A new way to create value in drug development

Unleashing Value

Biopharmaceutical companies engaged in the process of drug development need to become more adept at understanding the behaviour of both the patient and the healthcare system in which they participate.Companies that successfully incorporate a more human-centric approach will make better, well informed strategic decisions. The skilled application of multi factorial insight from clinicians, buyers and patients themselves will drive disruptive innovation and improve the performance of commercial strategy.

Phrases such as “patient centric”, “customer focused” or some variant thereof have become common parlance in the large pharmaceutical companies. Among smaller organisations in the biopharmaceutical drug development sphere, these terms are less ubiquitous with fewer companies actively thinking about the importance of this approach. Strasys’ insight framework (shown below) suggests that many companies ignore the wider opportunities available to identify unmet need in the context of patient journeys and the health system. As a consequence, there remain many under-exploited research opportunities from which biopharmaceuticals can identify transformative and disruptive innovations.

The challenge for our industry concerns how to leverage this information and drive additional returns from specific therapy areas. This paper elaborates on how that might be achieved and provides a framework for thinking about how leadership teams in biopharma should be implementing these insights into ongoing strategic decision making

Figure 1: Strasys’ insight framework for identifying unmet needs

Current 'human-centric' advocates are onto something

We see a drive to better understand patients coming from stakeholders such as patient advocacy groups and regulators. These organisations see additional value for the consumer when their views are integrated within the development process. However, it is also clear that when done properly, understanding and leveraging market insight is one of the principal determinants underlying the performance of commercial strategy. This is particularly true for the drug development industry. First understanding what matters most to patients, clinicians and buyers, then applying that insight intelligently is a vital component of clinical development, R&D productivity, commercial strategy and, in turn, regulatory approval.

Intangible insights

At present, there is no overall guide or universal consensus on how to ensure patient insight is used to inform and influence clinical development, commercial practice and the decisions biopharmaceutical companies must make about viable therapeutic candidates. Indeed, many executives that we work with struggle to translate the somewhat tenuous insight gathered from patients, clinicians and buyers into commercial strategy, the financial evaluation of portfolios, the NPV of current R&D projects, the in-licensing of new novel compounds or the commercial potential of partnership arrangements. How, for example, can a company consider variables such as…

the impact of disease on the individual’s life
the severity and chronicity of a disease
the broader health economy
the burden of living with and managing a disease

the impact of current treatment and care
costs in the healthcare system
patient goals, motivations, expectations and behaviours
HCP goals motivations, expectations and behaviours

all stakeholder perceptions of benefit and value
tolerance of harm in pursuit of benefit
attitude to risk and uncertainty

These factors will have a “make or break” impact on business models and future strategy. Moreover, they are the principal ingredients of successful decision making in the biopharmaceutical industry.

An accurate assessment and intelligent understanding of the market’s “unmet needs” serves as a basis to identify targets for interventions, spurring innovation as drug developers identify unique opportunities for therapeutic development. In addition, the in-licensing of novel compounds needs to be driven primarily by an evaluation of unmet need in order to help define commercial potential. Information about how patients assess available options for treating their disease can be used to inform a company’s decision-making about what projects would be a meaningful addition to the treatment space. Far too often consultants and executives in biopharma overlook these factors in their analysis and search for more, seemingly “concrete” variables upon which to base decision-making. However, real value is realised only at the point whereby an analysis of unmet need can be translated into tangible commercial aims.

The benefits of comprehensively and painstakingly understanding the patient experience are not limited to decisions about R&D investment and portfolio optimisation. Patient insight needs to be incorporated into the phased development of new drugs. Defining the “burden of disease” is a crucial component of developing meaningful endpoints that will matter to regulatory decision makers. This particular phenomenon is being driven by two emerging factors. Firstly, the declining compliance and retention of patients in clinical trials. CenterWatch has identified an increase in dropouts from clinical trials, currently averaging 15-40%, due, in large part, to a misunderstanding of the patient’s motivation for participating in the trial. Secondly, a concerted effort on the part of regulatory agencies to incorporate the patient perspective and quality of life impact into approval decision-making.

The future of regulation and pricing

The forces driving regulatory and pricing change continue to present growing strategic challenges to the biopharmaceutical industry. Fuelled by intensifying public scrutiny on pricing and rising health system costs, there is a growing legislative desire to safely accelerate the speed of new transformative medicines to market. To allow market access, health technology assessors are experimenting with new payment arrangements under collaborative legislation such as the recent ‘Fast track for cost-effective medicines’ scheme by the National Institute for Health and Care Excellence (NICE).

At the same time, the process of regulatory approval is also moving towards an iterative, evolving, and collaborative process among the sponsor, the company, and patients, with the aim of achieving a common understanding of benefit-risk in a patient-centric manner. These multi-track health ecosystem drivers place a requirement on biopharmaceutical companies, regulators and payers to develop approaches that result in win-win outcomes for all stakeholders. As such, companies seeking to advance drug development should not be confined to the clinical efficacy of a particular treatment, but should also consider acceptability, compliance and the impact of the treatment on the manifestations of the disease that most concern the patient, healthcare professionals and the health system.

This can be achieved by biopharmaceutical development becoming more adept at understanding the patient’s experience. This can be a messy and indeed uncomfortable process to initiate. It requires tinkering, creativity, curiosity, and an iterative approach. At its core needs to be a process of building empathy with the patient, clinicians and buyers in order to understand their pain points, perceptions of benefit and what they need or want.

The tools and methods needed to uncover this information will be unfamiliar to most biopharmaceutical organisations. In-depth ethnographical and explorative studies, engaging the patients in their everyday life so that an understanding of their use of current treatment options can be built and lessons from their lived experience of their disease can be learnt.

Efficacy at clinical trial is only half the game that biopharmaceutical companies play. We have seen numerous companies with great technology or promising therapeutic candidates fail because of an inability to properly comprehend or anticipate the underlying business model needed in producing commercially viable products. This often happens because of a lack of foresight regarding the wider unmet need that a therapeutic candidate seeks to solve. Developing long-term sustainable business models of the back of this provides the other half of a commercially successful approach.

New tools, new instruments and new conversations

New methodologies need to be established for accessing, analysing and evaluating the broader impact of market-led insight. At present, many are unsure of how to convert these insights into concrete commercial decisions. Strasys has already developed efficient and ready-to-use research tools, instruments and decision-making frameworks to rapidly evaluate these crucial variables and incorporate them into commercial decisions throughout the biopharmaceutical life cycle. These allow senior leaders to make better-informed decisions about where present and future value sits in their organisations. Moreover, these assets also provide new ways of communicating the vision and true value of biopharmaceuticals to all stakeholders.

Those companies which find ways to ‘connect the dots’ between their therapy areas and the drivers of sustainability and performance in their organisations will realise the greatest value in their portfolio.