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» Products & Services » » Medical Affairs » Medical Education

Innovative Medical Education in Rare Disease: Improving Reach, Awareness, Appropriate Diagnosis, and Better Outcomes

ID: POP-418


Features:

29 Info Graphics

54 Data Graphics

1380+ Metrics

131 Narratives


Pages: 91


Published: 2026


Delivery Format: Shipped


 

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919-403-0251

  • STUDY OVERVIEW
  • BENCHMARK CLASS
  • STUDY SNAPSHOT
  • KEY FINDINGS
  • VIEW TOC AND LIST OF EXHIBITS
Rare-disease medical education presents challenges distinct from broader therapeutic areas, driven by low prevalence, concentrated expertise, prolonged diagnostic journeys, and limited familiarity among PCPs. At the same time, traditional specialist-centric CME and congress models are no longer sufficient to support a fragmented, multidisciplinary care ecosystem.

This benchmarking report examines how biopharma organizations are re-designing rare-disease education to extend beyond Centers of Excellence and specialists to more effectively engage PCPs—and even extending outreach to pharmacists, genetic counselors, and other contributors within the broader diagnostic ecosystem. Based on comparative industry data, the research evaluates accredited and non-accredited education models, high-performing channels and formats, and content approaches that balance scientific rigor with scalability and accessibility.

The report also explores the growing strategic role of MSLs, society partnerships, and emerging applications of analytics and AI to identify education gaps, prioritize high-need segments, and assess learning transfer. Through benchmarks, peer insights, and practical guidance, the report helps organizations strengthen rare-disease education strategies that enable earlier diagnosis, appropriate referral, and improved patient care.

Industries Profiled:
Biopharmaceutical; Pharmaceutical; Manufacturing; Biotech; Consumer Products; Diagnostic; Medical Device; Health Care; Clinical Research; Laboratories


Companies Profiled:
Alnylam Pharmaceuticals; Bayer; Boehringer Ingelheim; Rigel Pharmaceuticals; Santhera; Servier; Sobi; Takeda Pharmaceuticals; Terns Pharmaceuticals; Travere Therapeutics; Alkermes; AstraZeneca; bioMérieux; Daiichi Sankyo; Eurofarma; Exact Sciences; GlaxoSmithKline; ImmunityBio; Incyte; Ipsen; Merck; Novo Nordisk; Orion Pharma; Sanofi; Santen; Seres Therapeutics; Summit Therapeutics; Vantive; Zimmer Biomet

Study Snapshot

Best Practices, LLC engaged 36 Medical Affairs leaders from 29 biopharma companies via survey and conducted 8 deep-dive interviews; analysis includes a Rare Disease segment with targeted exhibits (and an appendix of full rare-segment data).

Key topics covered in this report include:

  • Innovation Approaches for Rare Disease Education
  • High-Impact Stakeholder Engagement Models
  • Channels, Content, and Formats That Perform in Rare Disease
  • KPIs, ROI, and Measurement Maturity
  • Investment Trends and Resource Allocation
  • MSLs’ Role and Field Footprint in Rare
  • Analytics- and AI-Enabled Personalization
  • Future Outlook and Leadership Insights for Rare Education

Key Findings

Select key insights uncovered from this report are noted below. Detailed findings are available in the full report.

  • Most effective education formats:
    • For PCPs: All surveyed formats are rated at least somewhat effective by ~50% of respondents, with ~40% indicating N/A not used for each format
    • In-person CME at Congresses is top-rated (although just 20% deem it “highly effective”), followed by hybrid (17%), in-person CME outside of Congresses (13%, with Rare lower), live virtual (13%, with Rare lower), and on-demand virtual (10%, with Rare lower)
Table of Contents

Sr. No.
Topic
Slide No.
I.
Executive SummaryPg. 4
II.
Summary of Quantitative FindingsPg. 7
III.
Summary of Interview InsightsPg. 13
IV.
Education Stakeholders, Budget, Channels, Formats, Content and Strategies for Expanding ReachPg. 19
V.
Leveraging MSLs and Payer/Non-Provider SupportPg. 40
VI.
Measuring Impact, Analytics & AI, Lessons Learned, and Future PredictionsPg. 56
VII.
Appendix - Full Data for Rare Disease SegmentPg. 70
VIII.
About Best Practices, LLCPg. 91

