Pharma companies are embracing patient centricity in drug development more than ever before. Over the last decade, leaders in the industry have championed patient engagement, putting it at the heart of commercial strategies. It’s a hard task to get right though, as ICON’s Chris O’Toole explains, but the rewards can be huge on both sides.
That’s not to say that the industry is doing all it could do to when it comes to listening to patients throughout the development, regulatory, and post-approval life cycle of a medicinal product. It has however increasingly moved away from the view that the end-user of a medicine is a physician, rather than the person who is actually sick.
The moral and ethical case for incorporating the patient voice is beyond question – why would the person taking a new drug not be involved in that process after all? – but from a pragmatic perspective there are also considerable benefits to the drug developer.
Selecting the best design for a clinical trial programme – the most expensive part of drug development – is a key benefit of fostering two-way communication between developers and patient, along with improving patient recruitment, adherence to medication and retention.
Talking to people with a disease or medical condition can also tease out what matters to a patient, not simply what is scientifically provable. That in turn raises the chances that a drug makes it through development and onto the market and – once available – actually delivers real benefits to those it is intended to help.
It’s not simply about actively engaging patients to participate in the clinical development process though. The industry has made great strides forward in engaging with patient advocacy groups to really understand the daily challenges faced by patients and aid recruitment, for example.
It is also making greater use of real-world evidence – post-launch registries and epidemiological methods that gather information about how the drug works in real life as opposed to in a tightly controlled clinical trial. At the same time, there has also been a shift towards more use of decentralised trial protocols and the use of digital technologies – telemedicine for remote consultations, wearable sensor devices and e-diaries that collect data in real-time – to reduce the burden on patients posed by having to travel to investigation sites and potentially even enhance the data collected.
Support for patients needs to be an ongoing theme, and digital technologies can play a big part here, providing for two-way communication.
So, with all those clear benefits and tools to hand, what holds back companies as they try to take a patient centric approach? Challenges can occur internally due to organisational, operational, and personal investment goals, according to O’Toole.
Some companies have been able to generate a culture where the patient perspective is incorporated at a very high, operational level and – while others have been slower to do so – most are at the least planning and piloting initiatives.
In some cases, companies haven’t been able to coordinate the existing expertise they have in-house, with different projects operating in silos and in some cases unaware of what is going on elsewhere in the same organisation.
He cites one case involving an unnamed executive at a big pharma company who was given the role of person champion and sent out a company-wide email asking people with involvement in patient centricity and engagement to a meeting.
A related challenge is learning how to deal with a whole range of different data – primary and secondary, qualitative and quantitative research, and real-world evidence – and bring it all together in one place where it can be deployed to deliver real insights.
Companies are sometimes also overlooking health literacy, in other words how well a patient can understand medical information and failing to implement simple measures like making sure all materials directed at people are in plain language.
“We’ve got a whole industry talking way above people’s heads and the vast majority of people thinking, “I don’t want to look silly, so I’m not going to ask,” remarks O’Toole.
Meanwhile, a company can have all the altruistic goodwill in the world, matched by good business sense and the impetus to co-create and co-develop a new therapy with the patient in mind, but still find their multi-year patient engagement investment fall short due to organisational factors.
“There’s a compensation factor that gets in the way,” according to O’Toole. “If a manager has got a certain budget and is asked to invest in a patient-centred initiative that might not show a return until three to five years, the temptation is to put that spend into samples or advertising that delivers a quicker ROI. This decision has an immediate impact on them and fits the 18 month promotion window better.”
O’Toole’s Commercial Solutions team advocates an approach predicated on seeking out all available resources to maximise the windows into the patient’s life, including both internal and external sources. It also advises developers to capture all relevant data points along the patient journey – both quantitative and qualitative.
Research methodologies are evolving and as interest in gathering patient perspectives has grown, qualitative research – interviews with patients on entry and exit from a trial, for example – is now being encouraged by regulators and health technology assessment (HTA) bodies to enrich quantitative clinical results.
Developers may also be working with multiple external agencies, generating market research, patient insights, and patient-reported outcome data for example, that will do the job well but then hand it over for the sponsor to try to bring it all together and interpret the findings.
ICON’s approach – which O’Toole says makes it stand out in the sector – is to bring as many data sources as possible to the table.
