Assess efficacy, effectiveness and safety, and gather data for registration and market access.
Early clinical development initiates testing in humans, corresponding to Phases 1 and 2a exploratory studies. The investigational product is administered to small groups of healthy volunteers or patients to assess safety and to determine a safe dosing range. A clinical safety system is established to monitor the product’s safety profile and assess significant findings for reporting to regulatory authorities.
Concomitantly, subsequent trials are outlined and readied. Safety, clinical endpoints, patient populations, biomarkers and relevance of comparators are the principal topics of scientific advice meetings with regulators at this stage. Reimbursement environment analysis is undertaken in order to begin to apprehend reimbursement or access challenges, current perceptions of standard of care, defining unmet need and typical treatment pathways. Manufacturing processes are scaled up to provide sufficient drug product for larger clinical trials.
Design and conduct clinical trials
Define clinical development strategy.
Outline clinical development up to marketing, with for each trial.
Generate required evidence for regulatory approval & market access (HTA, pricing & reimbursement)
Develop clinical trial essential documents.
Ensure regulatory compliance of clinical trials.
- Should healthy volunteers or patients be recruited for Phase 1?
- What will be the market access roadmap?
- Does the development plan fit with Regulators’ and HTA’s evidence requirements?
- What are the costs and timelines to complete the program?
- Is the dose-range finding plan in Phase 1/2a studies adequate to identify the optimal dose for pivotal studies?
- Is a pediatric development plan necessary? What is the right time for this?
- When and how to scale-up manufacturing processes?
- Is all the CMC information available to assemble the IMPD?
- What is the best PRO (Patient Reported Outcome), the relevance of clinical endpoint and patient selection criteria from a patient perspective?
- How to understand the patients’ view on risk-benefit and trade-offs (e.g., potential side effects, quality of life)?