​Stephen Whitehead said:

“It is essential that once medicines are proven to be safe and effective, they find their way to patients, regardless of whether the treatment effects a handful of patients or millions. This report demonstrates yet again the problems UK patients face in getting medicines for rare diseases.

"It is a worry that so few medicines for rare diseases undergo health technology assessment, and an even greater worry that those that do, are rejected so often.

“We need to ensure that the system is better suited to meeting the needs of patients with rare diseases. If we look at England specifically, the role of the Advisory Group for National Specialised Services – which is now part of NICE – will be crucial to improving patient access.  It must provide a robust framework which includes assessment criteria such as societal value, contribution to research and budget impact, whilst cost effectiveness should not be the primary driver of decision making. Finally, it is crucial that an appropriately qualified, multi-disciplinary expert panel is assembled by NICE to support the evaluation process and that companies are fully involved throughout.” 

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Notes to editors

The ABPI represents innovative research-based biopharmaceutical companies, large, medium and small, leading an exciting new era of biosciences in the UK.

Our industry, a major contributor to the economy of the UK, brings life-saving and life-enhancing medicines to patients. Our members supply 90 per cent of all medicines used by the NHS, and are researching and developing over two-thirds of the current medicines pipeline, ensuring that the UK remains at the forefront of helping patients prevent and overcome diseases.

The ABPI is recognised by Government as the industry body negotiating on behalf of the branded pharmaceutical industry, for statutory consultation requirements including the pricing scheme for medicines in the UK.