From Discovery through Commercialization
Cell and gene therapies aim to treat, prevent, or potentially cure diseases. Both approaches have the potential to alleviate the underlying cause of genetic and acquired diseases. Although critically important, the cell and gene therapy landscape is especially challenging to navigate from R&D to regulatory approval and market access.
- Cell and gene therapies can have exaggerated and unexpected behavior, and animal models may fail to predict the potential for human toxicity or beneficial immune modulation
- Efficacy and safety issues have contributed to unsuccessful regulatory outcomes, creating uncertainty in regulatory pathways
- Cell and gene therapies pose pricing and market access challenges as systems wrestle with paying upfront for a lifetime of benefits
Certara’s Cell and Gene Therapy Practice de-risks and accelerates the entire process, from discovery to market access with an integrated approach and experienced team. We are uniquely positioned to anticipate risks early, provide concrete insights to make informed and critical decisions, and set your cell and gene programs up for success.
With extensive experience successfully supporting sponsors in more than two dozen cell and gene therapy programs, Certara’s Cell and Gene Therapy Practice offers broad and integrated experience and knowledge.
Integrated, commercial, best-in-class approach
From discovery through commercialization, our integrated team of experts leverages extensive experience and knowledge of development, regulatory pathways, commercialization, and lifecycle management to provide our partners with the support you need to advance your program and help to increase probability of success.
- With the most experienced team of experts in pharmacometrics, mechanistic modeling, regulatory science, and market access, we are dedicated to achieving and accelerating your success
- We use modeling and simulation across the entire drug discovery and development life cycle to increase confidence in critical decisions and help you reach your goals, smarter and faster
Agility, communication, quality, and trust are critical to our partnerships. Together with you, we are one integrated team that creates a robust strategy and delivers with seamless implementation
Getting the Dose Right - Certara Virtual™ Twin-QSP
Considered personalized medicine, each ‘therapy’ is based on individual patient data detail to repair or enhance faulty genes. Too low a dose might exclude that patient from any future benefit, while too high a dose can be very dangerous, since any immunogenic side effects may be irreversible and could last for years. Threading this needle requires a novel development approach.
Certara’s Virtual Twin-QSP platform has demonstrated success in multiple rare disease gene therapies in predicting and informing Phase I/II and III studies. We create a computer-simulated model of each patient (virtual twin), replicating the patient’s various attributes, including morphology that affect a drug’s fate in their body and hence its effects. Hundreds of virtual twins are simulated in virtual trials to evaluate the impact of different drug doses, schedules, and combinations so that we can predict an optimal dosing regimen for each patient.
Extensive experience in complex biologics
Certara provides substantive support in the development of Complex Biologic programs, including cell and gene therapies. We combine our deep knowledge of drug development with in silico modeling and simulation approaches to maximize program efficiency and increase the likelihood of success. We create and help clients execute on a Model-Informed Drug Development strategy, including:
- IND-enabling pre-clinical activities
- Early clinical development and regulatory strategy and program support, including Phase 1 and Proof-of-Concept trial design, and human dose projections for first-in-human trials.
- Opportunities to leverage in silico and quantitative methods for optimizing Phase II/III studies and streamlining post-marketing requirements.
Innovative pricing to expand market access
Cell and gene therapies pose pricing and market access challenges to manufacturers and payers as systems wrestle with paying upfront for a lifetime of health benefits.
Optimize value, positioning, and evidence synthesis across all stages of the reimbursement journey with Certara’s Evidence and Access team. Our combination of science and understanding of public health decision-makers’ challenges is the key to effective market access and pricing. Using an array of methods, we assess the value of health interventions.
Anticipating challenges ensures success with:
- Strategy aligned with health authority and payer/HTA expectations to eliminate unnecessary data collection activities and deliver development programs efficiently
- Adaptive response to changing market dynamics
- Understanding of sources of data and evidence for historical comparators that improve value communication and capture
- Support for both investor and investee needs as gene therapy manufacturers move across the funding trajectory
De-risking the regulatory process
Clinical efficacy and safety issues have played a major role in unsuccessful outcomes from a regulatory standpoint. As this is an emerging arena, there is much uncertainty in regulatory pathways, evidenced by recent FDA decisions.
