Uncommon Cures?

Happy Holidays from your friends at Uncommon Cures!

Enjoyed to chance to sit down with Pam Gavin (CEO at National Organization for Rare Disorders) and the Washington Post to talk about current topics in Rare Disease.

I recently joined Harsh Thakkar on the Life Sciences 360 podcast to share my insights on the hidden challenges facing clinical trials today, especially for orphan drugs.?In my two decades in academic medicine and at Uncommon Cures?,??I've witnessed firsthand the urgent need for bold leadership and groundbreaking innovation to push the boundaries of what's possible?in clinical trials. By introducing fresh perspectives, building diverse and talented teams, and relentlessly focusing on patient needs, I believe we can revolutionize the way clinical trials are conducted and ensure that every patient has access to the life-saving treatments they deserve. ? In this thought-provoking episode, we tackle critical issues such as: -The startling reality that over 55% of clinical trials fail?for reasons having nothing to do with the drug -How misaligned priorities among researchers, regulators, and sponsors can hinder progress -Innovative strategies to streamline clinical trials, enhance patient recruitment, and leverage cutting-edge technologies like AI and real-world data Listen to our conversation and join me in the mission to advance?clinical trial?solutions on a global scale. Check out the full episode at?https://lnkd.in/e8H4SY96.

I recently joined Harsh Thakkar on the Life Sciences 360 podcast to share my insights on the hidden challenges facing clinical trials today, especially for orphan drugs.?In my two decades in academic medicine and at Uncommon Cures?,??I've witnessed firsthand the urgent need for bold leadership and groundbreaking innovation to push the boundaries of what's possible?in clinical trials. By introducing fresh perspectives, building diverse and talented teams, and relentlessly focusing on patient needs, I believe we can revolutionize the way clinical trials are conducted and ensure that every patient has access to the life-saving treatments they deserve. ? In this thought-provoking episode, we tackle critical issues such as: -The startling reality that over 55% of clinical trials fail?for reasons having nothing to do with the drug -How misaligned priorities among researchers, regulators, and sponsors can hinder progress -Innovative strategies to streamline clinical trials, enhance patient recruitment, and leverage cutting-edge technologies like AI and real-world data Listen to our conversation and join me in the mission to advance?clinical trial?solutions on a global scale. Check out the full episode at?https://lnkd.in/e8H4SY96.

Had some requests to share the links to our Rare Disease Topic Posts from 2024. Here they are, please provide input and suggestions for topics you'd like to see in 2025! Best, The Team at Uncommon Cures Rare Disease in Latin America (with Juan Francisco Cabello):?https://lnkd.in/eQza-uAX Parent and Patient Driven Rare Disease Discovery and Research (with Simon and Nina Frost): https://lnkd.in/ebSjwrPj Ethics in Rare Disease Clinical Trials (whole team at UCC): https://lnkd.in/ezBXHptv Opportunities for FDA in a Changing Environment: https://lnkd.in/ey-fqBQi Why now is a good time to invest in Rare Disease: https://lnkd.in/e-DZjcui Clinical Trials Strategy Post 3 of 3,?Refining Clinical Trials Sites:?https://lnkd.in/eKPUJ7pd Clinical Trials Strategy Post 2 of 3, Innovating Drug Development : https://lnkd.in/eucjSxpQ Clinical Trials Strategy Post 1 of 3, Anthropophobia (Fear of Being told No) and the FDA:?https://lnkd.in/ervgGkra

Enrollment issues contribute to around 55% of clinical trial failures, highlighting a significant challenge in the industry. While complete control over all variables is impossible, there is a clear opportunity for improvement by reimagining the environments where these trials occur. Collaborating with Matthew Stares RIBA AIA FRSA, we embraced an approach centered on the "science of hope" to redesign clinical spaces. Our vision transcends conventional sterile settings, focusing instead on creating welcoming environments that meet regulatory standards while providing a sense of comfort and familiarity. Our innovative design features include: - Central communal areas with a kitchen and living spaces - Specialized zones for pediatric patients - Quiet spaces for neurodivergent individuals - Carefully selected materials ensuring both compliance and comfort Our work challenges prevailing industry beliefs by demonstrating that regulatory requirements offer ample scope for creative design solutions that prioritize patient well-being alongside clinical needs. I am confident in the positive impact of our approach, not only enhancing the experience for patients and families but also potentially reducing the failure rate of clinical trials by encouraging participation. Gratitude to Clinical Leader for showcasing our collaborative article, "Feels Like Home: Designing Sites For Patient Comfort," authored by Matthew and myself. We aspire to inspire others in setting higher standards for clinical trial environments. Explore the full article to delve into how our innovative designs are reshaping the landscape of clinical trial settings.

This is our last piece for the year 2024. Written in collaboration with Dr. Juan Francisco Cabello it focuses on the opportunities for rare disease research in Latin America.

Enrollment issues contribute to around 55% of clinical trial failures, highlighting a significant challenge in the industry. While complete control over all variables is impossible, there is a clear opportunity for improvement by reimagining the environments where these trials occur. Collaborating with Matthew Stares RIBA AIA FRSA, we embraced an approach centered on the "science of hope" to redesign clinical spaces. Our vision transcends conventional sterile settings, focusing instead on creating welcoming environments that meet regulatory standards while providing a sense of comfort and familiarity. Our innovative design features include: - Central communal areas with a kitchen and living spaces - Specialized zones for pediatric patients - Quiet spaces for neurodivergent individuals - Carefully selected materials ensuring both compliance and comfort Our work challenges prevailing industry beliefs by demonstrating that regulatory requirements offer ample scope for creative design solutions that prioritize patient well-being alongside clinical needs. I am confident in the positive impact of our approach, not only enhancing the experience for patients and families but also potentially reducing the failure rate of clinical trials by encouraging participation. Gratitude to Clinical Leader for showcasing our collaborative article, "Feels Like Home: Designing Sites For Patient Comfort," authored by Matthew and myself. We aspire to inspire others in setting higher standards for clinical trial environments. Explore the full article to delve into how our innovative designs are reshaping the landscape of clinical trial settings.

Breaking New Ground in Rare Disease Research: Carexso Partners with Uncommon Cures and PDC FZ LLC ? We are excited to announce our strategic partnership with?Uncommon Cures, a global leader in orphan drug development, and?PDC FZ LLC, a top-tier Contract Research Organization. This collaboration is set to revolutionize clinical trials for orphan drugs across the Middle East, providing new hope for patients with rare diseases who have limited treatment options. ? By combining Uncommon Cures’ expertise in drug development with Carexso’s and PDC’s established clinical and regulatory infrastructure, we aim to: ? ??Accelerate Clinical Trials?– Streamline processes to expedite life-saving therapies. ??Expand Patient Access?– Open pathways for underserved patient populations. ??Advance Medical Innovation?– Foster faster regulatory approvals in the region. ???Read the full press release here: https://lnkd.in/ebVvGVtY ? #ClinicalTrials #OrphanDrugs #RareDiseases #HealthcareInnovation #CarexsoPartnership #PatientFirst