A Deep Dive into Pfizer’s Rare Disease Engagement Framework
As rare disease teams navigate rising expectations from payers, regulators, and patients, the ability to prove the real impact of engagement efforts is becoming mission-critical. At the 3rd Measuring Patient Engagement Summit, Pfizer Specialty Care will unveil a multi-layered measurement framework designed specifically for rare diseases, showcasing how meaningful KPIs can connect education, behavior, and clinical outcomes with measurable value.
What Specific Metrics, KPIs, or Measurement Approaches Will You Be Sharing in Your Session?
In our session, we will share a multi-layered measurement framework we've refined at Pfizer Specialty Care for rare disease patient engagement, with a focus on hemophilia and emerging application to Sickle Cell and ATTR CM Therapies which includes:
- Reach & Engagement KPIs: Opt-in rates, education completions, webinar attendance, and next-step actions (e.g., resource downloads or peer connections)
- Comprehension & Knowledge Lift: Pre/post-education surveys assessing disease awareness, treatment understanding, and self-management confidence
- Behavioral & Clinical Impact: Adherence proxies (e.g., time-to-treatment initiation), site-of-care preferences, and aggregated insights on relapse recognition or quality-of-life improvements
- Non-Promotional Holistic Metrics: Patient satisfaction (NPS-style), Self Advocacy Confidence, and community strength indicators (e.g., peer support participation)
These combine leading indicators (engagement) with lagging outcomes (behavioral/clinical), all tracked via CRM enhancements, external surveying, and real-time conference insights.
What Concrete Tools, Frameworks, or Outputs Will Attendees Walk Away With?
Attendees will leave with a greater understanding of practical, adaptable takeaways and solutions for:
- A Patient Engagement Measurement Framework outlining the layered KPIs above, including how to balance leading/lagging indicators and non-promotional reporting
- Survey & Dashboard Idea's: Sample post-opt-in surveys and real-time dashboard elements (e.g., Polaris-style) for tracking opt-ins, satisfaction, and journey-stage insights
Can You Share One Example of How These Metrics Influenced a Real Decision?
In 2025, our hemophilia Patient Navigator + Community Engagement Lead model exceeded all Brand Activation goals for reach, comprehension, and opt-ins leading to great patient engagement and connectivity supporting why patient facing roles are critical to the organization.
This evidence directly influenced a senior leadership decision to expand the model: we secured approval for branded education permissions beyond PAGs, additional headcount (4 roles total), and investment in CRM enhancements for better dashboarding. Without the causal proof from Surveying insights and layered KPIs, the expansion would have faced greater scrutiny - metrics turned advocacy into strategic priority.
What’s One Common Mistake Teams Make When Trying to Measure Patient Engagement?
A frequent mistake is focusing too heavily on vanity metrics (e.g., raw reach numbers or webinar attendance) without linking them to meaningful behavioral or clinical outcomes. This creates a false sense of success while missing the full impact story.
For example, high opt-ins are great, but if they don't correlate with improved adherence, reduced diagnostic delays, or better quality-of-life scores, the program may not be driving real change. Teams often overlook causal inference methods aggregated surveying results or non-promotional holistic indicators (e.g., emotional well-being, peer connection strength), leading to under-investment in what truly matters to patients and payers.
Why Is This Topic Particularly Important for Teams Right Now?
If you're committed to expanding access to therapies and navigating the evolving landscape of global EAPs, the 6th Operationalize: Expanded Access Programs Summit is where you need to be.
Register today to secure your place, connect with peers, and help define the future of expanded access programs.
What Are You Most Looking Forward to at the Summit?
We are most excited about peer-to-peer learning and real-world case sharing. Hearing how other rare disease teams are tackling measurement challenges, especially causal inference, non-promotional KPIs, and integrating patient-reported outcomes will give us fresh ideas to refine our framework at Pfizer. We are also looking forward to the interactive discussions and networking; those moments often spark the best collaborations and innovations. We’re eager to share our hemophilia learnings and learn from others' successes and pitfalls in this evolving space.
Don’t Miss Out
Learn more about the summit this May by accessing the event brochure here. Additionally, tickets are currently available so if your team are looking to attend this year’s meeting, don’t delay and register today.