The Essential Drugs Concept

SETTING THE SCENE:

THE ESSENTIAL DRUGS CONCEPT

SESSION NOTES

PURPOSE and content

A growing number of pharmaceutical products are available on the world market and there has been an increase, both in the consumption of drugs, and in expenditure on them. In spite of this, many people throughout the world cannot obtain the drugs they need. There are also many people who have access to drugs but who do not get the right drug, in the right dosage, when they need it. The essential drugs concept was developed in response to these problems and continues to be central to policies and strategies which aim to address them. The essential drugs concept is central to the development of a national drug policy.

This session provides an introduction to the essential drugs concept. A short overview of the context in which it was developed and adopted is given. This includes a brief description of the main elements of primary health care (PHC) and the role of essential drugs. A short account of the current world drug situation follows, which includes problems relating to access to essential drugs and to irrational use. Current trends and challenges are discussed including the growing role of the private sector; changes in disease patterns; and the roles and interests of different actors.

OBJECTIVES

Upon completion of the module participants will be able to:

Understand the essential drugs concept in an historical perspective and in relation to primary health care.
Recognise the main components of national drug policy.
Identify the major actors in the pharmaceutical context and discuss the roles they play.
Recognise some of the main trends and challenges in the world drug situation today.

PREPARATION

Read the Session Notes.

Setting the scene: the essential drugs concept Session Notes

A. ESSENTIAL DRUGS – AN HISTORICAL PERSPECTIVE

A1. Pharmaceuticals in history

Pharmaceuticals are relatively new. Evidence-based medicine is even newer. The history of medicine goes back thousands of years and varies from continent to continent. But people have always (as long as records go back) attempted to influence their health and avoid or cure illness through the use of traditional medicine and herbal medicines. Many people all over the world turn to alternative, complementary and traditional medicine to maintain health or combat illness. However this course concentrates, to a large extent, on the use of pharmaceutical products – industrially produced medicines.

Pharmaceuticals and pharmacotherapeutics are relatively new. If we go back just a hundred years we enter an age in which modern therapeutic medicine, as we know it, was in its infancy. When discussing drug use and drug policy today it is useful to have some idea of the historical development of the pharmaceutical industry and the place which pharmaceutical products have in health care. Current debates about drug policy and drug use are easiest to understand if we look at them in the context of:

Changing ideas about health and health care services and systems;
The development of the pharmaceutical industry from small beginnings at the start of the 20th century into one of the most powerful industrial sectors by its end.

In the West, during the 19th century, there was an increasing concern with hygiene and a growing understanding of public health principles. However, the understanding of pharmacotherapy was very limited and there were few effective or safe medicines. Many common therapeutic interventions were hazardous and most were without any scientific basis.

A2. The development of modern pharmacotherapy

Before World War II there were relatively few effective medicines. Pharmaceuticals as we know them now hardly existed.

Smallpox vaccination was one of few effective measures available before the turn of the century. Around that time aspirin became available and was mass-produced. The pharmaceutical industry has its origins in pharmacy, chemistry and microbiology (see Chetley (1990) AHealthy Business. Zed Books, London).

Historically:

Pharmacy started with herbal medicines and patent remedies.

Chemical innovation during the 19th century (centred around the dye industry in Germany) led to the extraction of alkaloids and synthesis of organic compounds.

Increased understanding of germs and bacteria led to the development of compounds, which could kill bacteria and cure disease. (Salvarsan, an arsenic compound, was synthesised in 1910 and used to treat syphilis). Penicillin was identified in 1928 but not really recognised until 1939 when it ushered in the era of antibiotics.

After World War II developments came rapidly.

Early landmarks in drug development

1941 - Penicillin

1943 - Chloroquine

1944 - Streptomycin

1948 - Tetracycline and chloramphenicol

1954 - Sulphonylureas (diabetes)

1955 - First oral contraceptive trials

A3. Problems in providing access

The new breakthrough drugs were only available to a privileged minority. In spite of the rapid growth of the industry and the rapid proliferation of brand name drugs most people lacked the means to buy them. This led to the development of a market characterised by the following factors:

Differences between rich and poor countries;
Differences between urban and rural populations;
Proliferation of brand name products;
Lack of information/evidence about therapeutic value.

A4. Miracles turn into disasters

In addition to these elements there was almost no regulation of drugs, little understanding of how to measure their efficacy, and scant appreciation of potential risks. High expectations coupled with an optimistic view of possible benefits and ignorance about side effects created a dangerous situation.

