The pharmaceutical industry’s focus on health outcomes has become a global movement. As payers negotiate reimbursement contracts based on patient outcomes and the concept of risk-sharing evolves, capturing the right data – from clinical studies, clinical practice and real-world experiences – has become critical. The pressure to quantify value with demonstrable outcomes is being felt at every part of the product life cycle. Alongside it, the ongoing challenge of increasing shareholder value creates, on the face of it, conflicting strategic goals for corporate pharma. Where do you focus: profit or patients? Income or outcomes? The purists will argue that the latter should drive the former – but it’s a chicken-and-egg situation.
So how is the industry faring? It’s fair to say there’s room for improvement. An estimated two-thirds of drugs fail to meet revenue expectations in their first year. This could suggest that pharma’s self-proclaimed focus on outcomes is betrayed by a continued reliance on traditional models of commercialisation. Conversely, it could imply that the value companies’ products demonstrate is often misaligned with the needs of patients and payers. Certainly – notwithstanding the fact that product revenues are perhaps not the best measurement of patient outcomes – it confirms that the industry is still some way from establishing an outcomes-based operating model that chimes with the realities and dynamics of modern healthcare.
So how can pharma accelerate its journey towards outcomes-focused models? One approach might be to harness the never-ending proliferation of data and use real-world evidence (RWE) research as a more prominent driver of product development and commercialisation. While the use of RWE is not new, the industry could push the envelope of current business practices by being more proactive about bringing insights from a broader range of stakeholders into early clinical development and beyond. This article explores three ways in which pharma could better demonstrate that its products and services are improving patient outcomes – focusing on transformative R&D models, rethinking medical communications and taking simple steps to capture crucial RWE.
Best-in-class operating model
Ramita Tandon, executive vice president, ICON Commercialization
& Outcomes, says that current market dynamics present an opportunity for biopharmaceutical companies to develop a novel best-in-class operating model that responds to stakeholders’ rising evidentiary demands and moves beyond traditional norms to transform R&D productivity. “This new operating model must employ real-world intelligence to demonstrate improved comparative efficacy and cost-effectiveness to payers and rapidly innovate differentiated products. Two additional strategies support the feasibility of this new approach; patient-centric innovation – which can deliver transformative gains in the operational efficiency of studies – and vigilance for emergent technologies, which are pivotal for generating real-world intelligence and forging stronger connections with stakeholders. The model will open a number of opportunities. Under the new model, RWE is interpreted for clinical teams to align product development with specific unmet patient needs and hidden value opportunities. Insights reach R&D that ordinarily take years; beta-blockers, for example, found expanded use to control hypertension only after retrospective analysis of four decades of use in heart attack patients.”
Ramita says companies must take the lead to establish connectivity between the disciplines that analyse RWE and bridge the silos of clinical and commercial groups throughout the product life cycle. “Convergent teams can creatively deliver value by mapping key decisions against RWE from electronic health records, observational research, mHealth devices, data from patient communities, and emergent predictive big data initiatives,” she says. “This strategy facilitates deeper payer and patient-centric insights into optimal target indications, opportunities to improve care management and product feature profiles that align to potentially non-obvious patient populations. An R&D model enhanced by real-world data ensures momentum to market. Early insights, the nuances of a drug’s value to relevant stakeholders and the potential future scenarios that may influence stakeholders at launch can enable manufacturers to collect the appropriate evidence package at the right time and through the most efficient strategies. Furthermore, as formulary decision-making begins more than a year in advance, a strong prior communication strategy is critical to success.
Ramita believes the changing commercialisation environment is ‘a call to action’ for the industry to rethink the traditional R&D approach. Industry, payers and patients must, she says, partner to operationalise real-world evidence as intelligence to inform product innovation. “For both emergent and established companies, the R&D path can be simplified and enhanced by strengthening the connections between independent R&D and medical affairs organisations. Ultimately, the industry must rethink how to accelerate products to the clinic and attune them to patients’ most pressing needs.”
