Bringing you the latest cutting-edge research and commentary in bioscience.

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

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Journal

Welcome to the latest issue of Cell and Gene Therapy Insights – the open access, peer-reviewed publication that brings you the latest insight and commentary from leading experts from academia and industry.

Commercial Insights
Interview

How commercial biopharma experience is shaping cell & gene therapy facility design

Interview

Niranjan Kulkarni, Allan Bream & Grace Linton

Niranjan Kulkarni, PhD,
Niranjan is the senior director of CRB’s consulting group. He holds a doctorate and master’s degree in industrial and systems engineering from Binghamton University. He is also a certified Lean Six Sigma Master Black Belt. Niranjan has over 15 years of experience in business process and data modeling, operations and process simulations, process improvements, layout optimizations and supply chain management. He has worked with the pharmaceutical, biotech, food, chemical, semiconductor, electronics assembly and packaging, manufacturing and financial industries.

Grace Linton RA, AIA, LEED AP BD+C,
Grace has over 12 years of architectural experience in a variety of project types including over eight years in process architecture with CRB. Her experience encompasses domestic and international CGMP regulatory, process design and development, and facility design for biotech, pharma, and medical device industries. Grace’s combined knowledge of architecture, process, and sustainable design provides a strong basis for integrated facility design. Her ability to conceptualize ideas in BIM environment serves as an effective method of communication.

Allan Bream
Allan has more than 35 years of engineering and manufacturing experience including 30 years in the biotechnology industry. His expertise includes large-scale bacterial fermentation, mammalian cell culture, vaccines, downstream processing, protein purification and immobilization and CGMP facility design, operations and assessment.

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Future trends in commercial cell and gene therapy: the investor’s perspective

Spotlight Article

Interview

Gregory Bonfiglio

Market & Patient Access

Gregory Bonfiglio is the Founder and Managing Partner of Proteus, LLC – an investment and advisory firm focused solely on Regenerative Medicine. Formed in 2006, Proteus provides fund management and consulting services to the regenerative medicine industry. Proteus works with cell and gene therapy companies across all stages of development from early stage entities to large public companies, as well as governmental organizations pursuing RM initiatives. Mr Bonfiglio serves on the Board of Healios KK, one of the largest publicly traded RM companies in Japan. He is on the Investment Committee of the Centre for Commercialization of Regenerative Medicine (RM Translation Center in Toronto, Canada). He is on the Advisory Board of BioBridge Global (RM Translation Center in San Antonio, Texas). He is on the ISSCR Finance Committee. Mr Bonfiglio was an early investor in the RM field, and he continues to be actively involved in the space. He has over 35 years’ experience working with technology companies. He received his BA in Mathematics (magna cum laude) from Michigan State University in 1975, and his JD (magna cum laude) from the University of Michigan Law School in 1981.

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Staying ahead of the curve: NICE’s approach to HTA of novel fields of biotech innovation

Spotlight Article

Interview

Nick Crabb

Market & Patient Access

Nick Crabb had a 20-year career in analytical science, process technology and general management in the chemical, pharmaceutical and contract laboratory industries prior to joining NICE in 2010 as the associate director responsible for establishing and managing the Diagnostics Assessment Programme. In 2014 Nick was appointed to his current role where he oversees NICE Scientific Advice, the Science Policy and Research programme and NICE’s input to the European Network for Health Technology Assessment (EUnetHTA). Nick has broad scientific and policy interests relating to the evaluation of technologies and interventions to support the development of clinical, public health and social care guidance. His experience includes consideration of HTA issues arising from the availability of novel new products such as cell and gene therapies and work on methods issues relating to the evaluation of antimicrobials. Nick was also the co-chair of the evaluation and commissioning subgroup of the UK regenerative medicine expert group and led NICE’s contribution to a project on the assessment and appraisal of regenerative medicines that reported in 2016.

