Introduction

The regulation of pharmaceuticals is a form of risk regulation. The objective is to regulate risks in order to avoid the materialisation of potential harm to society in the field of public health. The risks related to pharmaceuticals mainly relate to availability, use, information, production and environmental impact. Risk regulation entails a balancing of risks and benefits. Such balancing requires the assessment of risks (risk assessment) and the management of risks (risk management). In other words, one needs to evaluate whether a risk exists, and if so, how to deal with it.

In an ideal world the risk associated with a medicinal product is very low and the benefits are very high. In most cases unfortunately such an ideal situation does not exist. Most medicinal products engender certain benefits, but not without sustaining considerable risks in the form of side-effects and/or adverse reactions.

Since the appreciation of risk is strongly related to personal conviction, views on how to regulate pharmaceuticals may differ considerably. In this context there are three groups of people generally distinguished in scientific literature: risk-averse people, people who are risk neutral, and risk-seeking people.¹

A risk-averse person does not like to take risks – he or she is better safe than sorry. Someone who is risk neutral does not prefer to take risks, but does not have a problem taking a risk either. A risk neutral person has the most rational approach to dealing with risk. Finally, a risk-seeking person will prefer to take a risk. A good example is a racing driver. Pushing a racing car to the limit entails considerable risk, but is worthwhile for the driver as he gets a kick out of it.

The regulation of pharmaceuticals tends towards the direction of the risk-averse. Although the goal is to make the evaluation of medicinal products as objective as possible both legislatures and evaluators prefer to be safe than sorry. Unfortunately, this approach often fails to bring about an optimal balance between risks and benefits. On the one hand the criteria used for evaluation are generally not representative of the real risks that exist in practice. On the other, the evaluation of medicinal products is executed in a very restrictive manner.

Based on the foregoing, risk regulation of medicinal products involves at least three issues which need further discussion:

•  Zero risk society
•  Risk-risk situations
•  Human behaviour.

Zero risk society

Governments and competent authorities regularly seem to strive towards creating a society which is free of risk in order to give consumers the feeling that they are well protected by the state. A negative side effect of such an approach is inefficient regulation which is very costly for society (i.e. administration, industry, and consumers).

Since no product is absolutely free of risk, patients and consumers may not be aware of the risks they are still facing despite the regulatory efforts of their administration. Instead of striving for a zero risk society, risk regulation needs to focus on efficient regulation and consumer information. This means that regulation should exclude hypothetical risks and be limited to risks that are realistic or real.²

Risk-risk situations

The term risk-risk situation refers to the situation where the alternative for the one risk represents another new risk.³ For example, if a certain pesticide is prohibited it may not present a risk anymore, but what if the alternative is even more harmful? Regulating risks goes further than targeting the specific risk at hand. One also has to examine the consequences of addressing a risk. Therefore a risk management strategy needs to take into account the effects of the measures that are being taken.

For pharmaceuticals the main parameters that need to be taken into account are firstly the harmfulness of the product, and secondly the chance that the harm materialises. If a product shows certain mild or moderate side effects which surface relatively often, it may still be better to allow that product instead of an alternative that has severe side effects that occur seldom.

Human behaviour

As mentioned above, the regulation of risk is inseparable from human behaviour. To avoid risks materialising there is a need for consumers to address potential risks properly. Human beings do not behave rationally in respect of risk (bounded rationality).⁴ Therefore, regulation of pharmaceuticals should not only focus on the quality and safety of the products involved, but also on the risk perception of patients and consumers regarding the product concerned.⁵ Therefore, appropriate risk communication can educate patients and consumers in taking the ‘right’ decisions.⁶

With the new communication mechanisms that exist nowadays on the internet, such as Wikipedia, Facebook, Twitter, YouTube, and thousands of other internet forums on which patients and consumers exchange information on medicinal products and medical treatments, governments, manufacturers, and healthcare providers have a (moral) responsibility to make sure that consumers and patients get an accurate picture. Paternalism should however be avoided at all cost. It will undermine the authority in the long run. Moreover, patients and consumers increasingly prefer to decide themselves on the treatment they want to use.

Risk regulation of medicinal products in practice

At the heart of pharmaceutical regulation lies the licensing system. The concept is simple. To gain market access one is required to obtain a license. The parameters under evaluation in such system are quality, safety, and efficacy. Data on quality, safety, and efficacy have to be provided by the manufacturer. Competent authorities subsequently make a risk-benefit analysis on the basis of which they decide to deny or grant a license.⁷ As most licensing systems were originally tailored to the characteristics of conventional synthetic medicinal products, they are generally not fit for the evaluation of non-conventional medicinal products from a practical and economic point of view.

Therefore special regimes adjusted to the specific characteristics of the products were developed worldwide to enable proper evaluation of non-conventional medicinal products – such as homeopathic and anthroposophic medicinal products. In most cases, these regimes derogate from the standard licensing procedure where necessary, while remaining within the framework of the conventional procedures. Examples of special regimes at EU level are the ‘special simplified registration procedure’ for highly diluted homeopathic medicinal products, and the ‘traditional use registration’ for herbal medicinal products.⁸

As licensing systems have an all or nothing character (you either get the licence or not), they rigorously influence market mechanisms (governments, consumers and producers).⁹ Hence, it is of cardinal importance that such pre-market control is applied as efficiently/restrictively as possible to avoid unnecessarily high costs for competent authorities, manufacturers, and consumers.

In the field of (non-conventional) medicinal products in the EU this is currently not the case. The approaches not only tend to be too theoretical, formalistic and costly, but they also appear to be disproportionate in the sense that sufficient protection may be generated by means that are less burdensome and inhibitive than the current ones. As a result, society on the one side has to bear a cost for pharmaceutical care that is too high. On the other side, patients and consumers are provided with a sub-optimal range of medicinal products to choose from – in fact the range should be larger.

Due to their less restrictive character post-market control mechanisms for example should be favoured if effective. An improved system of pharmacovigilance may be a good option to maintain safety of medicinal products on the market. Moreover, it can correct problems that did not surface at initial inspection.

Conclusion

The regulation of non-conventional medicinal products is a form of risk regulation that is currently too rigid to be efficient. The restrictive character of the pre-market control on medicinal products in combination with a (relatively) risk averse implementation of evaluation mechanisms does not provide an optimal balance between health protection and market access.

Improvement of post-marketing control mechanisms such as pharmacovigilance in combination with a stronger focus on ‘real’ risk in pre-market control and better risk communication to patients and consumers may bring about a more flexible and cost-efficient alternative for the current risk regulatory models. This would favour society in the long run as costs would go down and the number of products from which to choose would increase.

Johan Hulshof
Van Benthem & Keulen, Advocaten en Notariaat