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Fast & accurate MIC values are important, even more so when it is put to work.

Over the years, I've learned that everyone is only a degree of separation away from someone they know personally who is affected by a chronic condition. 73 million is not a small number, and that's just the number of individuals affected in the US alone. The list of diseases is long and growing, as are the days/weeks/months that pass for patients who are suffering. It's time to drive change as a community, and thanks to a generous donation from Metrodora Foundation, we finally can. Metrodora Institute is launching a landmark platform, called #MetX, to comprehensively study these complex disorders. **#MetX is built by patients for patients.** Whether you are personally affected or know of someone who is, this is a chance to make a difference and help by participating in research. Enroll now: https://lnkd.in/gyRteFJz This study can't be done in a silo and we have lined up tremendous partners across the industry to help us move faster. Steve Gardner, Rowan Gardner, and PrecisionLife team are bringing novel diagnostics and identifying drug candidates for clinical trials available to #MetX participants this year. Alex Boches, Tony Wyss-Coray, and the Teal Omics team are applying their cutting-edge technology to unlock key protein signatures driving biological changes in chronic conditions. With more partnerships on the way, groups are stepping up to be part of this transformation. We are working closely with foundations across the country that have served as thought leaders and patient champions across the growing community. As a start, Open Medicine Foundation (OMF), Sjögren’s Foundation, Food Allergy Science Initiative, The Ehlers-Danlos Society, Brain Inflammation Collaborative, Inc., and Solve ME/CFS Initiative are making sure our collective approach can serve as a network to improve access to clinical trials and support research. On a personal note, having witnessed the amount of suffering my family and friends have gone through with chronic conditions, I'm humbled to be a part of the change that is coming. Fidji Simo and Amy Rochlin, thank you for being a voice for patients everywhere and breaking down siloes through your leadership. While a study like this would have taken years to establish in traditional research institutes, your vision has made all of this possible in just a matter of months. John Wirthlin, Brent Goodman, Anne Maitland, Ellie Volckmann, and the rest of the Metrodora Institute team, I appreciate your partnership, dedication, and speed as we make all of this possible for patients. #precisionmedicine #migraines #eds #endometriosis #ibd #mastcell #foodallergy #asthma #sjogrens #scleroderma #pots #concussion #longcovid #mecfs #pans #chronicillness #visible #biobanking #rwd #patientadvocacy

Is your commercial team responsible for rare disease or ultra-rare disease therapeutics? If yes, mark your calendars for July 18 for this webinar with Peter W. Carbone, CEO of Mindset Effect Consulting, for critical insights into this market and effective strategies to engage HCPs. Specifically, you'll learn about: 📌 Defining rare (orphan) diseases. 📌 How L&D’s mission must be to always help commercial teams sustain the hope of rare disease patients. 📌 Ways L&D can lead the charge to becoming a stronger voice for rare disease patients. 📌 The mindsets required for rare disease commercial teams. 📌 Tips on how rare disease commercial teams can work to inspire key decision makers to act on behalf of rare disease patients. Register now! https://lnkd.in/dPKivTry Plus, attendees will be entered into a drawing to win one of 10 copies of Peter W. Carbone’s book, The Rise (Rare Disease) Selling Framework. #LTEN #webinar #raredisease #sellingstrategies #sellingframework

I saw this pic and it really struck a chord with me. Many of you will know about European Medicines Agency recent recommendation to revoke the license for OCA in PBC- a decision that still blows my mind. If this recommendation is carried through, it will absolutely do harm to some PBC patients. Absolutely. So, hopefully the European Commission will send it back for review. Beyond that, there is this insistence that patients MUST engage in placebo arm trials, even after a drug has a (conditional) license. EMA wants PBC patients - the ones whose disease has already progressed, and who are in the highest risk cohort- to voluntarily forgoe their opportunity to an active medicine, to let their disease progress, to let their biochemistry worsen- all in the name of science. First, do no harm. Once upon a time, the average life expectancy for someone diagnosed with PBC was 7 years. Successful treatments came along and that changed. BUT... EMA want PBC placebo arm to last 7 years. 7 years without successful treatment. What could possibly go wrong?!? The trials are designed to measure how many patients without treatment experience ascites, varisceal bleeds, decompensated cirrhosis, need for transplant and death. First, do no harm. COBALT was the first post-license PBC trial designed around this and it failed. Not the drug- the trial. It failed because patients would not comply. And neither they should. Patients withdrew and the trial collapsed. So would anyone be reckless enough to ask PBC patients in 2024 to forgoe medicine to have a sugar pill? Surely not, you would think. Because- first do no harm. Yet, it turns out Ipsen have had to do that same thing in PBC. Right now. 7 years. Over 200 patients in the placebo arm. In a world where we spend squillions trying to add a few months of life for cancer patients- again, rightly so- why is it OK to cut a few months off a PBC patient's life in the name of science? First, do no harm. So yes... this picture struck a chord with me. I am sure it will resonate with many #PBC patients- quite possibly in #patients in other #rare diseases too. Let me know your thoughts and comments below. I would love to know if your disease area faces this also. Maybe we can come together to highlight this issue as an entire community. Maybe you feel as unheard, as unseen as we do. Maybe together we can be that difference. Maybe, we can show that not one single patient should ever have their incurable disease progress in the name of science. After all... First, do no harm.

