Cell and gene therapy (CGT) represents a new frontier in the fight against many devastating diseases, including rare genetic disorders and certain cancers. It also poses unique challenges both for the investigators and companies pioneering this potentially transformative research and for the patients and families participating in it.
Complex study designs, challenging patient populations and distinct operational delivery requirements are the norm in CGT clinical trials. New treatment approaches are often unfamiliar to investigators, site staff and patients alike. CGT trials also present a multitude of complex logistical considerations, not the least of which is the long-term follow-up required to detect, record and adequately manage signals of early or delayed adverse reactions and to support pricing and reimbursement – a period of time that often coincides with changes in patients’ lives that can create significant challenges to patient retention.
The Syneos Health Cell and Gene Therapy Consortium
The Syneos Health Cell and Gene Therapy Consortium, built upon nearly two decades of experience in the field, is the hub for our knowledge and experience base in CGT preclinical/clinical development and commercialization. It brings together the cross-functional capabilities needed to navigate the challenges unique to CGT clinical trials, accelerate their development and deliver on their promise. The Consortium is where you will find staff with deep CGT and therapeutic area expertise, including clinical trial delivery teams and subject matter experts in regulatory, consulting, commercial, early phase and real-world/late phase R&D. The Consortium model ensures that our collective knowledge and insights are fully leveraged to deliver solutions specific to the needs of each trial.
Cell Therapy Trials
Gene Therapy Trials
With deep experience spanning more than two decades, including Phases I-IV and more than 25 trials at more than 500 sites since 2007, Syneos Health has delivered cell therapy trials in:
Since 2007, Syneos Health has conducted more than 20 gene therapy trials at more than 200 sites in therapeutic areas including:
Our Strategies for Efficient CGT Trial Execution
Forging New Experience
For CGT trials where therapeutic interventions and study protocols are often new to even the most experienced investigators and research sites, we provide enhanced levels of early site engagement (identification and feasibility assessment) and site start-up for novel therapies. This can include coordination as needed among multiple institutional units such as apheresis units and cell processing labs. CGT trials also often require specific clinical instruction related to the intervention (e.g., infusions, cell collection, etc.). In addition, we conduct focused patient outreach and education, working closely with patient communities and advocacy groups as well as academic medical centers and thought leaders to ensure the study will capture the most relevant data, identify enrollable patients, communicate the unique aspects of the study, and facilitate robust informed consent processes.
Delivering Operational Excellence
Building on insights gained over our years of hands-on CGT trial experience and leveraging the Syneos Health Trusted Process for delivery, our Consortium experts understand the operational complexities and challenges unique to CGT trials, including local IRB/IBC requirements, logistics management, cell processing and training needs. Our dedicated logistics and vendor management resources maintain tight coordination of additional functional units at sites and vendors.
Optimizing Patient Retention
The long-term follow-up required for CGT clinical trials adds significant complexity to the always-challenging issue of patient retention. We collect and analyze insights from patients and their families, disease communities and advocates to assess the burden of long-term follow-up trials from a patient journey perspective – that is, by understanding the real-world life experiences of patients over the course of the follow-up period, and often that of their families as well. We then plan for those real-world scenarios from the start, building in retention strategies that are adaptive and changeable over time.
Navigating Regulatory Complexity
The rapidly evolving regulatory landscape and new approval pathways and drug designations for CGTs require close attention to country, state and site requirements, multiple review board and specific site licensing requirements. For example, the US and Europe have taken quite different stances on the biosafety component of certain CGTs involving genetically modified organisms (GMO) or the use of recombinant nucleic acids.
Our Consortium of regulatory experts know this terrain intimately and use that in-depth knowledge to help inform CGT study designs, strategic decision-making and operational study planning.
Supporting Pricing and Market Access
Game-changing therapies are elevating the dialogue around treatment costs and leading to unique, frequent pilot arrangements with payers that can be the subject of media coverage, investor speculation and patient concerns. Our Consortium brings together pricing and communications experts to help companies execute complex and innovative arrangements, contextualize them for non-payer audiences, and support the environment in which negotiations occur.