How can 2020 be the year that R&D really delivers value, people get real about data, and pharma starts to develop even better medicines?

According to PWC, in 2020 the global pharmaceutical market is

predicted to more than double to $1.3 trillion. But with the world’s pharma industry having already invested over $1.36 trillion in R&D from 2007 to 2016, and with a predicted ongoing annual investment of $181 billion by 2022, some are questioning whether the medicines being launched will ever truly deliver value – to patients and to payers.

How might this question be answered in 2020? Andrew Rut, chief executive of MyMeds&Me, outlines the trends we might well see emerge in the New Year, which together could hold the key to unlocking the value of pharma R&D, and of the industry as a whole.

Data: quality, not just quantity

It is often remarked that data has become the new oil. But if this is the case, then (to coin a phrase) we clearly need better oil refineries. That is because it is not enough for the pharma industry to simply collect data and expect it to automatically produce better results. Indeed, much of what we think is robust data can in fact be quite misleading. With narrow indications and accelerated approvals the sample size of the trials is increasingly limited resulting in the

boundaries of efficacy and safety being very narrow.

Therefore, pathology data and adverse events during studies must be captured in a highly structured and systematic manner to ensure that shifts from what is expected in the disease cohort can be analysed for signals. Once on the market, is the default for regulators to demand risk management programmes focused on a better understanding of the benefit-risk profile of new medicines. This is where high value and high volume adverse event data comes to the fore, with its efficient and timely capture from the source essential. This serves to drive adoption of precision medicine and its economic consequences.

Precision medicine comes of age?

Good insights into disease and how it responds to treatment, enables precise targeting of interventions. With the advances in the various ‘omics’ , the potential of precision medicine over the coming year is hugely exciting.

The combination of therapeutic and preventive modality for disease that takes into account individual variability in genes, environment and lifestyle, so that medical treatment is tailored to the individual characteristics of each patient, is the cutting edge of medicine and healthcare. Indeed, some of the most promising cancer therapies currently in development are in the field of precision medicine.

However, implicit in this approach is not just better capturing of data, but (crucially) using that data to enable better and earlier decision-making at all stages in the R&D process. Medical teams must ensure that data is both trending in the ‘right’ direction but is also clinically meaningful versus established interventions. Precision medicine enables treatment of an individual and not simply the disease or illness.

Ultimately, therefore, I do hope it will be the New Year’s resolution of many drug developers to be more robust in their use of data in 2020. And, as alluded to, the reason I think they will is because of the economic consequences of precision medicine.

The economic consequences

Many cynics will of course grumble that whilst the impact on the individual patient of precision medicine can be high, so are the costs. This is a totally self-defeating and short-termist attitude. The days of blockbuster medicines are not over, but they have a much higher hurdle to overcome before reaching that status. The future lies in addressing individual patient needs in a way that delivers healthy return on investment and addresses disease areas considered perhaps less fashionable than, for instance, immune-therapeutics or cell and gene therapies. Indeed, with current returns on R&D investment of around 1.9%, the existing business model is clearly not working.

By enabling patients to monitor their treatment programmes in real-time and feed that data back to clinicians, and enabling this to be reported (securely and anonymised) at scale across much larger populations, there is the prospect of transforming not only adherence and safety, but to also feed back into the R&D process in a virtuous circle so that we can go on to create even better medicines and treatments.

So how do we bring it all together?

Better data through patient empowerment

There have been huge advances in the integration of AI and automation with healthcare. Indeed, only recently, Google’s move to buy Fitbit made many people wonder at the potential of integrating wearable tech with personalised health services. Completely understandably, privacy concerns have been raised, and we should of course debate those concerns openly.

But we should also turn that debate on its head.

How? I would argue that our starting point should not be asking what a tech company gets in return for access to data, but asking what the patient gets.

Indeed, the final trend that I am hopeful we shall see more of in 2020, is not simply more patient engagement, but true patient empowerment.

Patients need to have control of their data, and it needs to be made clear to them how they give consent and for what purposes. It cannot be beyond us to do that given all we have already achieved, and it feels as if we are at a tipping point in public opinion.

Return on investment

So as we approach the end of one year, I am very hopeful about what the next one brings.

I think there is the potential for greater strides in precision medicine, and I hope that this will be enabled and reinforced by better quality data collection, processing and its use in R&D decision-making.

This will not only be in the best interests of patients, but in the best interests of the pharma industry’s future growth, enabling it to better realise a return on the huge investment it continues to make in what remains such a vital area of human endeavour.