Faced with regulatory rejection in Europe despite FDA approval, a US-based pharmaceutical company turned to Kintiga to reframe its product’s value story in ophthalmology. By applying advanced statistical modelling, innovative outcomes research, and health economic techniques, Kintiga developed a new clinical narrative that demonstrated both clinical and economic value. This approach empowered local teams with a robust yet adaptable framework to engage with health technology assessment bodies and improve patient access.
Client Challenge
Demonstrating meaningful patient outcomes is critical for regulatory approval and market access. A US-based pharmaceutical company had received FDA approval for its ophthalmology product but encountered significant resistance from the European Medicines Agency (EMA), which questioned the product’s impact on patients’ everyday functioning. This regulatory setback highlighted the need for a more compelling and evidence-based value narrative tailored to European standards.
The EMA declined to grant marketing authorisation, citing insufficient evidence of the product’s real-world functional benefits. The clinical trials had focused on a primary endpoint that did not align with EMA expectations. Our client needed to pivot from its original clinical narrative and develop a new strategy that could convincingly demonstrate the product’s value in improving patient outcomes and justify its cost-effectiveness.
Kintiga Approach
A cross-functional team led a comprehensive and innovative outcomes research initiative, including:
- Analysis of the clinical data: Clinical plausibility of alternative avenues of association between vision and visual outcomes were assessed to determine the extent of the treatment effect.
- Feasibility of the new approach: Prior to further analyses, the identified alternative approach was scrutinised in relation to clinical opinion, literature and precedence
- Innovative outcomes research: The time-to-event approach highlighted that adverse visual events are less probable to occur in the treatment arm relative to placebo. Composite statistical methods including a multistate framework were used to predict the number of events in the fovea over time.
- Utility mapping: The classic measures of vision such as best-corrected visual acuity were supplemented by disease specific instruments that were more sensitive to changes in visual functioning. These outcomes were then translated to generic preference-based measures that are consistent with the axioms of health economic utility necessary for engagement with national health technology agencies.
- Markov modelling: Multiple sequential survival curves were used to model cohort transitions, using tunnel states to accurately capture the non-constant hazard relative to the preceding event.
Results
By using a comprehensive statistical approach and modelling techniques, the client was able to restructure their value story and demonstrate clinical and economic effectiveness of the product.
Kintiga’s well documented approach in technical reports and other documents gives local affiliates a thorough but flexible platform to engage with their health technology assessors in the face of previous regulatory challenges.
Find out how Kintiga can help shape your product’s value narrative, backed up by clinical and economic evidence. Contact us now at enquiries@kintiga.com.
