With a gene therapy for a rare disease in early development, the client was looking to understand how previous therapies in the same disease area have approached their health technology assessment (HTA) submissions, as well as the key challenges and critique previously received by HTA agencies in key markets across the globe. The client requested a review of relevant publicly available HTA documents in the markets within scope. The information gathered would help inform the design of their clinical and economic evidence development plans, and guide their future market access strategy.
We conducted searches in a wide range of countries as far afield as South Korea, Taiwan, Australia, Brazil and other Latin America markets, Canada and the USA; whilst also evaluating key European markets, including the EU5 and the Nordics. In total, we reviewed around 70 reports across 15 markets that met the inclusion criteria, pertaining to six different brands in three related indications.
The data were summarised into a data extraction table, and a PowerPoint deliverable presented the key findings per market and per product. This deck also presented the identified trends, conclusions and recommendations for the client. Our Global Market Access consultants worked with our in-house Health Economists to review and assess the economic data in a deep-dive section.
We presented the client with detailed information and conclusions to support their evidence generation plans. Our conclusions highlighted some of the expected clinical and economic evidence challenges typically associated with rare diseases, including lack of country-appropriate prevalence, clinical practice and costs data, sub-optimal patient numbers and trial designs, and consequent economic results uncertainty. We also observed that a shift from symptomatic to preventative treatments in this disease area was creating significant payer challenges, leading to restrictions in reimbursement.
Rather than simply delivering one report at the end, we first delivered an interim report on the key markets and indications of interest to the client in order to support some of their critical clinical trial protocol discussions as well as stakeholder engagement plan activities. Through that process, we applied the feedback on the deliverables to the final report. We are now working with the client to define a path forward to support our client’s internal discussions with ad-hoc updates of HTA decision reports as these become available, as well as conducting yearly updates to the deliverables.