I am delighted to use this week's blog to introduce one of the Centre's new analysts, Amen Sanghera. During his MSc studies in Nanotechnology and Regenerative Medicine (2013 -14) Amen was fortunate to gain first-hand exposure to the potential that precision medicine (PM) can have for patients. His blog explores this potential and considers the extent to which key stakeholders (payers, providers, physicians and patients) are ready to embrace the move towards precision medicine.
What is precision medicine?
PM is an emerging approach for treatment and prevention that takes into account a person's genetics, environment and lifestyle. It allows doctors and researchers to predict more accurately which strategies for a particular disease will work in which groups of people. It is in contrast to the traditional "one-size-fits-all" approach in which disease treatment and prevention strategies are developed for the average person, with less consideration for the differences between individuals.
While the term PM is relatively new, the concept has been around for many years. However, it is only in recent times that we have seen the first tentative steps towards turning the PM rhetoric into reality. Genetic profiling and accompanying diagnostic testing currently form the fundamental driving force in bringing precision therapies to clinical practice. These breakthroughs have allowed providers and physicians to understand diseases at a molecular level and specify a means for accurate diagnosis and the development of targeted therapies.
How has the industry's perspective changed?
PM was initially met with scepticism by the pharmaceutical industry, particularly from a business point of view. The industry saw spending resources developing therapies that treat specific sub-segments of patient populations as reducing the potential size of the addressable market. However, this view has changed over the past decade, thanks to the implementation of favourable regulatory policies and incorporation strategies in major markets. As a result, the percentage of PM drug approvals in the US increased from 22 per cent in 2014 to 29 per cent in 20151 and the number of PM products available on the market increased from 13 in 2006 to 113 in 2014.2
Oncology leading the way
Oncology is one area in which PM is making significant inroads, with the US regulators giving priority to PM for oncology treatment.3 Indeed, in 2015, 36 per cent of newly approved oncology drugs were PMs.4 The increased use of PM in oncology is partly due to being able to demonstrate its clinical advantages more readily in cancer treatment compared to other therapy areas, such as autoimmune diseases.
Clinical adoption of PM outside of oncology is slow, due to a number of challenges that have deterred key stakeholders from embracing the prescribing of PM therapies. These include:
- scientific and technical challenges such as overcoming clinical hurdles (demonstrating efficacy), developing therapies for a wider array of diseases, finding the right biomarkers, and a better comprehension of the benefits of molecular therapies amongst payers
- economic challenges which include incentive systems that are often slow to adapt to new trends in healthcare and are counterproductive in enabling innovation to succeed
- regulatory roadblocks that have made the approval process for PM unclear and convoluted, particularly in markets outside of the US such as the UK and Germany
- operational challenges which includes the need for healthcare infrastructures capabilities and technologies that support the standardised collection and safe storage of data, enable faster diagnostic testing; and the training of medical personnel on understanding diagnosis and precision treatments.
Economic considerations of precision medicine
There are also long term economical concerns on how the business model for PM will play out. Technologies to sequence large amounts of genetic information remain expensive (although reducing). The cost of pharmaceutical research and development is inherently high and potential returns on investment are constrained by the smaller size of the treatable population, leading to high prescribing costs. Furthermore, there is the cost of investment in the capability and capacity to obtain precision diagnostics and deliver personalised treatment when compared to traditional approaches that may not benefit a proportion of the population treated.4 On the other hand PM could lead to a drop in research and development costs as the rates of failure and time taken to prove efficacy reduce, ultimately lowering costs for patients.5 We are at a tipping point where the evidence to underpin the business model is only now emerging. Realising the potential of PM will need to be underpinned by more precise economics.
What will drive change?
In order for these issues to be resolved, a more concerted effort is needed to address the concerns of each of the key stakeholders. Specifically more data is required to understand the clinical and long term economic benefits of PM. As more and more leading institutions begin adopting PM therapies, best practice methodologies should begin to emerge thereby increasing adoption. This in turn should improve patient access to PM at both lower clinical and economical risk than is currently the case.
Healthcare remains an industry in which innovation outpaces regulatory policy, reimbursement processes and clinical reform. PM has only just begun to scratch the surface of its potential. Over the last decade we have seen how the perspective of industry has changed and with the current penetration of PM in oncology, we are seeing key stakeholder views also evolve. Ultimately, it is long term data (clinical and economical) that will help shape the development and adoption of PM. Whether that evidence base is strong enough to promote implementation of PM outside of oncology, only time will tell. However, with the implementation of multiple government backed programmes over the last five years, such as the UK's "100,000 genomes project",6 key enablers for change are now in place.
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