ICON understands the difference from first-hand experience, and has developed proven solutions to address all areas of cell and gene therapy development – from haematological and solid malignancies to rare, orphan and chronic diseases with increasingly sophisticated and diverse cell constructs.
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.
Cell and gene therapies insights
ICON's Cell and Gene Therapies team contributes regularly to industry publications and media coverage of cell and gene therapy clinical trials. Stay up to date with the latest trends in this therapeutic area through ICON's insights.
In this section
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Digital Disruption
- AI and clinical trials
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Clinical trial data anonymisation and data sharing
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Clinical Trial Tokenisation
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Closing the evidence gap: The value of digital health technologies in supporting drug reimbursement decisions
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Digital disruption in biopharma
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Disruptive Innovation
- Remote Patient Monitoring
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Personalising Digital Health
- Real World Data
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The triad of trust: Navigating real-world healthcare data integration
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Patient Centricity
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Agile Clinical Monitoring
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Capturing the voice of the patient in clinical trials
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Charting the Managed Access Program Landscape
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Developing Nurse-Centric Medical Communications
- Diversity and inclusion in clinical trials
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Exploring the patient perspective from different angles
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Patient safety and pharmacovigilance
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A guide to safety data migrations
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Taking safety reporting to the next level with automation
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Outsourced Pharmacovigilance Affiliate Solution
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The evolution of the Pharmacovigilance System Master File: Benefits, challenges, and opportunities
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Sponsor and CRO pharmacovigilance and safety alliances
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Understanding the Periodic Benefit-Risk Evaluation Report
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A guide to safety data migrations
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Patient voice survey
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Patient Voice Survey - Decentralised and Hybrid Trials
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Reimagining Patient-Centricity with the Internet of Medical Things (IoMT)
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Using longitudinal qualitative research to capture the patient voice
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Agile Clinical Monitoring
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Regulatory Intelligence
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An innovative approach to rare disease clinical development
- EU Clinical Trials Regulation
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Using innovative tools and lean writing processes to accelerate regulatory document writing
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Current overview of data sharing within clinical trial transparency
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Global Agency Meetings: A collaborative approach to drug development
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Keeping the end in mind: key considerations for creating plain language summaries
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Navigating orphan drug development from early phase to marketing authorisation
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Procedural and regulatory know-how for China biotechs in the EU
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RACE for Children Act
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Early engagement and regulatory considerations for biotech
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Regulatory Intelligence Newsletter
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Requirements & strategy considerations within clinical trial transparency
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Spotlight on regulatory reforms in China
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Demystifying EU CTR, MDR and IVDR
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Transfer of marketing authorisation
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An innovative approach to rare disease clinical development
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Therapeutics insights
- Endocrine and Metabolic Disorders
- Cardiovascular
- Cell and Gene Therapies
- Central Nervous System
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Glycomics
- Infectious Diseases
- NASH
- Oncology
- Paediatrics
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Respiratory
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Rare and orphan diseases
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Advanced therapies for rare diseases
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Cross-border enrollment of rare disease patients
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Crossing the finish line: Why effective participation support strategy is critical to trial efficiency and success in rare diseases
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Diversity, equity and inclusion in rare disease clinical trials
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Identify and mitigate risks to rare disease clinical programmes
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Leveraging historical data for use in rare disease trials
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Natural history studies to improve drug development in rare diseases
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Patient Centricity in Orphan Drug Development
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The key to remarkable rare disease registries
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Therapeutic spotlight: Precision medicine considerations in rare diseases
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Advanced therapies for rare diseases
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Transforming Trials
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Accelerating biotech innovation from discovery to commercialisation
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Ensuring the validity of clinical outcomes assessment (COA) data: The value of rater training
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Linguistic validation of Clinical Outcomes Assessments
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Optimising biotech funding
- Adaptive clinical trials
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Best practices to increase engagement with medical and scientific poster content
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Decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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Decentralised and Hybrid clinical trials
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Practical considerations in transitioning to hybrid or decentralised clinical trials
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Navigating the regulatory labyrinth of technology in decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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eCOA implementation
- Blended solutions insights
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Implications of COVID-19 on statistical design and analyses of clinical studies
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Improving pharma R&D efficiency
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Increasing Complexity and Declining ROI in Drug Development
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Innovation in Clinical Trial Methodologies
- Partnership insights
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Risk Based Quality Management
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Transforming the R&D Model to Sustain Growth
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Accelerating biotech innovation from discovery to commercialisation
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Value Based Healthcare
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Strategies for commercialising oncology treatments for young adults
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US payers and PROs
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Accelerated early clinical manufacturing
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Cardiovascular Medical Devices
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CMS Part D Price Negotiations: Is your drug on the list?
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COVID-19 navigating global market access
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Ensuring scientific rigor in external control arms
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Evidence Synthesis: A solution to sparse evidence, heterogeneous studies, and disconnected networks
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Global Outcomes Benchmarking
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Health technology assessment
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Perspectives from US payers
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ICER’s impact on payer decision making
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Making Sense of the Biosimilars Market
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Medical communications in early phase product development
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Navigating the Challenges and Opportunities of Value Based Healthcare
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Payer Reliance on ICER and Perceptions on Value Based Pricing
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Payers Perspectives on Digital Therapeutics
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Precision Medicine
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RWE Generation Cross Sectional Studies and Medical Chart Review
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Survey results: How to engage healthcare decision-makers
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The affordability hurdle for gene therapies
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The Role of ICER as an HTA Organisation
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Strategies for commercialising oncology treatments for young adults
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