Patient Advocacy Collaboration to Enhance HCP Education for Rare Metabolic Disease

Challenge

Rare metabolic diseases are often difficult to diagnose due to vague and overlapping symptoms, leading to significant delays in diagnosis and treatment. Non-specialist healthcare professionals (HCPs) frequently lack the awareness or tools needed to identify these rare conditions. A global biopharma company aimed to improve HCP education on recognizing rare metabolic diseases by collaborating with patient advocacy groups (PAGs) to develop and deliver targeted educational initiatives. The challenge was to build a scalable, patient-centric strategy that empowered advocacy groups to effectively engage with HCPs.

Approach

A patient-centered, collaborative strategy was implemented, focusing on leveraging the expertise and networks of PAGs to reach and educate HCPs:

1. Patient Journey Insights

Conducted qualitative interviews with 10 patients and caregivers to map their diagnostic journeys and identify missed opportunities for earlier diagnosis.
Insights revealed:
- Primary care physicians and general pediatricians were key touchpoints but often lacked awareness of rare metabolic diseases.
- Many misdiagnoses were attributed to overlapping symptoms with common conditions like gastrointestinal disorders or chronic fatigue syndrome.

2. PAG-Led Educational Campaigns

Collaborated with 5 patient advocacy groups to co-create educational materials for HCPs, ensuring alignment with real-world patient experiences and medical accuracy.
Delivered resources included:
- Clinical Case Studies: Real patient stories with diagnostic clues for HCPs to recognize rare metabolic conditions.
- Symptom Recognition Guides: Quick-reference tools highlighting hallmark symptoms and diagnostic pathways.

3. Advocacy Training and Capacity Building

Provided training sessions to PAG leaders and members on how to engage effectively with HCPs, including:
- Presentation skills to deliver impactful education at medical events.
- Insights on how to align patient stories with clinical evidence to drive HCP understanding.
Equipped PAGs with digital templates, presentation decks, and print materials to ensure consistency and scalability across different regions.

4. HCP Engagement Initiatives

In-Clinic Sessions: PAGs partnered with metabolic specialists to conduct in-clinic educational sessions for primary care physicians, pediatricians, and general practitioners.
Webinar Series: Hosted live webinars featuring metabolic experts and PAG representatives, emphasizing the importance of early symptom recognition.
Conference Presence: Supported PAGs to present at local and regional medical conferences, distributing tailored educational materials to attendees.

Outcome

The collaboration with PAGs delivered measurable results in improving HCP awareness and education:

Improved HCP Awareness:
- Educational campaigns reached over 800 HCPs through webinars, conferences, and in-clinic sessions.
Enhanced Knowledge:
- Post-session surveys showed that HCPs reported increased confidence in identifying symptoms of the rare metabolic disease.
Increased Referrals:
- Advocacy-led efforts contributed to an increase in referrals to metabolic specialists in targeted regions.
Empowered Advocacy Groups:
- PAG members reported greater confidence in engaging with HCPs, with 5 groups now independently organizing and delivering regular educational initiatives.

Key Takeaway

This case study highlights how collaboration with patient advocacy groups can drive meaningful improvements in HCP education for rare diseases. By equipping PAGs with the tools and training to lead educational efforts, biopharma companies can amplify their impact, build trust within the patient community, and accelerate pathways to earlier diagnosis and treatment.

Contact us today to learn how advocacy collaborations can enhance HCP education for rare diseases.