Unlocking the potential of precision medicine: Are we ready?

This week I delivered a keynote address at eyeforpharma Sydney 2018 on a subject which I’m personally very interested in – Precision Medicine.

In the past we knew less about the molecular causes of diseases and we worked as effectively as we could with generalities.

A medicine worked for a significant population in the trials – so we give it a shot. Kind of like a blindfolded archer, we know the targets exist, but we may need to reel off a few shots before managing to hit them.

Precision medicine is like taking the blindfold off. Seeing targets more clearly; identifying those patients that will benefit, and realising as a result a much higher chance of success.

I have been fortunate to work in this wonderful industry for more than three decades now and I’m excited to see the promise of Precision Medicine has become a reality.

Something I take great pride in is the fact our innovative products are helping millions of people around the world deal with some of mankind’s most devastating illnesses.

This is at the centre of everything we do – striving for better outcomes for our patients.

And the reason I am so interested in Precision Medicine is it is a key way in which we can drive this. Now and into the future.

One driver is the mapping of the Human genome. By sequencing the human genome, it is now discovered that variations and mutations are both predictors and signals of future or current disease. This in turn has unlocked the opportunity to diagnose disease and develop treatments in a more targeted way.

This particular area is known as genomics, but note the definition of Precision Medicine is indeed a broader one. There are other ‘omics’, epigenetics, gene editing technologies and the development of targeted therapies specific to an individual’s disease profile.

It also includes the use of diagnostics – complementary and companion – in the development of targeted health care solutions from disease prevention to interception and cure.

And of course it also includes the development of advanced therapies including cell therapies like CAR-T, which involves reprogramming someone’s own cells to more effectively fight cancer. Truly both precision and personalised medicine at an individual level.

The use of these technologies is on the rise. Now why is that?

Firstly, we are seeing immense advances in science – particularly in diagnostic technologies which allow a greater understanding of diseases and genetic mutations.

Secondly, we are also seeing a health system continually driven by needs for improved outcomes with value for money. We must ensure innovative and high cost therapies are used for the right people.

But finally and most importantly, these advances are also being driven by expectations of our patients. They want, need and expect better products to live longer, healthier lives

And while it is possible to perhaps talk about a lot of this field as something which is somewhat futuristic and, on the horizon, the fact of the matter is it is here right now.

A few years ago in 2015 the Tufts Centre for Drug Development based in Boston observed in one of their Impact Reports that Precision Medicines are much more ‘now’ than some may have though.

They observed 35 per cent of oncology drugs – about one third – are precision medicines with almost three quarters – 73 per cent – in development falling into this category.

Oncology is very much the dominant therapy area for Precision Medicine through the use of molecular diagnostics to characterise diseases – or what we refer to as Companion Diagnostics. But we are expecting to see this grow further across all therapy areas.

While the same report observed only 13 per cent of drugs across every therapy area can be considered as Precision Medicines, 42 per cent of what was in development at the time could be.

We only need to look to our counterparts in the United States to see what is waiting for us – and perhaps some lessons we should be taking on board.

In May of last year the Food and Drug Administration granted accelerated approval for an immunotherapy for patients with solid tumours that have a particular biomarker indicating disrupted ability of cells to repair DNA.

This approval was significant because it was the first for a cancer drug based on a tumour’s specific genetic features rather than its location in the body.

The FDA granted accelerated approval for this therapy’s new indication based on results from five uncontrolled, single-arm clinical trials. A total of 15 cancer types were identified among the 149 patients enrolled.

This “site-agnostic” indication being approved in the United States signals a real shift in that market to the way Precision Medicines are going to be evaluated in the future. There are certainly some lessons for us to draw in Australia from this particular development.

While Australian regulators and payors are evaluating how the Australian system needs to adapt to precision medicines there are of course a number of excellent things taking place in this market.

In the budget this year the Australian Genomics Health Futures Mission was announced. $500M aimed to boost Australia’s ability to become a world leader in this space.

As you might expect Australia has great science in genomics. The Australian Genomic Cancer Medicine Program is a world class approach.

The focus of the program is on rare or less common cancers. 52,000 people are diagnosed with rare cancers annually, this 30% of all cancers diagnoses but 50% of cancer deaths. With relatively poor access to clinical trial and treatments. 

The Program was conceived and established at the Garvan Institute and has now been expanded to other key cancer centers across the country.

This represents an investment in national infrastructure for future precision medicine. At the heart of this is the generation of evidence which can be used by the TGA and PBAC in the new era of targeted therapy to address underserved populations.

There are a number of benefits to this approach – it will assist in improving the quality of life and survival of cancer patients through nationally coordinated research-led, evidence-based care.

It will also grow the health economy through greater engagement with industry while innovating clinical trial designs and pathology platforms that anticipate the future of precision medicine in here in Australia.

But are we truly ready for all of this?

As with other medicines there are of course challenges around evolving our regulatory and reimbursement approaches. These challenges are well known to all stakeholders.

But when we talk about Precision Medicines we need to be aware of the way in which these challenges may be even further amplified – and that is both for industry as well as government and regulators.

Science is evolving in this space and technology will drive further insights, it is a constantly moving target and if reform is required we will have to anchor at a point in time and be prepared to adapt.

By definition – Precision Medicines are all about meeting smaller, more specific needs in terms of our patients with higher chance of success. This means smaller patient populations for increasingly innovative therapies.

For industry the challenge of shrinking patient populations presents a significant challenge for therapy development in a traditional sense.

We need to work in partnership across the sector to accommodate less traditional evidence approaches and develop methodologies to balance the associated risk associated with attributing value for money.

How do we best attribute value to companion diagnostics? Is our current mindset of one test per indication valid, or do we need to consider broader genomic screening? 

There is a wave of Precision Medicine coming. We need to collaborate across the sector to realise the benefit.

As the founder of my company Dr Paul Janssen often said: “There is so much more to be done; the patients are waiting.”

We must work to overcome these challenges. For our patients, speed is also of the essence.

#mycompany

Bruce Goodwin is Managing Director of Janssen Australia & New Zealand, a Board Member of Medicines Australia, and has recently been appointed to the Board of the Australian Genomics Cancer Medicines Program.

The views expressed here are entirely his own personal views and not necessarily those of the above organisations.