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European Biopharmaceutical Review

Editor’s Letter

“It is important that rare disease businesses bring treatments to market as early as possible to give patients the best chance,” say Sobi’s Hege Hellström and Neil Dugdale in our summer 2017 industry Q&A. This captures the essence of therapy research and development for these often life-limiting illnesses affecting an estimated 70,000 patient groups. Access to the therapy, not just producing and commercialising it, should be a priority.

‘Salami-slicing’ of mass market, non-rare diseases to create artificial orphan indications is increasingly blurring lines between big pharma and specialised focus, according to The Weinberg Group’s Matthew Weinberg and Emily Krulewitz. Its driver is seemingly pharmacogenomics and the ability to stratify patients inside a group based on their genome and consequent response to particular therapies, switching them to personalised medicines.

Hermann Mascher at pharm-analyt Labor reminds us of the transformational potential these patient-specific therapeutics offer, and that particular biochemical pathways’ missing or defective components can be both the biomarker to identify a disease’s subset and the therapy to restore that deletion. If these could be characterised by bioanalytical methodologies serving a range of conditions, more synthetic forms of these endogenous small molecules may come on stream in development as therapeutics.

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Dr Deborah O’Neil, Chief Executive and Scientific Officer, NovaBiotics Ltd
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Dr Deborah O’Neil
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