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orphan drugs
This page shows the latest orphan drugs news and features for those working in and with pharma, biotech and healthcare.
GSK’s Benlysta given orphan drug status by FDA for systemic sclerosis
GSK’s Benlysta (belimumab) has been given Orphan Drug Designation (ODD) by the US Food and Drug Administration for the potential treatment of systemic sclerosis (SSc), the company announced. ... The antibody will now benefit from the incentives given
Latest news
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The Holy Grail of healthcare pricing
Drugs with an orphan designation as the only FDA-approved indication. That’s quite a list of exclusions. ... No drug with an orphan designation or less than $200m in Medicare spending can be subject to price reductions.
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PMEA 2020: continuing to recognise excellence in improving patient outcomes
Excellence in Rare Diseases and Orphan Drugs categories. ... with support from Learner Adams Bones Ltd. 8. Excellence in Rare Diseases and Orphan Drugs.
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Building a brighter future for rare disease patients
Before 1983 only thirty-eight orphan drugs gained regulatory approval while by 2014 that figure had reached 468 designations covering 373 drugs. ... The calibre of the shortlist in the Excellence in Rare Diseases, and Orphan Drugs category at the PMEA
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AstraZeneca buys FDA priority review voucher from Sobi
The FDA’s PRV is used as an incentive for companies to invest in orphan drugs, by promising a cost reduction for the application fee for future products and a ... hundreds of millions for the most promising drugs.
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The most annoying ‘F’ word in healthcare
We know that some of the largest employers in the US participate in these plans and we also know that fail first policies are being applied to the coverage of drugs ... In other words, these policies are not reserved for the orphan disease drugs or the
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Approximately 81 fully matching, plus 94 partially matching documents found.
Latest Intelligence
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Assessing the impact of rarity on French HTA and pricing decisions
We analysed 22 non-oncology orphan drugs (ODs) that received EMA approval between July 2012 to the end of 2019, focusing on chronic, non-curative treatments. ... Specifically, the CT assessment – which in turn depends on submitting clinical evidence
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Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England
Comparison of orphan drug assessments from NICE versus the NHS. The question of how to adequately capture the holistic value of orphan drugs (ODs) for rare diseases is the subject of ... to drugs targeting ‘very rare’ diseases (typically ultra-orphan
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Successful product commercialisation: what can we learn from the rare disease setting?
Furthermore, orphan drugs are anticipated to account for 55% of the EU pipeline’s cumulative value in 2022. ... specialty or orphan drug status – all of which are typically associated with drugs targeting rare diseases.
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Biopharma and orphan drugs
Price concerns. Another argument in favour of the support given to the orphan drug industry is that rare disease drugs generate lower revenues than mass market medicines. ... According to a 2019 study, for example, the mean orphan drug cost per patient
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Cortellis Drugs to Watch in 2020 report
The 11 drugs are almost universally accelerated in their development by orphan drug status or other designations intended to spur closer cooperation between regulators and drug developers with the intention of ... The 11 drugs identified collectively
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Approximately 14 fully matching, plus 44 partially matching documents found.
Latest appointments
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New hires at Crescendo, Healx and Galecto
Prior to joining Healx, he worked as CMO at Aparito and as therapeutic area head of orphan drugs at Mereo BioPharma. ... Before that, he lead global R&D at Almirall, which involved 500 staff and the development and approval of several new drugs in a
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New hires at FibroGen, Saniona and Turbine
He has extensive experience of the regulatory, access, commercial and financial environment for developing orphan drugs in the US – a region which is directly relevant to Saniona’s next phase of ... The first members of the SAB bring 70 years of
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Orchard Therapeutics appoints Mark Rothera as CEO
market, and launching several orphan drugs.
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XenoGesis appoints business development consultant
business.”. Currently, Mogle serves as chief executive officer of RareMoon Consulting, a non-clinical development and regulatory affairs consulting firm specialising in orphan drugs she co-founded earlier this year. ... She has previously held
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Hansa Medical strengthens management team
In his new role, van Troostwijk will be responsible for overseeing the firm's commercial assessments of its lead project IdeS, an orphan drug in development for organ rejection. ... IdeS.”. Van Troostwijk brings over 20 years of sales and marketing
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Approximately 0 fully matching, plus 12 partially matching documents found.
Latest from PMHub
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Rare Disease Day – looking to the future with hope
Collaboration. Progress would not be possible without collaboration at all stages in the commercialisation of rare disease drugs, from clinical development to product launch, to those working hard to elevate the ... themes to advance their goal) recently
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Rare Disease Day – why it’s important to show your stripes
A standardised definition for rare diseases is currently lacking, with incidences typically defined by regional legislations developed to incentivise drug development (termed ‘orphan medicines/drugs’ by the FDA and EMA).
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OPEN Health’s virtual reflections on the World Orphan Drug Congress US
Orphan drugs and advanced therapies no doubt come with high expectations from patients, carers and clinicians. ... Additionally, as fully discussed during the conference, demonstrating the value of orphan therapies is essential for broad and ready access.
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Delivering change through better rare disease communications
Consequently, it’s vital that biotechnology and pharmaceutical companies cut through the noise to bring real understanding and informed decision making about the next generation of orphan drugs.
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Lucid Group has four programmes shortlisted for PMEA 2019
Category: Excellence in Innovation. DermDialogue: Bringing innovation to MSL‐led education. Excellence in Rare Diseases and Orphan Drugs.
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Approximately 5 fully matching, plus 19 partially matching documents found.
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