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We are all familiar with personalized medicine in oncology but future developments involving new technologies are likely to flourish in other disease areas. Peter Keeling and Steve Vitale of Diaceutics look at challenges and opportunities in the non-oncology space.
Oncology has always been the front runner in personalized medicine so this is where targeted therapies and diagnostics have, for the most part, been focused. However, there is new and exciting activity outside this area where biomarker and targeted approaches are proving successful.
There are now biomarker-driven therapies for diabetes and inflammatory diseases (such as asthma and rheumatoid arthritis) and neurological diseases (such as Alzheimer’s, Parkinson’s and multiple sclerosis). The total number of targeted drugs in this space has doubled in five years and while oncology still dominates as the single most important therapy area, development in other disease areas has increased exponentially.
Diagnostics in oncology tend to be linked to specific therapies based on single analyte detection (by PCR, FISH, IHC, etc.) from tumor or blood samples. Outside oncology, however, there will be multiple entry points for biomarkers that are not necessarily blood-based. Depending on the disease area there is a set of potential new diagnostic tools, such as wearables, that can be employed singly or in combination, making the non-oncology space a more complicated but competitive landscape. These tools may ultimately be employed to differentiate certain brands, drive faster regulatory approval and/or improve the value proposition presented to payers and patients.
Is the future already here?
Pharma is already gearing up for a non-oncology future, as demonstrated by a sharp increase in personalized medicine deals outside on oncology since 2011 (Figure 1). Deals between pharma and diagnostic or lab partners have started to converge in both spaces. This is why Diaceutics anticipates that non-oncology could be equally, if not more, as active as oncology. But there could be a change in the volume and nature of the agreements that pharma have with external partners in this space. Additionally, the focus of such agreements, which is towards technology partnering, may change, as shown by Novartis and Microsoft teaming up to further develop Kinect for multiple sclerosis (MS) assessment2.
Figure 1. Deals among 14 leading pharma companies (*biomarker discovery, companion/complementary diagnostic development).1
All this activity around partnering mirrors Diaceutics’ own experience as 50 per cent of our expertise has been built by working on personalized medicine challenges outside oncology. Since 2005, Diaceutics has completed multiple projects focused solely on non-oncology issues: broad, diagnostic, testing and lab strategies in HIV, Alzheimer’s, insulin resistance, sepsis, hepatitis C and neglected diseases such as TB and leprosy, plus financial modelling for rheumatoid arthritis, to name just a few. This demonstrates just how much development is already taking place outside oncology.
Expect to see a diverse testing landscape outside oncology
In MS, the gold standard for diagnosis is imaging, but there is significant work being done on biomarkers to identify patients, or measure therapeutic response to pharmaceutical interventions. Stratify JCV was developed to address a significant safety issue for a specific therapy, but many companies are trying to identify a prognosis biomarker to identify and track disease progression. In addition, sensitive wearable technologies can gather data on daily movement and motion to inform the clinical perspective. Clinical trials reveal substantial activity in this area, while health apps and devices are expanding from the consumer field into the diagnostic world.
Another area where we can expect to see a rise in the use of diagnostics is at opportune points early on the treatment pathway. In rheumatoid arthritis, for example, big issues restrict biologics to a small patient pool in the very late stages of the disease, when damage to the joints has already been done. But research shows that earlier identification and treatment can reduce joint damage and improve quality of life3. By identifying inflection points on the pathway, and incorporating existing and novel biomarkers, is it possible to create a perfect storm of diagnostic, education and novel therapy to get earlier treatment to the right patients that also benefits cost-management?
Collaboration with payers on this last point could provide the incentive to make sure tests are used as early as possible and have rapid uptake.
Non-oncology personalized medicine: a risky bet but a potential winner
The non-oncology space is more complex, potentially more expensive and risky, but get it right and there are significant access, market and patient share benefits. It is likely that pharma companies will start to craft a diagnostic entry into treatment pathways where they hold valuable assets. This could impact on product uptake, peak sales, access and pricing discussions, because there is a strong correlation between the choice of test, particularly early in the treatment pathway, and the potential to avoid costly outcomes later.
Personalized medicine in oncology has been a euphemism for high priced targeted therapies but this is not sustainable as a model outside where there is a much larger population. For instance, a new test in NSCLC could be used up to 80,000 times in its first year, but in cardiology or CNS we would need to add a few zeros to that figure.
Competitors in key disease areas are lining up some very interesting multi-test strategies. As a result, ‘one size fits all’ therapies will increasingly be competing with test-enabled therapies, and 2017 will be a key tipping point.
This article was published in Life Science Leader on 1 September 2016. http://www.lifescienceleader.com/doc/great-expectations-for-personalized-medicine-outside-oncology-0001