Whole people. Meaningful questions.
For professionals
From the evidence already available to the questions still unanswered, Rare Strategies helps organizations build a clearer research direction around the people a condition affects.
01
Bring together published literature, natural history, current care guidance and unanswered questions. Examine what is established, where findings disagree and what remains missing. Where guidance is absent, define the evidence and clinician-led collaboration needed to develop and evaluate recommendations with the community.
A starting point: an evidence summary, care-guidance gap map and prioritized questions.
02
Ask people living with the condition what would make a meaningful difference. Include family and caregiver perspectives alongside the person’s own voice. Accessible conversations, interviews and advisory work can turn those priorities into research questions and outcomes worth measuring.
A next step: a plan for lived-experience research and a map of meaningful outcomes.
03
Choose measures for a defined purpose, then assess their relevance, reliability and validity for the people and setting involved. Distinguish a person’s own report from caregiver observations. Account for missing information, development, treatment changes and participation burden. More tracking is useful only when it helps answer the question.
A next step: a measurement plan, collection protocol, data dictionary and analysis plan.
04
Examine unmet needs, variation and possible intervention targets. Consider alternative explanations and report uncertainty. Study differences within and between individuals alongside population patterns. Observational associations can identify questions to test; evaluating treatment effectiveness needs an appropriate study design.
A next step: an analysis that separates findings, uncertainty and testable hypotheses.
05
Develop a practical sequence of studies and decisions. Define priorities, study questions, outcomes, required expertise and milestones. Evaluate medical and supportive approaches with suitable research designs and relevant clinical expertise. Revisit the plan as evidence and community priorities change.
A next step: a research roadmap, protocol concepts and funding rationale.
A patient organization may need to identify research priorities. A research team may need a better way to measure change. A funder or development team may need to understand an evidence gap before choosing its next step.
We begin by defining the question, reviewing what is already known and agreeing the work needed. Potential outputs include an evidence summary and gap map, priorities informed by lived experience, a measurement and analysis plan, or a research roadmap with milestones.
Before work begins, we agree who needs to contribute, what will be delivered and how the findings will inform the next decision. Research proposals and funding plans can be reviewed against that shared rationale.
Discuss your next research decisionBegin with the person, then bring together the expertise the question needs. Sleep, movement, feeding, digestion, communication and participation may be relevant, depending on the condition and individual. Our initial interest in neurodevelopmental conditions includes health and life beyond the nervous system.
We can investigate better support while research into disease mechanisms continues.
Our interest includes the interaction of biology, development, clinical care and everyday circumstances. Changes in support or environment need to be evaluated for the person and question; they are not a substitute for disease-specific care.
Research should examine how responses differ between people. Individual treatment choices require appropriate evidence and qualified clinical judgment; a population average cannot prescribe a person’s care.
Evidence in Context is the Rare Strategies research initiative developing this approach, initially focused on neurodevelopmental conditions. Its direction connects disease knowledge, meaningful outcomes, daily-life context and more informative studies.
We are developing the questions, methods and collaborations for this initiative. Each proposed use will need appropriate validation and reproducibility checks.
Bring a question, a method or a publicly available paper. We welcome conversations about evidence synthesis, meaningful outcomes, natural history, longitudinal study design, measurement, statistics and relevant clinical disciplines.
Develop the work
Define the question, choose an appropriate design and make the reasoning inspectable. Separate exploratory findings from confirmatory tests. Plan for confounding, missing data, reproducibility and uncertainty.
Propose a collaborationResearch in focus
Propose a published paper for an accessible Science Notes feature. Selected work can be explained with accurate attribution, contributor permission where needed, a link to the source and a clear account of its limits.
Share published scienceParticipant-facing work requires applicable ethics review, consent, data governance and an accessible participation plan. Responsibilities, access, intellectual property, publication and contributor credit—including patient and caregiver contributions—should be agreed before work begins.
This is an invitation to explore collaboration. No paid vacancies or enrolling studies are advertised.
Measurement
A precise number still needs to answer a meaningful question.
Read the noteUnderstanding evidence
Why a relationship between two things does not, on its own, establish cause.
Read the noteDaily life
How lived experience can help shape natural history research.
Read the noteTell us the research area, the question and the decision it could inform.
Start a conversation through our inquiry form. Please leave out medical records, identifiable health information and confidential research data.
Open inquiry form