Articles in this Issue
Dr. John Ayres
Adoption of ICH Q8-11 has provided a more structured way to define product Critical Quality Attributes (CQAs), the design space, the manufacturing process and the control strategy for establishing acceptance criteria - all linked to the Quality Target Product Profile (QTPP). Utilizing these guidance documents, regulatory agencies have been increasingly effective in promoting a quality culture embedded in an integrated system of continuous improvement.
Susumu Uchiyama
While the relation of protein aggregates in biopharmaceutical products to the immunogenicity after administration is not fully understood and thus remains elusive, protein aggregates have been considered to be a potential risk for biopharmaceuticals as the aggregates may elicit an immune response.
Randy Hutt, PhD
This article is primarily about the Environmental Monitoring (EM) program that should be in place for biological products including bacterial and viral vaccines, although there is also mention of testing of raw materials and finished product as well. Biological products are derived from cells, tissues or microorganisms through cell culture growth/fermentation, extraction of substances of interest, isolation and purification leading to formulation and filling. Products include allergens, antigens, hormones, enzymes, monoclonal antibodies, immune animal sera, fermentation products and bacterial and viral vaccines.
Juan A. Hernandez Bort, PhD
Over the last few years, gene therapy (GT) has emerged as a promising medical tool to treat diseases. This novel approach is underpinned by the positive clinical results achieved in patients followed by regulatory authority approvals for in vivo product commercialization in both the USA and Europe; recently for Luxurna™ (Spark Therapeutics Incorporation) by the FDA) n 2017 and more recently by the EMA in 2018, followed by Tegsedi™ (Ionis Pharmaceutical and Akcea Therapeutics) and Onpattro™ (Alnylam Pharmaceuticals) in 2018.
One of the major technical challenges is the delivery of poorlysoluble drugs.
Pete Gilmore
Recent regulations designed to alter drug manufacturers’ relationships with PBMs may be just the tip of the iceberg when it comes to dissatisfaction with drug pricing practices. From proposals to require drug pricing transparency in patients’monthly explanations of benefits to caps on annual list price increases to expanded CMS’s negotiating power, there are a myriad of other regulatory and legislative proposals under consideration. The goal seems to be to drive drug manufacturers and PBMs to offer more up-front consumer discounts and reverse the trend toward annual price increases, particularly for costly specialty drugs.
Anvit Vasavada, M.S, Sunny Christian, MS, Neelam Sharma, M.S., Hemant N. Joshi, Ph.D., MBA
The purpose of this column is to highlight and summarize recent key patents in the pharmaceutical arena issued by the US Patent Office during March-April 2019.
Joshua S. Sharp
Protein higher order structure (HOS) analysis is one of the most complicated steps in biopharmaceutical analysis. Hydroxyl radical protein footprinting (HRPF) is a technology developed over the past three decades for characterizing protein higher order structure using mass spectrometry.1,2 Proteins are exposed to freely-diffusing hydroxyl radicals in solutions, and these hydroxyl radicals modify amino acid side chains based, in part, on their solvent accessibility.
Jonathan Jones, Jade Tuck, Sam Went, Philip Probert
Stratified medicine and personalized therapeutics offer a route to more efficacious patient treatment regimens. However, the diversity of therapeutics required, and the small market size for each, requires a more agile manufacturing method than those currently in use to ensure these products are readily available and affordable. Cell Free Expression (CFE), a method which utilizes biological components extracted from living cells to produce recombinant proteins, could be the solution to this manufacturing challenge.
Johannes Kiefer, Ph.D., Claudia C. Rullich
About four years ago, enantioselective Raman spectroscopy was proposed as a new tool for analyzing chiral media. In recent years, the concept was proven experimentally and different approaches to analyze the data were tested. This article shows how the technique can be implemented and it gives an overview of data evaluation strategies taking the solutions of a chiral pharmaceutical analogue as an example.
Frederic B. Ayers
Producing non-terminally sterilized parenteral products requires strict microbiological controls to mitigate potential contamination in highly controlled environments. A holistic Sterility Assurance program is the combination of all facility and process controls, practices, and procedures that ensure, with a high degree of confidence, that products are free of microbial contamination. One of most fundamental measures of Sterility Assurance program effectiveness is Environmental Monitoring (EM). Although there is not a direct correlation between EM data and product quality, it is a representative measure of holistic environmental contamination control and capability representative of multiple mechanism within a site’s Microbiological Control Strategy.