Patient-centric approaches and cross-therapeutic teams
drive success in cell and gene therapy trials.
Despite incremental market growth, cell and gene therapy (CGT) clinical development continues to present a complex set of challenges, such as managing logistics, navigating the constantly evolving regulatory landscape, and the complexities of patient participation in cell and gene therapy trials for patients.
At Medpace, our multi-disciplinary teams take an active approach to surmounting complex clinical and regulatory barriers common to the development of CGT clinical studies. Maximize your CGT trial budget and expedite trial timelines by partnering with our global, integrated team to unlock efficiencies across medical, regulatory, and operational considerations and requirements.
Diverse Therapeutic Expertise
The Medpace cell and gene therapy team offers cross-functional therapeutic expertise to accelerate the development of cellular and gene therapies. Our extensive CGT experience spans rare disease, metabolic, infectious diseases (ID), CNS, cardiovascular, pediatrics, diabetes, and medical device. We have also managed therapies that have gained marketing authorization approval. This depth of experience highlights our ability to manage the most complex and innovative trials.
Medpace Cell and Gene experience over the past 5 years:
Making the Complex Seamless®
in Global Cell & Gene Clinical Development
Medpace is a well-established innovator in the clinical development of cellular and gene therapies, having contributed to over 130 clinical CGT trials across numerous therapeutic areas and global regions.
We provide Sponsors with comprehensive strategies that address the rapidly evolving clinical, operational, and regulatory challenges, as well as the unique set of hurdles presented in crossover therapies. Additionally, our patient-centric approach enables our team to keep patients engaged throughout the entirety of the study.
“Implementing a strategy of integrated, cross-functional activities, which leverages best practices early in clinical development, can enrich the likelihood of success in executing cell and gene therapy trials.”Jeff Vassallo, PhD
Senior Director, Clinical Trial Management
Cell and Gene CRO Capabilities
Medpace, a full-service clinical research organization (CRO), has conducted over 130 advanced therapy trials, and applies this experience to help Sponsors advance their CGT therapies. Leverage Medpace’s cross-functional cell and gene therapy clinical development services from preclinical to post-approval.
- Extensive experience across various products (cell, gene, tissue-based)
- Network of globally trained investigators with the capabilities and experience to store, prepare and administer these unique products
- Investigational product experience, such as first-in-human (FIH), allogeneic and autologous cellular products, ex vivo and in vivo gene transfer, and gene editing
- Active member and supporter of Rare Disease UK
Patient-Centricity in CGT Trials
Medpace recognizes the significant burdens involved in patient participation in CGT trials. We place patients at the center of a trial to increase awareness, reach, and enrollment while helping yield successful outcomes in terms of study design, patient education and engagement, and study results.
Patient education is one of the most critical contributions to a successful patient recruitment and retention strategy. Educating patients on cell and/or gene therapy trials (potential benefits or risks, side effects, and adverse events) can help ease any hesitation or discomfort a patient may have about participating in a trial. Our dedicated clinical trial management teams follow a multifactorial approach towards awareness, which includes developing educational materials to ensure patients are informed throughout the study.
New Article – Lessons Learned in Emerging Cell and Gene Therapies
Making the Leap from Lab to Clinic?
Integrated Laboratory Services
Medpace offers a comprehensive array of cell and gene lab capabilities through its wholly-owned, fully-integrated business units to meet the constantly evolving demands of complex clinical studies. This provides cohesive, streamlined, and standardized trial management.
- Medpace Core Laboratories combines state of the art imaging services with Medpace clinical teams to provide efficiency through partnership. Our imaging and clinical trial experience ensures that imaging components are seamlessly integrated into the complex structure of CGT trials.
- Central Laboratories provides customized, quality central laboratory services and in-house expertise including genomic and molecular testing, flow cytometry, anatomical pathology, and immunoassays. Additionally, Central Labs provides specialized product storage and delivery capabilities often necessary for cellular and gene therapy trials.
Comprehensive Regulatory & Operational Expertise
Advanced therapy clinical studies are often delayed due to lack of therapeutic experts. Medpace has a team of therapeutically-aligned operations, regulatory and technical experts with relevant experience in cell and gene therapies, who work collaboratively with our specialized medical team in order to leverage expertise for the benefit of trial programs. The Medpace Technical Advisor team provides in-depth regulatory support regarding country and regional guidelines, develops environmental risk assessments and other technical documents required for submissions and assist sites with regulatory activities such as obtaining site level certificates and accreditations.
Achieving Successful Cell Therapy Outcomes through Integrated Expertise
Cell and Gene Therapy Experts
Medpace’s team of Medical Directors, Advanced Clinical Practitioners, and Clinical Trial Managers have led advancements in the field across tens of cell and gene therapy studies in the U.S., Europe, and Asia Pacific. The deep experience has given Medpace a robust strategy for avoiding and mitigating the risks related to common pitfalls that are faced by its cell therapy customers.
Success Stories in Cellular and Gene Therapy Research
Our strong relationships with investigators, key sites, regulators, institutions, specialized laboratories, and KOLs in Cell and Gene Therapy help facilitate strategic site selection, start-up, and recruitment efforts. The experience and expertise combined with long-standing relationships will readily translate into the successful and timely recruitment, conduct, and delivery of a study.
Cell Tracking & Logistical Support
With a history of over 130 advanced therapy trials, Medpace has developed a proven approach to manage complex cell trials, including cell tracking. Through our customizable process, we provide Sponsors with seamless logistics and cell tracking to ensure the Chain of Custody and Identity is maintained.
- Through our customizable process, we provide Sponsors with expertise on the logistics process, a study-specific portal, site-facing guidance, and rigorous site training to ensure that patients get the right product at the right time and under the appropriate storage conditions
- An integrated and intuitive cell therapy manual is developed for each trial; the manual is reviewed by medical monitors and advanced clinical practitioners who have advanced training and experience in cellular therapy to ensure it is conducive with institutional standards
Related Cell and Gene Therapy Insights
Successful Approaches from Rare Disease &
Cellular and Gene Therapy Product Approvals
Understanding and proactively addressing these common challenges and considerations ensures that a trial is best
positioned for success and will support the future goal of marketing approval.