Consultancy
Clinical Development Readiness
Early planning through execution and regulatory submission
Overview
Clinical development readiness means ensuring a rare disease drug development programme has the scientific, strategic and operational foundations in place before it enters clinical trials. For rare diseases this is particularly demanding: patient populations are small, trial design is complex, natural history data is often limited, and regulatory pathways require early and careful navigation. CortexBio helps companies assess whether their programme is genuinely trial-ready, and identify the gaps that need closing before committing to expensive clinical investment.
What we do
- Target Product Profiles — TPPs that align clinical development with commercial goals and patient needs.
- Clinical development strategy — pathways that balance scientific, regulatory and commercial considerations.
- Study design optimisation — trial designs tailored to rare and metabolic disease, including adaptive and framework approaches.
- CMC readiness — a manufacturing strategy that keeps pace with accelerated clinical timelines.
Where this creates value
The highest-value point to engage is before Phase 1 or Phase 2 planning begins — when there is still time to shape the development strategy rather than course-correct it. Companies benefit most when they have a promising early-stage asset and need to define the regulatory pathway, identify the right patient population, design robust endpoints, and align clinical strategy with what regulators and payers will ultimately require. CortexBio focuses on the points where poor decisions carry the highest cost: patient population definition, endpoint selection aligned with regulatory expectations, early regulatory engagement strategy, and a realistic assessment of what a development programme can achieve given the available evidence — resolving the gaps that would otherwise surface at regulatory review, while there is still time to act on them.
Why CortexBio
Readiness is a judgement, and judgement comes from having taken programmes the whole way. Dima Martini-Drew, MD, has directed the launch of four enzyme replacement therapy programmes across Genzyme, Astellas and Abbott, and is a founding industry member of the Critical Path Institute's rare and orphan disease programmes — 30 years of seeing which assumptions hold at regulatory review and which do not. That experience is what turns a readiness review from a checklist into a prioritised, defensible view of what a specific programme still needs.
Who this is for
Early- to mid-stage biotech and pharma teams with a promising rare disease asset approaching Phase 1 or Phase 2 planning, companies opening a rare disease programme without in-house orphan drug or natural history experience, and investor groups who need an independent read on whether a programme is genuinely trial-ready before committing further capital.
Frequently asked questions
What is clinical development readiness for rare diseases?
Ensuring the scientific, strategic and operational foundations are in place before a rare disease programme enters clinical trials — assessing whether it is genuinely trial-ready and identifying the gaps that need closing before committing to expensive clinical investment.
What is a Target Product Profile and why does it matter in rare disease?
A TPP defines what a drug needs to achieve clinically and commercially to be viable. Regulators expect to see it early, and it disciplines the programme around what actually matters to patients, clinicians and regulators — helping avoid costly pivots later.
How important is natural history study design in rare disease development?
Often critical — natural history data generates the baseline needed to understand disease progression, define meaningful endpoints and design comparator arms when randomised controlled trials are not feasible. Regulators increasingly expect it before approving trials in ultra-rare conditions.
When should a biotech engage a clinical development readiness consultant?
The highest-value point is before Phase 1 or Phase 2 planning begins, when there is still time to shape the strategy rather than course-correct it.
How does CortexBio reduce risk in rare disease clinical programmes?
By applying experienced strategic judgement at the points where poor decisions carry the highest cost — patient population definition, endpoint selection aligned with regulatory expectations, early regulatory engagement strategy, and a realistic assessment of what a development programme can achieve given the available evidence. The goal is to identify the specific gaps that would cause problems at regulatory review, and resolve them while there is still time to act.
How an engagement works
A readiness engagement typically starts with a structured review of the programme against the questions a regulator will ask: is the Target Product Profile defined and defensible, does the natural history evidence support the proposed endpoints, is the study design realistic for the population size, and is CMC on a timeline that will not become the bottleneck. That review produces a prioritised list of gaps rather than a generic audit, so the company can see exactly what needs to close and in what order before committing further capital.
Where the engagement continues past the initial assessment, work is scoped around the specific deliverable each gap requires — a revised TPP, a study design option paper, a regulatory engagement strategy — so the relationship stays accountable to closing named gaps rather than running open-ended.