Rare Disease

We estimated the long-term risks and benefits of disease modifying therapies. Benefits were favored by natalizumab with minimal increased risks in the negative anti-JC virus population.

Matthias Cheung, PhD, FCSHP, FASHP, a principal at Advanced Rx Consulting, LLC, and an adjunct professor of pharmacy practice at the University of the Pacific, Thomas J. Long School of Pharmacy and Health Services, discusses the circumstances and factors that catalyzed the passage of current legislation and regulatory policies for orphan drugs, rare diseases, and ultra-rare diseases.

Experience of a pediatric integrated delivery system with the surge from the 2009 H1N1 pandemic is described, emphasizing scale, scope, and flexibility at multiple locations.

This cost-utility analysis reports on the effect of quality of life on the value of screening all new patients with colorectal cancer for Lynch Syndrome.

An evidence-based managed care budget impact model shows that incorporating TSI testing into existing Graves%u2019 disease hyperthyroidism diagnostic algorithms reduces costs and shortens time to diagnosis.