Through a variety of tools, the company’s commercial consulting unit incorporates the patient experience and journey in their process of gathering information and evidence to craft strategy in a variety of areas, such as scientific, path to market, regulatory, and payer/reimbursement.
ICON’s strength in this area is drawn from this multi-faceted access to patients and people living with medical conditions, which provides powerful insights to their customers, he says.
The commercial unit can provide insights on pricing, market access and reimbursement, and health economics outcomes research, at the scale that accompanies being one of the largest contract research organisations in the world.
Moreover, ICON now has an additional source of US patient-level data from electronic health records and insurance claims, through Symphony Health, which came from the acquisition of PRA in July. Coupled with that is its association with the non-profit Mapi Research Trust, a unique asset that includes a library of more than 40,000 clinical outcome assessments and hundreds of patient-reported outcomes questionnaires.
“These provide distinct and varied insights into the patient and can be helpful in planning commercial activities, as well as clinical programmes,” according to O’Toole.
The best development plans that bring together nonclinical, clinical, regulatory and commercial expertise – with input from the patient throughout – can improve efficiencies, cut costs, shorten timelines, and increase the chances of success for a new programme.
Bringing all these data points together demands leadership and cross-functional collaboration to ensure buy-in to patient centred strategy – particularly if pharma companies want to achieve this at an operational scale.
“There has to be a lead and that has got to come from the top,” according to O’Toole. “There’s got to be a mandate and a direction, but that accountability has to be pushed outwards so that all the teams are aligned.”
Finally, another obstacle to going beyond the pilot phase in engaging with patients may be that it is simply hard to quantify a return on the investment, even taking into account the fact that reimbursement and regulatory bodies are increasingly looking for patient centred approaches in their deliberations on new drugs.
Recent data shows however that patient engagement activities – particularly those with the potential to reduce the need for clinical trial protocol amendments and/or improve trial enrolment, adherence, and retention – can add considerable financial value to a development programme.
One study (1) led by the Clinical Trials Transformation Initiative (CTTI) in the US has attempted to put a value on the benefit of engagement by coming up with a way to calculate the net present value (NPV) and expected net present value (ENPV) for a clinical project, taking into account cost, time, revenue, and risk.
Based on a premise of $100,000 spent on an engagement programme, the model calculated a 500-fold return on the investment in ENPV – tens of millions of dollars – that was equivalent to accelerating a pre-phase 2 project by two and a half years, and a pre-phase 3 project by 18 months.
To put that in perspective, that return is higher than would be accrued, say, if a sponsor carried out studies in children to get an extra six months of market exclusivity.
They add that according to their model “even if the benefits of increased probabilities of technical and regulatory success were far smaller, simply the NPV and ENPV increases from the time savings alone far exceed the investment.”
As the dynamics of patient centricity in drug development and clinical trials continue to evolve, pharmaceutical companies have the opportunity to put in place the resources, structures, and business practices to help make patient focus actionable.
(1) Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project; Therapeutic Innovation & Regulatory Science 2018, Vol. 52(2) 220-229
Chris O’Toole is vice president of ICON’s Commercial Solutions business, which provides pricing, market access, reimbursement, global medical communications, health economics and outcomes research, strategic regulatory, and real-world evidence services. Prior to joining ICON Chris was chief partnership officer at Health Monitor Network where he led relationships with patient advocacy organizations, medical societies, and the pharmaceutical industry. He also served as co-chair of the Point of Care Communications Council (PoC3), whose mission is to advocate for the effective use of the point of care channel to advance health and healthcare outcomes. Chris’ experience also includes senior management positions with a number of medical marketing and communications consultancies. Earlier in his career he held sales and marketing positions in the international pharmaceutical industry.
ICON is a global provider of consulting, and outsourced development and commercialisation services to pharmaceutical, biotechnology, medical device and government and public health organisations. ICON’s focuses on the factors that are critical to clients – reducing time to market, reducing cost and increasing quality – and its global team of experts has extensive experience in a broad range of therapeutic areas. ICON has been recognised as one of the world’s leading Contract Research Organisationsthrough a number of high-profile industry awards. With headquarters in Dublin, Ireland, ICON employs approximately 38,000 employees in 151 locations in 46 countries. Further information is available at www.iconplc.com.