Clinical research organisation (CRO) Ergomed has launched a Rare Disease Innovation Center to support biotech and pharma companies with their drug development plans.
Rare diseases are thought to affect around 300 million people across the world. Conducting clinical trials in rare disease areas can be especially difficult due to the rarity of patients, alongside issues such as a limited pool of experienced investigators, heterogeneity of indication, lack of established endpoints, and poorly understood natural history.
Ergomed’s Rare Disease Innovation Center has been designed to provide solutions to address the challenges faced by rare disease sponsors to help them reduce clinical trial timelines, optimise patient experience, and bring more treatments to market.
The center features a consortium of industry partners and subject matter experts dedicated to developing tailored solutions for rare disease focused companies and patients. These include partners such as Trialbee, mdgroup, THREAD, Clinone, Medidata, a Dassault Systèmes company, Rare Patient Voice, Patient Wing and others.
“Rare disease drug development is the fastest growing area in drug development with a 12-14% annual growth rate, accounting for about one third of the drugs in development. We’re excited about the opportunity to launch our Rare Disease Innovation Center to leverage Ergomed’s experience from doing hundreds of rare disease studies in conjunction with innovative approaches from our industry partners to help our sponsors overcome challenges in rare disease drug development” said Zizi Uzezi Imatorbhebhe, senior vice president, Global Strategy and Development, Head Rare Disease Innovation Center, Ergomed.
Specifically, the center will aim to support companies in designing more and performance analytics to predict key milestones and scenario analyses. The center will also aim to utilise real world evidence for synthetic control arms, patient selection, biomarkers, end point selection, patient registries and other areas. Companies will also be assisted in selecting the right innovations, such as decentralisation and other technologies, to identify and retain rare disease patients.
LaQuinta Jernigan, executive vice president, North American at mdgroup, partner to the Rare Disease Innovation Center, said: “Rare diseases represent an area of significant unmet medical need – these more than 7,000 conditions have a huge impact on people’s lives, yet treatment options are often limited. And while the advent of cutting-edge therapeutics, such as gene therapy, presents exciting new opportunities, drug development in this space is challenging. Recruitment can be difficult, and retention can be tough.
“At mdgroup, we wrap our arms around participants by combining a personalised approach to clinical trial management, patient care, and logistics with cutting-edge technologies and data systems. That means going above and beyond to support people in the best way for them – whether that’s facilitating participation within their own homes, supporting them to travel safely to and from study sites, or developing a hybrid model. It all adds up to increased engagement and fewer dropouts.”