Lack of regulation and adequate safety measures resulted in a series of disasters, including:

1930sSulphanilamide scandal resulted in the establishment of US Food and Drug Administration (FDA). In 1937 at least 73 people died as a result of taking a sulphanilamide elixir containing ethylene glycol. The resulting public outcry resulted in the establishment of the US Food and Drug Administration and to requirements for drug safety testing. (Only in 1962 were regulations tightened to require proof of efficacy)

1960sThalidomide (8,000 children in 46 countries seriously handicapped)

1950 - 1970sDiethylstilbestrol (DES) tragedy

1970sClioquinol caused widespread neurological disease in Japan.

A5. Major challenges became evident

This situation brought out a number of distinct challenges to be met. The first was to make useful drugs widely available. Secondly to make sure that drugs were properly used in such a way that their benefits can be maximised and risks minimised. Thirdly to harness drug development to serve the needs of public health and the people who need them most.

These challenges have stayed with us and are at the core of the drug policy debates occurring during the last three decades.

“Thirty years ago modern health technology had just awakened and was full of promise. Since then its expansion has surpassed all dreams, only to become a nightmare. For it has become over sophisticated and over costly. It is dictating our health policies unwisely; and what is useful is being applied to too few.”

Halfden Mahler, then WHO Director-General addressing the World Health Assembly in 1978.

Frustration about proliferation of pharmaceutical products and inequitable access led to the development of the Essential Drugs Policy.

A6. Essential drugs

Essential drugs are those that satisfy the health care needs of the majority of the population; they should therefore be available at all times in adequate amounts and in the appropriate dosage forms (WHO).

The essential drugs concept was not any one person’s discovery but was developed out of a number of experiences in countries seeking to meet the challenges described above.

1940s

Norway defined a list of priority drugs which should be made available to everyone by state health services.

1960s and early 70s

Sri Lanka, Cuba, and Costa Rica experimented with lists of essential drugs and bulk procurement.

1974

The UN adopted a resolution and programme of action of a New International Economic Order which introduced a new concept of, and commitment to development.

The International Labour Organization (ILO) adopted the idea of defining basic needs. This concept provided a focus for countries trying to ensure equitable access to essential goods and services.

1975

WHO defined ‘essential drugs’ – this was a response to the ILO challenge to multilateral organisations and countries to prioritise basic needs.

1976

First Model List of Essential Drugs produced and promoted by WHO.

1978

Declaration of Alma Ata – calling for focus on Primary Health Care. This was a logical progression. The focus on basic needs drew on the experience of countries such as China that had tried to promote access to health care through community involvement and barefoot doctors.

WHO called for “a virtual revolution to bring about changes in the distribution of power, in the pattern of political decision-making, in the attitude and commitment of health professionals and administrators in ministries of health and universities and in people's awareness of what they are entitled to”.

Primary health care was defined as including (at least):

education about prevailing health problems and how to control and prevent them;
promotion of food supply and proper nutrition;
adequate supply of safe water and basic sanitation;
maternal and child health care (including family planning);
immunisation against major infectious diseases;
prevention and control of locally endemic diseases;
appropriate treatment of common diseases and injuries;
provision of essential drugs.

1981

WHO Action Programme on Essential Drugs is established. This action is a result of the recognition within WHO that the essential drugs concept could revolutionise access and use of drugs. There was also the understanding at WHO that it was an idea that would need support and promotion at the international level and at the national level to assist countries in implementing it.

1982

Bangladesh adopted a national drug policy. One of the poorest countries in the world embarked on a bold experiment to tackle the double problems of irrational use and lack of access to essential drugs.

1985

Rational Use of Drugs - WHO Conference of Experts in Nairobi. At this meeting the need for both prescribers and consumers to know about and use drugs appropriately was the central theme - an important shift given that, until Nairobi, attention had gone mainly to strengthening selection, supply and distribution systems.

Gaining ground

Since the beginning of the 1980s the essential drugs concept has become one of the cornerstones of international and national health policy - influencing decision-making in not only developing but also industrialised countries. The selection and rational use of drugs is accepted as a key principle of health service quality and management in both the public and private sectors. WHO has vigorously promoted the essential drugs concept and the rational use of drugs - at first through the Action Programme on Essential Drugs which became a powerful advocate for the new policies. National Drug Policies were promoted by WHO and others as a guide to action and a key framework within which to coordinate the various policy components needed to guarantee access to and rational use of drugs. The next section of this module deals in more detail with National Drug Policies and looks at some country examples.

Figure 1. Access to essential drugs

B. NATIONAL DRUG POLICY

Some of the early attempts to implement the essential drugs concept focused on supply and technical aspects such as procurement, storage and distribution. At the same time, there was a growing awareness of the problems of rational use. These involved their complexity, the need for an integrated approach and for a comprehensive national drug policy as part of health policy. A drug policy is needed to:

determine standards and values which will guide actions in the drug sector;
define global objectives which should be met;
identify which strategies will be pursued to meet these objectives.

