Survey Data: Rare Neurological Disease Education Needs
The National Organization for Rare Disorders (NORD®) partnered with Medlive and surveyed U.S. neurologists actively treating patients, asking what topics, formats, and channels would genuinely help them serve patients better.
Here’s what we found…
SURVEY DATA
Neurologists Want More Education on Rare Disease Diagnosis and Emerging Therapies
Over 70% of neurologists surveyed say they want more education on diagnosis and recognition, emerging therapies, and treatment selection and sequencing for rare neurological conditions.
% of Neurologists "Very Interested" by Topic
- Diagnosis and recognition 76%
- Emerging therapies 76%
- Treatment selection and sequencing 72%
- Molecular/genomic testing 54%
- Adverse-event management 52%
- Clinical trials 48%
- Supportive/palliative care 46%
- Multidisciplinary care 42%
Key insight
Education Gaps Span the Continuum
Diagnosis and recognition at 76% may reflect how rare neurological conditions often present with symptoms that mimic common neurological disease, making recognition especially difficult. Once identified, neurologists face a growing field of emerging therapies many haven't encountered, making treatment selection just as difficult. Treatment selection and sequencing just behind at 72% indicates that once a diagnosis is made, neurologists still face meaningful uncertainty about what to do next, an echo of the same treatment-complexity signal seen in other rare disease specialties.
IN THEIR OWN WORDS
Themes from open-ended responses
Q: For what neurological disease related topic would you most like additional education?
- 55%Specific Rare Neurological Disease Subtypes
- 21%Emerging Therapies & Mechanisms
- 13%Diagnosis, Workup & Treatment Sequencing
- 11%Hereditary Risk & Molecular Testing
EMERGING THERAPY INSIGHTS
Newest Mechanisms, Lowest Confidence
Q: Which categories of emerging or recently introduced therapies for rare neurological disease are you least confident discussing with patients and caregivers?
- Gene-editing therapy (CRISPR-based) 66%
- Gene-replacement therapy (viral vector–based) 56%
- RNA interference / gene-silencing therapy 38%
- mRNA-based therapy 36%
- Substrate-reduction / pharmacologic chaperone therapy 36%
- Enzyme or protein replacement therapy 34%
- Precision small-molecule therapy 30%
- Immune-targeted biologic therapy (mAbs) 16%
0%70%
Key insight
The Newest Therapy Classes Need Foundational Education
When we asked which emerging rare neurological disease therapies neurologists feel least confident discussing with patients, the top two mechanisms they named are also the newest to neurology practice. Gene-editing therapy (66%) and gene-replacement therapies (56%) need foundational, category-level education so clinicians can make informed decisions as the evidence on each one develops.
WHO THEY TRUST
Why the Messenger Matters
- 86%of neurologists treating rare neurological disease trust their medical association or professional conferences for clinical information
- 68% trust their peers/colleagues or KOLs for clinical information
“Neurologists overwhelmingly point to professional conferences, medical associations, and peers as the sources they trust most for clinical information. This highlights the value of independent education delivered through trusted channels and designed around the needs clinicians themselves identify.”
— Katie Kowalski, Associate Director of Education, National Organization for Rare Disorders (NORD)
AI AND RARE NEUROLOGICAL DISEASE CARE
Where Neurologists Want AI to Help
Interest concentrates on three jobs: reading an image or biomarker faster, catching a differential earlier, and flagging rare disease patterns buried in EHR data, each named by roughly half of respondents. Clinical trial matching draws real interest too. Only 4% see no near-term use for AI in their practice.
- 54%AI-supported imaging or biomarker interpretation
- 46%AI-assisted differential diagnosis
- 44%AI tools that flag rare disease patterns in EHR data
- 32%AI in clinical trial matching
TIME AND PLACE
Medlive Platform Engagement Insights
- 83.3%of learning sessions happen on desktop
- 2 PMis the peak engagement hour
- Saturdayis the busiest day of the week on the platform
Desktop v Mobile
- Desktop 84.3%
- Mobile 15.7%
Device share does vary by audience, as rare neurologists are the most likely to view educational content on a desktop computer among the specialties we surveyed.
In response
Building Education that Aligns with Clinician’s Needs
Visit the programs below to see how NORD brings trusted rare neurology education to clincians through the Medlive platform.
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