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Despite recent initiatives, the UK is failing adequately to address the inequities in healthcare provision for children with rare lung disease
Money drives research—whether it is for profit by drug companies or for academic status by universities. It is therefore not surprising that little has been done in the past for patients with rare lung diseases, where there is little financial incentive for drug companies or the small population does not merit sufficient grant funding for adequately powered clinical studies. This problem is further compounded in children where research is perceived to be even more difficult. Over the recent years, largely due to extensive lobbying from independent patient support groups, there has been a concerted effort by the European Parliament and the USA government to address these inequities.
The French originally coined the term “orphan” disease, now defined as a disease with a prevalence of less than 5 in 10 000 individuals, and they have been instrumental in addressing the many problems faced by patients suffering from these diseases. It is estimated that there are 7000 rare diseases affecting 27 million people in Europe and a further 27 million in North America. The adoption of recent laws such as the loi sur l’égalité des droits et des chances, la participation et la citoyenneté des personnes handicapées (law on equality of rights and opportunities, the participation and the citizenship of the handicapped) by the French Parliament makes specific reference to rare diseases. In addition, European Parliament legislation in 1999 on orphan drug development provides access to treatments for patients with rare diseases and encourages industry to develop new treatments by providing financial inducements. Similar legislation has been in place in the USA since the 1983 Orphan Drug Act. In the USA the Rare Disorders Act of 2002 formally established a statutory …
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