Building With, and For, Patients [STAT Summit 2026 Review #26]

Report 26. Building With, and For, Patients

Part I. English Version

1. Event / Date & Time

Event: Building With, and For, Patients | Sponsored by argenx

Conference: STAT Summit 2026

Date & Time: October 15, 2026 | 11:40 AM EDT

2. Participants

Speakers

- Sandrine Piret-Gerard — Chief Commercialization Officer, argenx

Moderator

- Torie Bosch — Editor, First Opinion, STAT

3. Summary

Patient Co-Creation: argenx described involving patients in decisions from research and trial design through commercialization.

Individualized Treatment: Experiences of people with myasthenia gravis informed a preference for flexible dosing adapted to fluctuating symptoms.

Beyond Product Approval: Patient input can help shape service delivery, education and treatment access as well as clinical endpoints.

4. Main Discussion

Co-Creation Across the Development Lifecycle

Piret-Gerard distinguished formal patient-centric language from active co-creation with patients. She said argenx seeks patient input from initial research decisions through clinical development and commercialization. This includes recurring patient panels rather than collecting feedback only after a product is launched.

Piret-Gerard contrasted a broad statement of patient centricity with actual patient participation in design decisions. She described conversations with people living with myasthenia gravis, a neuromuscular disorder whose symptoms and limitations can fluctuate substantially between and within individuals. Patients characterized their disease as highly individual and asked for dosing that could adjust to changing circumstances rather than a rigid schedule. Such a request complicates protocol design because regulators, investigators and manufacturers must still define safe and reproducible use. The company said it incorporated this input into the development of its FcRn-targeting treatment, with flexibility reflected in product information. The example illustrated patient engagement as a source of design requirements, not simply a communication activity after a product is developed.

Pierre-Girard described “co-creation” as inviting patients to shape decisions from early research through commercialization rather than asking for feedback after a product has been designed. Her example was generalized myasthenia gravis, an autoimmune neuromuscular disorder whose symptoms fluctuate across individuals and across time. Patients explained that they needed flexibility in treatment rather than a rigid universal schedule. That message challenged the predictability preferred in conventional drug-development protocols, yet the company considered it sufficiently important to incorporate into development thinking. The distinction matters: patient involvement was not presented as a branding exercise but as an input that can alter the treatment regimen and the outcomes that researchers decide to measure.

Myasthenia Gravis and Flexible Dosing

In developing efgartigimod (Vyvgart) for myasthenia gravis, the company learned that symptom patterns vary substantially from one patient to another. Patients preferred treatment schedules responsive to symptom fluctuations rather than rigid fixed regimens. Incorporating that preference increased development complexity but helped align treatment design with lived experience.

The discussion extended the same logic across research, clinical studies and commercialization. Patients may value the ability to plan work, family life and treatment visits as much as a measured improvement on a clinical scale. For rare diseases, small populations can have a broad range of experiences, and a narrowly defined trial endpoint may fail to capture the treatment burden relevant to individuals. Piret-Gerard described repeated patient-panel conversations, rather than a one-time survey, as the means by which preferences were brought into the process. This approach still requires scientific evaluation and appropriate regulatory review; patient preference alone cannot establish efficacy. The practical point is to integrate the voice of the people who live with a disease before determining how a therapy will be delivered and supported.

The company used the experience with efgartigimod, marketed as Vyvgart, to illustrate a patient-focused product strategy. Pierré-Gérard linked clinical benefit to how treatment is delivered and accommodated in a person’s daily life. In rare and heterogeneous diseases, endpoints that look similar on average may conceal substantial differences in individual burden. Repeated patient panels can therefore identify practical requirements that may not emerge from clinician-led assessment alone, including the timing of dosing, frequency of treatment visits and what a meaningful period of symptom control actually means to a patient. These were descriptions of argenx’s development philosophy, not a comparative demonstration that one consultation method improves efficacy.

Commercialization as Part of Patient Care

The discussion broadened patient participation beyond trial protocols to education, treatment logistics and access. Piret-Gerard argued that commercialization decisions also shape whether a medicine can be used successfully in practice, particularly for patients with chronic rare diseases.

argenx outlined a growth strategy linked to additional conditions and indications rather than a single product launch. Piret-Gerard mentioned a Vision 2030 ambition of reaching 50,000 patients, a scale she described as substantial in a rare-disease business. She referred to favorable trial news in myositis and anticipated Phase 3 information in another program. These were company statements about development progress and objectives, not guarantees of future approval or access. The discussion also touched on expanding therapeutic expertise from neurology into adjacent immune-mediated conditions. Building relationships with patient organizations can help identify unmet needs but must be accompanied by clinical testing, physician education and sustainable patient services as indications broaden.

Commercialization was portrayed as a continuation of the development relationship. Patients may face uncertainty about recognition of symptoms, specialist access, reimbursement, injection or infusion logistics and transitions between care settings. Those barriers can determine whether a therapy that performs well in a clinical trial delivers real-life value. The speaker argued that patient services and information should therefore be designed alongside evidence generation rather than left to a separate postapproval team. Such an approach may also yield information about burdens missed by trials, although those observations need structured methods before they can be used as reliable comparative evidence.

Further Development Across Indications

Commercial delivery was treated as part of product performance. A medicine that requires repeated administration may create burdens that patients did not anticipate during a trial; support teams need to explain the regimen, help with navigation and understand barriers beyond the biological disease. The speaker connected these services to a broader notion of co-creation, in which patients remain participants in decisions as treatment reaches practice. She distinguished this from claiming that all patients want the same type of flexibility or services. The session suggested a practical industrial model for rare diseases: collect specific lived-experience evidence, adapt development where feasible, validate the resulting regimen and continue learning after launch.

Pierré-Gérard described a longer-term ambition to serve more people with rare autoimmune diseases and mentioned an aspiration to reach 50,000 patients by 2030. In this context, the number was a company goal, not a current count of patients treated. She also discussed ongoing clinical development in additional indications and results expected from Phase 3 programs. The strategy links an existing therapeutic mechanism to broader populations, while preserving attention to differences among indications. Expanding access without diluting patient involvement will require consistent engagement mechanisms, appropriate trial endpoints and scalable support programs, not only additional regulatory approvals.

5. Insights

Patient-Informed Development: Repeated patient input can improve the relevance of dosing schedules, endpoints and support services.

Implications for South Korea: Korean rare-disease programs could examine structured patient panels from protocol development through treatment introduction.

Collaborative Care Ecosystem: Industry, clinicians and advocacy groups can co-design practical treatment pathways that reflect varied patient needs.

Report 26. Building With, and For, Patients_eng.pdf

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