Phase 3 study design assessment and recommendations

Our client was part of a joint development programme with a US company and had concerns around the suitability of their Phase 3 study design for European and other HTA markets, especially given the unique considerations related to eye diseases and sight loss. In addition, the QoL measures in the study design were insufficient for robust HTA modelling.

We carried out an internal evaluation to identify specific issues related to eye diseases and sight loss, which was crucial to avoid overlooking nuances that could impact the study outcomes, such as the potential rebound effect of QoL to false highs. Informal advice from payers was sought to ratify the proposed study modifications.

We proposed new HRQoL measures that were not only deemed acceptable for modelling, but also expected to demonstrate advantages in the specific context of rare disease. We sought formal scientific advice on behalf of our client for NICE, G-BA and HAS, and presented the proposed changes in a business case to the joint board of co-development.

The modifications to the study were successfully adopted and were in line not only with the requirements of the co-developers but also HTA bodies in Europe, thus supporting with the acceptability of the study in European and other HTA markets. The incorporation of new HRQoL measures showed the advantages in specifically addressing rare disease and enhanced the overall value of the joint development programme.