Regulatory Affairs

Hurdles Ahead for Cell and Gene Therapy Makers

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…

The CRISPR Saga (So Far)

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…

Orphan Drug Designation: Securing the Significant Benefits

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)…

Maximizing European Market Access: Guidance for Young Biopharmaceutical Companies

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…

An Ethical Option for Shelved Drugs

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…

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

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…

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

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…

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

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…

Preparing for a Virtual Audit

Audits are a vital quality-management tool of the biopharmaceutical industry. Whether the objective is to verify supplier or partner qualifications, contribute to corrective and preventative actions (CAPA), or fulfill regulatory compliance requirements, conducting proactive auditing is key to successful operations. Over the past year, virtual audits —also known as remote or distance audits — have enabled biopharmaceutical companies to meet compliance and quality assurance demands despite COVID-19–related travel restrictions and social-distancing protocols. Notwithstanding the challenges of virtual audits, the benefits…

Untapped Potential of Tissue Engineering: The Three Obstacles Holding It Back

Regenerative medicine is the interdisciplinary field comprising tissue engineering, cell therapy, and gene therapy. These biopharmaceutical modalities, also referred to as advanced therapies, are growing rapidly, characterized by groundbreaking therapeutic advances that have the potential to change how healthcare providers deliver care. As Figure 1 shows, cell and gene therapies have gained traction over the past decade, as evidenced by large increases in investment and the number of marketed products. By contrast, tissue engineering investment and product commercialization has lagged…