Translating Cell Therapies: Academic versus Industry model
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This document discusses the differences between academic and industry models for cell therapy clinical trials. Academia focuses on early stage "first-in-human" pilot studies using autologous cell therapies for rare diseases, while industry aims for large-scale allogeneic therapies for prevalent diseases. Key differences include scale, funding, and regulatory pathways. There is a need for more academic Good Manufacturing Practice cell therapy facilities to help translate research into applications, though gaps exist in quality control and commercialization expertise. Increased collaboration and education can help bridge gaps between academia and industry.
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• Regulatory expectations for empty/full analysis
• Key technical requirements for running an AUC assay and how to interpret the data from the results generated
• How the AUC assay fits into the larger picture of AAV characterization
Orphanetics presentation
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Process Impurities: Don’t Let PEI or HCP Derail Your BioTherapy
View our webinar here: https://bit.ly/2lKNdWX
Many different impurities are present in or generated during biotherapy manufacturing. This webinar will address how process contaminates can arise from raw input materials, occur as residual processing agents, or form as reaction by-products. We will review strategies within product characterization to de-risk the manufacturing process, including the use of routine and high complexity assays; and the recommended testing to meet regulatory requirements for clinical submission. Learn methods to avoid costly pitfalls and implement procedures to expedite product quality decisions at critical junctures in your development plan. We will discuss two types of therapies:
Cell & Gene Therapies
Polyethylenimine (PEI) is a transfection agent used in nearly all cell and gene therapy products. We will review the regulations and the liquid chromatography with charged aerosol detection (LC-CAD) methodology to demonstrate PEI removal during the production process.
Monoclonal Antibodies (mAb) and Cell & Gene Therapies
During mAb manufacturing and inherent to Cell & Gene Therapies, a significant proportion of process impurities arise from the host cell used to express the drug. Host cell protein (HCP) impurities, present at PPM-levels, are a major immunogenicity risk because they can elicit an unpredictable immune response in patients. We will review why their complex and diverse nature makes them challenging to monitor, and theho best practices, specifically HCP identification by mass spectrometry, for detection.
Learning points:
1. Accurate detection and characterization of residual PEI in cell and gene therapy products
2. Effective detection and characterization of residual host cell proteins (HCP) in mAbs
3. Available technology and assays for quantifying process impurities
4. Current regulatory requirements for detecting, quantifying, and removing process impurities during biotherapy manufacturing
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Florence Bine-Scheck, Senior Medical Director at VCLS, shares her insights on the clinical development of Advanced Therapy Medicinal Products (ATMPs)
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The document discusses the EHR4CR project, which aims to develop a trustworthy service platform to access clinical information from electronic health records (EHRs) to improve clinical research. The project has developed a scalable platform used in pilot programs across 11 major hospitals in 5 countries. It is working to expand this platform into a pan-European network to support clinical trial feasibility studies, patient recruitment, and other research activities. Key stakeholders include hospitals, pharmaceutical companies, and the newly formed European Institute for Innovation through Health Data, which will help govern the platform. The goals are to increase clinical trial efficiency, reduce costs, and improve patient care through better use of EHR data for research.
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This 424-page report provides a comprehensive analysis of the US coagulation testing market, including major issues, trends, forecasts, and business opportunities. It reviews 40 coagulation procedures, placement estimates for automated systems, a comparison of 50 leading analyzers, and forecasts for reagent and instrument sales. The report also profiles major suppliers and provides strategic recommendations for players in the market.
DEC 3 2015 Mats Sundgren
1) The EHR4CR project developed a platform to facilitate clinical research by enabling access to de-identified patient health records across countries, systems, and sites.
2) The platform supports protocol feasibility testing, patient recruitment, and data capture/exchange through a network of hospitals in multiple European countries.
3) Going forward, the EHR4CR Champion Program aims to further validate and scale up the platform through partnerships with additional hospitals and research organizations, overseen by the newly formed European Institute for Innovation through Health Data.
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The document discusses the development of a surgical simulation training system called ReSuture. It notes that surgical complications can be reduced by 50% with simulation training. ReSuture aims to provide a realistic and cost-effective training platform for open vascular surgery using lifelike materials and a pumping system to simulate blood flow. The startup is seeking $250,000 in funding to validate its first product, expand its advisory board, and market the simulation system to medical schools and hospitals to improve surgical training outcomes.
