A disease is generally considered to be rare if it affects one patient per 200,000 people (1) and most rare diseases affect far fewer than this. However, collectively rare diseases are relatively common, affecting 350 million patients worldwide (2). The path to diagnosis for these patients is often a long, difficult battle and even once the diagnosis is made, it is likely there will be no suitable treatment available. For 90% of rare diseases, there is no approved therapy (2). There is, therefore, a pressing need to develop new, effective therapies that can bring hope to rare disease patients. However, the clinical development environment for life-threatening, rare diseases is fraught with challenges. By their very nature, rare indications have few patients and limited sample size. This scarcity of patients also results in a lack of available information and knowledge about the disease from the best endpoints, to the treatment effect size or the variability of response between subgroups.
Earlier this year, the FDA also released guidance to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs.
At the 2019 Challenges in Rare Diseases Clinical Trials Symposium and East training, Cytel is partnering with Alexion to bring together expertise from academia and industry to help tackle this important issue. This meeting aims to move the rare disease development agenda forward and help promote new, robust strategies to address these complex challenges. Over the years, Cytel has been offering a unique platform through these annual meetings that bring together industry experts, thought leaders and applied statisticians. The discussions revolve around innovations in clinical trials and guide the development of the industry's leading clinical trial design software, East. This year, the symposium will be held in Boston on November 6 & 7.
Don’t miss the opportunity to learn from leading trial design and implementation innovators including keynote speaker, Pfizer’s Kannan Natarajan who will share his experiences on the use of historical control information, natural disease history records, and Real World Data (RWD) to enrich the design and analysis of clinical trials in rare disease. Our other noted speakers will address various topics around the central theme. The speaker panel includes Alex Dmitrienko, Amy Pace, Clare Elkins, Pantelis Vlachos, Jianchang Lin and David Kerr. Click here to learn more about the speakers and access the presentation abstracts.
The EUGM offers a unique perspective on clinical trials by combining methodological and practical scientific talks with the ability to gain hands-on training with East software.
Day 1: Wednesday, November 06, 2019
The sessions will open with a keynote talk from Kannan Natarajan "Drug Development in Rare Diseases - Need for Innovation in Statistical Thinking", followed by a range of presentations covering hot topics like: "Historical Control Borrowing: Overview, Advancement and New Methodologies", The PREVENT Trial for NMOSD and "Clinical Trial Optimization with Applications to Rare Disease Trials".
Day 2: Thursday, November 07, 2019
In the hands-on East training day participants will benefit from practical training in Cytel's East software.
2) Kym M Boycott, Lilian PL Lau, Christine M Cutillo, and Christopher P Austin. International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases.