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Rare Disease Medical Writing, Statistics and Regulatory Documentation
Pubrica supports sponsors and clinical research teams developing treatments for rare diseases with medical writing, statistical analysis, regulatory documentation and publications. Projects include single-arm trials with external or natural-history controls, n-of-1 and crossover designs, registries and orphan designation dossiers. We prepare protocols, statistical analysis plans, clinical study reports, submission summaries and manuscripts, and document how closely natural-history cohorts match the trial population. A designated project lead coordinates the protocol, analysis plan, study report and publications to keep data interpretation and messaging consistent.
We identify the reporting guideline and extension that fit your study design during the free scoping read.
- Assigned by indication
- One named lead throughout
- Reporting guideline identified
- Mutual NDA on request
- You remain the sponsor
Scope of Work
Rare disease study types, guidelines and analysis issues
Small patient populations often rule out conventional randomised designs, so rare disease programmes rely on external controls, natural-history data and registries. The design determines the reporting guideline and the points assessors and payers examine.
Typical study types
Typical study types
Single-arm trials with external or natural-history controls, n-of-1 and crossover designs, registries and orphan dossiers.
Reporting guidelines
Guidelines and extensions applied
CONSORT extensions for small populations, STROBE for natural-history studies, and regulatory guidance on small-population clinical trials.
Key analysis issue
Points reviewers examine closely
External controls: whether a natural-history cohort is comparable enough to support the planned comparison.
Common review points
Common review points in rare disease submissions
Assessors reviewing rare disease programmes frequently raise the points below. We check each one before documents are filed.
Points we check before filing
Services for this area
Services for rare disease programmes
Services are grouped by programme stage. Device and diagnostic work follows MDR and IVDR rather than ICH and is covered on the relevant industry pages.
59 services, by stage of the programme
Services can be ordered individually or as a programme. A designated project lead coordinates the documents so that data and messaging stay consistent.
Who We Serve
Who we support in rare disease
The free scoping read is written for the team that requests it, whichever function it sits in.
Teams we work with
Our compliance and guideline standards
Guidelines applied at the current edition
Each deliverable names the guideline and extension your design requires, and the edition used. Writing to the current edition reduces the risk of findings linked to superseded guidance.
Standards applied in this area
Where AI is used, and where it is not
Used for search and formatting. Not used to write your science.
This policy applies in every therapy area and to every deadline.
Our AI policy
How it works
Five steps, starting with a free scoping read
We review your programme before providing a quote.
One
Scoping read
Free, within two business days: what the programme needs, who would lead it and the expected cost.
Two
Named lead
A lead matched to the indication is assigned before work starts and coordinates the programme throughout.
Three
Outline approval
Every section and its source, approved before drafting.
Four
Drafting and review
Independent review, QC and reference verification, each signed off separately.
Five
Handover
The deliverable with its working material, revised within the agreed scope.
After
Follow-up questions
Reviewer and health-authority questions are answered by the writer of the document.
Our Experts
Who works on rare disease projects
Writers are assigned by indication, not by availability. They hold a master’s degree or doctorate in life sciences, medicine or pharmacy and have at least five years in the field. Regulatory writers work to the guidance that governs each document.
Team roles
Sample work
Sample deliverables
Published where the sponsor agreed, redacted where they asked.
Benefits
Benefits of indication-matched teams
What clients receive on every programme.
What you get
Packages
Priced per programme, not per page
A 4,000-word study report and a 4,000-word overview involve different work, so we do not price by word count. The free scoping read gives you a programme-level figure for your budget.
First
Scoping read
Free, two business days. A written assessment of what the programme needs.
Then
A written scope
Documents, standards, named team and timeline, agreed before work starts.
Quoted
Per programme
One price for the agreed scope, with revisions included within the agreed scope rather than billed per round.
Rare disease FAQs
Questions from rare disease teams
Yes. Leads are assigned by indication and have worked in this area. They know that the comparability of external and natural-history control cohorts is a frequent review point and address it before the document is written, not after it comes back. If no one on our panel is a suitable match, we tell you and decline the project.
The study design determines the guideline, not the indication. For rare disease work this is usually a CONSORT extension for small populations, STROBE for natural-history studies, and regulatory guidance on small-population clinical trials. We confirm the applicable extension in the free scoping read, because revising a finished report to a different guideline is harder than writing to it from the start.
External control cohorts assembled without documented comparability, so the treatment effect cannot be separated from patient selection. We check this on every rare disease programme before filing, when it is easier and less costly to correct.
Payers want natural-history data as well as trial data, and registries set up late are a common gap. The scoping read identifies this early, while the evidence plan can still be adjusted.
Yes. A designated project lead coordinates the protocol, statistical analysis plan, clinical study report and publications to keep data interpretation and messaging consistent. Health-authority questions are answered by the writer of the document concerned.
We do not operate your pharmacovigilance system. We write the risk management plan and periodic reports, but case processing and reporting to authorities stay within your own PV system under your qualified person. We do not conduct trials, audit or certify GCP or GMP compliance, or claim authorship of your research. You remain the sponsor throughout.
Related Areas
Therapy areas often linked to rare disease programmes
When a programme spans two therapy areas, we assign a lead for each area.
Other therapy areas
Discuss your rare disease project requirements
Share your indication and timeline. The free scoping read takes two business days and gives you a written view of what your rare disease programme needs and who would lead it.