By Dr. Richard Williams
In our daily lives most of us devote little thought to how our medicines get to us. We tend to take for granted the fact that when we have a headache, or suffer some back pain, we can go to the pharmacist and buy a pill to mitigate the problem. In the face of endemic or even pandemic diseases we tend to assume that there will be a timely solution - often in the form of a vaccine available at our local doctor’s surgery.
For the majority of us, experience of the pharmaceutical industry is limited to these periodic encounters. Our knowledge of the industry is largely confined to the medicines we consume, and perhaps the limited information on the accompanying leaflet, if we have the time or the inclination to read it. Some may even have a vague notion about clinical trials, but few know what these really involve.
However, in recent years the profile of the industry has become more prominent in public discourse, particularly since the Covid-19 global pandemic, and not always in the most positive way. This public questioning is to some extent symptomatic of a general lack of a detailed understanding of how pharmaceutical research and development operates, what it sets out to do, how it does it, and the internal and external technical, legislational and business constraints within which it must work.
I came to this project as someone with extensive experience in both molecular biology research and the discipline of building and running technology-based businesses, but with little deep knowledge of the pharmaceutical world. My participation in the writing of this book in support of the two principal authors was that of a naive reader. Nevertheless, the process has furnished me with a deeper understanding of the industry, the unique challenges it faces and the ways in which it continues to meet those challenges in an ever-changing technical and commercial environment.
There are a number of key features of the modern pharmaceutical industry for which I have gained a much deeper appreciation while working on this book. First among these is the absolute primacy and value of data. A pharmaceutical product is much more than the pill and associated leaflet in the branded box. These are the parts of the product that the end customer experiences, but this represents the very small tip of a very large iceberg. Throughout the process of production of a new therapy, from initial target and drug discovery, through pre-clinical and clinical testing, transition to manufacturing, and lifetime in, and final retirement from the market-place, a vast amount of extremely complex and diverse data must be generated, recorded, validated, integrated and analysed. At each stage of research and development a new layer of data is created, attached to and wrapped around the physical product. These layers must be linked together into what can justifiably be called a whole data product. In a very real sense, the data is the product, and without it no pill could ever reach the pharmacy counter.
The second feature is that this vast array of diverse, integrated data must be managed, made available and usable within a technical environment capable of generating and supporting it. In particular this must be achieved for the duration of a drug’s development cycle and presence in the market (many years), within a systems estate and technical architecture that must constantly evolve to accommodate new advances in technology, laboratory technique and analytical capability. In addition it must service new clinical trends such as the move towards personalised medicine. This challenge is especially highlighted with the rise and development of new technologies such as AI, which are now driving extremely rapid changes in the generation of data, the automation of processes and in decision-making.
Third is the sovereign importance of accountability. In today’s world the pharmaceutical industry operates within an environment where every single action, operation, decision and outcome must be fully recorded, traceable, attributable and subject to scrutiny at every step of the research and development process. External health authorities impose stringent requirements on the industry to ensure that any therapy entering the market has undergone sufficiently rigorous testing to assure its safety for human consumption, and that all associated data and decisions are readily reviewable. Changes in relevant legislation in different jurisdictions must be constantly monitored and accommodated in order to maintain compliance.
Finally, there is the ever-present problem of risk management. All businesses must manage risk, but this requirement is particularly amplified within the highly competitive and regulated pharmaceutical environment. Product research and development timelines stretch to years and require extremely high investment in terms of effort, resources and finance, with high probability that an initially promising therapy will fall by the wayside before ever reaching production. In a highly competitive market the application of new approaches, such as the use of AI to decrease time-to-market and improve product portfolio management and investment decisions, can be crucial to commercial viability and success.
The vast majority of this activity lies below the water line and is invisible to the public at large. This book sets out to provide an insight into these and other issues, and convey an integrated view, to those working within different areas of the industry, and to interested outsiders, of the main strands that must come together within modern pharmaceutical research and development to deliver effective, timely and safe therapies to an increasingly demanding world.