Preparing a successful regulatory submission for this challenging therapeutic area requires significant expertise, effort, and attention to detail. A large, diverse team, numerous tools, planning, and processes must work together in harmony for a successful outcome. Complexity, risk, time, and costs typify every cell and gene therapy submission. Without careful execution and the ability to navigate changing regulatory requirements, your submission may face costly delays.
Certara Regulatory Science, also known as Synchrogenix, assures and accelerates regulatory success through a unique combination of customized:
- regulatory strategy
- document authoring
- medical communications and publications
- operations solutions
Synchrogenix’s proven record of success is built on its expertise, and an unrivaled commitment to quality and compliance combined with the use of advanced technologies to enhance speed and efficiency.
Piet van der Graaf is Senior Vice President and Head of Quantitative Systems Pharmacology at Certara and Professor of Systems Pharmacology at Leiden University. From 2013-2016 he was the Director of Research of the Leiden Academic Centre for Drug Research. From 1999-2013 he held various leadership positions at Pfizer in Discovery Biology, Pharmacokinetics and Drug Metabolism and Clinical Pharmacology. He was the founding Editor-in-Chief of CPT: Pharmacometrics & Systems Pharmacology from 2012-2018 before becoming Editor-in-Chief of Clinical Pharmacology & Therapeutics. Piet received his doctorate training in clinical medicine with Nobel prize laureate Sir James Black at King’s College London. He has been awarded the 2024 Gary Neil Prize for Innovation in Drug Development from the American Society of Clinical Pharmacology and Therapeutics (ASCPT) and was the recipient of the 2021 Leadership Award from the International Society of Pharmacometrics (ISoP). Piet is an elected Fellow of the British Pharmacological Society and has published >200 articles in the area of quantitative pharmacology and drug development.
Maximilian Vargas, PhD MBA, is a Vice President, US Access Strategy within Certara’s Evidence and Access Group since 2012. He brings both scientific training and experience in healthcare product development that are important for evidence-based value and access strategies for pipeline and marketed products.
Oxana has more than 15 years of experience in the healthcare industry including the last 10+ years in regulatory affairs. She specializes in global regulatory strategy and drug development for rare diseases, pediatrics, and biosimilars, with a focus on Chemistry, Manufacturing and Control (CMC). She has hands on experience with regulatory meetings and various types of submissions to EMA, FDA and Health Canada.
Piet van der Graaf is Senior Vice President and Head of Quantitative Systems Pharmacology at Certara and Professor of Systems Pharmacology at Leiden University. From 2013-2016 he was the Director of Research of the Leiden Academic Centre for Drug Research. From 1999-2013 he held various leadership positions at Pfizer in Discovery Biology, Pharmacokinetics and Drug Metabolism and Clinical Pharmacology. He was the founding Editor-in-Chief of CPT: Pharmacometrics & Systems Pharmacology from 2012-2018 before becoming Editor-in-Chief of Clinical Pharmacology & Therapeutics. Piet received his doctorate training in clinical medicine with Nobel prize laureate Sir James Black at King’s College London. He has been awarded the 2024 Gary Neil Prize for Innovation in Drug Development from the American Society of Clinical Pharmacology and Therapeutics (ASCPT) and was the recipient of the 2021 Leadership Award from the International Society of Pharmacometrics (ISoP). Piet is an elected Fellow of the British Pharmacological Society and has published >200 articles in the area of quantitative pharmacology and drug development.
Maximilian Vargas, PhD MBA, is a Vice President, US Access Strategy within Certara’s Evidence and Access Group since 2012. He brings both scientific training and experience in healthcare product development that are important for evidence-based value and access strategies for pipeline and marketed products.
Oxana has more than 15 years of experience in the healthcare industry including the last 10+ years in regulatory affairs. She specializes in global regulatory strategy and drug development for rare diseases, pediatrics, and biosimilars, with a focus on Chemistry, Manufacturing and Control (CMC). She has hands on experience with regulatory meetings and various types of submissions to EMA, FDA and Health Canada.
