Precision Medicines - Why?

Precision medicine is the talk of today, with a cancer drugs fund for Wales in the news and our own 'Stopping The Clock' campaign launching, calling for fair and prompt access to precision medicine for people with cystic fibrosis.

But what is 'precision medicine' and why does it matter? Dr Janet Allen, Director of Strategic Innovation, digs deeper.

On 20 January 2015, President Obama made this announcement in his State of the Union address to the US nation: 

“Tonight, I’m launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes – and to give all of us access to the personalised information we need to keep ourselves and our families healthier.”

This was an exciting, bold statement that frames the future of treatments where individuals are treated as individuals. Recently Francis Collins, Director of the USA National Institute of Health, gave his vision of how this will be delivered, at the Faster Cures conference at the Milken Institute (pictured). He described the programme as “big, hairy and audacious – and so it should be.” 

Dr Collins argued that the time is right to do this mainly as a result of technologies coming together that will be able to inform treatments; for example genomics, environmental effects, personal wearable sensor technology. In the past, new treatments were approved based on the law of averages. For instance, how does this treatment lower the average person’s cholesterol compared to no treatment?

We need to move away from this to a state where the right drug is given to the right patient at the right time to achieve the right effect. 

This is the aim of precision medicine; we need to convert our thinking on how to keep people healthy and not see healthcare as only having a role in treating ill-health. A key component in the development of precision medicine is to engage with people as equal partners and not as subjects for study. This thinking will transform the way we do scientific research and is already creating the concept of ‘citizen scientists’. All of this will not happen overnight but the very mention of precision medicine in the State of the Union address creates momentum and it is clear that the National Institutes of Health are up for the challenge and ready. 

If you think that precision medicine is only for the ‘big’ conditions such as heart disease and cancer, see the extended quote from the State of the Union address: “21^st century businesses will rely on American science, technology, research and development. I want the country that eliminated polio and mapped the human genome to lead a new era of medicine – one that delivers the right treatment at the right time. In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable.” 

These are truly interesting and exciting times and we need to ensure that cystic fibrosis remains in the vanguard of developing precision medicine to benefit each and every individual with the condition. This is audacious but that shouldn’t stop us. As another speaker said:

·         Don’t let perfection be the enemy of good.

·         Don’t be afraid of getting started.

·         Don’t be afraid to learn.

Find out more about our 'Stopping The Clock' campaign at www.cysticfibrosis.org.uk/stopping.