Rett Syndrome, rare diseases and UK research

Crompton, Helen (2001) Rett Syndrome, rare diseases and UK research. UNSPECIFIED. Manchester Metropolitan University. ISSN 1471-857X

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Abstract

This paper examines policy issues surrounding patients who suffer from rare diseases and the implications of the biotechnology 'revolution' in relation to priority setting and rationing in the National Health Service. It shows how the key players in rationing decisions - politicians, bureaucrats, managers, consumers and the media, are shaping research into rare diseases and the threat to autonomous researchers by the increasing centralisation of NHS R&D. It also shows the increasing power of patient and professional lobby groups to influence policy decisions, aided by the new information and communication technologies. The lack of government support for the nascent UK biotechnology industry, in contrast to the USA, not only impacts on industrial success but may also hinder medical advances. The recent European Orphan Drug Regulation (2000) and the supporting Framework V programme may have an impact on treatments for rare diseases. Orphan drugs are defined as medicinal products for the treatment of rare [orphan] diseases, whose costs of development are inevitably disproportionately high in relation to the volume of products likely to be sold, since there are only a few sufferers needing treatment.