The changing engagement paradigm: elevating patient voices for better care

The changing engagement paradigm: elevating patient voices for better care

By Suki Kandola, Envision Pharma Group

The pharmaceutical landscape is changing, with the patient perspective moving to center stage. The industry is increasingly recognizing the importance, and the benefits, of actively involving patients in shaping value and access (V&A) strategies. This changing perspective is driven by a deeper understanding of patient insights, experiences, and priorities, which are now considered essential for developing healthcare solutions. Despite the benefits to both patients and developers, incorporating patient voices in V&A strategies presents unique challenges.

This article discusses some key factors shaping the changing engagement paradigm and explores potential solutions for elevating patient voices in V&A strategies to deliver improved patient care.

Empowering the unheard voices

In patient advocacy, the larger, more prominent organizations associated with well-known diseases have been the dominant voices in healthcare discussions, due to their substantial resources and wide influence. Undoubtedly, their contributions have been invaluable in raising patient voices but their perspectives may not always encompass the full range of patient experiences.

Smaller patient advocacy groups, and even individual patients, offer unique and pertinent insights that are often overlooked. Smaller groups can represent communities including:

• Rare diseases
• Underserved populations
• Those with specific disease and treatment experiences

The experiences of these groups may not have been fully captured by larger organizations. These “unheard voices” can raise awareness of unmet clinical needs, highlight potential treatment gaps and offer invaluable real-world evidence about disease management and how patients interact with the healthcare system.

It has been difficult for developers to interact with grassroots and smaller advocacy groups, making their insights harder to capture, analyze, and act on. Reasons for this include limited resources, lack of formal networks, geographic dispersion, and concerns about regulatory compliance or the potential risks associated with working with less established organizations. However, these insights are equally as instrumental as those from larger advocacy organizations, particularly for developers seeking to differentiate their products and V&A strategies in increasingly crowded markets. Ignoring these perspectives can lead to a disconnect between the solutions developed and the actual needs of patients. By actively seeking and integrating unheard voices into V&A strategies, developers can:

• Discover authentic patient insights: These groups often provide more direct and unfiltered insights into patient experiences, needs, and challenges. Engaging with them allows companies to understand the nuanced perspectives of specific patient populations, which can inform patient-centered product development and marketing strategies.
• Uncover unmet patient needs in niche patient populations: Including identifying areas where innovation is required and discovering where existing therapies are falling short.
• Inform research and development: Designing research and clinical trials to develop new treatments that address unheard patient priorities and real-world experiences.
• Shape commercial strategies: Tailoring marketing, communications and commercial strategies using the insights gained from unheard patient populations.
• Demonstrate value and improve access: Targeting gaps identified and providing compelling evidence about the impact of new treatments on patient outcomes and quality of life to payers and providers.

Navigating the evolving regulatory landscape

The regulatory landscape is also transforming, with regulatory bodies embracing a more patient-centric approach. The U.S. Food and Drug Administration (FDA) [https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence] and the European Medicines Agency (EMA) [https://www.ema.europa.eu/en/news/use-real-world-evidence-regulatory-decision-making-ema-publishes-review-its-studies] are actively encouraging the collection of patient-reported outcomes (PROs) and real-world evidence (RWE) to support drug development and approval.

Compared with traditional randomized controlled trials (RCTs), RWE is collected from diverse data sources, including electronic health records, insurance claims, patient registries and wearable devices. Analyzing information from a wide range of sources offers a holistic understanding of treatment performance in real-world settings, accounting for diversity among patients, comorbidities and treatment patterns.

While RCTs remain the benchmark for establishing efficacy and safety, their controlled environments often limit the generalizability of results in real-world populations. RWE provides insights into long-term outcomes, comparative effectiveness and the impact of treatments on diverse patient groups. This means that RWE complements and enhances RCT data. Insights derived from RWE are essential for healthcare decision-makers, including payers, regulators and clinicians. These stakeholders require comprehensive data to make informed choices about coverage, reimbursement and treatment guidelines.

PROs can be used to capture patients’ perspectives on their symptoms, functional status and quality of life, offering a direct assessment of the impact of treatments from their unique perspective. The FDA and EMA emphasize the value of understanding the long-term impact of treatments from the patient’s perspective. By analyzing product effectiveness and capturing PROs, developers can gain a deeper understanding of patient experiences and foster shared decision-making between patients and healthcare providers. A comprehensive approach, combining RWE and PRO, can ultimately enhance V&A strategies and lead to improved patient outcomes.

The shifting regulatory focus is positive, benefitting both patients and developers. However, varying implementation and interpretation of guidelines across different regions and diseases can create challenges for pharmaceutical companies. Adopting a proactive and strategic approach can help navigate this complex environment, including:

• Early and continuous patient engagement: Involving patients from the earliest development stages helps ensure research and clinical trials are designed to meet their needs.
• Robust data collection and analysis: Using validated PRO and RWE collection methods, pharmaceutical companies can generate high-quality and patient-relevant data.
• Clear and compelling communication: Articulating the value of new therapies to regulatory bodies, payers and providers with a comprehensive picture of patient outcomes and real-world impact.

Elevating patient voices by embracing patient-centricity

Empowering unheard voices and the changing regulatory landscape underscore the growing importance of prioritizing patients in pharmaceutical V&A. By actively listening to diverse and overlooked patient voices and generating robust evidence of patient benefit, pharmaceutical companies can develop more valuable therapies, navigate the evolving regulatory landscape, and ultimately improve patient access to essential treatments.

Although the changing engagement paradigm presents many opportunities, it can be challenging for developers to incorporate into V&A strategies. However, embracing a strategic approach can help elevate patient voices and develop healthcare solutions that truly meet patient needs to ultimately deliver meaningful outcomes. This involves:

• Patient-centricity: Placing patients at the heart of every decision. Strategies should be meaningful, impactful and aligned with patient priorities.
• Evidence generation: Generating robust evidence through the strategic use of patient-centric data, which is vital for demonstrating value to stakeholders.
• Value communication: Effectively communicating the value of therapies, including developing compelling value propositions that resonate with payers, providers and patients.

One solution to help embrace patient-centricity is to collaborate with strategic partners with the experience and resources necessary to develop V&A strategies. Leveraging these partnerships provides access to specialized services and support on the journey toward patient-centricity. From developing and implementing comprehensive patient engagement strategies tailored to a company’s specific goals to guiding the strategic use of PROs and RWE to support value demonstration, clear and compelling V&A propositions can help showcase the unique benefits of therapies.

Fueling a patient-centric future

By embracing the changing engagement paradigm, developers can develop healthcare solutions that genuinely address patient needs. Taking a patient-centric approach to V&A strategies not only helps navigate the evolving regulatory landscape, it can also empower patient voices that have gone unheard. Ultimately, prioritizing patient voices enables companies to create solutions that resonate with patients, lead to improved outcomes and ultimately foster a more patient-centric healthcare ecosystem.

Suki Kandola is global head of commercial strategy, value & access at Envision Pharma Group.