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Rare disease development is complex, characterized by distinctive challenges due to small patient populations spread across many geographies, limited disease data, and narrow site pools. For sponsors, successful studies involve striking a balance among cost, quality, and innovative approaches that improve trial efficiency and patient engagement. Read our checklist to explore 9 best practices for optimizing the efficiency and quality of rare disease clinical trials.
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Today’s Big NewsApr 25, 2024 |
| By Max Bayer Bristol Myers Squibb is cutting $1.5 billion in costs through 2025, including layoffs to more than 2,000 employees in 2024. The pharma says it will reinvest the capital into high-performing assets. |
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By Gabrielle Masson After BioMarin’s new leadership conducted a “strategic R&D asset review,” the company is prioritizing three of its “most productive” assets, leaving four candidates on the wayside. |
By James Waldron Keen watchers of Deloitte’s annual reports on Big Pharma will have noticed a depressing trend—ever-increasing costs for developing drugs over the past decade as the return on investment sinks. |
Sponsored by NSF NSF TraQtion software combines decades of quality expertise with real-time digital insight in one trusted, cloud-based digital solution. |
By Nick Paul Taylor AstraZeneca’s first-quarter results put a twist on a fairy tale, telling the lesser-known story of GOLDILOX and the porcupine. The drugmaker axed a cardiovascular disease prospect after wrapping up the phase 2 GOLDILOX trial while stopping work on early-stage assets bought in from Ionis and Redx Pharma. |
By Nick Paul Taylor Sanofi is narrowing development of one of its 5 billion euro ($5.4 billion), “pipeline-in-a-product” assets. The French drugmaker axed Sjögren’s syndrome from the list of targeted indications for frexalimab after seeing phase 2 efficacy data—and reported a phase 3 flop for one of its tarnished former top prospects. |
By Annalee Armstrong Regeneron is signing another major deal in gene editing—and, this time, it’s Mammoth. Mammoth Biosciences, that is. The two companies will collaborate on in vivo CRISPR-based gene editing therapies in a deal worth $100 million upfront plus $370 million in milestones per target. |
By James Waldron Cidara Therapeutics already knew that Janssen planned to palm off its obligations to their flu prophylaxis candidate to another entity. Now, it turns out that entity will be … Cidara. |
By James Waldron Both Destiny Pharma and Santhera Pharmaceuticals used their earnings announcements this morning to explain why a tighter clinical focus will come at the expense of one of their potential assets. |
By Helen Floersh Parexel has struck a multi-year deal with Palantir Technologies to integrate its artificial intelligence systems into Parexel clients' clinical trial operations. The companies did not disclose financial terms. |
By Nick Paul Taylor Biogen and Eisai are ready to ramp up their Leqembi sales and marketing push. After a slow start, Biogen is now planning to grow its U.S. field force by 30% as it works with Eisai on direct to patient and caregiver omnichannel marketing campaigns. |
Fierce podcastsDon’t miss an episode |
| | This week on "The Top Line," we are joined by Annalee Armstrong, Senior Editor at Fierce Biotech, and Ben Adams, Senior Editor of Fierce Pharma Marketing, to discuss Fierce's take on March Madness. |
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| eBookTo make every rare disease trial count, we design patient-centric, relevant strategies for sponsors and caregivers to produce impactful results every time. Sponsored by: Emmes Company |
| WhitepaperDiscover the secrets behind successful patient engagement Sponsored by: ProofPilot |
| WhitepaperAntibody biomarkers are redefining precision medicine, from diagnosing diseases earlier to stratifying patients – download your FREE white paper today! Sponsored by: Sengenics Corporation LLC |
| WhitepaperThe use of prefilled syringes to administer biotherapeutics is beginning to gain traction because they offer a range of stability, efficacy, and patient safety benefits. Download this white paper to learn how Lonza has developed a novel workflow to ensure confidence regarding functionality in PFS and autoinjectors. Presented by Lonza |
| eBookUnlock the future of monoclonal antibody (mAb) manufacturing with a complete guide to enhancing productivity and quality for exceptional outcomes. Sponsored by: Thermo Fisher Scientific |
| Whitepaper This paper assesses the current state of the oncology market, reviewing key expectations for 2024. It addresses CAR-T therapies, KRAS, radiopharmaceuticals, & targeted protein degradation. Presented by Blue Matter, strategic consultants in the life sciences |
| WhitepaperChoosing the right contract lab can help drug developers accelerate their timelines and generate cost-efficiencies. For insights into what to look for when selecting a GMP/CMC lab partner read this white paper which delves into a range of factors that can support your needs in both the short-term and in planning for the future. Sponsored by: PPD®️ Laboratory Services |
On Helix Date: 4 July 2024 - Location: Babraham Research Campus, Cambridge UK |
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