PRIME PATIENT

Oncology R&D: the patient insights we’re still missing, and how to catch up

Imagine you are a pharma executive working in oncology R&D. The landscape is rapidly becoming more competitive and overcrowded. You are under pressure to bring solutions now, and for your entire career, you have been taught that things must be done a certain way. Patient engagement is viewed by many as a nice-to-have rather than a necessity, and some may even consider it a passing fad or commercially unfeasible – in any case, you’re already satisfying regulators and journals with lay summaries and a few engagement projects.

In this pressured environment, are you going to be drawn towards taking a risk by giving patients more power to influence trials? Some may say yes, but many would feel more inclined towards traditional, comfortable R&D methodology. After all, pharma are the experts in getting drugs through trials and onto the market, right?

In fact, the old way is dying out. Evidence shows that, despite historical success, the oncology R&D pipeline is unsustainable, not only becoming increasingly difficult and costly, but also fast approaching relative stagnation. The numbers are striking – in just the 13 years following 2003, R&D costs were estimated to have doubled, and with failure rates as high as 90% and time to market stagnating at ~12 years for the past three decades, the R&D innovation need is urgent. Healthcare systems and patients are suffering the consequences, as drug prices and times to market increase, and access to treatments is consequently hindered in the context of an ageing population carrying an ever-rising chronic disease burden. Indeed, increasing costs pose pharma’s biggest R&D challenge today.

Luckily, the tools required to escape from this downward spiral already exist – enter patient engagement. However, despite R&D being the area of pharma most widely accepted as benefiting from the patient voice, few trials are truly patient-centric. Fortunately, patients can provide valuable input across the drug development process, and the costs of accommodating this are low within the scale of investment required for R&D overall. If drug developers, regulators, payers and patient associations can collaborate to arrange deeper and earlier engagement that is structured, well-defined and consistently applied, a triple win for pharma, patients and society is certainly achievable.

Early patient input leads to improvement

Clinical trial planning is an essential stage for patient input. Patients can provide insight on unmet needs, protocol design, priority endpoints, trial conduct, data interpretation and dissemination, risk-benefit analysis, risk management, and treatment value as relevant to payer discussions, all of which can strongly influence decisions made.

Patients who have participated in previous trials can also point to pros and cons of prior studies, which provides a useful benchmark for new trials. Patient input on protocol design is particularly vital, as the design of a trial may exclude target patients before the study has even begun. For example, sponsors may not be aware that the energy levels of patients with long-term fatigue may be strongly affected by the time of day, and thus the time of study appointments (or lack of predetermined clarity around this) may make the trial inaccessible.

There is no substitute for asking patients directly. Both sponsors and physicians have been shown to identify different desired outcomes to patients, and while outcome preferences may overlap, oftentimes patients will suggest a key outcome that was not previously suggested by any other party. Notably, some patients may prioritise quality of life over the typical endpoints of progression-free or overall survival. While some may consider patient perspectives as easy to imagine or simply common sense, researchers do not know what they do not know, and unexpected revelations are far from incidental.

As well as engaging with patients directly, it is also critical that companies sustain long-term relationships with these patients and clearly communicate steps taken in response to insights provided. Not only does this protect against engagement appearing tokenistic, but it also embeds a sense of purpose for the patient, rewarding them for sharing their personal perspectives and encouraging further engagement with industry.

Incorporating the patient voice at early stages brings numerous benefits to R&D. These can be immediate, such as higher chances of ethical approval, and also longer term. In 2017, AstraZeneca hosted a clinical trial simulation to facilitate patient input prior to study commencement. Consequently, 60 recommendations on how to improve the experience were provided by patients, which saved AstraZeneca significant time and costs compared to if those amendments had been made further down the line. In addition, the trial was less of a burden on patients, which would make the trial design more appealing to patients approached for recruitment if it were to be adapted for future studies.

Expanding beyond the AstraZeneca simulation, patient insights can be included in study planning for as little as €10,000 – a very low cost compared to that of running a multicentre trial. Indeed, this is a small price to pay for the protection these insights afford against errors which could delay a trial by several months – not to be sniffed at considering the harshly competitive nature of the oncology pharmaceuticals market.

Furthermore, it is in pharma’s interest to engage with patients to ensure they are health- and trial-literate so the most informed insights as possible can be provided. With a third of older adults in England shown to struggle with comprehending basic health-related information, only 12% of Americans possessing health literacy, and various studies demonstrating patient misunderstanding of research aspects including trial aims, side effect risk, and likelihood of personal benefit, the need for pharma to step up on patient education is evident. Furthermore, if patients enter a trial with unrealistic expectations, they are more likely to become disappointed or frustrated and consequently drop out.

Sponsoring attendance to the European Patients’ Academy (EUPATI) Expert Training Course, which educates patients and patient advocates in medical R&D to the level of becoming qualified partners in drug development, is a gold-standard intervention, but solutions can be more low-key. Building a simple platform through which lay health and (particularly) trial information may be efficiently shared with patients by HCPs and using literacy screening findings to inform targeted patient education content would greatly complement existing patient trial materials. By investing in patient education, pharma can expect to see returns in the depth of insights gained from involving patients in study design.

Overall, consulting with patients is essential in ensuring that trials are relevant and beneficial to them, while also refining treatment value, reducing later-stage protocol amendments, and saving time and costs.