Last week marked Rare Disease Week🌈, culminating in the FDA's Public Meeting for Rare Disease Day 2024. Below are 8 essential insights, highlighting the regulatory initiatives of the FDA and strategies aimed at advancing the development of treatment options for rare diseases👉 💪 CDER Engagement Initiatives: The FDA's CDER has launched programs like the Accelerating Rare Disease Cures (ARC) and LEADER 3D Initiative to support rare disease drug development through regulatory knowledge and support. 💪 CDRH Patient Perspectives on Home Use Devices: Efforts to integrate patient perspectives into regulatory processes for device quality and accessibility, in collaboration with the National Organization for Rare Disorders (NORD). 💪 Project Catalyst: Aims to expedite the availability of directed and novel cancer treatments through guidance and educational resources for small pharmaceutical companies and academic life science incubators. 💪 START Pilot Program for Rare Disease Therapeutics: An initiative to expedite the development of therapies for rare diseases without approved treatments, focusing on significant regulatory milestones and therapy approval progress. 💪 Decentralized Trials: The FDA is focusing on improving patient accessibility to clinical trials by overcoming geographical, mobility, cognitive, and socio-economic barriers through decentralized trials, including telemedicine visits, direct distribution of products, electronic informed consent, and home visit mobile trial units. 💪 Digital Health Technologies (DHT): The use of wearable devices, ePRO instruments, continuous monitors, and software for medical analysis is being emphasized to provide richer, continuous data for better disease understanding and treatment evaluation. 💪 Pragmatic Trial for Rare Cancers: Project Pragmatic aims to simplify clinical trials, making them less burdensome for patients and more reflective of real-world experiences, exemplified by a streamlined lung cancer trial focusing on essential outcomes. 💪 Medication Guide Patient Medication Information (PMI): A proposed rule in 2023 aims to simplify patient understanding of medication use through a one-page document format for outpatient prescription drugs, enhancing patient information accessibility. These takeaways highlight the FDA's comprehensive approach to enhancing rare disease research, drug development, and patient engagement through innovative trials, regulatory initiatives, and collaborative efforts.🚀

Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub Public Meeting FDA’s Rare Disease Innovation Hub, in collaboration with the Reagan-Udall Foundation for the FDA, will host a public meeting on October 16, 2024. The meeting will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work. During this public meeting, FDA seeks to gather stakeholder input in the following areas: Cross-cutting rare disease-related, scientific, regulatory, or policy issues (those not related to a particular disease or condition) that should be prioritized for consideration by the Rare Disease Innovation Hub Rare disease-specific (but not application-specific) scientific, regulatory, or policy issues that should be prioritized for consideration by the Rare Disease Innovation Hub Rare disease-related activities or initiatives currently being undertaken independently by CDER or CBER that you believe would benefit from being undertaken by the Rare Disease Innovation Hub as a joint activity Approaches that the Rare Disease Innovation Hub should follow for engagement with patients and caregiver groups, industry organizations, and scientific/academic organizations #fda #raredisease #innovation #policyissues #regulatory https://lnkd.in/g5-QNpGU

RESTORATiVE303 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the safety and efficacy of VE303 in patients at high risk for recurrence of CDI after completing a course of antibiotics for a prior CDI episode. The trial is being conducted at approximately 200 sites in 22 countries, across four continents. The primary endpoint is the comparison of CDI recurrence rates at eight weeks in the VE303 and placebo groups.  https://lnkd.in/dhtCMd8b

**Only put off until tomorrow what you are willing to die having left undone** **Imdusiran, VTP-300, and low-dose nivolumab show promise in treating chronic hepatitis B** In a recent Phase 2a clinical trial for chronic hepatitis B (cHBV), Arbutus Biopharma and Barinthus Biotherapeutics presented encouraging preliminary results. The trial showcased significant reduction of HBsAg (hepatitis B surface antigen) levels with the use of Imdusiran, VTP-300, and low-dose nivolumab. The announcement came during the AASLD 2024 conference, where the companies unveiled new data from their IM-PROVE II trial. These findings offer hope for patients suffering from chronic hepatitis B, a potentially life-threatening condition affecting millions worldwide. By leveraging cutting-edge treatments such as Imdusiran, VTP-300, and low-dose nivolumab, healthcare professionals can work towards mitigating the severity of chronic hepatitis B and improving patient outcomes. This breakthrough brings optimism to the medical community and sparks anticipation for further advancements in cHBV treatment. 🌟💪 Don't let Fear of Missing Out hold you back! Act now to stay ahead of the curve in healthcare investing. Take advantage of Health Savings Accounts (HSAs) to invest in innovative companies like Arbutus Biopharma and Barinthus Biotherapeutics. By combining financial growth with contributions towards your health and well-being, you can secure a bright future for yourself and your loved ones. 💰📈 #hsa #investing #healthcare #health #family #wellness

📢 New Live Webinar Alert! 🔗 https://bit.ly/3XDvnVa ⏰ 2:00 PM Eastern Daylight Time 🗓️ Thursday, October 3rd, 2024 🎓 One (1) ARRT Category A CE Credit & One (1) AHRA CRA Credit While gadolinium-based contrast agents (GBCAs) are essential to performing MR imaging, guidance from the US Food and Drug Administration (FDA) and the American College of Radiology (ACR) includes the recommendation of limiting a patient's gadolinium exposure. One technique for reducing gadolinium exposure is to use a high- relaxivity gadolinium-based contrast agent (GBCA) that can be administered at a lower dose than a conventional GBCA without deteriorating image quality. Gadopiclenol recently approved by the FDA is a high-relaxivity GBCA, designed to reveal high-quality images at half the conventional dose of other GBCAs. This educational program provides the clinical rationale for adopting the use of Gadopiclenol for pediatric MRI. Through case study reviews, Azam Eghbal, MD, will share clinical insights to achieve optimal image quality using a lower administered gadolinium dose when performing pediatric MRI. A focus on the selection and use of a high relaxivity, lower dose GBCA in vulnerable patients, receiving multiple MR contrast injections, will be supported by clinical evidence and imaging protocols. Audience participation will be encouraged during the Q & A session following the presentation.