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orphan diseases

This page shows the latest orphan diseases news and features for those working in and with pharma, biotech and healthcare.

Raising awareness and encouraging support on Rare Disease Day 2020

Raising awareness and encouraging support on Rare Disease Day 2020

These include:. New requests for applications for the Orphan Products Grants programme, which supports natural history studies and clinical trials for rare diseases. ... Raising awareness is central to our mission to improve outcomes for people affected

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  • Biopharma and orphan drugs Biopharma and orphan drugs

    Rare disease regulations. Twenty one of the 48 novel drugs approved in the US were for rare or orphan diseases – those that affect fewer than 200, 000 citizens. ... Clinical complications. Given the rarity of orphan diseases, one of the greatest

  • Industry needs an aligned position on the future of the EU OMP Regulation Industry needs an aligned position on the future of the EU OMP Regulation

    The Pharmaceutical Strategy is likely to include innovation-boosting measures and plans to revise the EU Regulation on Orphan Medicinal Products (OMP Regulation). ... of orphan diseases).

  • BRIC markets BRIC markets

    drug list, list of 24 orphan diseases, list of seven nosologies, and other programmes, depending on region.

  • The changing nature of approvals – what does the future hold? The changing nature of approvals – what does the future hold?

    Small and mid-sized companies can concentrate on specific development areas, innovative products and diseases with smaller target populations. ... However, rare diseases and orphan drugs are also emerging as important focal areas.

  • The trials and tribulations of rare opportunities The trials and tribulations of rare opportunities

    Pharmaceutical industry interest in developing treatments for rare diseases is increasing, for understandable reasons. ... Humira, the world’s best-selling drug, has multiple orphan designations. Likewise Soliris (eculizomab) – which generated

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Dovetail

About Dovetail We’re award-winning specialists in healthcare collaboration that makes patients’ lives better. Combining expertise with a passion for collaboration,...

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Working together against COVID-19
Analysing social media conversations to see what HCPs are saying online about the global pandemic...
Vicky Bramham, Managing Director at OPEN Health PR shares tips on media relations during COVID-19
Vicky speaks with freelance health journalist Jacqui Thornton who gives her expert view on how best to approach media relations during this unprecedented time....
Are biobetters a market access opportunity?
Biobetters are biologics that have been improved compared to the marketed originator, for example in efficacy, safety, tolerability or dosing regimen....

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