Greater success from recruitment to product launch

Evidence suggests that if clinical trials enrol patients at higher rates, the speed of treatment evolution and corresponding improvements to cancer population outcomes increases. Unfortunately, 80% of trials face difficulties with recruiting the target number of participants, and approximately 30% of patients who are recruited drop out. However, by making trials more comfortable for and relevant to patients, implementing patient insights can speed enrolment (and thus time to regulatory submission) and improve retention.

One study found that integrating patient centricity into oncology trials resulted in an average recruitment time saving of 37% (reduced time = reduced costs), also noting that interestingly, given the significant benefits of innovation in cancer therapy, oncology trials appeared to be less commonly innovative (with innovative trials defined as patient-centric, adaptive, precision medicine- or RWD-based) than trials in neurology or rare disease.

Patient centricity will become an even more valuable asset to trial enrolment in future, as recruitment pressures are projected to rise with the advent in precision medicine and consequent shrinkage of patient pools. Recruitment competition is already high, with 38% of all compounds in the visible 2019 preclinical pipeline being oncological (an increase from 23% in 2000), and only half as many breast cancer patients as would be required to fill active clinical trials diagnosed each year.

Traditionally, recruitment has been limited by the geography of investigator sites, which deprioritises patients and limits the generalisability of results. By going directly to patients and identifying investigators close to them, researchers may be able to improve enrolment and retention as patients may have to travel less to reach the investigator site. In future, the patient experience will become an increasingly key differentiator within the competitive trial landscape, exerting even more influence on recruitment and retention.

Methods of making trials more appealing to patients include a hybrid design, whereby patients can provide data from home (e.g., via wearable devices) as well as in the clinic, and arranging transportation for patients when clinic visits are required (e.g. for scans). In addition, access to a clear, single point of contact for patients before, during and after the trial would facilitate positive relationships between patients and sponsors, ease the addressing of any concerns or questions and reduce the overall mental burden of trial participation, potentially reducing dropout rates.

It is logical that if pharma companies were to make consistent, collaborative efforts to integrate patient preferences into their study designs, more successful recruiting (e.g., through greater trial appeal, positive brand recognition and word of mouth among patient networks) and keeping patients enrolled throughout would be much more likely than if these efforts were not made. In the longer term, integrating patient insights can benefit drug developers beyond recruitment and retention, as the chances of providing regulatory and reimbursement decision makers with compelling patient-relevant evidence are significantly higher. Moreover, drugs developed using patient-centric trials have been shown to not only have a 19 percentage point increase in phase II and III likelihood of launch compared to drugs developed in standard trials, but to also be more likely to be adopted by payers.

Patient engagement is key to staying competitive

Finally, the most long-term benefit to pharma of patient insights in oncology R&D is the future of the drug company. The pharmaceutical landscape is changing, and companies will do well to work with, rather than against, this evolution.

In the flourishing patient economy, where patients are increasingly empowered and equipped to collaborate with industry, deepening and expanding upon patient engagement will be essential for drug developers to be considered relevant and trustworthy in an industry increasingly demanding of value demonstration and transparency.

Companies that do not advance in this direction may be left behind in years to come. Indeed, drug developers competing in an increasingly crowded oncology space will need to make the most out of technological innovation and meet evolving relationship and value expectations if they hope to remain competitive. Patient engagement may be new to some, but expertise in this field is taking strong roots. Undeniably, the evidence is there to support patient centricity as a standardised business essential across all subsectors of pharma.

The ROI

To revolutionise pharma as a whole, the return on investment/engagement cannot be communicated solely within R&D. Importantly, the ROI/E is not only identifiable across, for example, commercial and market access subsectors, but also clearly measurable. Hence, financial rewards await companies if they have the foresight to persist with the long-term investment, as patient centricity ultimately results in better products and outcomes. For commercial teams, it may be intriguing to see that money spent on patient engagement could produce more than a 500-fold return in investment via the consequent reduction of protocol amendments and improvements to trial recruitment and retention. Furthermore, 86% of pharma executives were shown in a 2015 survey to agree with the statement “a focus on patient centricity is the best route to future profitability”. Looking at market access, patient support programs have been shown to improve adherence by 29% and cut disease-related and all-cause medical costs by 35%.

Moving forward

That’s all very well, but how does one navigate the complexities of engaging patients directly? Patient engagement frameworks (yes, even covering compliance), toolkits, case studies, guides and recommendations are arising and evolving all the time, and Prime Global Medical Communications will soon be launching PEP Talks as a best practice training resource and safe space where pharma can rapidly get up to speed with the how and why of patient engagement.

Thus, dismissive avoidance of patient engagement – e.g., “It’s not compliant/legal for us to talk to patients directly” (note: it’s promoting to patients which is not allowed), “It’s better for HCPs to do any patient work”, or “We don’t know where to start” –  no longer holds the power it once did. It’s time for a pharmaceutical culture shift, whereby company-wide infrastructure is established across the industry to anchor the patient at the heart of all business strategy. After all, drug developers will be left behind if they neglect to jump on board.

The bottom line: patient engagement in oncology R&D, although implemented to a small degree, remains a vastly untapped resource and opportunity to secure a triple win for pharma, patients and society.

About the author

Olivia Kersey

Olivia Kersey is a patient engagement strategist (with a particular interest in health and trial literacy) contributing to Patient360 Insights programmes within the Prime Patient Center of Excellence at Prime Global. Patient360 is a service that delivers long-standing patient insights and engagement strategies focusing on the ABC – access to medicine, behavioural change, and care support. This service highlights how to overcome obstacles to creating a standard of patient engagement care and implementing this standard into medical guidelines. Patient360 also helps to bridge the patient-pharma relationship and support business sustainability.

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