First-in-human readiness

• Study concept and objectivesThe target population, the objectives, the initial endpoints and the evidence the first study has to generate, including whether and how to enrich for a biomarker.
• Dose-finding approachWhether the escalation and expansion design will produce the information needed to choose a dose to take forward, not only a maximum tolerated dose, and how nonclinical and pharmacology input feeds that choice.
• Cross-functional dependenciesWhat is firm, provisional or missing across the nonclinical package, CMC supply, regulatory strategy, biostatistics and clinical pharmacology.
• Sponsor operating modelWho owns medical review, safety, vendor oversight and program decisions in a small company.
• Data flowHow information moves from the visit to the CRF to the reviewer, and whether emerging safety, exposure and activity can be seen together.

A company entering the clinic for the first time has to build a clinical function, choose vendors and make design decisions that shape the whole program, usually with a small team. An early study that cannot say which dose to take forward, or whose data cannot be reviewed coherently, can cost months.

• Preparing a pre-IND discussionor a first clinical concept.
• Before selecting a CROand other vendors.
• Defining clinical governanceand medical-review responsibilities for a small sponsor.
• When the concept and the available supply, safety package or timeline do not fittogether.

1. Map the dependencies Each element of the first study, with the function that owns it and its current status.
2. Assess the gaps What is missing, what is provisional and what could change the concept.
3. Prioritize by milestone What has to be settled before the IND, before vendor contracts and before the first site is activated.
4. Hand over to owners A roadmap with owners and decision dates for each open item.

• A clinical readiness gap assessment
• An owner-and-dependency roadmap to the next milestone
• A ranked list of unresolved clinical questions
• An initial map of oversight and clinical-review responsibilities

• StructureA clinical-scope assessment, with a broader multidisciplinary review assembled to suit the program and the availability of specialist contributors.
• What we need from youThe development summary, target product profile, draft clinical concept and current functional plans or vendor assumptions.
• Roles and limitsA full readiness determination needs nonclinical, CMC, regulatory, statistical and clinical-pharmacology expertise. A clinical review is not IND clearance and does not replace those assessments.