Progress in haemophilic care: ethical issues

Care of haemophilic patients has dramatically improved during the past 40-50 years. Clotting factor concentrates have become much safer. Expensive treatments such as prophylaxis, immune-tolerance induction, the use of bypassing agents in inhibitor patients, and the replacement of joints have become available. However, there is still a shortage of factor concentrate, and we are yet to discover the most cost-effective treatment for patients. There is also a wide gap in the availability of these treatments between developing and developed countries, and a risk that patients are treated in unnecessarily expensive ways in some parts of the world compared with those who suffer from a lack of treatment in other parts, thus creating the ethical dilemma of whether less safe but more affordable concentrates should be used in developing countries. Other issues that raise ethical problems are the use of molecular genetics and gene therapy. It is proposed that international working groups, preferably under the auspices of the World Health Organisation and the World Federation of Hemophilia, take responsibility to solve the ethical issues in haemophilic care, the basis of which should be to promote the education of modern care and to set up clinical studies, including studies on health economics, so that treatment and care are cost-effective and based on sound evidence.