When existing medicines no longer offer a cure, people want new medicines as soon as possible. At the same time, they want the medicines to be safe. How should the “conditional approval” of medicines take place in a socially responsible way? 

There is currently the possibility of rapid, “conditional approval” of medicines for severe and as yet untreatable diseases. However, a medicine’s efficacy and safety must be monitored properly after it is put on the market. This project demonstrated that there is much to be gained by actively involving patients and physicians in the monitoring process. Success factors such as short lines of communication and a shared story about the desirability of the process were identified.

Using and monitoring conditionally approved medicines within a protected regulatory framework and on a small scale (see box) is a method that could be applied more widely, say the researchers. It can supplement or provide an alternative to the standard large-scale procedures for monitoring new medicines, but this is only socially responsible if the research is properly embedded in close and well-defined networks of patient organisations, medical professionals, pharmaceutical companies and researchers.

Why might wider use of this method be beneficial?

1. Close contact between patients and doctors can reveal causal relationships that may be overlooked in research based on large databases.
2. New information on the effect of a medicine could immediately be incorporated in patient treatment and further development of the medicine.
3. Patients appear to be happy with the opportunities the new medicine offers and are willing to take the associated risks.

Critical questions

There are a number of critical success factors, however, such as creating close involvement by all participants, short lines of communication, good information provision and a shared story about the desirability of conditional approval as an alternative or in addition to the standard process. The participants must also be coached in providing a good explanation in response to critical questions.

Once these factors are met, the researchers believe a safe framework can be created for experimentation on the basis of initiatives from patients, patient organisations and physicians. Government agencies would, in that case, have to assume a facilitative rather than a directive role.

Different models of participation

In procedures for “conditional approval”, the benefits of rapid availability are weighed against the health risks. This is only justified if there is an effective system of “pharmacovigilance”, or monitoring a medicine’s effects after it has been put on the market. In practice, however, there are inadequate legal frameworks for this. Because there are few incentives for stakeholders to report negative side effects, for instance, this often does not happen.

In this project, the research team cooperated with stakeholders in the medical profession, which resulted in an interdisciplinary conceptual framework for the research. The research project focused on the role of users (from patients to physicians and critical citizens’ groups). Participation by these groups will result in an improved process and, according to the projections, more socially responsible pharmaceutical innovations.

It emerged from the research that different models are possible for participation by user groups. The researchers indicated prerequisites and conditions under which certain models work better than others.

Ethical conflict

The research team identified a number of ethical issues in the initial phase. In all cases, there was a conflict between managing the health risks and the potential health benefits of the new medicine. For example:

• One of the requirements for early approval is that the effects of the medicine should be researched more extensively than normal. In practice, however, research results hardly ever lead to withdrawal of the medicine at a later stage. The pressure from companies and patients is simply too high.
• Proper medical research requires a control group that is given a placebo. If the medicine is already on the market because of early approval, it may be unethical to withhold it from patients who need it.
• In the case of medicines for an emergency situation, such as an influenza epidemic, the dilemma is how to work around the principle that treatment is only permitted if patients voluntarily accept the risks.

These types of ethical issues were pivotal in developing alternative methods for conditional approval and pharmacovigilance.

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Prerequisites for conditional approval of new medicines

The European Medicines Agency (EMA) recognises three categories of medicines eligible for conditional approval:

1. medicines for diseases that are life-threatening or seriously weaken people, such as HIV;
2. medicines that are needed in an emergency situation, such as an influenza epidemic;
3. medicines known as orphan medicines, such as medicines for rare cancers.

Medicines in the third category concern pharmaceutical drugs for a condition that is rare, which means it is not commercially interesting for the pharmaceutical industry to invest in it.

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