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Presentation-French-clusters-Adebiotech-BMsystems-manuel-gea09052016-vonline
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A new intellectual product named Lean Enablers for Clinical Laboratories (LEfCL) is described. It is a collection of 136 best practices for clinical laboratory operations, containing collective wisdom on how to practice laboratory management, focusing on simultaneous improvements in test quality, productivity, laboratory capacity and stakeholder satisfaction, and reductions of test turnaround time and cost. The methodology follows an awarded method established in Systems Engineering and represents an extension of that engineering knowledge into clinical practice. The product has been developed by experienced directors from Kaiser Permanente regional clinical laboratory while pursuing a graduate Certificate in Lean Healthcare at Loyola Marymount University. LEfCL are organized into six well-known Lean Principles: Value, Value Stream Mapping, Flow, Pull, Perfection and Respect. The paper also identifies 46 Challenges that prevent success. Results of Lean improvements measured over the period of 2014-2016 are quoted for five laboratories. The LEfCL are not intended to become a mandatory practice. Instead, they should be used as a checklist of good practices. LEfCL are formulated as a comprehensive set of practices, intended for future training of new hires and as reference material. As such, they include both basic and advanced practices.
Lean Enablers for Clinical Laboratories
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2013 US Hospital Coagulation Testing Market
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This document provides a 973-page report on diagnostic testing markets outside main hospital laboratories, such as emergency rooms, operating rooms, and intensive care units. It includes analysis of market dynamics, size, growth trends, technologies, and business opportunities. The report segments the market and provides test volume and sales forecasts for over 300 procedures. It also reviews leading point-of-care diagnostic instruments and emerging technologies. Strategic recommendations are provided for new product opportunities and alternative market expansion strategies.
ReSuture Pitch Deck
This document discusses the development of a surgical simulation training system called ReSuture. It notes that surgical complications can be reduced by 50% with simulation training. ReSuture aims to provide a lifelike and cost-effective training platform for open vascular surgery using patent-pending lifelike materials and a pumping system to simulate blood flow. The startup is seeking $250k in funding to validate its first product, expand its advisory board, and market the simulation system to medical schools and hospitals to reduce errors and costs from avoidable complications in vascular surgery.
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The document discusses the concept of BBMRI Expert Centres, which are proposed as a new public-private partnership model to improve the use of biospecimens for research. The expert centres would provide specialized medical and scientific expertise, standardized analysis platforms, and quality management to facilitate data sharing between academia and industry. This model offers industry access to expertise and reduces sample shipping needs, while avoiding issues around commercialization of samples.
Are we using the correct quality goals?
Setting quality goals / specifications is a debatable issue since 1999. I am trying here to show the options and the continuos trials from several professional bodies to reach a consensus in this matter.
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Build Trust and Security:
Establish a safe and supportive environment where children feel comfortable expressing themselves.
Encourage Expression:
Enable children to articulate their thoughts, feelings, and experiences.
Promote Emotional Understanding:
Help children identify and understand their own emotions and the emotions of others.
Enhance Listening Skills:
Develop children’s ability to listen attentively and respond appropriately.
Foster Positive Relationships:
Strengthen the bond between children and caregivers, peers, and other adults.
Support Learning and Development:
Aid cognitive and language development through engaging and meaningful conversations.
Teach Social Skills:
Encourage polite, respectful, and empathetic interactions with others.
Resolve Conflicts:
Provide tools and guidance for children to handle disagreements constructively.
Encourage Independence:
Support children in making decisions and solving problems on their own.
Provide Reassurance and Comfort:
Offer comfort and understanding during times of distress or uncertainty.
Reinforce Positive Behavior:
Acknowledge and encourage positive actions and behaviors.
Guide and Educate:
Offer clear instructions and explanations to help children understand expectations and learn new concepts.
By focusing on these objectives, communication with children can be both effective and nurturing, supporting their overall growth and well-being.
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HIMMS TIGER resources are available to assist Health Informatics education.
