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Policy

A total of 130 posts are filed under Policy
Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials
  Despite growing acceptance that patients have the clearest view of the health outcomes that matter, drugs to treat rare diseases often… Continue Reading
What the FDA’s New Framework Means for Regenerative Therapies
Earlier this month, the U.S. Food and Drug Administration announced a comprehensive policy framework for the development and oversight of… Continue Reading
FDA Orphan Drug Designation in November 2017
The following is a list of U.S. Food and Drug Administration orphan drug designation issued in November 2017… Continue Reading
Why the Tax Bill Poses A Double Threat to My Family
My son Jack spent the first four weeks of his life screaming in pain and crying from hunger. When he was two-weeks-old, the pediatrician… Continue Reading
Measuring the Value of Therapies for Ultra-Rare Diseases
  The pricing of drugs is characterized by a tension between providing incentives to drug companies to invest in the development of… Continue Reading
That Other Thing in the Tax Bill Rare Disease Patients Should Worry About
The rare disease community has organized to fight a provision in the Republican tax bill that would repeal the Orphan Drug Tax Credit, an… Continue Reading
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