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Market access in the era of personalized cell & gene therapy

Spotlight Article

Interview

Edward Abrahams

Market & Patient Access

Edward Abrahams, Ph.D., is the President of PMC. Representing innovators, scientists, patients, providers and payers, PMC promotes the understanding and adoption of personalized medicine concepts, services and products for the benefit of patients and the health system. It has grown from its original 18 founding members in 2004 to more than 200 today. Previously, Dr. Abrahams was the Executive Director of the Pennsylvania Biotechnology Association, where he spearheaded the successful effort that led to the Commonwealth of Pennsylvania’s investment of $200 million to commercialize biotechnology in the state. Earlier, he had been Assistant Vice President for Federal Relations at the University of Pennsylvania and held a senior administrative position at Brown University. Dr. Abrahams worked for seven years for the U.S. Congress, including as a legislative assistant to Senator Lloyd Bentsen, as an economist for the Joint Economic Committee under the chairmanship of Representative Lee Hamilton, and as a AAAS Congressional Fellow for Representative Edward J. Markey.

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Value demonstration and advanced therapies: the ARM perspective

Spotlight Article

Interview

Janet Lynch Lambert

Market & Patient Access

Janet Lynch Lambert joined ARM in 2017 as the organization’s first CEO. With more than 25 years in public and private sector management, Janet is an experienced government relations and business professional with an extensive record of accomplishment. Janet most recently served as the Acting Head of Engagement for the All of Us Research Program at the National Institutes of Health and as head of the Outreach Office in the Office of the NIH Director. Prior to joining NIH, she was Vice President of Government Relations and head of the Washington office of Life Technologies, aiding the company in its growth from $300 million in annual sales to more than $3 billion. Prior to Life Technologies, Janet held leadership positions in government relations, marketing and business development at large and small life science organizations, including GE and InforMax. Her experience also includes legislative and staff leadership positions in the U.S. Senate and House of Representatives. Janet received her MBA in International Business from Georgetown University and her B.A. in Political Science from Stanford University. She lives in the Washington, D.C. area with her husband and two daughters.

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An outcomes-based, innovative reimbursement mechanism for curative medicines

Spotlight Article

Interview

Omar Ali

Market & Patient Access

Omar Ali qualified as a hospital pharmacist and has extensive experience as an NHS formulary pharmacist reviewing cost effectiveness of medicines for access and reimbursement at regional and national level. He was a former adviser to NICE on the ERG for adoptiona and impact of new medicines. He is currently working towards a PhD in value-based agreements and innovative contracting for new medicines, last year authoring on a paper providing a methodological framework for CAR-T reimbursment based upon remission (pay for performance model) over time.

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Perspective

Health Technology Assessment of Gene Therapies in Europe and the USA: Analysis and Future Considerations

Spotlight Article

Perspective

Tingting Qiu, Eve Hanna, Monique Dabbous, Borisov Borislav & Mondher Toumi

Market & Patient Access

Gene therapies constitute a new concept of transformative therapies, administered once in a lifetime. The value assessment of these innovative therapies constitutes a challenge for health technology assessment (HTA) bodies. The HTA reports for all seven gene therapies that have to date been granted a market authorization in the European Union (EU) and/or the United States (US) were examined to understand the rationale behind their assessment outcomes and to explore the differences in value assessment across US, England, Scotland, France and Germany. In England, Imlygic® was accepted for use with the manufacturer agreeing to the application of a discount to the list price under a patient access scheme (PAS), while Strimvelis® was recommended due to its cost–effectiveness estimate being considered as reasonable under the highly specialized technology (HST) evaluation. KYMRIAH® and Yescarta® were approved for use within the Cancer Drugs Fund (CDF) in England, conditionally, as long as managed access agreements are upheld. In France, KYMRIAH®, Yescarta®, and Luxturna® were considered as having important actual clinical benefit. In France, GLYBERA® was considered to have ‘insufficient’ benefit due to its unsustainable and heterogeneous treatment effects. In Germany, the extent of the added benefit of GLYBERA®, KYMRIAH®, and Yescarta® was evaluated as ‘non-quantifiable’ as the submitted evidence made reliable, comparative assessments difficult. In Germany, Imlygic® was assessed to have no added benefit due to the selection of inappropriate comparators. In Scotland, KYMRIAH® was accepted for B-cell acute lymphoblastic leukemia treatment with a PAS, while Yescarta® and KYMRIAH® for diffuse large B-cell lymphoma were rejected due to unjustified cost–effectiveness estimates. In the USA, KYMRIAH®, Yescarta®, Luxturna®, and Zolgensma® were evaluated as having substantial net health benefits, however, a high certainty of conclusion for the assessment of Zolgensma® was established. Although the limitations in pivotal studies resulted in substantial uncertainties regarding long-term treatment benefit, there was still a possibility for gene therapies to gain acceptance from HTA bodies. Most importantly, further evidence collection becomes the critical key, not only to reduce the uncertainty in reimbursement decisions, but also to increase the public’s confidence in the use of gene therapies.

