Researchers have created a fundamentally new class of HIV-fighting molecules by combining two separate antiviral mechanisms into a single engineered protein. Early laboratory results, reported
As reported on BioPharmaDive, UCB has reported positive results from a head-to-head clinical trial showing that its anti-inflammatory biologic Bimzelx was more effective than AbbVie’s
Editor’s Note: Patient Worthy is proud and honored to share Jennifer’s story with cystic fibrosis and colon cancer, originally published on the Cystic Fibrosis Research
The biotechnology sector experienced significant positive developments this week, with Xenon Pharmaceuticals’ seizure medication achieving a decisive late-stage victory while regulatory pathways accelerated for several
As reported on Healio, glucagon-like peptide-1 (GLP-1) receptor agonists, best known for treating diabetes and obesity, are emerging as unexpected players in breast cancer care.
CAR T- cells are immune cells that are engineered in a laboratory to attack cancer. The therapy has worked as planned for certain blood cancers
Artios Pharma has achieved a significant milestone in cancer treatment development with the U.S. Food and Drug Administration’s granting of Fast Track designation to ART6043,
As reported on drugs.com, Bristol Myers Squibb (BMS) has announced encouraging interim results from the Phase 3 portion of the SUCCESSOR-2 clinical trial evaluating oral
As reported on FierceBioTech, the U.S. Food and Drug Administration has released its complete response letter (CRL) detailing why it declined to approve Regenxbio’s experimental
As covered on PharmaBiz, Roche has reported topline results from the phase III persevERA Breast Cancer study, which evaluated the oral selective oestrogen receptor degrader
Editor’s Note: Patient Worthy is proud to share this article from our friends at the Courageous Parents Network. A parent often can sense when there
Daiichi Sankyo has taken a significant step forward in expanding treatment options for early-stage breast cancer patients by submitting a supplemental New Drug Application (sNDA)
As reported in a recent article by Pharmaceutical Technology, Bristol Myers Squibb (BMS) has secured US Food and Drug Administration (FDA) approval for Sotyktu (deucravacitinib)
Editor’s Note: Patient Worthy is honored to share this caregiver story with you, originally written by Aisling Melton, Parent Champion at Courageous Parents Network. In
GlaxoSmithKline has achieved a significant regulatory milestone as China’s National Medical Products Administration granted priority review status to linerixibat, an investigational treatment for cholestatic pruritus
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has accepted and granted Priority Review to AstraZeneca and Daiichi Sankyo’s supplemental Biologics License
Two of Intellia Therapeutics’ clinical trials that had been on hold since October 2025 have now been released by the FDA. According to The New
REGENXBIO encountered a significant regulatory obstacle when the FDA issued a complete response letter regarding its biologics license application for RGX-121, an investigational gene therapy
Michael Bovarnick is a prostate cancer patient advocate and founder of The Prostate Education Project, a patient-led initiative dedicated to helping men and their families better understand prostate cancer, treatment options, and life during and after therapy. After navigating his own journey through surgery, radiation, and hormone therapy, Michael began sharing practical, experience-based guidance to help others feel less alone and more informed. His work focuses on education, quality of life, and empowering patients to take an active role in their care. If you are interested in sharing your prostate cancer journey, click the link: bit.ly/4dV7gru #ShareYourStory #PatientWorthy #pc #prostatecancer #prostatecancerawareness
Risk and benefit of therapies have a new scale in rare, progressive, fatal diseases. Individualized treatments must be evaluated differently.
Critical Path InstituteAs precision medicine continues to evolve, so too must the frameworks we use to evaluate benefit–risk, especially for patients facing severe, rare conditions with limited or no treatment options.
Join us on April 8 at noon (Eastern) for a timely and important discussion: Scalable Approaches to Patient-Centered Benefit–Risk: Defining Patient-Focused Drug Development in Precision Medicine
This session will feature patients and families sharing real-world experiences that are shaping how benefit–risk is understood in the context of individualized medicines. Their perspectives will provide critical insight into how decisions are made when stakes are high and options are limited.
Together, we will explore:
- How traditional development models translate to personalized contexts
- Patient and family perspectives on real-world benefit–risk decisions
- How benefit–risk evolves as new therapies become available
- Opportunities for collaboration across patients, regulators, and industry
- As traditional population-based models are increasingly challenged, this conversation will highlight how incorporating patient experience can help inform more responsive and scalable approaches to drug development.
Register now to be part of this important discussion: f.mtr.cool/mxnehuucqz
#CPath #Collaboration #DrugDevelopment #PrecisionMedicine #DataSharing #GlobalHealth #PFDD
Critical Path Institute #individualizedmedicine #rarediseaseExpectations around submissions of eCOA-related materials at trial start up are a persistent challenge globally. In this Educational Series session, regulatory experts from the Electronic Clinical Outcome Assessment Consortium’s membership will explore the expectations of the EU’s Clinical Trial Regulation and the use of the EU’s Clinical Trial Information System from an eCOA trials perspective. Are we over-preparing for EU-CTR submissions? Are eCOA source screen reports and translations required for this regulatory approval? Join us to hear the experts weigh in, and bring your own queries for further discussion.
eCOA Exchange #6
eCOA and the EU’s Clinical Trial Regulation
Host: Scottie Kern
Participants:
Pierre Mermet-Bouvier, Senior Manager, eClinical Strategy & Delivery, ICON
Pierre-Frederic Omnes, Executive Director, Life Sciences, TransPerfect
Date/Time:
Thursday, April 2nd, 2026
10-11 AM ET
Registration Link here: f.mtr.cool/foxjaavyhq
#CPath #Collaboration #ElectronicClinicalOutcomeAssessment #DrugDevelopment #DataSharing #GlobalHealth
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