Rare Disease - Episode 8

Episode 8 - Long-Acting Agents and Considerations

As explained by Hugh Fatodu, RPh, MBA, current data reveal that hemophilia treatment centers provide better outcomes for patients, lower the number of emergency department visits, and decrease rates of hospitalization. Unfortunately, not all areas of the United States have access to these facilities.

Maria Lopes, MD, MS, continues the discussion by explaining that in addition to limited access to these facilities, the lack of consistency between existing hemophilia treatment centers is a major gap. Dr Lopes concludes that transparency is important when caring for patients, and that all variables that affect consistencies of the cost and value of care within these facilities need to be addressed.

And as a result of increased home-care events, Mr Fatodu also explains that there is a need to address the frequency of administration of medicines that currently affect patient adherence levels. Developing medications that have lower requirements for frequency of administration is necessary for improving patient adherence.

Michelle Petri, MD, MPH, explains that in order to eliminate these challenges a demand for research and new data is necessary. Dr Petri questions who would become responsible for paying for the research necessary to develop this new data.