International Pompe Association

Dr. J.M.A. van den Boer-van den Berg
Ethicist
Erasmus University Medical Centre Rotterdam, Dept. of Clinical Genetics, Dept. of Ethics
Leiden University Medical Centre, Dept. of Clinical Genetics
The Netherlands

Ethics

People with chronic diseases and handicaps are not only confronted with medical problems, but also with ethical ones. In general one may say ethics has to do with the behaviour of people, especially when that behaviour affects others. Ethics studies moral rules, such as: do not kill, do not cause pain, do not disable, do not deceive, or to sum up: ‘do no harm’. However there is more to morality than just moral rules. Morality also consists of ideals, such as: prevent death, prevent pain, prevent disabling, prevent deceit, or in other words: ‘prevent harm and do good’. But rules and ideals only provide a moral framework, a way of thinking, and not a concrete solution. Moreover, it is obvious that at one time or another every moral rule may be broken. In health care and certainly in the field of scientific research one runs a risk, by following one rule while simultaneously breaking another. Scientists and physicians are confronted with questions such as: Is it permissible to harm present people in order to do good to people in the future? People who are participating in clinical trials are confronted with questions too. What do I expect and will my expectations come true? Do I want to take the risk of harming myself (or my child)? New medical options create new moral questions, and those need new answers.

Medical responsibility

The twentieth century has brought many changes in health care. Rapid scientific evolution became closely associated with growing personal freedom. Nowadays people are, within boundaries, free to live their own lives. But this freedom has its price of increasing responsibility. Fundamental choices appear to be difficult. It is rarely immediately evident which is the ‘right decision’ in a particular situation. This is also true for people with diseases like Pompe’s disease, because on the one hand therapy seems to be approaching, and on the other hand much is unknown, unclear, and uncertain. When medicine develops, hope grows that someday new medication or therapy will be found, and somehow treatment or cure will be possible.

Autonomous choices

Patients and parents of patients want most of all to prevent a premature death or to stop the disease process. But they are not certain if and how they can achieve what they want, and therefore they do not know how to choose. For that very reason it is of the utmost importance to give patients who participate in clinical trials (and their parents, if children are involved) all the support and protection they need. They must get the opportunity to make their own autonomous choices. If a doctor respects a patient, he or she will give not only full information but also full support. Together they can reach a decision that fits into the patient’s life. A decision which will hopefully be right not only in the short but also in the long term. That is why respecting freedom needs other values as well. Values such as empathy and compassion. Researchers and physicians have to keep the interests of patients in mind, take them seriously, never give idle hope, and never claim certainty.

Conclusion

In the field of clinical trials rights, safety, and most importantly the well-being of patients are the most important issues, and they have to prevail above the interests of science and society. Together with scientific progress we have to keep moral progress in mind, in trying to create not only a civilised society but also a decent one.