In Silico Trial Design in Development of Rare Disease Cell and Gene Therapies
Speakers: Abie Ekangaki, Ph.D.Vice President, Statistical Consulting Kenneth Ndugga-Kabuye, MD, FACMGVice President, Cell & Gene Therapy
Speakers: Abie Ekangaki, Ph.D.Vice President, Statistical Consulting Kenneth Ndugga-Kabuye, MD, FACMGVice President, Cell & Gene Therapy
Premier Research is built on a foundation of rare disease research. With decades of relevant experience in developing novel therapies, devices, and diagnostics, we make rare a common focus. Our aim is to provide Sponsors of novel rare disease therapies with proactive solutions to ensure trials stay on course and fast-track clinical outcomes to deliver...
Small patient populations. Limited data on disease. Patient enrollment and retention challenges. Narrow site pools. These are some of the most common hurdles sponsors face in rare disease drug development. The right CRO partner can help turn these challenges into solutions. When launching your next RFP, keep this checklist on hand to ensure you’re covering...
Small patient populations. Geographic dispersion. Competing studies. Sound familiar? If you’re a sponsor of a rare disease study, you know all too well the challenges that come with recruiting patients. These challenges lead to immense pressure to design and implement studies that limit the burden of participation, while keeping patients engaged and generating high-quality data...
As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders, sponsors need to be proactive in demonstrating their commitment to addressing unmet therapeutic needs. Beyond...
April 20, 202311:00 a.m. EDT There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease. The majority of CGT approvals have been based on small, open-label, non-randomized, single-arm studies which either evaluate treatment effectiveness without a comparator or utilize a historical control comparator to evaluate treatment efficacy. Among...
MORRISVILLE, N.C., MILAN, ITALY, TRIESTE, ITALY — Premier Research, whose mission is to help the most innovative biotech and device companies take their best ideas from concept to commercialization, and InSilicoTrials, a leader in the use of artificial intelligence (AI) and computational modeling and simulation (CM&S) to accelerate development of new therapies and medical devices, have...
Individually, rare diseases by definition have a low prevalence, but collectively, the societal burden and impact of these uncommon conditions is significant. The majority of rare diseases are genetic in origin, and advances in genomic sequencing tools and technologies have driven an increase in the identification of rare disorders. Currently, one out of every 10...
In 2020, the European Joint Programme for Rare Diseases developed a guide on patient partnerships in rare disease research projects. This guide still rings true today, and we summarize in this blog the key points for sponsors of rare disease studies to consider when engaging patients and their representatives in study planning. If you’re developing...
It comes as no surprise that putting patients first is imperative for achieving success in rare disease development programs. However, to what extent can sponsors integrate patient input into their study design while still securing the high-quality data they need? Although a bit of a balancing act, it is possible to design a study that...