Clinical Development and Evidence Generation
What this domain tests
This domain covers how companies close evidence gaps beyond the pivotal programme: investigator-initiated studies, real-world evidence, registries, patient-reported outcomes and expanded access. Expect questions on which evidence answers which question and its limitations.
Core rules
- Company-sponsored studies are protocol-controlled and sponsor-audited; investigator-initiated studies are proposed and conducted by external investigators, reviewed by an internal committee for scientific merit, safety, regulatory and budget fit, with clearly defined publication and pharmacovigilance responsibilities.
- An evidence-gap analysis maps what payers, regulators, HCPs and guidelines need against what exists; the output is a prioritised integrated evidence plan.
- Real-world evidence draws on registries, electronic health records, claims, chart review and patient surveys. It is powerful for long-term safety, adherence, subpopulations and resource use, but is vulnerable to confounding and missing data.
- Patient-reported and other clinical outcome assessments require documented content validity and psychometric properties before use as endpoints.
- Expanded access and named-patient programmes provide treatment, not marketing, and data use is governed by local rules.
Worked example
A payer asks for local cost and adherence data that the pivotal trial did not collect. The team designs a retrospective chart review linked to claims, with a pre-specified analysis plan and endpoints chosen to match payer questions (persistence, hospitalisations, comorbidity burden). The output supports the dossier as complementary, and the team does not present it as equivalent to randomised evidence.
Traps that cost marks
- Presenting observational results as if they carried randomised-trial certainty.
- Using an expanded access programme as a promotional or data-generation channel.
- Running a study without pre-specified endpoints or an analysis plan.