Therapeutic function – the conventional use of medicines to prevent, treat and cure disease

 Placebo function – to show concern for, and to satisfy the patient

 Coping function – to relieve feelings of failure, stress, grief, sadness, loneliness

 Self-regulatory function – to exercise control over disorder or life

 Social control function – to manage behaviour of demanding or disruptive patients, hyperactive children

 Recreational function – to relax, enjoy the company of others, experience pleasurable feelings

 Religious function – to seek religious meaning or experience

 Cosmetic function – to beautify skin, hair and body

 Appetitive function – to allay hunger or control the desire for food

 Instrumental function – to improve academic, athletic or work performance

 Sexual function – to increase sexual ability

 Fertility function – to control fertility

 Research function – to gain knowledge and understanding of human behaviour

 Diagnostic function – to help make a diagnosis

 Status-conferring function – to gain social status, prestige, income.

Defining rational use

The rational use of medicines has been defined as the safe, effective, appropriate and economic use of medicines. This definition seems to be clear and straightforward, but the definition of safe, effective and the other components needs consideration. Safety relates to aspects like relative and absolute safety. All medicines have side-effects, some less and some more, such that they may be viewed as more or less safe. Safety has to be assessed from many different angles, e.g. the severity of the disease, the available treatment options including medicines and other options, long or short-term treatment, whether the medicine is to cure or control symptoms, and over-dosage risks.

Effectiveness relates to the question of how well the medicine works in daily practice when used by unselected populations and patients having co-morbidities and other medications. Efficacy relates to the maximum effect of the medicine in a particular disease, particularly when it is optimally used in selected patients with as few confounding factors as possible, such as co-morbidities and other medicines used simultaneously.

Appropriateness refers to how a medicine is being prescribed and used in and by patients, including aspects such as appropriate indication, with no contraindications, appropriate dosage and administration. Duration of treatment should be optimal and the medicine should be correctly dispensed with appropriate and sufficient information and counselling (see Ch. 25). To achieve the intended effects, the medicine also needs to be correctly used by the patient.

The economic aspect refers to a cost-effectiveness approach which needs to be applied, where all factors are assessed (see Ch. 22). A somewhat more expensive medicine may be preferable to a less expensive medicine, for example, because it has better treatment outcomes or fewer side-effects. Additionally, hidden costs, such as a need for more extensive laboratory tests, may increase the total cost of a particular treatment.

National medicines policy (NMP)

The rational use of medicines requires that appropriate and effective structures and processes are in place. The starting point and frame of reference is the NMP, which, particularly in developing countries, defines the policies and key issues that need consideration and the policies relating to medicines and their usage. More developed countries often already have many key issues and policies regarding medicines and their rational use already in place and therefore, the NMP receives little attention. However, the global pressure on healthcare funding, especially the increasing medicines budget, has created the momentum to look more closely at medicine policies in industrialized countries too. When trying to understand the general principles that can be applied to all countries it is helpful to use the guidelines that have been proposed for developing countries, and from there, to understand how the system works and what might be the strong and weak points in each particular country.

The NMP can be seen as a guide for action, including the goals and priorities set by the government, and their main strategies and approaches. It also serves as a framework in the coordination of different activities. Depending on cultural, historical and socioeconomic factors there are differences in objectives, strategies and approaches between countries, but some common components can be identified. The goals for an NMP can be divided into:

Ensuring the safety of medicines

Why is it important to regulate and control the medicine sector with special laws and regulations? The medicine sector is of concern to the whole population. Most citizens will use medicines and related services on a regular basis and therefore the functioning of the sector is of common interest. There are many parties involved – patients, healthcare providers, manufacturers and sales people – requiring detailed rules for interaction and functioning. The consequences from the lack of medicines or their misuse might be serious.

Legislation and regulation include different health-related laws, pharmacy law, trademark and patent laws, criminal law, international treaties (e.g. on narcotic and psychotropic drugs) and governmental decrees. Sometimes there may be a lack of political will or a weak infrastructure to enforce the laws. When looking at the legal situation in the medicine sector in different countries, the problems seem to be more often in the enforcement of legislation than in the lack of legislation.

Registration of medicines is a key tool in assuring the safety, quality and efficacy of a new medicine being introduced on to the market and in defining the legal status of the medicinal product. The infrastructure that will assure quality, safety and efficacy involves the licensing and inspection of manufacturers, distributors and their premises, and setting professional working standards. There is wide international cooperation in this field among the different component authorities. Nevertheless, every now and then the media have reports about counterfeit products and toxic products sold to the public, sometimes with disastrous consequences (see Chs 22 and 51).

Ensuring the availability of medicines

Medicines’ availability is one of the key requirements in a well-functioning pharmaceutical system. This includes a functioning manufacturing and importation system of medicines, and good procurement and distribution practices. These functions are often taken for granted in industrialized countries, while in developing countries they are key issues for a functioning system. In developing countries the maintenance of a constant supply of medicines, keeping them in good condition and minimizing losses due to spoilage and expiry are issues that need to be solved to assure the availability of medicines to the population.

