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New medicines are needed to treat disease, to prevent people getting a disease, to improve people's health and wellbeing and to save lives. Every year in the UK, well over one billion medicines are prescribed and dispensed, out of the more than 18,000 licensed preparations. Medicines and vaccines have helped deliver incredible improvements in patient health – from doubling cancer survival in the last 40 years, reducing heart disease deaths by 75% since the 1960s and transforming HIV/AIDS into a chronic manageable condition. The UK has exceptional scientific strengths in early-stage research, with world-class scientists creating sophisticated systems that capture aspects of human biology previously impossible to study.
Most new medicines are developed and manufactured by the pharmaceutical industry. Researching, developing and manufacturing medicines contributes to the health of the economy through exports and providing a wide range of interesting, well paid, jobs.
Typically it can take approximately 10-15 years to discover, develop and test a new medicine. It is not a straightforward process and many projects fail for one reason or another - the medicine may not work as well as expected, or the side effects are too great - it is a high risk business. However when everything goes well the outcome for patients is a new medicine which might treat a disease, supress it's symptoms or prevent it taking hold.
Some projects fail because the basic science didn't work out, or the chemists weren't able to make a new molecule that had all the right properties. Other projects might fail because of unexpected side effects once large numbers of patients are being treated. It is important to identify potential problems as early as possible in the process because research and development is very expensive, particularly once clinical trials start. Although estimates vary, it typically costs in excess of £1 billion to develop a new medicine.
Ideas for new medicines come from all sorts of places. Sometimes there is a traditional plant that has been used to treat a disease. Identifying why this plant works and what the actual substance is that is affecting the disease is one source of new medicines. Alternatively, there may be ways to improve an existing treatment, or increasing knowledge of the human genome may hold the key to discovery of more targeted and personalised medicines. Approaches to medicines discovery are supported by knowledge of how the body works, what proteins it makes, and how they work.
One fast moving area of medicines development is AI‑-assisted discovery which can have multiple touch points with the medicines development pipeline.
Scientists who discover new medicines might work in universities, in research labs run by charities or research councils, in small biotechnology companies or in the laboratories of large pharmaceutical companies. However, wherever they work, they are aiming for the same thing - to discover medicines to improve the health of patients.
Most medicines being developed by pharmaceutical companies never make it through the research and development process to become marketed medicines, and there are many reasons for this – hence new medicines are expensive because of the high cost of development and the high likelihood of failure.
Estimates suggest that for every 25,000 chemical compounds tested, on average 25 of these will have gone into clinical trials and five received approval for marketing. And only one will recoup the cost of its development.