RWE: small can be beautiful
Alison Currie, senior clinical payer specialist, WG, believes the need to satisfy both the regulators and reimbursement agencies with evidence that demonstrates efficacy and value has pushed pharma increasingly towards ‘big data’. “There’s an emphasis on conducting huge clinical trials with statistically robust endpoints. But some diseases will never achieve such scale. In those diseases there’s a strong argument for conducting very small studies to determine whether what was discovered in a rigid clinical setting can be replicated at a local level. For example, by using a therapy in 30 patients in a particular Trust we can see what happens in practice; what do patients say and do? What effect does the therapy have on them? Are they returning to their GP or being readmitted to hospital? This information can help Trusts commission services, providing real-world evidence of the impact a treatment has on local patients and services. We need to accept that while RWE may relate to a tiny local population, it can be high quality and generate learnings that could translate in other localities. By definition, the very nature of ‘real-world’ is that the way practice is conducted in Manchester may be very different to how they do it in Truro. But there are simple ways of measuring it and adapting it accordingly.”
So how can pharma companies progress? Alison thinks they should carry on improving the data they collect for large-scale reimbursement processes – and this could involve early engagement with reimbursement authorities to ensure they’re collecting the right data to demonstrate value. “But to support that, and to differentiate themselves, they should consider doing some local work that’s perhaps more like an audit than a study. By agreeing audit points and monitoring how patients and clinicians are behaving, it’s possible to collect local data that indicates real-life practice. We can make it easy for hospitals to collect the data through simple questionnaires that are completed in clinic or at the end of a patient consultation. This enables pharma companies to give trusts something they can roll-out themselves – and everyone benefits from the data. Then, when that product is being reviewed in Truro or even in France, we can share data of how it’s being used in its real-world population. It also means you’re allowing for the subtleties of the local healthcare system.”
Measure for measures: improving metrics
Dennis O’Brien, CEO, Lucid Group, believes there’s an enormous gap between where we currently are with measuring outcomes for patients and where we need to be. “Today’s metrics are weak. Some medicines do a good job of demonstrating cost-effectiveness to enable market access. That’s great, but what next? In terms of services, metrics tend to focus on whether patients like them. We create thousands of patient leaflets because market research says patients like them. We produce symposia for HCPs and then measure them by asking if they enjoyed the meeting. We invest millions in iPad tools for the clinic to help patients and physicians, only to evaluate them by confirming whether they used them or liked them. These are all weak metrics. As we dream of a future where wearable tech allows us to track all aspects of patients’ behaviour, a sobering glance at the present shows we’re nowhere near utopia.”
To get there, says Dennis, we need to focus on behaviour change. “We need to understand what behaviours we need to change and the barriers that prevent progress. Once we understand this, we need to create interventions that help patients overcome those barriers and exhibit the behaviours we need. If we’re picking the right behaviour, we should naturally be improving outcomes. The question is: how do we know what the right behaviour is? We need a different methodology.”
The clues, believes Dennis, can be found in health economics. “There’s a growing evidence-base that pharma can harness to drive better outcomes. Careful evaluation of health economic literature can highlight the interventions that have had an impact on patients’ lives and inform what we should be doing in clinical practice. All we need to do then is work backwards to identify the behaviour and build interventions designed to change it. The metrics take care of themselves.
“We may be some way from leveraging data from wearable technology – but what we do have is a wealth of outcomes-based information in health economic literature that can be used to develop our strategic approach to education and services. This can only help us develop interventions that we know will make a difference to outcomes.”
The final outcome
So where do we go from here? Developing a sustainable outcomes-based operating model is a strategic challenge that starts right at the very top of pharma. If companies truly want to be patient-focused, their development of brands and services needs to start at that point – with the patient – and work backwards. Typically, companies appear torn between short-term financial gain and making a different to patients’ lives. Claims to be patient-centric are often contradicted by strategies that focus purely on the numbers and chase quarterly targets as a barometer of success. It’s understandable, but it has implications right down the line in terms of metrics and patient outcomes.
Success depends on companies defining – and remaining true to – their overall purpose. If, when you launch a product, your ambition is to maximise sales, the development, research and commercial questions will be very different to what they need to be if your ambition is to demonstrate the drug makes a different to patient lives. Defining that focus early on – and anchoring to it throughout – will shape your model, dictate your metrics and help you pursue the demonstrable outcomes that will drive commercial success.
It’s the million-dollar question: where’s your focus – income or outcome? If it’s the latter – and it should be – it’s perhaps time to think a little differently.