Business Case

Therapeutic landscape characterization for a CNS-targeted Enzyme replacement therapy, laying the groundwork for future clinical activities

Our client, a mid-size pharma company developed an enzyme replacement therapy for an ultra-rare disease, expected to have significant difficulty identifying suitable patients and sought LSC’s help identifying providers familiar with a substantial patient population. With patients widely dispersed, LSC developed an approach to understand key prescribers and treatment sites in several global markets, ultimately developing a medical information plan to approach these sites and lay the groundwork for future clinical activities. As a result, our client was able to successfully target and inform prescribers of the potential of enzyme replacement therapy for this ultra-rare disease.