Measuring the Value of Therapies for Ultra-Rare Diseases

  The pricing of drugs is characterized by a…

Duchenne UK Collaborates with Industry to Speed Payer Value Assessments
Rare Daily Staff Duchenne UK has launched a collaboration with five pharmaceutical companies developing treatments for Duchenne muscular… Continue Reading
Akcea Initiates Mid-Stage Trial of Therapy in Patients with Rare Hyperlipidemias
Rare Daily Staff Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals focused on developing and commercializing drugs to treat patients… Continue Reading
New Rare Disease Center Opens in U.K. to Treat Rare Genetic and Skin Conditions
Rare Daily Staff The Rare Diseases Centre at St Thomas’ Hospital, a first-of-its-kind center in the United Kingdom to treat patients with… Continue Reading
ICER Issues Modified Framework for Assessing Value of Treatments for Ultra-Rare Diseases
Rare Daily Staff The Institute for Clinical and Economic Review, an independent non-profit research institute that analyzes the evidence on… Continue Reading
Trump Taps Former Drug Industry Exec to Head HHS
Daniel S. Levine President Donald Trump said he would nominate Alex Azar, an attorney and former drug industry executive who served in the… Continue Reading
Dynacure Licenses Ionis Antisense Drug for the Treatment of Centronuclear Myopathy
Dynacure, a 2016 spin-off from the Institute of Genetic and Molecular and Cellular Biology in France, entered into a license agreement with… Continue Reading
Transgenic Stem Cell Therapy Restores Skin of Boy with Rare Genetic Condition
Rare Daily Staff A seven-year-old boy in Germany who lost 80 percent of his skin to a potentially lethal rare genetic condition that causes… Continue Reading