    List of Charts & Exhibits

    I. Executive Summary

    • Executive brief: Strategic imperatives for medical education in rare disease
    • One-page summary of background, methodology, and key topics
    • Participating benchmark companies and rare-segment overview
    • Key quantitative benchmark findings
    • Highlights from deep-dive interviews

    II. Innovative Education: Stakeholders, Budget, Channels, Formats, Content, and Strategies for Expanding Reach

    • Priority external stakeholders for medical education
    • Medical education program strengths and weaknesses
    • Effective medical education channels for specialists
    • Executive narratives around medical education activities
    • Effective medical education channels for primary care providers (PCPs)
    • Executive narratives around PCP engagement
    • Side-by-side comparisons of effective channels for specialists and PCPs
    • Executive narratives around efficient education strategies
    • Medical education budget allocation across CME, non-accredited IME, and other modalities – Total benchmark class
    • Medical education budget allocation across rare disease segment
    • CME funding at congresses – Total benchmark class
    • CME funding at congresses – Rare disease segment
    • Top CME formats for expanding reach among specialists
    • Top CME formats for expanding reach among PCPs
    • Side-by-side comparisons of effective CME formats for specialists and primary care providers
    • Effective medical education content types for specialists
    • Effective medical education content types for primary care providers
    • Executive narratives around identifying and meeting the needs of specialists and PCPs
    • Side-by-side comparisons of effective medical education content types for specialists and PCPs
    • Innovative forms of medical education

    III. Innovative Education & Engagement Approaches: Use of MSLs, Payers/Non-provider Support

    • Proven tactics to improve education program reach
    • Success stories in expanding education reach
    • MSL utilization to improve the reach and impact of medical education programs
    • Field team sizing benchmarks and average MSLs per indication – Total benchmark class
    • Field team sizing benchmarks and average MSLs per indication – Rare disease segment
    • Executive narrative around developing an integrated educational campaign
    • Key social media platforms for peer-to-peer exchange among specialists and PCPs
    • Engagement frequency for each social media platform
    • Frequency of education-related support provided to payers
    • Adoption of creative pilots for non-providers (payers, IDNs, patients/advocacy)

    IV. Measuring Impact, Analytics & AI, Lessons Learned and Future Predictions

    • KPIs for measuring impact of key medical education tactics
    • Current performance levels among specialists
    • Peer tips for pre- and post-event outcomes tracking
    • Industry perspectives on medical education performance assessment
    • Current performance levels among primary care providers
    • Side-by-side comparisons of current performance levels among specialists and PCPs
    • Use of analytics & AI to drive smarter education approaches
    • Lessons learned around effective formats for expanding reach and driving care improvements
    • Lessons learned around strategic alignment for expanding reach and driving care improvements
    • Lessons learned around measurement practices and vendor selection for expanding reach
    • Predicted evolution of medical education over the next 2–3 years

    V. Appendix: Full Data for Rare Disease Segment

    • Priority external stakeholders for rare disease medical education
    • Strengths and weaknesses of rare disease medical education programs
    • Effective education channels for specialists
    • Effective education channels for PCPs
    • Comparison of effective medical education channels for specialists vs. PCPs
    • Effective CME formats for expanding reach among specialists
    • Effective CME formats for expanding reach among primary care providers
    • Comparison of effective CME formats in rare disease segment - specialists vs. PCPs
    • Top medical education content types for driving engagement with specialists
    • Top medical education content types for driving engagement with primary care providers
    • Comparison of effective medical education content types in rare - specialists vs. PCPs
    • Proven tactics to improve the reach of rare disease education programs
    • Use of MSLs to expand reach and improve the impact of medical education programs
    • Social media platforms used for peer exchange in rare disease
    • Engagement frequency by social media platform
    • Frequency of education-related support provided to payers
    • KPIs to measure rare disease medical education tactics
    • Current performance levels among specialists in rare disease segment
    • Current performance levels among PCPs
    • Use of analytics & AI to drive smarter education in rare segment