Faster access to treatments and diagnostics for rare diseases?

11 December 2009

The UK National Health Service (NHS) has launched a consultation on a proposed pilot scheme to speed up access to new therapeutics for patients with rare diseases. Normally, novel treatments must be thoroughly evaluated and approved according to strict criteria by the National Institute for Health and Clinical Excellence (NICE), the body that produces guidance on treatments and interventions for the NHS. In some cases – and particularly in the case of rare diseases that individually affect only relatively small numbers of people – it may not be possible to generate adequate data for the rigorous demonstration of cost-effectiveness required by NICE (and hence for NHS funding of the treatment) – for example, because the smaller numbers of patients make demonstration of efficacy slow even where novel therapeutics may actually offer significant benefit in terms of patient outcomes.

Many genetic diseases fall into the category of rare diseases, although in fact collectively their public health impact (and the burden they place upon health services) is significant, so that moves towards a ‘one size does not fit all’ system that can respond more flexibly to different requirements to assess needs and costs for less common conditions are highly desirable.

The new ‘Innovation Pass’ scheme, announced earlier this year by the UK Government as part of the new Office for Life Sciences (OLS) Blueprint (see Central Office of Information news) will allow selected new therapeutics to be made available via the NHS from 2010/2011 for three years. It is hoped that this will simultaneously allow patient access and generate data that could support subsequent full NICE appraisal, with priority to be given to areas of the greatest patient need. 

The Department of Health is now running a consultation for stakeholders to comment on the plans. The consultation notes that the Innovation Pass, currently intended to focus on treatments, could also be extended to include medical technologies and diagnostics, and specifically queries whether this is advisable. This might, for example, include new genetic tests.

The consultation closes on 8th February 2010.

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