Why Behavioral Science Is Needed On Day One To Support Patient-Focused Drug Development

By William Hind and Lisa Campbell

There’s an argument that pharma companies can never be truly patient-centric because the philosophy runs counter to their commercial interests. But that is to assume that drug developers are ambivalent about their ability to transform patients’ lives. The middle ground is more likely, and to achieve a better balance life science leaders have an opportunity to employ behavioral science in pinpointing exactly what patients want from a new therapy, or incarnation of a drug, beyond quality, safety, efficacy, and affordability.

It is no coincidence that momentum is growing around behavioral science’s role in promoting the latest patient breakthroughs to stakeholders right across the healthcare ecosystem, from the earliest stages of drug discovery and development. This is about performing formal patient research into the subtlest as well as more overt needs of target populations, and capturing real scientific evidence that will inform everything from modes of treatment delivery to commercial as well as patient and clinician communications.

Last year, Astellas Pharma established a dedicated behavioral science consortium to help understand the importance of human behavior for patient outcomes and the benefits of using behavioral science to improve them. The Consortium’s raison d’etre, through scientific research, is to uncover some of the most common patient and caregiver challenges with a view to delivering more of what matters to them.

Health organizations, too, are developing a keen interest in behavioral science. The World Health Organization (WHO) recently appealed for experts in the field to join its global Technical Advisory Group on Behavioral Sciences, as part of research into health behaviors and the potential global impact of behaviorally-informed health interventions.

Informing Clinical Development Planning And Design

Taking a more holistic view of a proposed new or repurposed product is part of the premise of patient-focused drug development (PFDD), a movement that strives to bring the rounder patient experience to the fore from the early stages of a treatment’s design and development.

Patient-focused considerations will be critical to the take-up of novel treatments like cell and gene therapies. They also can be subtler, linked to how a treatment is administered.

For decades the accepted approach to treating severe epilepsy^1,2 in children was to administer a muscle relaxant — by means of a suppository — within the first 10 minutes of a seizure. Not only is such an application tricky to achieve during an epileptic episode, it also introduces social sensitivities given that convulsions could occur at school, requiring intervention by a member of staff.

So when a drug company (then ViroPharma) introduced an equivalent product (Buccolam) that could be given orally, via a syringe, the more socially-acceptable mode of delivery offered attractive differentiation.³ It followed that identifying and communicating this effectively would be the key to maximizing the new product’s take-up.

Uncovering The Subtler Drivers Of Medical Decisions

In the case of novel therapies, which lack a wealth of historic precedence and may seem a step too far in pursuit of the promised clinical benefits, harnessing behavioral science to identify precise patient priorities makes strong commercial sense. Armed with these insights, drug design and strategic teams can improve the design of relevant early research and inform narrative creation. In addition to solidifying the opportunity to transform the patient experience, capturing and quantifying patient preferences from day one will maximize the product’s market success.

PFDD offers particular value in the rare disease space, where industry, regulators and broader stakeholders share an appreciation for having patients define the most relevant endpoints for clinical drug trials (ultimately defining the marketing authorization claim). Behavioral science can help showcase that value to developers – of having patients shape clinical development programs.

Indeed, the 2023 regulatory guidance series developed by the U.S. Food and Drug Administration (FDA) aims to enhance the inclusion of the patient’s voice in medicinal product development and regulatory decision-making,⁴ paving the way for changes in drug development practices globally. This will be important if advanced therapies such as cell and gene treatments are to have maximum impact and commercial success.

Disrupting Established Thinking, Challenging Fears

A major potential barrier to uptake in the case of new, breakthrough therapies is patients’ fear of the unknown – of putting something into the body that could be there for 15 years or more, with long-term effects which are hard to predict for now. Seeing those specifics reflected in early data affords the biopharma industry a powerful opportunity to address such fears head on in their product design, and in associated messaging to financial supporters, healthcare providers, patients, and their caregivers. The treatment could mean an end to a lifelong medication regimen which up to now has compromised the patient’s overall quality of life. Leaving aside the huge resource savings for the health provider, that is a massive patient win in terms of overall experience and hope for the future.

Supported by the earliest patient evidence, drug development pathways can be more optimally streamlined, with the assurance that no opportunities are missed along the way (that may not be recoverable later in clinical development).

Without a clear and timely view of patient priorities, on the other hand, medical communications will inevitably default to the clinical evidence alone (linked primarily to the drug’s efficacy), potentially failing to  speak to the factors that really matter to patients in their day-to-day lives.

Coming back to modes of drug delivery as a simple but important example, it turns out that these present as a factor in a whole range of treatments. Now that the potential of GLP-1 agonists (medicines originally used to treat type 2 diabetes) has been recognized for lowering obesity, new consideration must be given to whether clinically overweight populations will tolerate regular injection. Building such considerations into clinical studies would have helped discover and address this issue.

About The Authors:

William Hind is CEO of specialist life sciences consultancy, Alpharmaxim, which he founded in 2001. With over 35 years’ experience in the pharmaceutical industry, Hind helps cross-functional biopharma teams communicate effectively with healthcare professionals and medical affairs professionals about new medicines and treatment regimens.

Lisa Campbell, senior director at SSI Strategy, a global life sciences consultancy, served as a medic (working in obstetrics and gynecology in the U.K. NHS for 13 years), and then for the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) for a decade in its clinical trials unit. SSI Strategy is a boutique management consulting company with particular expertise in cell and gene therapies.