Stephen Whitehead added:

"There is a robust framework already in place to evaluate these medicines developed by the Advisory Group on National Specialised Services (AGNSS) and it will be important that this framework is fully embraced by NICE. The inclusion of criteria in this framework such as societal value, contribution to research and budget impact, are vitally important when assessing medicines for very rare diseases and must be given prominence in decision making. Estimates of cost effectiveness are notoriously difficult to establish for these kinds of medicines and are generally not useful to aid decision making.  

“It will be important that an appropriately qualified, multi-disciplinary expert panel is assembled by NICE to support the evaluation process and that companies are fully involved throughout. We look forward to engaging with NICE to ensure that a system is set up which enables patients with very rare diseases to benefit from the newest and most innovative medicines.”

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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.