From molecule to medicine

Published: 20-Aug-2018

Dr Sheuli Porkess, Deputy Chief Scientific Officer at the ABPI explains the importance of pharmaceutical research and what is involved in developing new medicines

A report this week from the Office of Health Economics shows the amazing impact medicines have had on the NHS and also further afield.

The antipsychotic chlorpromazine, first used in the NHS in 1954, paved the way for deinstitutionalisation and community-based care for people with mental illness.

In 1948, there were almost 400,000 cases of measles in England and Wales, and 327 people died. By 2015 the number of cases of measles in England and Wales had fallen below 1200.

These medicines and others, had a variety of benefits including better clinical outcomes, saving lives, improving quality of life, greater health service efficiency and wider societal impacts.

But making medicines is a complicated and costly business. It costs billions of pounds and can take decades. Successes can change the world; failures are an inevitable part of the discovery and development process. But when medicines get through the development process, they can clearly change millions of lives.

There are broadly three stages to creating a new medicine: research, development and approval. Here’s how it works:

Drug Discovery and Development

The process usually starts with chemical compounds or biological molecules. With advances in technology in the last few years, it is now possible to screen compounds that have the potential to become treatments faster than ever before. AstraZeneca – a British pharmaceutical company – launched a new screening robot in 2016 called NiCoLA-B, which is able to test 300,000 compounds a day. Its job is to find those chemicals that show the slightest potential of being useful as a medicine.

The research stage benefits hugely from collaborative partnerships between the pharmaceutical industry, charities and universities, all working together to find a potential medicine. This stage can take 4-5 years and takes about 22% of the total budget it takes to find a treatment. Each compound has a less than 0.01% chance of success.

Preclinical Research

From a batch of about 10,000 compounds screened in the drug discovery phase, only about 10-20 go into the preclinical phase, where scientists determine how safe a medicine might be through testing in cells and animals as well as using computational models.

Clinical Research

If any of those 10-20 compounds show real potential of being turned into something useful, they are developed into a medicine that will move into clinical trial stage. There are three steps: Phase I involves about 20 to 100 volunteers. If medicines are successful here, they will move onto Phase II where they are tested in people with the disease.

Phase III can include up to 5000 patients. Going through the three phases can take 6 or 7 years. About 65% of the money it takes to make a medicine is spent in the development stage.

Phase IV clinical trials are after the medicine has a licence and are there to help monitor the medicine’s safety and help clinicians better understand how the medicine works in everyday life, not just in clinical trials.


The final stage is when regulators review the medicine and it can get market authorisation – which shows the medicine is safe and effective. By this point, the manufacturing of the medicine has been scaled up. Only 1 medicine of 5000 – 10,000 compounds discovered will make it to this stage.

The approval processes last anywhere from 6 months to 2 years. The medicine is continually monitored once it starts being prescribed for patients.

Researching and developing medicines takes a lot of time and work along the way; there is no guarantee that any particular medicine will make it through the various stages of this highly regulated process. Once medicines get through this system, their impact can be huge.

Of course, the pharmaceutical industry is pioneering new ways to find treatments. The future looks exciting and how diseases are detected, diagnosed and treated is set to change significantly.

Advances in medical technology and the miniaturisation of diagnostics, wearables and devices will have a huge impact on our lives and could help people with chronic diseases to remain out of hospital.

Advances in understanding how cells monitor and repair damaged DNA enables us to develop game-changing treatments for cancer. Progress in immuno-oncology sees patients own immune cells used to attack cancer cells and stem cell therapy is treating rare sight conditions.

Now AI and synthetic biology can be used for treating malaria, HIV and hepatitis. Gene-editing technology is happening in labs right now, identifying new disease targets, accelerating the discovery of novel treatments.

Passionate pioneers, such as those who invented the groundbreaking treatments in the report, have always been at the heart of our industry and it’s exciting to imagine what their successors could achieve in the next 70 years.

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