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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 26, 2024 |
| By Max Bayer Bristol Myers Squibb is closing a 256,000 square-foot research facility in Redwood City, CA as part of the company's larger restructuring. Scientists at the site homed in on studying the tumor microenvironment. |
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By Nick Paul Taylor A $4.9 billion hole has appeared in Gilead’s pipeline. After years of setbacks, the big biotech dealt a final blow to its anti-CD47 monoclonal antibody magrolimab Thursday by removing the remaining solid tumor trials from its pipeline. |
By Zoey Becker The company opted not to renew its Biotechnology Innovation Organization (BIO) membership for its next fiscal year. |
Sponsored by NSF NSF TraQtion software combines decades of quality expertise with real-time digital insight in one trusted, cloud-based digital solution. |
By Max Bayer,Gabrielle Masson,Andrea Park Last year was a difficult one for ex-Stanford president Marc Tessier-Lavigne, Ph.D. An investigation into his prior research from the campus’ student newspaper ultimately led to his resignation. But the once-chief scientific officer of Genentech is now back in biotech. |
By Gabrielle Masson,Max Bayer We really didn't want to have to create another Layoff Tracker this year. But here we are, launching the third annual iteration tracking biopharma layoffs. |
By Kevin Dunleavy Ten years after dipping its toe into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained an FDA approval for the acquired treatment. The U.S. regulator has endorsed Beqvez for adults with the bleeding disorder hemophilia B. It becomes the first FDA-approved gene therapy for Pfizer, which will charge $3.5 million for the single-dose drug. |
By Ayla Ellison,Eric Sagonowsky,Kevin Dunleavy This week on "The Top Line," Fierce Pharma Senior Editor Eric Sagonowsky and Senior Writer Kevin Dunleavy are diving into our annual top pharma companies by revenue special report. |
By Angus Liu Novartis has started cutting ties with Chinese contractors amid a potential threat from the BIOSECURE Act. A depression drug candidate that Neurocrine licensed from Takeda succeeded in a phase 2 trial. AstraZeneca and Daiichi Sankyo's Enhertu is on the cusp of a key readout in HER2-low breast cancer. |
By Dave Muoio Two academic studies published this week sought to characterize cross-market deals and whether the FTC is taking a strong enough stance on blocking uncompetitive deals. Both painted an unflattering picture of post-merger prices and were refuted by the hospital lobby. |
By Conor Hale Researchers at the University of Washington’s Institute for Protein Design have put forward a paper showing its AI algorithms can help create millions of never-before-seen drug-like peptides—a field that Big Pharmas have described as the next wave of drug discovery. |
Fierce podcastsDon’t miss an episode |
| | This week on "The Top Line," Fierce Pharma Senior Editor Eric Sagonowsky and Senior Writer Kevin Dunleavy are diving into our annual top pharma companies by revenue special report. |
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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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