In every industry, across every sector, there are words and phrases that appear to have become ubiquitous. They are used so many times that their meaning seems to fade. How many times have we seen the word ‘unprecedented’ used to describe the current COVID-19 pandemic? Or heard governments refer to their approach to the ‘new normal’ after the crisis as being ‘led by the science’?
The pharmaceutical industry is not an exception, and ‘patient-centricity’ is an example of one of these ubiquitous phrases. Of course, this is with very good reason: every company is ultimately working to develop treatments for patients that need them. But when we examine research practices or approaches to investigating new therapy options more closely, it has to be said that we all can do better. We can work harder to ensure that patients’ real needs are better understood, and that patients are more involved and put front and centre of everything we do.
The patients of today are not the same as the patients of yesterday.
In our modern world, patients are more proactive, informed and involved in treatment decision making. This is a positive development, but it means that their needs are different too. And we must make sure we respond to this – ‘patient-centricity’ may be a term that is used a lot, but ensuring that we engage patients’ views in everything we do is arguably more necessary than ever before.
One thing is certain: approaches to oncology – whether through clinical trials, research goals or even our overall strategy for patient access and care – need to evolve to reflect this new landscape.
How have patients changed?
For so long, the core goal that has informed our trial endpoints is overall survival (OS), with longer life expectancy being the primary aim. While this still remains a key priority, increasingly we are also focusing on improving the quality of our daily life (QoL) for patients alongside this. People living with cancer, and their close family, want to be able to spend their time doing the things that matter to them, with the ones they value the most.
In other words, we all want to add life to our years, as well as years to our life, where possible. In both health and disease.
Thanks to the internet, growing health awareness and the relentless commitment of patient advocacy groups, patients are more empowered than ever before. They want to be involved at each stage of their treatment, throughout the disease pathway. This gives us more opportunities to move away from the traditional model where OS is the focus, and towards solutions that are matched to their real-world needs and find equilibrium that balances both QoL and OS. This balance should underpin our future research.
Better endpoints and bigger data
The earlier you can identify the cancer, the better the chances for an early intervention, and therefore better outcomes. This knowledge guides our research ambitions, where we apply what we call the ‘Go West’ principle. This means we don’t just focus our efforts on late stage disease, when people are at their most sick, but look to develop interventions at earlier disease stages – ie. ‘going west’ – to make a real impact.
It certainly poses key challenges, including the definition of new innovative endpoints in such ‘western’ disease stages that could accurately reflect a clear benefit for both the patient and the health system. Moreover, identifying patient-relevant endpoints or outcomes to ensure that QoL is a key focal point is something that we need to actively investigate.
In prostate cancer, we have already seen developments in trials incorporating early intermediate endpoints such as metastasis- free survival. As metastases are a major cause of symptoms, complications and death – particularly in prostate cancer – delaying the onset of metastases has the potential to translate to an increase in survival, but importantly also to a delay in symptom progression.
Beyond this, the use of intermediate endpoints can also move beyond OS and take into account things that OS does not. For example, symptomatic progression- free survival accounts for the real-world experience of the patient, while factoring in next-generation imaging into endpoints will also be important given the critical role this technology plays in diseases such as prostate cancer.
As we seek to advance science in order to develop new cancer treatments, these intermediate endpoints, with value on their own regardless of the existence of surrogacy, are becoming more widely accepted by regulatory and health technology assessment agencies, with alternatives including failure of cure, symptomatic progression, progression-free survival, disease-free survival, or minimal residual disease. Of course, adoption of alternative endpoints requires a wealth of supporting evidence, for which big data and artificial intelligence could play a significant role.
By being able to harness patient data already being collected on a daily basis through apps or wearables and analyse it in more precise and meaningful ways, we may be able to achieve a new gold standard when it comes to research and knowledge generation, achieving levels of granularity on data capture never seen before, and so a profound understanding and monitoring of the patient status on the target outcomes.
Janssen is exploring artificial intelligence (AI) to help analyse the granularity of the structured and unstructured prostate cancer patient level data collected in electronic health records (EHRs) in several European, Middle Eastern and African countries, integrating physician written text and notes and standard structured clinical records. This allows us to more accurately understand and describe the epidemiology, diagnosis, management, outcomes, and prognostic and predictive factors of response of people with prostate cancer across all disease stages. We can then identify correlations that may otherwise have remained hidden, and we can then incorporate the evidence into our own research. This Big Data/AI approach widens the scope of the scientific method as it’s been understood for decades, as knowledge generation can now start from pure unbiased observation, and not just from a pre-specified hypothesis that is limited by the existing knowledge at a given time.
We are also seeing innovations across the technological spectrum: from the Internet of Things, to predictive algorithms that select the right patient populations based on expected outcomes. All of these will help us improve how patient-centric our work is and, though it is inspiring to think about how much more we can do, it also puts things in perspective. Because that also means there’s so much more we have to do.
Adding life to years
Patient-centricity has always been a watchword of the oncology industry. But as our approach to the precision medicine era evolves, with alternative endpoints considered and new technologies embraced, I for one am hopeful that this phrase will come to mean even more.
This is a time of enormous change and with it brings enormous opportunity. We will have to be bold, but patients are waiting. These are patients who need our help to add life to their years, as well as years to their life – and striving to do this is non-negotiable.
Joaquín Casariego is EMEA Therapeutic Area Lead Oncology at Janssen
