Regulatory Affairs

CMC Strategies for Expedited Program Development — Regulatory Perspectives: Session 1 of a CMC Strategy Forum

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In response to increasing demands for expedited availability of biotherapeutics around the world — and with the ultimate goal of patient benefit — health authorities have developed accelerated regulatory pathways to reduce development timelines to product licensure. Because of the complexity and unique nature of each program and product modality, some solutions must be worked out case by case between sponsors and regulatory agencies. On 13 October 2020, CASSS–Sharing Science Solutions presented a virtual session overviewing currently available regulatory pathways…

May 20, 2022

Managing Host-Cell Proteins: Robust Risk-Assessment Frameworks for Process-Related Impurities in Biological Products

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Although biomanufacturing processes are designed to generate highly pure drug substances, some host-cell proteins (HCPs) copurify with target proteins and thus remain in finished drug products. Biopharmaceutical developers are keenly aware that such impurities must be minimized to protect patients. HCPs can activate several kinds of immune responses in treated patients, including production of antidrug antibodies and induction of cross-reactivity with therapeutic proteins (1–5). HCPs also can diminish drug efficacy, potency, and/or stability (6, 7). Thus, regulatory guidances such as…

May 20, 2022

Hurdles Ahead for Cell and Gene Therapy Makers

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Significant growth of the cell and gene therapy (CGT) pipeline in recent years demonstrates the enormous potential of these modalities to treat or even cure otherwise intractable diseases. Several CGT products have been approved for clinical use over the past five years. More than 75 such products have come to the market around the world so far. They include chimeric antigen receptor (CAR) T-cell therapies that involve genetic engineering of patient cells ex vivo as well as in vivo gene therapies…

April 19, 2022

The CRISPR Saga (So Far)

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Since January 2016 (with a brief interlude as described below), the Patent Trial and Appeal Board has been attempting to adjudicate the proper inventorship of CRISPR technology in (to date) six separate patent-interference proceedings. (Scientific “priority has been decided, for now, by the awarding of the Nobel Prize in Chemistry to Jennifer Doudna and Emmanuelle Charpentier in 2020; see the “Priority Claims†box). CRISPR-based gene editing was hailed as the “Breakthrough of the Year†in 2015 (1), and the scientific…

April 19, 2022

Orphan Drug Designation: Securing the Significant Benefits

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When preparing an application for orphan drug designation (ODD) in the European Union (EU), you need to consider key eligibility criteria that are different from those required by the US Food and Drug Administration (FDA). One factor is justifying the “significant benefit†(defined below) of your drug product over existing therapies for an orphan condition. Significant benefit contributes considerably in securing a successful ODD application if the correct approach is taken — as described in two European Medicines Agency (EMA)…

March 16, 2022

Maximizing European Market Access: Guidance for Young Biopharmaceutical Companies

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Achieving efficient and profitable market access for next-generation pharmaceutical products is extremely challenging. The number of drug launches is rising every year, taking competition levels higher with them. And because these novel products tend to be more tightly targeted to smaller patient populations than the “blockbuster†drugs of old, their pricing/reimbursement terms need to be tailored to match. This is especially the case with highly complex biologic drugs, which typically are expensive to research and develop. Below I offer a…

March 16, 2022

An Ethical Option for Shelved Drugs

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A key priority in today’s investment world is corporate adherence to environmental, social, and governance (ESG) requirements. A company’s ESG score serves as a marker of the organization’s values and as a disclosure mechanism for investors to consider. Many companies now consider ESG scores when making strategic choices, and my group has identified a tangible option. In 2019, the Children’s Tumor Foundation and CureSearch for Children’s Cancer, launched the Bridge initiative (1) in partnership with FasterCures, a division of the…

February 4, 2022

The Biosimilars Action Plan: Promoting Faster and More Extensive Adoption of Biosimilar Drugs

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The pace with which biosimilar drugs have been adopted in the United States has frustrated (and displeased) policymakers (1). After passage of the Biologics Price Competition and Innovation Act (BPCIA) (2) as part of the Affordable Care Act of 2010 (3), policymakers intended and expected significant reductions in expenditures for this class of biopharmaceuticals (4). The Federal Trade Commission (FTC) had predicted that the percentage of savings would be lower than that of the <90% reduction in costs for small-molecule…

December 8, 2021

eBook: Creating CMC Strategies for Pandemics and Beyond — Perspectives on the Impact of the COVID-19 Pandemic

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The COVID-19 novel coronavirus pandemic has highlighted the biopharmaceutical industry’s need to create and implement chemistry, manufacturing, and controls (CMC) strategies that can expedite drug development during times of crisis. This bold topic was the premise of the 2021 CASSS Well-Characterized Biotechnology Products (WCBP) forum titled “Special Edition: Creating CMC Strategies for Pandemics and Beyond.” Held virtually on 25–28 January and 1–4 February 2021, the eight-day event addressed manifold considerations for SARS-CoV-2 virus prevention, diagnosis, and treatment. Five hundred attendees…

November 30, 2021

Implementation of Established Conditions: Learnings from a “Sharing Science Solutions†Workshop

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The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guideline Q12 (1) (step 4 sign-off in November 2019) is in the process of being implemented in a number of regulatory regions. The document provides additional frameworks for pharmaceutical life-cycle management. It is intended to support globally harmonized regulatory tools such as established conditions (ECs) and product life cycle management (PLCM) documents to facilitate postapproval changes to chemistry, manufacturing, and controls (CMC). Although a harmonized framework…

October 28, 2021