Technology assessment of a gene therapy

Abstract

One suggested motive for massive international research programmes (financially comparable to the moon landings) into the analysis of the human genome is the potential for improved medical diagnosis and treatment of the 4,000 known singlegene hereditary disorders. This paper presents a technology assessment framework containing some costs and benefits to analyse the merits of attempting to apply any new knowledge from this programme to the development of a novel medical treatment: the use of what is called somatic cell gene therapy (an application of genetic engineering) to a most unpleasant condition affecting some one in 16,000 live birth's (Lesch-Nyhan syndrome). The authors' conclusion is that any such treatment seems unlikely to work well, for technical reasons; that the consequential benefits to sufferers are unlikely to be great. Financial and non-financial costs and risks will arise for both patients (from side-effects, etc) and for the wider community. Thus this aspect of the human genome programme seems unlikely to increase social welfare.