Patients were involved early in STAR and at the beginning of LEAP. Facilitated discussion with patients allowed Alexion to gain an in-depth understanding of the Moments that Matter (MTMs), key experiences that significantly impact patients. Immersive Simulations of the stakeholder ecosystem enabled a 360° evaluation of the impact of living with a rare disease; these simulations included all stakeholders and allowed patients, caregivers, and patient advocates an equal voice to all others. Patients and/or patient advocacy group (PAG) leaders who participated in a STAR Therapeutic Patient Forum or LEAP Immersive Experience were invited to provide personal observations and examples of how Alexion shifted its culture from product-focused to patient-centred. Patients were asked to assess the value and limitations of these programmes and feedback was gathered to demonstrate the importance of understanding each patient’s experience. The patient authors may disseminate this manuscript as per their preferred formats and channels (including social media or websites) and, as appropriate, per Alexion compliance guidelines.

Although plain language communications are increasing, discussions regarding clinical data still largely centre on providing knowledge to HCPs, culminating in a treatment recommendation. Alexion realised that if the goal was to focus on the needs of the patient, drug development needed to be reframed around the question of ‘what does the patient need?’ This shift in viewpoint is aligned with the US Food and Drug Administration non-binding recommendations on ‘Patient-Focused Drug Development: Methods to Identify What Is Important to Patients’ [18].

Alexion conducted a gap analysis to identify the needs within the business and the processes required to be patient-centric during medicine development and commercialisation. The organisational structure was reconsidered, and new functions were created. The driver evolved from ‘what impact will our drug have on symptoms’, to ‘we need to understand what it is like to live with this disease, to engineer meaningful solutions’. This resulted in a cultural shift where the journey starts by actively listening to individual patients.

STAR is Alexion’s patient value incubator that helps embed patient-centricity into drug development. STAR engages global teams across indications early in the clinical development process and invites patients, caregivers, and stakeholders to partner with Alexion. STAR aims to identify MTMs and prioritise where they can create the greatest value.

STAR Therapeutic Patient Forums utilise four core pillars to deeply understand and empathetically appreciate the MTMs, co-create strategies and solutions, and implement plans (Fig. 2).

The foundation of the STAR Patient Value Incubator. HCP health care professional, MTM Moments That Matter, STAR Solutions To Accelerate Results for patients

Pillar 1: Learn and Align

A comprehensive internal knowledge assessment is conducted, and employees are encouraged to be agnostic of their function to drive an understanding of the disease. This establishes the baseline level of awareness for each patient’s lived experience across disciplines. This assessment is published into a ‘STAR Factbook’, a detailed outline of the experience of living with or managing a rare disease. The STAR Factbook is a key resource for Alexion employees.

Pillar 2: Experience and Prioritise

Learning from external stakeholders about their unique role within the patient journey. Patients, caregivers, and other participants take part in facilitated discussions to capture MTMs, which are characterised as an intense experience, emotion, and/or impact. These insights form the core of the methodology.

Pillar 3: Innovate and Test

The broader, in-depth, understanding developed in the first two pillars informs strategic choices facing the global early medicine strategy and clinical development teams. This creates an opportunity to highlight solutions that could create differentiated stakeholder value.

Pillar 4: Implement and Engage

Finally, the Alexion teams, patients, and stakeholders co-create ideas and plans to positively affect each patient’s lived experience and improve outcomes. STAR connects disparate initiatives and fosters collaboration between teams and patients early in development.

Alexion’s LEAP Immersive Simulations enable granular patient insight from late-phase development through to lifecycle management. These experiential interactive simulation sessions are designed to deeply immerse employees in each patient’s lived experience through the MTMs identified in STAR, providing a narrative for an indication as a medicine approaches late phase or regulatory approval.

During a LEAP Immersive Simulation, participants come to appreciate the diverse perspectives within the stakeholder ecosystem, including patients, carers, physicians, and payers through thoughtfully developed personas. Teams are presented with pivotal scenarios including (but not limited to) symptom onset, diagnosis, and patient access, facilitating real-time reflections and impactful solutions. LEAPs are conducted across countries, in different languages, and Alexion cross-functional country teams are encouraged to ‘take a LEAP’.

Participants engage in role play, focusing on multiple interactions or decision making aligned with real-world choices that a patient, caregiver, or stakeholder need to make. External participants include patients, caregivers, PAGs, and HCPs. Employees are encouraged to put themselves in the shoes of the patient and stakeholders and consider how they would feel in those pivotal moments. As the simulation progresses, participants work together to find solutions to challenges and prioritise strategies for country initiatives. By working collaboratively with all stakeholders at the country level, MTMs can be assessed in the context of the geographical setting and patient demographics, highlighting factors that could go unnoticed at the global level.

Patient and other stakeholder involvement in both STAR and LEAP are executed based on appropriate compliance processes. The key compliance considerations that we evaluated included transfer of value, fair market value, honoraria, and necessary policies, processes, and documentation. To ensure both programmes are managed and executed compliantly, Alexion established a patient engagement center of excellence. This team oversees all patient and advocacy activities in partnership with the legal and compliance functions.

Regarding patient engagement, initial outreach is made by Alexion patient advocate professionals who are the formal liaisons to patients, caregivers, and patient advocacy organisations. Once a patient has confirmed interest in participating in one of the programmes, contracting (based on country-specific legal requirements) is completed. Transfer of value such as honoraria is subject to country regulations and reviewed by governance functions. Payment to patients is based on country-specific fair market value guidelines. Engagement in these activities can be a short-term defined period (e.g., 3 months) or a long-term commitment depending on the patient’s preference to be involved in subsequent activities.

The patient’s preference is the key factor in determining whether they participate virtually or in person. In general, most patients engage virtually, via Zoom. Anecdotally, this virtual participation minimises potential burden related to travel and/or disruption of the patient’s healthcare regimen. Virtual participation mitigates the risk of transmitting infections to patients who may be immunocompromised, and also enables patients from different geographies who may speak different languages to participate.

LEAP simulations are developed in English. Country teams translate the simulation into local language prior to conducting their sessions. The output is stored in local language at the country level and shared at the global level in English allowing for an integrated view. For STAR, we utilise the simultaneous language translation function available within the Zoom platform. The meeting materials are translated into the language spoken by participating external stakeholders (i.e., patients, caregivers, physicians, etc.) using a translation software available to AstraZeneca/Alexion employees. The final output is developed and provided in English. The summary reports are developed for and provided for internal review purposes only, therefore are not sent to the external stakeholders.

A visual representation of how STAR and LEAP function, and the interplay between them, can be seen in Fig. 3.

The Alexion–patient partnership driving a new era in the pharmaceutical industry. LEAP Learn, Evolve, Activate and deliver for Patients, STAR Solutions To Accelerate Results for patients

After every STAR and LEAP event, data are gathered through an anonymous employee survey, which includes quantitative metrics and qualitative feedback via free-text fields.