Patient Knowledge Bias: How diverse patient insights can help drive pharma success and improve healthcare access

The world of #patientengagement has moved at pace over the last 5 years.? It was not long ago that the general discussion with many pharmaceutical companies was around why patient insights should be included in R&D and commercialization.? Return On Investment (ROI) was often a topic of conversation, with views varying over whether patient engagement really made a significant difference to getting a product approved, or increasing patient access once approved.

The conversation in R&D is changing though, as most companies now see that patient engagement is fast becoming a requirement due to various guidelines being launched across the world, and the general expectation that patients should be involved in their healthcare journeys.? ?For example, demonstration of Public & Patient Involvement (PPI) is now often required for a clinical trial to be approved to start in some countries.?

Pharmaceutical companies are also seeing that there is an expectation that patients are included across the full life-cycle of patient care, particularly when communicating with the public and Healthcare Professionals (HCP’s), submitting to authorities to demonstrate need and value, building relationships with patient groups, and as part of launch and country-level expansion.

Whilst the standard inclusion of patient perspectives is a big step forward, the challenge with being directed to include patients in guidelines, or not having trials approved due to lack of patient engagement (or even credibility issues by not including patient perspectives), is that there is a potential for patient engagement to become a check-box exercise where the minimum is undertaken to be able to say that patient input has been included.

Usually the fastest way to meet this minimum is to engage with a small number of patients, often identified through patient expert groups or disease specific patient organisations.? The pharmaceutical company gets fast access to patients that are often highly knowledgeable in relation to their disease, the way treatments are developed, and the healthcare system that they are part of. The added benefit of working with these types of patients and patient groups is that it can help build credibility and relationships for when the product is launched.?

However, relying solely on these “more knowledgeable patients”, while convenient, can lead to an incomplete understanding of how a treatment will work across the full spectrum of users. Broader patient perspectives enable insights into different pathways, real-world experiences, and communication preferences, ultimately helping to improve access and support successful launches that reach all patients effectively.

Expanding the patient pool of insights to be more representative not only meets the expectations of regulators, but can also offer a lot of added value to the pharmaceutical companies that choose to engage and listen to more patients.?

Risks of Relying on Informed Patients Only

Informed patients — those familiar with medical terminology, treatment protocols, or the healthcare system — are often asked to provide detailed feedback on their experiences. However, limiting research to only these patients can skew results, as this group may not represent the needs or preferences of the general population. These patients are often more proactive, health-literate, and informed about their treatment options, which can make their experiences atypical compared to those who may have less experience navigating the healthcare system.

These patients may be more likely to understand complex treatment regimens, handle side effects, or follow specific medical advice, whereas other patients might find the same processes challenging or confusing. If feedback focuses too heavily on these informed patients, crucial details about treatment accessibility, ease of use, and support needs may be overlooked, leading to a product that’s less effective or accessible to the broader patient population.

Additionally, informed patients might underreport or better manage side effects that could be more problematic for those who are less familiar with their conditions or treatment options. This discrepancy can result in treatments appearing easier to manage than they actually are, leading to challenges post-launch when the treatment reaches a broader audience.

The Value of Diverse Patient Insights

Gathering perspectives from a wider group of patients helps capture a fuller range of experiences and challenges. Patients bring diverse backgrounds, varying levels of health literacy, and different degrees of experience with managing their conditions. Broadening the sample pool allows R&D, launch and commercial teams to see how treatments function across different demographics, highlighting challenges that might be invisible in a more specialized sample.

These broader insights are invaluable when refining communication strategies, understanding real-world side effects, and designing treatments that genuinely accommodate varied needs. By including those less familiar with medical processes or those who may find treatment instructions challenging, companies can ensure that their products are suited to the everyday realities of all patients, not just the most knowledgeable ones.

Supporting the Full Range of Patient Journeys and Treatment Pathways

Patients navigate healthcare in unique ways depending on factors like prior experience, condition severity, lifestyle, and support networks. By including more representative patients, research can capture this range of patient journeys and identify different treatment pathways, ensuring that products align with real-world usage and not just a single subgroups model of treatment.

For instance, patients who are new to managing a chronic condition may prioritize simplicity and ease of understanding in their treatment regimen. In contrast, more experienced patients might value flexibility and customization. With a broader number of patient, researchers can gain insights into what works best across these varied scenarios, creating treatments and guidelines that are more universally accessible and effective.

This approach also highlights the types of support that may be needed during and after the launch, as those with less healthcare familiarity may require additional resources, instructions, or follow-up. Addressing these needs during R&D and during the pre-launch phase increases the likelihood of smoother, more widespread adoption once the product is available.

