Cell & Gene Therapy (CAGT) Consulting and Services
ProPharma's Cell and Gene Therapy expert consulting team provides a wide range of consulting and services to help clients navigate the complex and ever-changing landscape of cell and gene therapy development. Also known as Advanced Therapy Medicinal Products (ATMP), our cell and gene therapy team includes scientists and engineers with hands-on experience in the development of CAGT, ex-regulators from several Agencies, and senior regulatory consultants with global experience across multiple therapeutic areas.
We have experience and capability to support the development of cell and gene therapies including, among others, CAR-T cells (autologous or allogenic), TCR-T cells, tumor-infiltrating lymphocytes (TILs), γδ T cells, stem cells, therapeutic vaccines, AAVs, adenovirus, or the most innovative gene editing products. We have worked on multiple therapeutic areas, with outstanding expertise in oncology, hematology, and rare diseases.
Our services include regulatory support, CMC development, manufacturing technology and quality and compliance, non-clinical and clinical development, market access and reimbursement, financial advice, and safety services. These services are oriented to both developers and manufacturers. By partnering with us, clients can be confident that they have access to the knowledge and expertise they need to successfully bring their innovative therapies to market. Read on to learn more about our services and how we can help you achieve success in cell and gene therapy development.
Regulatory Support
Our Cell and Gene Therapy expertise includes a multidisciplinary scientific team of regulatory consultants, with strong nonclinical, CMC, and clinical backgrounds and extensive experience in the development of CAGT products. The team can guide you to customize and accelerate your development plan with consideration of the state of the art for similar products, either approved or under development. Our experts can facilitate any type of interactions with global regulatory agencies, including INTERACT and ITF meetings, PRIME and Breakthrough Therapy designation, and pre-IND or general Scientific Advice meetings.
CMC Development
Our experts can support CMC development covering multiple aspects as required by developers (both industry and academia) or manufacturers (CDMOs or hospitals). Assuring high-quality of the starting materials, identifying critical quality attributes, developing and validating methods, assessing comparability among different manufacturing batches, or choosing the proper release specification margins are only some of the many aspects where our experts can help.
Manufacturing Technology and Quality Compliance
ProPharma supports the design and qualification of new or expanded manufacturing sites and ensures that the site is licensed to legally manufacture products to treat patients. Our process modeling team has developed validated methodologies to achieve efficiency, effectiveness, and scalability following the relevant GMP and ISO standards. We have worked with multiple hospitals and research institutions to develop qualified spaces for effective CAGT manufacturing, which included collaborating with all the major equipment suppliers.
From early concept development through each clinical phase, product launch, and commercialization, our Quality and Compliance Consultants partner with clients around the world to ensure GMP and quality compliance requirements are met, and patient health and safety is assured and improved.
In addition, we have thorough expertise in auditing GMP facilities for the manufacture of different CAGT products using our global team of GxP Auditors, including former regulatory agency inspectors and qualified auditors who are proficient in conducting mock inspections, internal audits, vendor and supplier auditing, and gap assessments.
Nonclinical Development
Our cell and gene therapy consultants support the nonclinical development of multiple types of viral vectors and cell-based therapies. We ensure that a robust nonclinical data package exists to de-risk clinical development and support approval. We have hands-on experience in the design of proof-of-concept studies as well as toxicology packages, including biodistribution and shedding analyses. Our non-clinical experts further participate in the interactions with the regulators by reviewing briefing books for scientific advice and attending meetings with the Regulatory Agencies.
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Clinical Development
At ProPharma's Cell and Gene Therapy Center of Excellence, we can design and support the most innovative designs for successful clinical development in bespoke populations, including natural history studies, synthetic control arms, virtual models, or long-term follow-up studies. We have extensive experience in a broad range of therapeutic areas, mainly oncology, and multiple rare diseases. ProPharma strongly believes in the power of Real-World Data (RWD) to support the lifecycle of CAGT, and our team is ready and capable to collect, analyze and discuss these data. Moreover, we have digital solutions to enable decentralized models of clinical trials.
Reimbursement
Demonstrating clinical and cost-effectiveness for CAGT products, as required for reimbursement, is very challenging. High short-term costs combined with limited data on efficacy and effectiveness create uncertain cost-effectiveness and, consequently, heterogeneity in the requirements by the payers. ProPharma's Cell and Gene Therapy Center of Excellence has the capabilities to support market access within Europe and the United States and assure success in reimbursement strategies. Moreover, our team is a unique partner to support early engagement with Health Technology Assessment (HTA) bodies, since our clinical regulatory experts and market access specialists closely collaborate to help you streamline the development and launch of new therapies.
Financial Aspects
Clients while in the early development phase of their CAGT product often ask us to help them create a financial plan which they can bring to investors to show when funding is required, how much funding is needed, and what the benefit of the funding is. Specific questions will include when to invest in infrastructure versus investing in analytics, or other key packages. Our Cell and Gene Therapy Center of Excellence works with you to ensure funding requirements are understood, utilized efficiently, and results-oriented.
Pharmacovigilance and Medical Information
The Cell and Gene Therapy Center of Excellence includes experts in pharmacovigilance to ensure that adverse events are handled correctly both during development and post-approval. Also, our team of Medical Information further ensures that patients and service providers have the correct information to ensure positive outcomes and a central contact with multilingual capabilities to expand globally.
Get Help from ProPharma's Cell and Gene Therapy Consultants and Experts
Partner with us to develop a comprehensive plan for your cell and gene therapy. Our team of experts is committed to helping you achieve success and bring your therapy to market. Contact us today to learn more.