As medicines increase in sophistication, the prices increase, and many new products are too expensive for much of the population if no mechanisms like price control or reimbursement/insurance systems are in place in the country. Economic availability of medicines continues to be a major policy issue for all countries at the present time.

Use of medicines

Medicine (drug) use or utilization studies and pharmacoepidemiological studies have aimed to describe the users of medicines and quantify their usage. On a population, or macro-level, factors influencing medicine consumption include among others: size of population, age and gender distributions, occupational structure, income levels, availability of health services, number and type of health facilities and personnel, along with social insurance and reimbursement mechanisms.

Medicine use studies have been used to identify ‘irrational medicine use’, such as the overuse of psychotropics and antibiotics, where there has been inappropriate prescribing and unnecessary extended treatment periods. There has also been interest in the ‘underuse’ of medicines for major chronic diseases such as hypertension, diabetes and hypercholesterolaemia. Underuse of medicines, together with misuse, whether deliberate or unintentional, has been one of the main focuses of patient adherence studies. From these studies we know something about the use of medicines and the clinical, social and economic consequences.

The use of medicines should be seen within the context of a society, community, family and individual, recognizing cultural diversity in concepts of health and illness or how medicines work. To try and understand the medicine-related behaviour of patients, qualitative research methods such as in-depth interviews have been used. These studies have focused on people’s thoughts about their medicines, their motivation to take or not take the medicine, their attitudes and beliefs about the medicines and their experiences of treatments. Some general consumer behaviour models have been used to explain non-prescription and prescription purchases. In one American study the medicine attributes that consumers rated as important included possible side-effects, physician recommendation, strength, prior use, price and the availability of generic versions. Medicines are not ordinary goods and consumers appear to understand this. According to one purchase theory, purchase motivations can also be characterized as being either transformational (positive) or informational (negative). Positive purchases are made to enhance or generate a positive situation or state of mind (e.g. clothes, music) and negative purchases to minimize or prevent negative situations (e.g. car service). Negative purchases are based on (rational) choices like perceived benefits and convenience (and therefore require more information), while positive purchases are more emotional and based on subjective appeal and positive shopping experience. Research has shown that OTC medicines and vitamins are neutral on the positive–negative dimension and oral contraceptives highly negative. This type of research is still not very well developed within the pharmaceutical field.

Like general illness behaviour, medicine use occurs in a social context. Choosing self-medication or consulting a healthcare professional to obtain prescription medicines is not based solely on symptoms or clinical aspects. The concept of ‘social knowledge’ has been used to describe collective understanding, arising in a group or population, which is based on available information and prior experiences. Family members, friends, work colleagues and their experiences, books and the media in addition to our own experiences, form the basis of social knowledge of medicines. Montagne in 1996 described some interesting social conceptions or fundamental principles about medicines in people’s minds, which he calls ‘pharmacomythologies’. It is a common belief among lay people that a specific medicine produces only one ‘main’ effect, which is positive. Other effects are considered as negative or ‘side’-effects. Likewise, it is believed that a medicine produces the same main effect every time it is taken and in each person who takes it. This means that medicine effects are caused by the taken medicine and the effect of the medicine is a property residing inside the chemical compound and not a function of some change in a living human body. This perception leads to the belief that medicines cure diseases, rather than often only treating symptoms.

The general health behaviour models and theories previously presented – such as the health belief model, theory of reasoned action, social learning theory, conflict theory and behavioural decision theory – can all be used to explain certain types of behaviour related to taking medicines. Basic decision-making and problem-solving skills are important components of patients’ seemingly rational and irrational behaviours. As presented earlier, the choices do not always follow the criteria of medical rationality, but can be justified as rational by the individual patient. It may be useful to consider rationality as a continuum rather than seeing something as either rational or not. The degree of rationality an individual exhibits is also influenced by social knowledge and the micro- and macro-environment, as described earlier, as well as the actual health problem.

Improving public understanding of medicines

During the last few years there have been attempts in both developed and developing countries to improve the knowledge and understanding about medicines among the general public. This can be seen as an attempt to influence and improve (from a medical point of view) social knowledge related to medicines and health in general. National campaigns such as ‘Ask about your medicines’ are good examples of this kind of activity. A more balanced partnership between consumer-patients and healthcare providers is one of the goals in such activities. This has been referred to as concordance (see Ch. 18). A better appreciation of the limits of medicines and a lessening of the belief that there is a ‘pill for every ill’ are examples of the goals of such efforts.

Advertising and other commercial information may fail to give objective information about medicines and patients need to be encouraged to be more critical of such information. Early education in schools about health and medicines would improve health knowledge, along with guidance on using appropriate information sources. To facilitate informed choices on the use of medicines, public education should be accompanied by supportive legislation and controls on the availability of medicines. Non-governmental organizations, community groups and consumer and professional organizations should be involved in the planning and implementation of such programmes to make them seem more relevant to their target audience. Healthcare professionals need to be prepared for their role in developing a partnership with their patient to optimize treatment options.