Designing Treatment Options that Suit Diverse Needs

When patient research includes patients with different levels of knowledge, pharmaceutical companies gain a more balanced view of treatment preferences and adherence factors. ?Feedback from patients less familiar with medical terms or dosing schedules may show ways to make treatments easier to use and understand.

Broader insights can guide product design decisions, such as whether a drug should be available in multiple formats (e.g., pills, injections) or if instructions need to be simplified. These modifications increase the likelihood that patients will follow their prescribed regimens, ultimately leading to better health outcomes. By taking into account the preferences of patients from many different backgrounds, companies can create products that are inclusive, accessible, and suited to diverse healthcare needs.

Effective Communication that Reaches All Patients

A wide range of patient perspectives also informs better communication strategies that account for different levels of health literacy. ?Better informed patients may be comfortable with medical terminology, but many others may need simpler language, visual aids, or additional guidance to understand their treatment. Larger numbers of patients with diverse backgrounds reveal what patients need in terms of clarity, support, and reassurance, which is essential for reaching as many people as possible.

Clear, accessible communication reduces barriers to care, especially in communities where medical knowledge may be limited. It also helps minimize misunderstandings or fears that may arise from inadequate or complex instructions. By understanding and addressing these needs, pharmaceutical companies can develop communication plans that promote equitable access and help patients and healthcare professionals make informed choices about treatment options.

Ensuring Launch Success with Broad Data

A successful launch depends on the treatment's performance with a wide variety of patients. Relying on specific patients alone risks creating a product that may not perform as well across the general population, potentially leading to adjustments, additional research, or unforeseen challenges. ?A larger, more inclusive sample strengthens confidence that a product will satisfy the diverse expectations of real-world patients.? Pharmaceutical companies that prioritize perspectives from a broad patient group may reduce the risk of post-launch setbacks.

Ways to Gather Fast, More Representative Patient Perspectives

Like other omnichannel approaches, patient insights can be gathered in a holistic and structured way.? ?Patients that are highly knowledgeable of their disease, which may include those actively involved in patient organisations, are an important stream of perspectives. Afterall, a percentage of patients will be in this category and their views will enable pharmaceutical companies to effectively communicate, access and support patients that fall within this category.? ?However, the majority of patients for many indications may not fall into this category, and so additional approaches should be developed to enable greater representation of perspectives.?

Many patients now have access to the internet, and are often utilising social media platforms in their day to day life.? This can be a gateway to quickly reaching many more patients, and to provide some balance to perspectives gathered from expert patients or those from other patient organisations.? Larger-scale quantitative digital surveys can be used as a fast way to increase representation and understand experiences across the wider patient population.? There will still be other groups of patients with differing experience in certain demographics and communities that are maybe not as easily accessible via digital routes, but community based approaches can also be also be used to gain trust and gather their perspectives.?

In addition, there are opportunities to utilise other repositories of patient data, such as patient healthcare records and insights from other stakeholders such as healthcare professionals and claims databases.? These will give one view of the patient experience and journey, and not all countries have this kind of data easily available.

The objective is not to gather perspectives from every patient, but to not become reliant on the experiences, needs and challenges of one small subset of patients that may not be reflective of the wider population, or use datasets that only provide one viewpoint.? ?By focusing on only one subset of patients or data, results may not be representative of other groups of patients that form the majority, and could even lead to biased data that negatively impacts patient access, support and trust. ?

Conclusion

There is a clear need to engage with, and understand the perspectives of, patients that have a high degree of knowledge of their disease and those that are part of patient organisations. However, relying solely on these patients can leave gaps in understanding that could compromise a treatment’s effectiveness, accessibility, and appeal. ?

Including a broad sample of patient perspectives in R&D, launch and expansion planning —across different levels of experience and familiarity with healthcare—enriches insights into patient journeys, pathways, and preferences. This approach allows for treatments that align more closely with real-world patient needs, supports effective communication, and can ultimately contributes to a smoother and more successful launch to enable greater patient access. ?

Whilst it may be difficult at the moment to represent every patient demographic and community, the objective should be to at least have data representative of the majority of patient types.? Taking steps to engage and gather insights from more than just a handful of patients should be a next step that is already readily achievable. ?By integrating a wide range of perspectives, companies can create products that provide meaningful, accessible benefits for all patients, which in turn provides greater value to pharmaceutical companies.

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About me: Having started my career in academic research and teaching at Guys Hospital, London over 30 years ago, I then moved into the commercial world of Clinical Research Organisations and Healthcare Consultancies. I have a strong interest in looking at ways to more effectively and efficiently use real-world data to better support and involve patients, both during the development of new treatments and once launched. I am part of the leadership team at DontBePatient Intelligence, which focuses on the design of patient and healthcare research and in accessing patients both quickly and in appropriate numbers to enable decisions to be made using scientifically robust methods on representative data. Follow me or the DBPi page for more insights every week.