PURPOSE: To explore the conditions and methods under which real-world data on rare diseases may be robustly borrowed from US and Chinese sources, given the known limitations of European country-specific sources.

DESCRIPTION: Randomized controlled trials have short time-horizons, and large sample sizes or randomized comparators are not always feasible in rare disease settings. Thus, the evidence base increasingly being presented to payers for these conditions can be inadequate to robustly determine clinical and cost-effectiveness. This uncertainty can delay critical decisions on reimbursement, impacting patient access. While “value-based” arrangements can be made between payers and manufacturers, establishing real-world outcomes for rare conditions remains challenging. HTA submission in Europe that aims to leverage real world outcomes faces several key questions: Can outcomes based on small numbers of patients be trusted? How can this be used to reliably address uncertainties in evidence?

The moderator will describe the problem and illustrate how US data have been used to support post-authorization safety mandates in Europe (10 minutes). This will set up a discussion on conditions under which US data can serve as a benchmark for real world outcome evaluations in Europe (15 minutes). The audience will be polled to rate the acceptance of these from the research and stakeholder perspective. Then, a review of the available methodological approaches will be presented (propensity score matching, inverse probability of treatment weighting and quantitative bias analysis), with the audience voting on their familiarity with the methods and if they consider them robust (10 minutes). Finally, a case study of a rare condition (e.g., spinal muscle atrophy) leveraging Chinese RWD will be presented (15 minutes) – the results will be contextualized in the prior European findings and the audience will be engaged to assess face validity. The workshop will conclude with the summary of the results and a Q&A (10 minutes).