Cell and gene therapy trials - delivering differently

Regulatory journey

ICON offers CGT regulatory consultancy from IND gap analysis and preparation, CTA pre-submission meetings (including GMO’s), EU Joint HTA-EMA scientific advice to BLA preparation and filing. To date, ICON has taken three oncology GGT programs from early phase to successful BLA filing and have facilitated Orphan Drug designation for 60 therapies. Regulatory requirements vary from country to country, and in many countries there are specific regional requirements. In CGT, ICON has proven experience in successfully navigating the complex regulatory environments such as Cartagena in Japan. Additionally, ICON has extensive experience in supporting accelerated access designations such as PRIME in EMA, RMAT, Fast Track and Breakthrough in the US and Sakigake in Japan.

An early pioneer in CAR-T trials, ICON realised these trials had to be delivered differently and formed a Cell and Gene Therapy Solutions Group to help clients navigate the unique complexities and mitigate risks. With over 400 dedicated CGT professionals globally, we have a formal structure that includes CGT principals, medical monitors, global logistics consultancy and dedicated CRAs to support all trials using the proprietary tools and best practices based on our extensive experience and innovation.

Patient journey

Cell and gene therapy trials require additional resources for patients and their caregivers and for the sites supporting them. Unlike traditional trials, CGT programs can experience multiple starts and stops resulting in patient rescheduling. Concierge services for lodging and transportation must be closely coordinated. In multi-dose therapies, patients who are discharged may require home health services that provide additional resources for sites to maintain a close connection and to manage side effects such as later developing neuro-tox or B cell aplasia. ICON’s home health group has extensive experience in managing patient hub services for CGT, CNS, rare and orphan programs. In a recent poll of patients and caretakers, 75% indicated that access to home health support was “very important” in their decision to enroll in the trial.

Site journey

As CGT trials expand, the demand for experienced sites has created a bottleneck of resources due to limited and overloaded CGT-experienced centers. Accelerated study start-up is the norm in CGT and site recruitment is critical path. To meet these timelines, ICON has developed an extensive repository of detailed, global site intelligence allowing the identification and recruitment of sites outside of the typically targeted candidates with CGT experience and resources. We’ve learned many lessons through the years that allow us to quickly identify red flags. We maintain data on CGT-specific resources and capabilities such as which sites are already FACT/JACIE accredited and can use that intel to our sponsors’ advantage.

In study startup and execution, ICON uses proprietary best practices and tools to relieve the burden on sites that are often running multiple programs for multiple sponsors. An example is our data tracker. Sites are often overwhelmed and unprepared for the sheer volume of data in CGT trials. Data accumulation in CGT is radically different than traditional curves. Our tracker helps sites predict data volumes for better resource management. To further alleviate site burden, ICON’s Firecrest™ portal serves as a centralised communication platform giving sites access to protocolspecific training, online visit by visit guide and centralised documentation.

Data management

Critical to study timelines is proper site preparedness for how data are captured in CGT trials vs. “typical” trials. Unlike traditional trials where data accumulates over the course of the study, CGT studies are characterised by a high data burden during the first three months, requiring the development of unique tools to support informed decision making.

ICON has created proprietary tools, methods and dashboards enabling management and monitoring teams to not only predict and visualise the data volumes based on the enrolment and dosing, but to readily respond to it operationally, including assigning additional support at sites unable to cope with the site data backlog. Data readiness workflow templates are also used to visually describe the study team responsibilities and to provide a detailed overview of the timelines and activities to support a rolling lock process and early site closure process on the project.

Product journey

CGT trials represent the most complex logistics and ecosystem coordination in the history of medicine. ICON’s logistics strategies are designed to support patient and product safety through precise management of these “living therapies.” Whether the program is autologous or allogeneic, Phase I through commercialisation, all programs require realtime management and monitoring of chain of identity, chain of custody and chain of condition. ICON routinely works with best in class service providers to mitigate risks and support patient safety by controlling and monitoring the product journey – from initial cell collection through the manufacturing process and eventual administration to the patients.

Supporting transparency & accountability

ICON supports sponsors from large pharma to emerging biotech. Our flexible and agile approach allows us to become an extension of sponsors and sites, supporting programs from early phase to commercialisation. We have developed a repository of special tools to assist sites and program managers in tracking key performance indicators to drive accountability on all levels.