Rare Disease Medical Affairs Impact Equation: Linking Activities with Strategic Outcomes to Drive Real-World Impact

Industries Profiled:
Biopharmaceutical; Pharmaceutical; Health Care; Biotech; Clinical Research; Laboratories


Companies Profiled:
Alnylam Pharmaceuticals; Boehringer Ingelheim; Eton Pharmaceuticals; Grifols; Jazz Pharmaceuticals; Otsuka; Sanofi; Syndax Pharmaceuticals; Takeda

Study Snapshot

This research captures insights from 12 Medical Affairs executives across 9 biopharmaceutical organizations specializing in Rare Diseases. In-depth interviews with senior leaders offer nuanced perspectives on how Rare Disease Medical teams link activities with strategic outcomes and organizational priorities.

Key topics covered in this report include:

• Rare Disease Leading and Lagging Indicators of Medical Impact
• Frameworks for Strategic Impact Measurement
• Integration of Real-World Data and Evidence
• Role of Medical Insights in Launch and Access Success
• Rare Disease Medical Affairs KPIs and Performance Measurement

• Top Measurement Hurdle Hinges on Too Many Market Variables To Clearly Link Action to Outcome: 83% of surveyed Medical organization cite this is a major challenge. Meanwhile, 50% cite limited resources/data as a critical challenge, and 42% indicate “we don’t know what outcomes to measure.”

• Managing Leading and Lagging Indicators Over Time: Leading indicators are revisited monthly or quarterly by 33% of organizations – more than any other interval surveyed. Lagging indicators are most often assessed on quarterly basis (42% of orgs) or ad-hoc basis as needed (33% of orgs).

• One-page executive summary
• Benchmarked companies
• Executive insights and takeaways
• Rare Disease Medical Impact Cycle: Connecting activities to strategic outcomes
• Key quantitative highlights
• Insights vs. impact pathway

• Top-rated activity metrics across Rare Disease Medical Affairs
• Case study: Evaluating MSL impact across multiple rare disease workstreams
• Top field activity metrics
• Top non-field activity metrics
• “Cycle of Impact” examples: How Rare Disease awareness and education translate into measurable engagement outcomes

• Benchmark linkage rates across Rare Disease teams
• Most linkable medical activities to strategic outcomes
• Most linkable field activities to critical external outcomes
• Cycle of impact: Real-world examples showing the impact of medical insights
• Most linkable non-field activities to critical external outcomes
• “Cycle of Impact” examples: Real-world influence of congress presentations and post-congress engagement

• Case study: Using AI to uncover care gaps and optimize Rare Disease MSL deployment
• Prioritization and linkability of strategic outcomes
• Gap analysis: Importance vs. measurability of impact metrics
• Linkability trends across lagging indicators
• Linking real-world data outcomes to medical activities
• Connecting provider-level outcomes to tactical medical engagement
• Measuring Medical’s influence on launch strategy, guidelines, and access
• Cycle of impact: Real-world examples showing the impact of patient support
• Cycle of impact: Real-world examples showing the impact of disease awareness and knowledge retention through field and digital engagement
• Key challenges in linking Rare Disease tactics to strategic outcomes

• Benchmarking activity metrics across new vs. mature rare disease products
• Tailoring measurement frameworks by product lifecycle stage
• Evolution of medical KPIs over time
• Navigating competing strategic objectives across medical levels
• Critical RWD sources to validate Rare Disease impact
• Tracking data granularity to strengthen activity-to-outcome linkage

• Geographic distribution of benchmark participants