Business

Collaboration Agreements: Critical Issues and Common Pitfalls

The number of collaboration arrangements for emerging technologies is increasing significantly, especially among companies developing COVID-19 diagnostics, vaccines, and therapeutics. Consequently, a massive amount of capital was invested in the biopharmaceutical industry in 2020. These deals range in value from the low millions to several billion dollars, representing significant risks and value for investors. Some recipients of that capital are seeking partners with whom to collaborate, sharing both the costs and risks associated with their activities and bringing innovative technologies…

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…

Intellectual Property and COVID-19

The COVID-19 pandemic is the greatest global health threat caused by a virus since the influenza pandemic of 1918 (and, before that, innumerable smallpox outbreaks throughout history). So far, the number of infections and deaths has not reached levels seen during the “Spanish flu” pandemic. However, travel, global trade, and modern factors such as misinformation on social media have increased infection rates and risks of infection. As of 15 July 2021, the World Health Organization (WHO) had reported 188,655,968 confirmed…

eBook: Rare Diseases — Biopharmaceutical Challenges Presented By Relatively Small Patient Populations

By definition, an orphan disease affects a small percentage of the population. However, with some 7,000 such conditions identified so far, they collectively have a significant impact on global health. An estimated 350 million people are affected worldwide by a rare disease — altogether more than the population of the world’s third largest country (the United States). Some well-known biopharmaceutical companies are devoted to developing treatments for rare diseases. However, the vast majority of such diseases have no treatments approved by…

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…

No More Sleepwalking: New Mindsets for Manufacturing Cell and Gene Therapies at Commercial Scale

Cell and gene therapies (CGTs) offer potential cures to some of the most challenging illnesses of our time. The number of such therapies approved for market is set to surge in the next 10 years (1). Yet current manufacturing approaches are not fit for purpose. Biomanufacturing must adapt to prevent the industry from unintentionally sleepwalking into causing harm to patients. Some urgently needed changes could come with learning about the mindset of the medical-devices industry. Background and Current State After…

Microbiome-Based Therapeutics: Negotiating Key Development Challenges

Microbiome-based therapeutics have evolved significantly in recent years, with several promising candidates advancing through the clinical pipeline. That progress is the result of growing evidence showing that targeting and manipulating the microbiome could improve human health and treat more than 25 conditions by restoring healthy bacterial populations (1). The human microbiome comprises a diverse community of microorganisms, helpful and harmful. It differs by person based on factors such as genetics, environmental influences, diet, and immune function. Microorganisms play a role…

eBook: ADCs — Evolving Links in the Biopharmaceutical Pipeline

Antibody–drug conjugate (ADC) developers both old and new are talking about the next generation of drug candidates coming through their pipelines. In April 2021, Zynlonta (loncastuximab tesirine, from ADC Therapeutics) became the eleventh such product to receive approval from the US Food and Drug Administration (FDA). But with dozens of ADC candidates currently in clinical trials, those 11 products represent the tip of the ADC iceberg. In this eBook, Dan Stanton (founding editor of BioProcess Insider) explores ADC production history,…

Total Global Capacity Finally Shows Improved Productivity

Since 2018, global bioprocessing capacity has grown from 16.5 million liters (1) to 17.4 million liters. Although output has continued to expand at around 12% overall, that rate represents a significant slowing in capacity growth as the industry moves toward greater productivity and efficiency. Trends that we have tracked in the BioPlan Associates annual report of biopharmaceutical manufacturing capacity and production (2) for over 17 years correlate with that finding. Titers are increasing; single-use technologies have reduced the need for…

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…