Case Study | Rare Disease

Mid-size Pharma Accelerates Early Clinical Trial Engagement Of Patients With A Rare Neurological Disease


A mid-size pharma company sought an experienced advisor who could help them engage patients for an open-label extension clinical study in a rare neurological disease.

Client Challenge

A growing pharmaceutical company needed to facilitate early engagement of patients and accelerate recruitment for an open-label extension clinical study in a rare neurological disease. To accomplish this, they envisioned a patient-centric process of product development and clinical trial progression to include the patient perspective in the study’s protocol.

Our Approach

To address the challenge, Alira Health’s team proposed to establish a European Patient Advisory Board (PAB). To create it, we focused on building relationships with selected regional, national, and international neurological disease patient associations and their key patient representatives.

The goal was to map, select, and coordinate with the right patient associations; review and co-create the trial protocol with selected patient associations; and propose insights-backed actions.

Recruited patient representatives for the PAB:

We mapped patient associations in Europe at the regional, national, and international levels to find and contact eligible organizations. We selected patient representatives from the appropriate associations based on strategic criteria that contributed to fulfilling the activity’s objectives, such as reach, geographical presence, and language.

Delivered discussion guide and educational material:

We developed educational materials for patient representatives to acquire basic awareness about clinical trials and the specific goals of the activity before the meeting. We also developed a discussion guide in consultation with the client to ensure the PAB covers all topics of interest.

Managed the PAB end-to-end:

We organized a preliminary meeting with patients to review and clarify the success of the educational materials and the goals and structure of the PAB itself. Alira Health team members coordinated and moderated the PAB in collaboration with the client.

Analyzed insights and proposed next steps:

We collected and analyzed key patient perspectives and feedback on the clinical protocol (e.g., study design, trial feasibility, burdens on patients, language clarity). Then, we discussed the extracted information with the client to better understand the patients’ experience and their input on the protocol, allowing for the eventual adaptations and modifications to the protocol with the patients’ needs in mind. We then delivered actionable recommendations supported by patient feedback.


We helped the client make the open-label clinical study more accessible and feasible to patients with rare neurological diseases by incorporating the patients’ viewpoints. This accessibility enabled the accelerated recruitment and retention of patients into the study. The patient association mapping provided a panoramic view of all patient associations focused on rare neurological diseases with their key patient representatives.

The large-scale mapping helped the pharma client prioritize a selected number of patient associations for the PAB in this project. It also generated immense value by providing insights into key patient groups for future engagements. Partnerships with these associations helped to expand the client’s reach and involvement in clinical trials, and they intend to continue their engagement with the selected patient associations.

Key Success Factors

Rigorous mapping of all European patient associations and relevant stakeholders in addition to developing an active PAB was fundamental to the client’s goals. Our broad approach to mapping, rather than focusing only on the top players, proved indispensable to finding the right patient representatives and, ultimately, developing actionable recommendations backed with insights that the client sought. Finally, the ability to leverage in-house clinical operations expertise on PROs and endpoints played an essential role.

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Business Impact

Alira Health helped the client make the rare disease clinical study more accessible and feasible to patients through a Patient Advisory Board. The patient relationships generated helped to expand the client’s reach and involvement in clinical trials.

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