How Patient Experience Data Informs 5 Business Strategies

How does Patient Experience Data inform the 5 strategies throughout a medicine's lifecycle?

More than decade ago, the European Patients' Academy for Therapeutic Innovations EUPATI published the Toolbox on patient involvement throughout the medicine's development continuum. This brilliant tool helped many stakeholders with understanding of critical transition/decision-making points where patient insights and advice could be a driver and proof of value to be potentially delivered by a new asset. As a part of working groups and several cross-sectoral think-tanks I reviewed those points many times and must admit their practical relevance and good reference for other documents and educational tools, such as PFMD How To Guide for patient engagement in the early development and preclinical phases, Medical Affairs Professional Society (MAPS) Medical Affairs Textbook, and many others.

Having addressed the questions "How?" and "Where?", we have started to focus on "What?", and it becomes apparent that Patient Experience Data (PED) is addressing this question with full scale of its diversity and heterogeneity. We do collect PED in different formats and modalities, design evidence generation activities, embed patient reported and clinical outcomes measurements as endpoints in clinical trials, initiate patient preference studies, support registries and review electronic health records, conduct advisory boards with patient experts and join community advisory boards; finally, we are piloting the methodologies for patient experience mapping (PEM) within the healthcare pathways or generic patient journeys. However, it is important to understand when the PED has the biggest strategic impact, or, in other words - how could PED inform the strategies within the medicine's lifecycle.

There are 5 strategies where the PED impact is obvious and proven by many published/presented case studies and good practices across the industry:

Integrated asset development strategy driven by Target Product/Value Profile (TPP/TVP)

1. Clinical Outcomes Assessment (COA) Strategy

2. Clinical Development Strategy

3. Integrated Evidence Generation Strategy

4. Launch Strategy/Launch Excellence

Something new? I don't think so. All strategic elements and contents are well defined by the corporate functions - from early research teams to medical affairs. The only question is whether PED has been collected, analysed, then informed the decision making and planning there? Well, the situation is different from department to department, from company to company, from asset to asset. PED usage is inconsistent and its impact has not been well-proven or even appropriately documented. For example, the patient advisory board on study design or informed consent form review by patient experts are pretty common activities across R&D, or patient surveys or questionnaires are the parts of integrated evidence generation plan developed by medical affairs. At the same time, patient advisory board or consultations on TVP attributes, discussions around early risk/benefit profile or most meaningful outcomes assessment measurements aren't common and massively under-explored.

So, let's briefly discuss WHAT could PED specifically bring to each strategy?

1. Ideally, Integrated Asset Development strategy should be considered as overarching long-term strategy supporting whole development continuum to address desired characteristics/attributes of target product/value profile (TPP)/TVP), that's why its co-creation with patients from the start is the key factor for future success. There are several steps to establish strong partnership with patient communities/patient experts and start discussions about early research priorities, unmet needs, burden of a medical condition/conditions and expectations/incremental value to be delivered by new treatment (find more details in PFMD How To Guide https://pemsuite.org/How-to-Guides/Early-Discovery.pdf). Therefore, even at this early stage, the deep search, collection, generation, sorting-out and categorisation of PED, then holistic patient experience journey mapping (PEJM) are required. As an output. there will be the set of TPP characteristics or patient-driven attributes of TVP, which R&D teams plan to achieve throughout an asset development continuum. Team may also have the first blueprint of an asset's risk/benefit profile and key outcomes meaningful for patients to be measured.

   
   

2. Clinical Outcomes Assessment (COA) strategy - another strategy to be informed by PED earlier rather than later, which is currently not in place for many cases across the industry. Clinical development teams usually start thinking about the most appropriate clinical and patient-reported outcome (PROs) as study endpoints when developing a design for phase II or phase III. It is too late, as teams try to select general or condition-specific already validated tools, which may not be meaningful for people who live with the condition, nor completely reflect the incremental value of an innovative treatment. There is no time to develop and validate new tools, so teams explore such approach again and again. However, if COA strategy is being co-developed with patient experts well before the start of any clinical development programme, it allows to look precisely into the most burdensome symptoms and key areas of impact to be addressed first, discuss them together and suggest possible ways to measure them. PFMD PED Navigator tools https://pemsuite.org/ped-navigator/ might be helpful for this process to avoid any missed opportunities. Most importantly, regulators are keen to start discussions around COA strategy as early as possible.

   
   

3. Clinical Development strategy has traditionally been supported by patient advice and insights over the last decade, however, there are limited cases of systematic use of PED and PEJM as well as proof of their real impact at strategic rather than operational level of studies' execution. For example, one of the patient survey's findings was insufficient support of study participants with study-related information. To address this gap, the study sponsor and CRO developed additional materials for study participants and their families, and diversified communication channels (operational level). More strategically, it may require development of the unified communication platform and site management tools for several studies, condition-specific educational resources or AI-driven digital solutions to navigate patient journey. We shall remember, that from the patient perspective, participation in a clinical study is a complex experience, first and foremost medical/treatment-related, but also psychological, social, infrastructure-related and other types of experience. Therefore, patient experts should be involved at the stage of strategic clinical development planning and decision-making - to address many questions about design, timeframes, recruitment and retention, logistics, patient support services and materials, feasibility assessment, recommended sites and investigators. The formats of standing advisory panel, community advisory board or patient steering group could be considered as optimal to inform a clinical development strategy.

   
   

4. Integrated Evidence Generation strategy. Going forward, at the later stages of medicines development continuum, evidence gaps become more visible prompting to develop an integrated evidence generation strategy and aiming to understand broader aspects of a medicine's use and regulatory requirements. A good evidence generation strategy should include PED generation activities and PEJM development or upgrade, if such had been developed earlier. PED and PEJM could address many questions at this stage: real-world service gaps, pathway disruptions, key barriers and challenges with the condition management, patient preferences, satisfaction with the treatment, diversity of patient population, co-morbidities, co-medications, tolerability, and other aspects. More diversified and multiply sources of PED, highly heterogenous data and insights require technical/digital solution for their collection, consolidation, analysis (including AI-driven), update and data presentation/reporting. There is going to be an area of highest priority for the industry over the coming years.

   
   

5. Launch strategy and launch excellence. Historically, patient insights, pathway analysis and patient journey research have been in focus as a part of launch preparation. Market research teams planned and conducted ethnography, focus groups with patients and caregivers, social listening projects and other activities to better understand specific condition management patterns, sometimes pretty fragmented in terms of dedicated therapeutic group or health technology. Taking continuity of PED generation and PEJM updates throughout the medicine development continuum, this could add more value and better contribute to launch strategy with holistic understanding of patient experience living with a condition. Such approach requires closer cross-functional collaboration and exchange between the teams on the PED and PEJM leverage points. Alongside global/generic PEJM, the nationally adapted and translated PEJMs should be developed in close partnership with national patient communities. This will help the national teams with launch readiness.

To summarize, PED generation and PEJM development create unique value and opportunity to support strategic planning and decision-making from the start of a medicine's research and development - towards medicine's maturity. This work is impossible without long-term, trusted partnership with patient communities and patient experts. With pretty focused and currently fragmented scope of patient engagement/co-creation at the clinical development and later stages, we miss the opportunity to prevent or mitigate risks, better tailor unmet patient needs and address their expectations, improve outcomes which are really meaningful for patients and their families. Therefore, early co-development of target profiles and integrated asset strategy as well as COA strategy driven by PED and PEJM are key points to be considered by the industry and other healthcare stakeholders.

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