for local conditions and are appropriate. It should be supported by adequate yet simple systems of postmarketing surveillance, adverse drug event monitoring, and independent drug information. It should be thoroughly underpinned by a sound and appropriate legal system while consistent with national and regional policy and legislation. All its actions should be directed to promoting public health, and should be readily and openly explicable as such.

The national drug regulatory authority's contribution to research

A research agenda should be developed taking into consideration the following issues:

• Definition of the role of a national drug regulatory authority in the developing world, and of the international drug regulatory environment, with special reference to new drug development, research, access, and affordability of essential medicines for important and common diseases.

• The special case of tropical disease policies in endemic areas of the world, with particular reference to research, development and deployment of drugs, the nature of policy implementation, impediments to progress, and the costs of therapy and prevention.

• Consolidation of the respective roles of the pharmaceutical industry, United Nations agencies, nongovernmental organizations, bilateral agreements, ministries of health, and the communities themselves in providing essential medicines for important and common diseases which affect the country or region. This includes research and development of novel medicines for diseases such as malaria, viral infections, and tuberculosis.

• A developing world perspective of scientific, ethical and intellectual property issues pertaining to the use of traditional medicines and herbal preparations.

• An examination of alternatives to conventional industry-based approaches to new drug development and marketing.

Promoting essential standards for new drug development

An important role exists for the national drug regulatory authority to support and promote standards for new drug development to combat diseases such as malaria and tuberculosis and to support the manufacture of essential generic medicines. The following points will need consideration.

• There can be no compromise on basic standards of quality, safety and efficacy. After all, unsafe and ineffective medicines are costly and dangerous. The same applies to poor quality medicines.

• Fast track approval of important new products should not compromise any of these standards. Marketing of needed medicines can further be expedited by processes of mutual recognition between regulatory agencies and acceptance of decisions taken by other trusted agencies.

To meet these expectations, an expanded role and vision for key national drug regulatory authorities is beginning to emerge. This is evidenced by the recent WHO initiative to develop rectal artesunate for malaria in children. The US Food and Drug Administration, the UK Medicines Control Agency and the Swiss Intercantonal Office for the Control of Medicines have each agreed to deliver accelerated marketing approval in the interests of public health - a perspective which goes well beyond national responsibilities.

The establishment of the International Conference on Harmonization (ICH) presents a challenge to international public health objectives. The declared structure and purpose of ICH - which is made up of representatives of drug regulatory authorities of the European Union, Japan and USA and the pharmaceutical industry - does not take particular account of the special needs of the developing world. Standards have been set through ICH guidelines which, although excellent and helpful in developing innovative new medicinal products, have been interpreted as rules. Beyond an observer status, WHO and countries not included in the ICH are effectively excluded. In a sense, ICH is counterproductive to approaches for development of critically required new drugs by groups such as WHO and the non-ICH countries.

It is a further challenge to this new public health perspective that national drug regulatory authorities must also foster the development of local industry in a manner that promotes public confidence, supports excellent essential standards and is free of special arrangements between government and industry.

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