A New Approach to Rare Disease Clinical Trials
Originally published on Triple Pundit Research studies that test potential treatments for rare diseases have long faced excessive costs, prolonged timelines and a staggering failure rate. One frequently cited 2019 analysis found that over half of randomized controlled trials studying rare diseases — defined as conditions affecting fewer than six patients per 10,000 people — are ultimately […]
Why 55% of Clinical Trials Fail – The Truth About Drug Development
Have you ever wondered why so many clinical trials fail before reaching the market? In this episode, we dive deep into the hidden challenges of clinical trials with Rob Freishtat, President and Co-Founder of Uncommon Cures and a professor at George Washington University School of Medicine and Health Sciences. Rob shares insider knowledge on why […]
Rob Freishtat- Revolutionizing Clinical Trials in Rare Disease: Efficiency and Patient-Centric Solutions
In this episode, Leanne Woelke chats with Rob Freishtat, Co-Founder and President of Uncommon Cures and former emergency physician. Rob shares his journey into the world of rare disease clinical research and his mission to transform the clinical trial landscape. They discuss the unique challenges of diagnosing and treating rare diseases and the critical role […]
Rare Disease Research in Latin America: A Landscape of Promise and Potential By: Juan Francisco Cabello, MD and Marshall Summar, MD
Latin America offers a dynamic and promising environment for rare disease research, characterized by growing momentum within the medical community to meet the unique needs of those affected. Collaboration across countries and sectors is unlocking new opportunities for innovation and progress. The region’s rich diversity—encompassing social, cultural, epidemiological, and economic contexts—presents a unique foundation for […]
Feels Like Home: Designing Sites For Patient Comfort
Originally published on Clinical Leader By Rob Freishtat, MD, MPH, president and co-founder, Uncommon Cures, and Matthew Stares, senior vice president, global real estate, architecture and development, MillerKnoll More than half (55%) of clinical trial failures are a result of enrollment issues, according to GlobalData. Those issues are myriad, but, commonly, many patients feel overwhelmed by […]
Uncommon Cures and Carexso Partner to Expand Orphan Drug Clinical Trials in the Middle East
Originally published on EIN Presswire CHEVY CHASE, MD, UNITED STATES, December 17, 2024 /EINPresswire.com/ — Chevy Chase, MD, 17 December 2024 – Uncommon Cures, an organization at the forefront of accelerating the development of orphan drugs, has entered into a strategic collaboration with Carexso, a leading Site Management organization based in the United Arab Emirates […]
Parent and Patient-Driven Discovery and Research in Rare Diseases
By Simon Frost and Marshall Summar Rare diseases, defined as conditions affecting fewer than 200,000 individuals in the United States, present significant challenges to the healthcare system. With limited financial incentives for pharmaceutical companies and sparse knowledge about these conditions, families and patients have by necessity often taken the lead in driving discovery and advancing […]
The Ethics of Rare Disease Clinical Trials: Balancing Innovation, Urgency, and Responsibility
Introduction Rare disease clinical trials are a vital intersection of scientific discovery and ethical responsibility. These trials provide hope for patients with life-altering conditions but also present unique ethical challenges. With small patient populations, urgent unmet needs, and, experimental treatments researchers and sponsors must carefully balance the needs of patients with rigorous standards of safety, […]
Uncommon Cures and TASK Partner to Advance Clinical Trials for Orphan Drugs Across Europe and South Africa
Chevy Chase, MD, 2 December 2024 – Uncommon Cures®, a pioneering organization dedicated to accelerating the development of orphan drugs, has announced a strategic collaboration with TASK, a leading global clinical research organization specializing in various therapeutic areas. This collaboration will combine Uncommon Cures’ innovative approaches to orphan drug development with TASK’s extensive clinical trial […]
An Opportunity for FDA and Rare Disease
Rare disease research and drug development are at a transformative crossroads, with the FDA uniquely positioned to lead the charge in accelerating innovation. By embracing legislative support, fostering collaboration with the NIH, and adopting flexible regulatory models, the FDA can redefine the future of clinical trials for rare diseases. This shift will not only address […]