B1. Goals of a national drug policy

The general goals of a national drug policy are to ensure:

Access:equitable availability and affordability of essential drugs
Quality: the quality, safety and efficacy of all medicines
Rationalthe promotion of therapeutically sound and cost-effective use of drugs use by health professionals and consumers.

The more specific goals and objectives of a national policy will depend upon the country situation, the national health policy, and political priorities set by the government. In addition to health-related goals there may be others, such as economic goals. For example, an additional objective may be to increase national pharmaceutical production capacity. The policy should be concerned with efficiency, equity and sustainability.

Objectives of the South African National Drug Policy

Health objectives

availability of essential drugs

ensure the safety, efficacy, and quality of drugs

ensure good dispensing and prescribing

promote rational use through the provision of necessary training, education and information

promote the concept of individual responsibility for health, preventive care and informed
decision-making

Economic objectives

lower cost of drugs in private and public sectors

promote the cost-effective and rational use of drugs

establish a partnership between government bodies and private providers

optimise the use of scarce resources

Development objectives

improve knowledge, efficiency and management skills

reorient medical, pharmacy and paramedical education

support the development of local industry and local production of essential drugs

promote the acquisition, documentation and sharing of knowledge and experience.

Source: National drug policy for South Africa, Department of Health, 1996

B2. Components of national drug policy

Selection of essential drugs

Drug selection, preferably linked to national clinical guidelines, is a crucial step in ensuring access to essential drugs and in promoting rational drug use, because no public

sector or health insurance system can afford to supply or reimburse all drugs that are

available on the market. Key policy issues are:

the adoption of the essential drugs concept to identify priorities for government involvement in the pharmaceutical sector, and especially for drug supply in the public sector and for reimbursement schemes;
procedures to define and update the national list(s) of essential drugs;
selection mechanisms for traditional and herbal medicines.

Affordability

Affordable prices are an important prerequisite for ensuring access to essential drugs in the public and private sectors. Key policy issues are:

government commitment to ensuring access through increased affordability;
for all drugs: reduction of drug taxes, tariffs and distribution margins; pricing policy;
for multi-source products: promotion of competition through generic policies, generic substitution and good procurement practices;
for single-source products: price negotiations, competition through price information and therapeutic substitution, and Trade-related aspects of intellectual property rights (TRIPS) compliant measures such as compulsory licensing, “early workings” of patented drugs for generic manufacturers and parallel imports.

Drug financing

Drug financing is another essential component of policies to improve access to essential drugs. Key policy issues are:

commitment to measures to improve efficiency and reduce waste;
increased government funding for priority diseases, and the poor and disadvantaged;
promotion of drug reimbursement as part of public and private health insurance schemes;
use and scope of user charges as a (temporary) drug financing option;
use of and limits of development loans for drug financing;
guidelines for drug donations.

Supply systems

The fourth essential component of strategies to increase access to essential drugs is a reliable supply system. Key policy issues are:

public-private mix in drug supply and distribution systems;
commitment to good pharmaceutical procurement practices in the public sector;
publication of price information on raw materials and finished products;
drug supply systems in acute emergencies;
inventory control, prevention of theft and waste;
disposal of unwanted or expired drugs.

Regulation and quality assurance

The drug regulatory authority is the agency that develops and implements most of the legislation and regulations on pharmaceuticals, to ensure the quality, safety and efficacy of drugs, and the accuracy of product information. Key policy issues are:

government commitment to drug regulation, including the need to ensure a sound legal basis and adequate human and financial resources;
independence and transparency of the drug regulatory agency; relations between the drug regulatory agency and the ministry of health (MoH);
stepwise approach to drug evaluation and registration; definition of current and medium-term registration procedures;
commitment to good manufacturing practices (GMP), inspection and law enforcement;
access to drug control facilities;
commitment to regulation of drug promotion;
regulation of traditional and herbal medicines;
need and potential for systems of adverse drug reaction monitoring;
international exchange of information.

Rational use

The rational use of drugs means that patients receive medicines appropriate for their clinical needs, in doses that meet their individual requirements, for an adequate period of time, and at the lowest cost to them and their community. Irrational drug use by prescribers and consumers is a very complex problem, which calls for the implementation of many different interventions at the same time. Efforts to promote rational drug use should also cover the use of traditional and herbal medicines. Key policy issues are:

development of evidence-based clinical guidelines, as the basis for training, prescribing, drug utilisation review, drug supply and drug reimbursement;
establishment and support of drugs and therapeutics committees;
promotion of the concepts of essential drugs, rational drug use and generic prescribing in basic and in-service training of health professionals;
the need and potential for training informal drug sellers;
continuing education of health care providers and independent, unbiased drug information;
consumer education, and ways to deliver it;
financial incentives to promote rational drug use;
regulatory and managerial strategies to promote rational drug use.

Research