Rare Disease
Life Impact of Pain – Pain Series Part 2 of 3
Click here to see Part 1 of of this 3 part pain series. If you suffer with chronic pain, you not only live with the unrelenting
Illumina and Labcorp Deepen Collaboration to Broaden Access to Precision Oncology Testing
- March 26, 2026
Opinion Piece: Insurance Abuse
Eftilagimod Alfa Setback: Immutep Halts Landmark Lung Cancer Trial Following Futility Analysis
Dizal’s Zegfrovy Delivers Phase 3 Breakthrough in EGFR Exon 20–Mutant NSCLC
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THE LATEST FROM PATIENT WORTHY
Obesity
Monthly Obesity Treatment Gets Easier: Lilly’s New Zepbound Pen Simplifies Weight Loss Injections
Patients using Zepbound for weight management now have a simpler option for administering their medication. As reported by PharmaLive.com, Eli Lilly received FDA clearance for
Prostate Cancer
Redefining Cancer Treatment: Podcast Interview with Dr. Charles Link on Sync-T and the Future of Immunotherapy
On this episode of Wait, How Do You Spell That?, host Bree Clare sits down with Dr. Charles Link, a pioneering oncologist whose career has
Bladder cancer
“At 44, I Thought I Was Starting Over” – Kristian’s Bladder Cancer Story
At 44 years old, I was ready to hit the reset button. In March of 2025, my partner and I stepped away from our careers.
Ovarian Cancer
British Columbia Researchers Develop a System to Reduce Ovarian Cancer by 80%
Ovarian cancer is a serious gynecological cancer with no reliable screening test, resulting in limited treatment options and a low number of survivors. Each year
Fibrodysplasia Ossificans Progressiva (FOP)
Breakthrough in Rare Bone Disease: FDA Fast-Tracks Garetosmab for Fibrodysplasia Ossificans Progressiva
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for garetosmab, marking a significant
Datroway Receives U.S. Priority Review for First‑Line Treatment of Metastatic Triple‑Negative Breast Cancer
AstraZeneca and Daiichi Sankyo’s antibody‑drug conjugate Datroway (datopotamab deruxtecan) has been granted Priority Review by the U.S. Food and Drug Administration (FDA) for use as
Urea cycle disorders
More Than a Diagnosis: Gabriela’s Journey With Lysinuric Protein Intolerance, Urea Cycle Disorder, and Unshakable Faith
As a little girl, I believed miracles only existed in movies. They felt distant, beautiful stories meant for someone else. I never imagined that my
Psoriasis
Dual Approach to Disease: Eli Lilly’s Zepbound-Taltz Combination Demonstrates Breakthrough Results in Psoriasis Treatment
Eli Lilly is making strategic moves to expand its obesity medication Zepbound beyond weight management, demonstrating promising results when combined with its autoimmune therapeutic Taltz
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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
