Late-stage clinical development primarily aims at demonstrating efficacy, safety and cost-effectiveness. It corresponds to Phases 2b and 3 confirmatory studies. The product is tested in larger clinical studies, often as compared with the therapeutic ‘gold standard’ or standard-of-care (SoC), if any. The collection of safety data on a larger scale enables the confirmation of the product’s safety profile.
Phase 3 trials are conducted in a large number of countries in view of supporting approval in broader markets. Health economics data are also gathered to demonstrate the product’s value to payers and support post-approval reimbursement.
Client Goals
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 (FDA, HTA, pricing & reimbursement)
Develop clinical trial essential documents.
Ensure regulatory compliance of clinical trials.
Challenges
- Is(are) the planned pivotal study(ies) adequate to support both registration and positive US FDA and HTA appraisal?
- How to obtain endorsement from regulators and HTA on the late-stage clinical development strategy?
- How to measure healthcare resource use associated with the disease?
- How to articulate a development plan across multiple regions, with potentially different requirements?
- How to confirm/improve the product’s benefit/risk ratio?
- How to maximize patient recruitment and retention strategies in clinical studies?
- Which clinical endpoints would address both regulators’ and payers’ requirements?
- How can disease awareness be optimized?
- What must be done for launch readiness?
- How to validate manufacturing processes and methods?
- How to design/ adapt manufacturing for commercial launch (upscaling)?
- Which post-launch evidence will the payers require?
- Are there any early access opportunities?
Solutions 
