Being rare is often a blessing: a rare gem stands out from a pile of stones, a rare talent can garner prestige and success, and a rare personality can make for an influential leader or a cultural icon. But in medicine, the rarity of a disease often prevents patients from accessing effective therapies. Suffering from a rare disease means a longer diagnostic journey, a smaller patient community, and less approved treatment options. Advancing the availability of therapies for rare diseases necessitates raising awareness, driving policies, and progressing therapeutic approvals. At Catalyst, we’re proud that our work supports each of these pillars:
Building Awareness
Members of team Catalyst have moderated panels discussing the sustainability of rare disease therapies to amplify the patient voice. We’ve also led educational efforts to build industry- and agency-wide awareness of emerging technologies that are at the forefront of rare disease treatment.
Driving policies
There are ways of getting things done, and then, there are our ways! Team Catalyst uses our strong agency relationships to understand how to effectively craft and drive policies that help our clients build platforms to influence policies that favor the advancement of treatments and interventions to address unmet medical needs.
Advancing treatments
A large portion of Catalyst’s work involves strategic regulatory support to help sponsors developing therapies for rare diseases gain regulatory approval.
We love our work since it creates a positive impact on the lives of individuals and families living rare diseases.