The Medical Affairs strategy faces multiple challenges, the first being a large number of stakeholders needing specific evidence not found within the clinical trial publication. Endpoints in this trial were regulator-specific but largely irrelevant to payors – where is the reduction in healthcare utilization, for example? Secondly, these clinical trial endpoints do not always correlate with disease severity; where the quality of life is measured, it is done using generic quality of life tools that failed to include many of the domains relevant to rare disease patients, and therefore risked underestimating any true impact on their quality of life. Finally, the prescribers are in perpetual need of information on patients outside the marketing authorization – on those in the real world.
In order to ensure that a medicine/drug can enter the market and it will be reimbursed, it is highly recommended to design and implement rare disease registries, developing an expert opinion, and understand how disease-specific tools can provide more relevant patient information than generic ones.
- To present and discuss a variety of Real-World Evidence generation strategies: design and implementation of rare disease registries, developing expert opinion
- Focused on the multi-stakeholder approach, ensuring that each Real-World Evidence asset will be targeted to the appropriate group(s).