‘The Times they are a Changin’ in Pharmaceutical R&D
Bob Dylan may not have written his song with the pharmaceutical industry in mind, but it seems like an appropriate description of what is happening in the world of drug development. While most approved medicines are small chemical molecules that can be administered orally, the majority of the top ten best-selling branded drugs worldwide are large proteins, such as monoclonal antibodies, and these must be injected.
This is a big change, since not so long ago, pharmaceutical companies were reluctant to develop drugs of this type because of the complexity and expense associated with biological products. However, once the technical difficulties of producing monoclonal antibodies as medicines were overcome and patients began to feel the benefit, industry attitudes changed, leading to the situation we have today. In other words, ‘nothing succeeds like success’. Now this is happening all over again, this time with highly technical biotech medicines, or advanced therapy medicinal products (ATMPs) to use the European Medicines Agency (EMA) terminology. ATMPs can be human cells that have been modified using genetic engineering, or gene therapy products that replace faulty genes (e.g. in cystic fibrosis) or that silence unwanted gene activity (e.g. CCR5 to prevent infection with HIV).
A few years ago, it would have inconceivable for a major pharmaceutical company to develop and market a cell product derived from individual cancer patients, but that is exactly what Novartis has done with white blood cells genetically engineered to attack childhood leukaemia and lymphomas. These chimeric antigen receptor (CAR) T cells are marketed as Kymriah, and while some manufacturing issues have emerged since approval in 2018, their ability to drive previously untreatable cancers into sustained remission has contributed to the rapidly growing field of immunotherapy.
What does all this mean for the translator? Firstly, the relevant terminology and jargon is biased towards biotechnology and immunology rather than the chemistry used with conventional small molecules. Although it may seem that there are quite enough acronyms in pharmaceutical R&D already, new ones based on fundamental molecular biology are appearing on a regular basis: ‘clustered regularly interspaced short palindromic repeats’ (CRISPR) anyone? There are also implications for the formal drug nomenclature of biological products, particularly in the era of biosimilars, i.e. drugs such as monoclonal antibodies in a generic form which is not chemically identical to the original branded medicine.
In conclusion, my experience of running courses on the pharmaceutical industry and related subjects for medical translators and interpreters has shown me that despite the challenging nature of the above material, even those without much background scientific knowledge can benefit from an understanding of the basic concepts from a subject matter point of view.
The upcoming webinars on the pharmaceutical industry cover the key aspects of drug development, with webinar 3 being the most relevant for this topic. I have also written a detailed guide to biotechnology that can be accessed via the PharmaGuide website.
The webinars take place on 6, 15 and 22 May and may be booked here.