Regulatory Affairs

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…

Manufacture and Regulation of Cell, Gene, and Tissue Therapies: Part 2 — Regulatory Guidances

The US Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Medicines and Healthcare Products Regulatory Agency (MHRA), and Japan’s Pharmaceutical and Medical Devices Agency (PMDA) all offer support and guidance for developers of advanced therapy medicinal products (ATMPs). Some agencies have issued guidelines to help companies through different stages of product development — from research and development to marketing authorization and postauthorization activities. Such guidelines are updated regularly as more knowledge becomes available from the development and…

Emerging Strategies for Drug-Product Comparability and Process Validation: Part 2 — Validation, Legacy Products, and Lifecycle Management

This two-day CASSS CMC Strategy Forum explored many technical, practical, and regulatory facets of biological drug-product (DP) analytics, process validation, and comparability. Part 1 of this report summarized the discussions on drug-product analytics and comparability in BPI’s March 2021 issue (1). Here we report on day two presentations and discussions on validation, legacy products, and lifecycle management. Session Three: Drug-Product Validation The morning session focused on principles of process validation with examples of challenges specific to drug products. New Risk-Based…

The EU MDR Deadline Delay: What Does It Entail for Pharmaceutical Companies?

The life-sciences industry has been working hard to meet the deadline for compliance with the European Union’s Medical Device Regulation (EU MDR, 2017/745) (1). Doing so has been a challenging journey for many companies. Therefore, the full-year postponement of the final application date has been a welcome development, particularly in view of the new and extraordinary challenges stemming from the COVID-19 global health crisis. The extension has instigated other important changes, so it is critical that life-science businesses familiarize themselves…

COVID-19 As a Catalyst for Changing Orphan-Drug Regulations

A long-awaited (and for months withheld) evaluation of the European Union’s Orphan Drug Regulation (ODR) finally reached the interested public in August 2020 (1). Its publication during the public consultation of the European Commission’s “Pharmaceutical Strategy” was well planned because the latter discusses policies on access, availability, and affordability of new medicines. However, the ODR evaluation shows not only that the intentions behind the legislation have not been fulfilled, but also that its generous incentives (extension of market exclusivity and…

The Power of Industry Collaboration: Driving Harmonization of Regulatory Requirements

The biopharmaceutical industry continues to develop advanced manufacturing processes, systems, technologies, and facilities. Regulations play a significant role in assessing and approving marketing authorizations for drug products that are submitted for approval. The industry and regulators together should form a coordinated, streamlined process that delivers much-needed medicines around the world. Global Complications The complex global nature of biopharmaceuticals sometimes means that progress is not always as smooth as it could be. One main reason is that regulatory agencies often differ…

Fetal Bovine Serum Source Countries: Comparing Regulatory Animal Health Infrastructures

The motto of the European Serum Products Association (ESPA) — “Serum Saves Lives” — reaffirms the essential role that animal serum plays in cell-culture–based research and applications to protect the health of both human and animal populations. Animal serum, especially fetal bovine serum (FBS), needs to be available in abundant supply and at affordable prices to medical and veterinary facilities all over the world. With one billion cattle globally, the supply of FBS should be plentiful, but not all countries…

Accelerated Pathways for Authorization of Medicines in Europe and the United States

Before a medicinal product can be considered suitable for patients, it must go through laborious testing and cost-effectiveness analysis. In addition, all medicinal products must be authorized before they can be sold on the market and thus made available to patients (1). This is the case in the European Union (EU) and European Economic Area (EEA) countries as well as in the United States (US). Every year, a number of medicines receive marketing authorization. In their wake, however, several thousand…

Virtual Audits: A New Reality in the World of COVID-19

The novel coronavirus disease 2019 (COVID-19) pandemic has every industry seeking out ways to accomplish time-sensitive activities using a number of virtual approaches. This is certainly true in the biopharmaceutical sector, in which good manufacturing practice (GMP) audits are required to manufacture medicinal drug products for human use. Examples include supplier/vendor audits, mock inspections, and preapproval and prelicense inspections (PAIs and PLIs) conducted by sponsors and regulatory authorities. Auditors usually perform such activities on site and only sometimes remotely. In…

Cell and Gene Therapies Get a Reality Check: A Conversation with Anthony Davies of Dark Horse Consulting Group

As founder of cell and gene therapy (CGT) specialist firm Dark Horse Consulting Group in California, Anthony Davies speaks from a quarter century of experience including former positions at Onyx Pharmaceuticals, Syrrx, ZymeQuest, Serologicals, Geron Corporation, Capricor, and 4D Molecular Therapeutics — and he currently serves on the board of directors for TrakCel and the scientific advisory boards for Akron Biotech and BioLife Solutions. In his plenary address at the Phacilitate 2020 Leaders World conference (part of Advanced Therapies Week…