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New Patient Charter for rare diseases launched

22 October 2014 David Bennett

The charity, Genetic Alliance in the UK, launched its second Patient Charter at Great Ormond Street Hospital, London, this week (22 October). The emergence of specific treatments for rare, usually monogenetic conditions has been one of the most prominent developments in health care during the past decade. Payers are still adapting existing policy apparatus and organization designed for common diseases to cope with the rising number of new rare disease treatments.

The aim of the charter is to provide systematic input of patient evidence into the appraisal of new treatments. Compared to common diseases, the special value of the patients’ evidence arises from the often limited data available on the total impact of a rare disease on the individual and their family.

Another important objective is to clarify and simplify the current complex processes by which patients gain access to the treatment they need. The UK currently lacks a developed and streamlined process for evaluating rare disease treatments for reimbursement. This means that an increasing number of patients are likely to be forced to make individual applications to secure access to a licensed medication. As one patient representative at the London meeting noted, the patients are often the least able to campaign for treatment access due to the severity of their medical condition.

One of the influential agencies responsible for appraising these treatments in the UK, NICE, will be limited to evaluating three rare disease treatments per year even once their process is finalized. There is an adage that justice delayed is justice denied. The same could be said about the implementation of health policy.

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