Module 3 · Part 1 · Planning

Scientific Advisory Boards

Build a Scientific Advisory Board (SAB) that provides genuine scientific direction, not just credibility optics, and structure it to serve your registry's long term needs.

Patient Registries 101 · Dr. Danielle Boyce · EpilepsyLive

Why your registry needs a SAB

A well-constituted SAB is not a prestige accessory.
Scientific validity: Ensures your data elements, study design, and analysis plans meet publishable standards
Regulatory credibility: FDA and EMA look favorably on participant led research with independent scientific oversight
Industry credibility: Pharma and biotech partners evaluate SAB composition when deciding whether registry data is worth licensing
Academic credibility: Journal reviewers assess whether the registry was designed with appropriate scientific rigor
IRB support: IRBs are more comfortable with participant led research when there is independent scientific oversight

Why your registry needs a SAB (cont.)

Endpoint development: Helps identify and validate clinically meaningful endpoints for future trials

Core roles to fill

Disease specific clinician scientists (2 to 4) , Physicians or scientists with direct expertise in your disease.
Biostatistician (1) , Essential for designing data collection that produces analyzable results.
Participant/caregiver representative (1 to 2) , Ensures the registry captures outcomes that matter to participants, not just outcomes that are easy to measure.
Clinical trial methodologist (1) , Advises on how registry design can support future trial endpoints, comparator arms, and recruitment.
Bioinformatician or data scientist (1) , Critical if you are collecting genomic, proteomic, or other high-dimensional data.
Regulatory scientist (1, optional but valuable) , Advises on FDA/EMA guidance, natural history data acceptance, and the regulatory pathway from registry to trial support.

Core roles to fill (cont.)

Ethicist (1, optional) , Particularly valuable for registries collecting genomic data, pediatric data, or planning broad consent.

What to look for in members

Active publishing record in your disease area
No conflicts of interest that would compromise independence (industry employment is a common conflict, can be managed with disclosure and recusal policies, but must be explicit)
Genuine interest in participant led research (not just a CV line)
Willingness to commit meaningful time, quarterly meetings minimum, plus ad hoc consultations

Charter

Every SAB needs a written charter covering
Mission and scope of the SAB's authority
Composition requirements and term limits
Meeting frequency and quorum requirements
Conflict of interest disclosure and management policy
Compensation policy (honoraria, travel)

Charter (cont.)

Relationship to the advocacy organization's board of directors
Decision making process (advisory vs. binding)

Meeting cadence

Full SAB meeting: Quarterly (90 minutes minimum), or semi-annually for very busy members
Working groups: Monthly or as-needed for specific projects (e.g., data element committee, publication committee)
Annual meeting: In-person or hybrid intensive; review registry progress, set annual priorities, discuss publications

Compensation

SAB members should be compensated.
Meeting attendance: $500, $1,500 per meeting
Manuscript review/authorship work: $500, $2,000 per project
Travel expenses reimbursed at cost
Compensation levels vary significantly by organization size and the seniority of advisors.

Data element review

The SAB should formally review and approve your core data element set before data collection begins.
Are these elements sufficient to answer our scientific questions?
Are these elements consistent with the literature and with other registries in this disease space?
Are there validated instruments or scales we should use rather than custom questions?
What are the minimum data elements required for a natural history analysis?

Publication oversight

Define clearly before the first publication
Who is eligible for authorship vs. acknowledgment when registry data is published?
Does the advocacy organization have a right of review before submission?
How are participant coauthors identified and supported?
What is the embargo policy for data shared with industry partners?

Endpoint development

For rare diseases pursuing FDA approval, the SAB should actively participate in FDA meetings (e.g., Type B or C meetings, Natural History Workshop) and help develop clinically meaningful endpoints…

How to recruit SAB members

Start with your clinical network , Physicians treating your community are natural candidates. Ask your most engaged participants who their best doctor is.
Conference outreach , Attend disease specific conferences and approach researchers whose work aligns with your registry's goals.
NORD and Global Genes networks , Both organizations connect participant groups with scientific advisors.
Reach out cold with a strong pitch , Many researchers are genuinely motivated by participant led science. A clear, compelling ask with defined time commitments gets more yeses than you expect.

Checklist

[ ] Drafted SAB charter
[ ] Identified required expertise areas
[ ] Recruited disease specific clinician scientists
[ ] Recruited biostatistician
[ ] Included participant/caregiver representative(s)
[ ] Established conflict of interest policy

Checklist (cont.)

[ ] Defined compensation structure
[ ] Scheduled inaugural meeting before data collection begins
[ ] SAB has reviewed and approved data element set
[ ] Defined publication policy with SAB input

Key resources

NORD Scientific Advisory Board Resources
Global Genes RARE Advocacy Resources
FDA Natural History Workshop Proceedings
PCORI Stakeholder Engagement Resources
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