Developing and updating a GVD and PVP: Joined up thinking

Our client launched a gene therapy for an ultra-rare neurological disorder, for which new evidence is continually generated. Because the patient population is so small, even one new case study can make a large difference to the strength of value messages. Due to a lack of data within the literature with regards to important aspects of the disease (i.e., epidemiology and mortality), new data is being released continually. Wave 1 submissions have all already been made and HTA feedback and questions have been harvested, meaning HTA success rate has become a measure of success.

We have taken responsibility for creating and updating our client’s GVD, PVP, objection handler, and data tracker when new data becomes available. We took a flexible attitude towards not only the value materials, but also the messaging within them. This approach has given us the opportunity to ensure Wave 2 submissions are better-focused.

This ultra rare disease leads to rapid decline and shortens the lives of children dramatically if not treated. Conversely, the gene therapy is transformative, so our messaging focuses on both scientific data and emotion. Our project lead is now considered 1 of 2 global experts in the client’s own data.

Affiliate teams in second-wave countries have been able to avoid some of the pitfalls and issues raised by the early launch markets. This has been facilitated by linking value messages to data and highlighting potential issues, aligned with successful mitigation strategies. The suite of access materials follows a common thread, resulting in excellent feedback from country teams and external HTA consultancies.