Case Studies / Advisory board for rare disease specialists…
Medical Affairs KOL & Expert Engagement Rare Disease

Advisory board for rare disease specialists: end-to-end scientific event support from slide design to expert consensus report

Challenge
A rare disease treatment brand needed to align specialist clinicians — the primary managers of the condition in most markets — on practical management recommendations. No existing forum brought this expert group together across markets.
Approach
Designed and managed a full advisory board for rare disease specialists: scientific programme, participant materials, slide deck, structured facilitation, and a peer-reviewed consensus report delivered within one week of the meeting.
Result
Expert consensus report with actionable management recommendations delivered within 1 week; report adopted by participating specialist departments as internal reference.
The challenge

The clinical experts who matter most are not always the physicians

For this rare metabolic disorder, specialist clinicians are the primary ongoing clinical managers. They make the day-to-day treatment decisions and are often the first point of contact when patients experience management difficulties.

A brand launching a new treatment for this rare condition understood that specialist clinician alignment was critical for clinical adoption. But no structured forum existed for bringing this expert group together across markets — advisory boards in the rare disease space tended to focus on physicians.

The brand needed to convene this expert group, facilitate a structured scientific discussion, and produce a consensus document that specialists themselves would recognise as credible and practically useful.

Advisory boards in rare disease often overlook the specialists who manage patients most intensively. The clinical expert closest to the patient is not always the physician.

Our approach

What we did

1
Participant identification and recruitment
Identified specialist clinicians across multiple markets using rare disease patient registry contacts, specialist centre records, and professional society rosters. Conducted pre-engagement calls to confirm scientific interest and availability.
2
Scientific programme design
Developed a structured 1-day programme covering 4 clinical topics: patient selection for new treatment modalities, dietary adjustment protocols, monitoring parameters, and transition of care for adult patients. Topics defined in consultation with 3 scientific committee members.
3
Slide deck and stimulus material production
Produced all meeting materials: scientific background decks for each topic, clinical case vignettes for discussion, and voting tools for real-time consensus capture. All materials reviewed and approved by scientific committee.
4
Facilitation and real-time documentation
Provided on-site facilitation support. Captured discussion outputs in real time using structured note-taking and electronic voting for consensus items. Identified 12 areas of consensus and 4 areas of ongoing divergence.
5
Consensus report production
Drafted a structured consensus report within 3 working days of the meeting. Circulated to all 18 participants for comment. Incorporated revisions and obtained sign-off within 1 week of the advisory board.
Result

Measurable impact

The consensus report was delivered to all participants within 1 week of the advisory board, documenting areas of consensus across all clinical discussion topics. Several participating specialist departments adopted the report as an internal reference document for their management protocols. The brand's Medical Affairs team used the consensus document as the foundation for their HCP education programme across all participating markets.

18
Specialist clinicians across multiple markets
12
Actionable consensus recommendations documented within 1 week
3
Participating departments adopting report as internal reference protocol
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// Query: ribociclib OS data MONALEESA 2023–24
search("ribociclib overall survival", {
  years: [2023, 2024],
  output: "structured_table"
})
// 847 records → 23 relevant
Processing 847 records...
Evidence Summary
MONALEESA-2 updated OS (NEJM 2023): median OS 63.9 mo vs 51.4 mo (HR 0.76, 95% CI 0.63–0.93). Benefit maintained across all pre-specified subgroups...