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The diversity in regenerative medicines regulations in Europe, USA and Japan

Spotlight Article

Perspective

Tingting Qiu, Monique Dabbous, Lylia Chachoua, Claude Dussart & Mondher Toumi

Market & Patient Access

Substantial efforts have been made to increase harmonization across regulatory authorities for the regulation of regenerative medicines (RMs), yet variations still exist in market authorization (MA) processes with regards to terminology, product classification, and evidence requirements. Regulatory and MA processes were examined in the EU, the USA and Japan. RMs are evaluated under similar regulatory frameworks as either traditional medicines or biologicals by the Food and Drug Administration in the USA, with a risk-based approach acknowledging the RM’s specificities. In the EU, RMs are regulated under the centralized procedure by European Medicines Agency (EMA), with an additional step that a draft opinion is prepared by Committee for Advanced Therapies (CAT) prior to a final MA opinion from Committee for Medicinal Products for Human Use (CHMP). The Pharmaceuticals and Medical Devices Agency (PMDA) in Japan has shifted the regulatory paradigm of RMs with a time-limited, conditional MA pathway to accelerate patient access and increase global competitiveness. Opponents argue that such a system may be too permissive in potentially exposing vulnerable patients to treatments with questionable efficacy and safety. The FDA and EMA have shown more willingness to accept real world evidence (RWE) to support MA applications. RWE was more commonly used in post-market surveillance by the PMDA in the past, but efforts are underway to explore the possibility of using patient registry data for MA application. To avoid the use of unauthorized RMs, the FDA has temporarily established the Tissue Reference Group (TRG) Rapid Inquiry Program (TRIP) to support sponsors in the regulation of specific RMs, along with the release of final guidelines to explain the principle of ‘minimal manipulation and homologous use’. The PMDA requires a specific expert committee review for RMs (including autologous RMs for homologous use) administered in medical practices and research based on effects on human health. The ‘hospital exemption’ rule in the EMA still needs more clarification to ensure its harmonious implementation across different Member States. Expedited approval programs in three jurisdictions showed disparities in eligibility criteria, application timelines and incentives provided to sponsors.

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Breakthrough therapies, breakthrough economics in the era of cure

Spotlight Article

Perspective

Stephanie Farnia, Lisa Mostovoy, Carole Redding Flamm & Naomi Aronson

Market & Patient Access

Cellular immunotherapies and gene replacement therapies herald new therapeutic categories and renewed discussion of how transformative therapies should be integrated into the US healthcare system. All stakeholders will need to participate in processes focused on long-term healthcare system stewardship.

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Genetic-based therapies: looking ahead to ensuring access to a cure for cystic fibrosis

Spotlight Article

Perspective

Lisa B Feng, Jacqueline V Erdo & Mary B Dwight

Market & Patient Access

Recent scientific advancements have accelerated research for a cure for cystic fibrosis (CF). Research is underway for genetic-based therapies such as gene therapy, gene editing, and RNA therapy. However, great optimism is countered by concerns about how and if patients will have access to a future cure. As health care decisionmakers look to solutions for paying for and ensuring access to curative therapies, they should: integrate patient preferences into new payment mechanisms for a cure; design insurance benefits to incentivize highly effective therapies; and ensure equity.

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