Why behavioral science is needed from day one to support patient-centric drug development

William Hinde and Lisa Campbell

There is an argument to be made that pharmaceutical companies can never be truly patient-centric because their philosophy is at odds with commercial interests. But that assumes that drug developers are ambivalent about their ability to change patients’ lives. A middle ground is more likely, and to achieve a better balance, life sciences leaders have an opportunity to leverage behavioral science to identify exactly what patients want from new treatments and medicines beyond quality, safety, efficacy, and affordability.

It is no coincidence that from the earliest stages of drug discovery and development, there is growing momentum amongst stakeholders across the healthcare ecosystem around the role of behavioral science in driving the latest patient breakthroughs – by conducting formal patient surveys about the most subtle and more obvious needs of target populations, and collecting real scientific evidence that informs everything from how treatments are delivered to commercial needs and patient-clinician communication.

Last year, Astellas Pharma launched a dedicated Behavioral Science Consortium to understand the importance of human behavior to patient outcomes and the benefits of using behavioral science to improve patient outcomes. Reason for existenceThe aim of is to uncover, through scientific research, some of the most common challenges for patients and caregivers and provide them with what matters most to them.

Health organizations are also taking a strong interest in behavioral science: The World Health Organization (WHO) recently invited experts in the field to participate in its Global Technical Advisory Group on Behavioral Sciences as part of a study on health behaviors and the potential global impact of behavior-based health interventions.

Clinical development plan and design information

Taking a more holistic look at proposed new or repurposed products is part of the premise of Patient-Centered Drug Development (PFDD), a movement that aims to bring a more comprehensive patient experience to the forefront from the earliest stages of treatment design and development.

Patient-centric considerations are essential for the introduction of new treatments, such as cell and gene therapies, and more nuanced considerations may also be required in relation to how the treatment is delivered.

An approach that has been accepted for decades to treat severe epilepsy^1,2 In the case of children, the method used was to administer muscle relaxants via suppository within 10 minutes of the seizure, but this is not only difficult to administer during an epileptic seizure, but also poses social problems, such as the possibility of convulsions occurring at school, which requires intervention by teachers and other staff.

So when a pharmaceutical company (then called ViroPharma) introduced a comparable product (Buccolam) that could be administered orally via syringe, the more socially acceptable method of administration became an attractive differentiator.³ Therefore, identifying and communicating this effectively proves to be key to maximizing adoption of new products.

Uncovering the nuances of medical decision-making

For new therapies that don’t have much historical precedent and may seem like a step too far to pursue promised clinical benefits, leveraging behavioral science to pinpoint patient priorities can make huge commercial sense. Armed with these insights, drug design and strategy teams can improve the design of relevant early studies and inform narrative creation. In addition to solidifying opportunities to transform the patient experience, capturing and quantifying patient preferences from day one can maximize a product’s market success.

PFDDs are particularly valuable in the rare disease space, where industry, regulators and broader stakeholders have come to recognise that patients define the most relevant endpoints for clinical drug trials (ultimately defining the claims for marketing authorisation) and behavioural science can help demonstrate that value to developers. patient Develop clinical development programs.

Indeed, a 2023 regulatory guidance series produced by the U.S. Food and Drug Administration (FDA) aims to strengthen the inclusion of patient voices in drug development and regulatory decision-making.^Four Paving the way for change in global drug development practices, which is crucial to ensuring advanced therapies such as cell and gene therapies achieve maximum efficacy and commercial success.

Disrupt existing thinking and challenge fears

A major barrier to the introduction of groundbreaking new therapies is patients’ fear of the unknown – the fear of putting something in their bodies for 15+ years with long-term effects that are currently difficult to predict. Seeing these details reflected in early data provides the biopharmaceutical industry with a unique opportunity to address these fears head-on in product design and associated messaging to funders, healthcare providers, patients and caregivers. This treatment could mean the end of a lifelong medication regimen that has historically undermined patients’ overall quality of life. Aside from the enormous resource savings for healthcare providers, this is a huge win for patients in terms of overall experience and hope for the future.

Being supported by the earliest patient evidence allows the drug development pathway to be more optimally streamlined, ensuring that opportunities are not missed along the way (that may not be recoverable later in clinical development).

On the other hand, without a clear and timely understanding of patient priorities, medical communication will inevitably rely solely on clinical evidence (mainly related to drug efficacy) and may miss the factors that really matter in patients’ everyday lives.

A simple but important example goes back to the method of drug administration, which has been found to be a factor in a variety of treatments. Now that the obesity-reducing effects of GLP-1 agonists (drugs originally used to treat type 2 diabetes) have been recognized, new consideration must be given to whether clinically overweight people can tolerate regular injections. Incorporating such considerations into clinical studies should help to uncover and address this issue.

About the author:

William Hind is CEO of Alpharmaxim, a boutique life sciences consulting firm he founded in 2001. With over 35 years of experience in the pharmaceutical industry, Hind helps diverse biopharmaceutical teams effectively communicate with medical professionals and healthcare administration professionals about new medicines and treatments.

Lisa Campbell, Senior Director at global life sciences consulting firm SSI Strategy, was a physician (13 years in obstetrics and gynaecology in the UK NHS) and then spent 10 years in clinical trials at the UK Medicines and Healthcare products Regulatory Agency (MHRA). SSI Strategy is a boutique management consulting firm with expertise in cell and gene therapy.