Pre-Clinical and Clinical Trials

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Progress Toward Commercial Scale and Efficiency in Cell Therapy Bioprocessing

Regenerative medicine includes both cell and gene therapies. Currently 672 regenerative medicine companies operate around the world, and 20 products have been approved by the US Food and Drug Administration (FDA). Of 631 ongoing clinical trials by the end of 2015 (1), over 40% are in oncology, followed in prominence by cardiovascular and infectious diseases. Here I focus on gene and cell therapy bioprocessing in which the final products delivered to patients are cells. Cell therapies are either autologous (derived…

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Automation of CAR-T Cell Adoptive Immunotherapy Bioprocessing: Technology Opportunities to Debottleneck Manufacturing

Continued clinical efficacy demonstrations of cell-based immunotherapies (iTx) such as chimeric antigen receptor T cell (CAR-T) therapies has made the prospect increasingly likely of an immunotherapy product achieving conditional market authorization in the short term. For example, Novartis and the University of Pennsylvania’s lead candidate (CTL019) for treating a range of hematological malignancies received breakthrough status from the US Food and Drug Administration (FDA) in 2014, permitting access to an expedited drug development pathway for high unmet medical needs (1).…

Figure 1: Clinical trial logistics, planning aspects

Clinical Supply Chain: A Four-Dimensional Mission

A clinical supply chain fulfills perfectly all four characteristics of what Packowski describes as a “VUCA” (volatility, uncertainty, complexity, and ambiguity) world (1). In commercial markets, supply chains depend predominantly on consumer orders. For global drug development programs, both investigators and patients can be considered end consumers. The international journey of a specific investigational medicinal product (IMP) includes all of the following: global sourcing of comparators, manufacturing, storage, distribution, site/patient (consumer) management, and return and destruction of the IMP. Application…

Figure 1: Assessing your therapies' reimbursement potential

Planning for Commercial Scale of Cell Therapy and Regenerative Medicine Products, Part 2: Clinical Efficacy, Reimbursement, and Needle-to-Needle Logistics

Cell therapy is an emerging pillar in healthcare with the potential to provide curative solutions to a wide range of indications. The biological complexities through which cell technologies exert their clinical impact (especially those used in immunotherapies for cancer) provide opportunities for novel modes of immune regulation, cell targeting, and payload delivery. Cells also can serve as vehicles for genetic content, which the gene therapy industry is now investigating. Since early 2004, Invetech has worked with organizations dedicated to cell…

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Cancer Innovation Forum Calls for Improving US Research “Ecosystem”

Accelerating the commercialization of promising new cancer treatments relies on ensuring that patients — individually and collectively — are actively involved throughout research and drug development. This was the consensus of leading scientists, advocates, and government officials meeting in Washington, DC, at the first national policy forum convened by the Cancer Innovation Coalition (CIC). A collaboration of cancer stakeholder organizations and others working through a national campaign called Project Innovation — which is spearheaded by National Patient Advocate Foundation (NPAF)…

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A Critical Mission: Clinical Trial Material Storage and Distribution

As if manufacturing of investigational medicinal products (IMPs) weren’t challenging enough already, the appropriate storage and distribution of sensitive biological products can be an adventurous journey itself — especially if not carefully planned and managed. No one solution fits all situations. Many things must be evaluated during planning stages. For example, what is more important: time to delivery or quality of transport and product integrity until drug can be administered to a patient? It is important to understand key storage…

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Biosimilars Awaken CROs

Biosimilars are revolutionizing the bioprocessing industry. Over the years, company CEOs have pushed different models, expanding and closing down huge amounts of internal manufacturing, research and development, and quality control facilities. Sometimes services were pushed to Asia, only to get brought back a few years afterward when a new CEO was appointed. Mergers and acquisitions further complicated those ideas. Consequently, many biopharmaceutical companies now have “gaps” in their drug development service provisions. Filling those gaps will fall on contract research…

Transformative Healthcare: The National Biomarker Development Alliance

A newly launched independent, nonprofit organization — the National Biomarker Development Alliance (NBDA) — will broadly engage leaders in industry, academia, patient groups, and government from across the United States. It was announced on 13 January 2013 at the National Press Club by the Research Collaboratory (RCASU) at Arizona State University (ASU). The mission of the NBDA is to address the complex and urgent challenge of creating standards needed for end-to-end, systems-based biomarker research and development (R&D). The alliance is…

Activatable Immunoconjugates for Target Cancer-Cell–Specific Diagnosis and Therapy

In cancer treatment, early diagnosis and targeted therapies are assumed to yield the highest cure rates. However, most current methods are limited by their low sensitivity to early disease and a lack of specificity for targeted cell killing. Newly developed, activatable immunoconjugates assist in the accurate detection of cancer through in vivo imaging with high target-to-background contrast (1,2). They also provide for the possibility of highly specific, light-mediated treatment with minimal effects on healthy cells surrounding tumors (3). In fact,…

Benefits of Business Process Management

The biopharmaceutical sector is synonymous with innovation. There is a general sense, however, that biomanufacturers often don’t “have their houses in order” when it comes to adopting efficient processes, communications, and reporting tools. Indeed, many companies still rely on outdated methods to support areas such as research and development (R&D), manufacturing, clinical trials, and sales and marketing. That is especially true now that the biopharmaceutical sector is undergoing an acquisitory revolution of both rival companies and products. The industry has…