The State of Pharmaceutical Regulatory Compliance: Global Changes and Challenges in Large Trading Regions

There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies, especially for single gene mutation disorders. The US Food and Drug Administration (FDA) has more than 700 active Investigational New Drug Applications (INDs) for gene and cell therapies and in 2017, the FDA approved two cell-based gene therapies and it is anticipated that gene therapy will become a mainstay treatment for many rare diseases.

Chemical raw materials are used in multi-tons per batch during biopharmaceutical manufacturing. Managing such quantities in a pharmaceutical production environment can be challenging: Handling and weighing procedures can be time-consuming and costly. In addition, the formation of dust and caking/clumping of chemicals can pose a significant safety risk to employees.

Lipid nanoparticles are widely adopted vehicles for oligonucleotide-based therapeutics and vaccines.

This event will showcase how nations have coordinated pharmacy support for person-centred care in the fight against COVID-19 with a focus on strategy, policy and practice.