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Translating Cell Therapies: Academic versus Industry model
- 1. Translating cell therapies: Academic versus Industry model Alexey Bersenev, MD, PhD Advanced Cell Therapy Laboratory, Yale-New Haven Hospital, Yale University www.celltrials.info The BioProcessing Summit, Boston, Aug. 4, 2015
- 2. • Academia vs. industry – clinical trials global trends • Key differences between academic and industry models • Gaps and solutions Outline of the talk:
- 3. Global trends: sponsorship of clinical trials http://dx.doi.org/10.6084/m9.figshare.1504124
- 4. Global trends: Total number of MSC trials http://dx.doi.org/10.6084/m9.figshare.1504131
- 5. Global trends: Total number of trials, involved adipose-derived cells http://dx.doi.org/10.6084/m9.figshare.1504586
- 6. Is industry in favor of allo- model? NO! Total number of registered clinical trials http://dx.doi.org/10.6084/m9.figshare.1504311
- 7. Measuring output: published results of clinical studies in regenerative medicine – 2014 sample (n=116)
- 8. • focus on “first-in-human”, pilot studies • early stages of development • delivery: cell therapy as procedure • favored model – auto? • favored medical innovation regulatory path • diseases with low prevalence • low scale • no attention to pharmacoeconomics, relies on “efficacy boost” • focus on commercialization and technology transfer • delivery: cell therapy as mass- produced drug • favored model – allo? • all phases of trials under FDA • diseases with high prevalence • large scale • pharmacoeconomics will determine product development Academic versus industry cell models – key differences: academic industry
- 9. Medical Research Personalized Medicine First-In-Human Serum Cell Culture Patient File/ Binder Scale Out Closed System Cost of Goods Automation Large Scale Bioprocess Batch/ Lot Unit Operations Academia vs. Industry – cultural/ language difference: Academia Industry
- 10. developer FDA local IRB FACT AABB Traditional academic and industrial pathways CAP HCT/P biologic/ drug 361 351 No GMP cGMP need for cell culture/ gene modifications
- 11. • Booming of stem cell and maturity of cell-based technologies on research side – ready for translation! • Clinical resources in big academic hospitals – pioneering physician are eager to be the first • Technological tools for clinical cell processing are available • Pharma and Biotech are reluctant to enter the field on early stages The need for academic GMP cell therapy facilities:
- 12. Academic GMP cell therapy facilities: • Trend: increase number of academic facilities • AABM – Association of Academic Biologics Manufacturers http://www.aabmonline.org ~ 45 registered, mostly in US • AGORA: ATMP GMP Open Access Research Alliance http://agora-gmp.org/ - 8 centers listed • EATRIS: European Infrastructure for Translational Medicine ATMP Platform http://www.eatris.eu/ – 14 GMP facilities in EU • Estimated total # of facilities in US 50-100 and EU – few hundreds
- 13. • Usually not a subject of FDA inspection not always follow strict GMP standards • Scale and capacity: much smaller than industry • Acting as a hospital core facility must to accommodate all types cell/gene therapy trials • Usually under-funded data not publishable, so no return Academic GMP facilities – key differences:
- 15. Centralized vs. decentralized cell manufacturing facilities cGMPcGMP decentralized centralized collection/ processing OR/ APH closed system/ automation OR/ APH release delivery collection delivery processing release
- 16. • Licensing —> Tech Transfer • Contract manufacturing • Validation studies Academic GMP – Industry interactions:
- 17. Key to success in tech transfer: 1. Academics must understand needs of industry for successful commercialization and regulatory compliance 2. Industry to leverage the knowledge of academics
- 18. Academic GMP - more gaps: • Lack of QC, QA and Regulatory • Research-like mindset • Training • Funding: The cost of GMP compliance is underestimated in academia
- 19. Advice for academics: • Invest time in developing cell therapy programs in academia – training, GMP facilities, QA/ regulatory affairs teams • Start pre-clinical design and Phase 1 with pharmacoeconomics analysis think of cost- effectiviness, COGS, BLA, reimbursement • Utilize (1) translational centers to help and (2) industry consultants • Get involved in industry collaborations and tech transfers huge experience!
- 20. Advice for industry: • It will be slow —> be patient • Educate academics for your own benefit • Invest early —> product/ process development. Phase 2 could be too late • Utilize academics more for validation studies