The Prescription Drug User Fee Act (PDUFA) allows the FDA to collect fees from drug makers that produce specific human drug and biological products. These fees help to support the new drug approval process. In general, applications for products that have been designated as a drug for a rare disease are exempt from this fee. The statutory authority for PDUFA expires in September 2012.
At that time, new legislation will be required for FDA to continue collecting user fees for the prescription drug program (the renewal of this Act is called PDUFA V). The FDA will hold the first of many meetings to gather input in advance of the next PDUFA, giving stakeholders an opportunity to weigh in on user fees the agency charges for drug review and how those fees are used by the agency.
The July 1 meeting is open to consumer advocacy groups, health care professionals and scientific and academic experts, the FDA says in a notice in the Federal Register.
An example of PDUFA’s relevance for families impacted by a rare disease diagnosis is the drug Acthar, marketed by Questcor, which is used in treating patients with infantile spasms (“IS”), a rare form of refractory childhood epilepsy, and opsoclonus myoclonus syndrome, a rare autoimmune-related childhood neurological disorder.
Opsoclonus Myoclonus Syndrome is an extremely rare auto-immune disorder that affects as few as 80 children a year in the United States. It is caused by a neuroblastoma, a cancerous tumor, or a virus. It is thought that antibodies in the immune system designed to fight off the tumor (or virus) end up attacking the brain as well. Opsoclonus” is an unusual disorder of eye movement in which both eyes dart involuntarily (dancing eyes). “Myoclonus” simply means brief muscle jerks and “ataxia” indicates incoordination. The syndrome has been called by many other names, such as “Kinsbourne syndrome,” “dancing-eyes-dancing-feet” or opsoclonus-myoclonus syndrome (OMS). Most children are less than two years old when diagnosed. There is no cure for OMS. (A Global Genes Project Organization)
Facebook Groups—
Opsoclonus Myoclonus Syndrome Awareness
OMS Families – Opsoclonus Myoclonus
The onset of infantile spasms (sometimes characterized as West Syndrome) is usually in the first year of life, typically between 4-8 months. The seizures primarily consist of a sudden bending forward of the body with stiffening of the arms and legs; some children arch their backs as they extend their arms and legs. Spasms tend to occur upon awakening or after feeding, and often occur in clusters of up to 100 spasms at a time. Prognosis is dependent upon the underlying disorder. Many underlying disorders, such as birth injury, metabolic disorders, and genetic disorders can give rise to spasms, making it important to identify the underlying cause. In some children, no cause can be found.
Infantile Spasms and TSC: A Devastating Diagnosis

From Twitter—
@bsangel: it’s not for a prize or anything, it’s just about spreading infantile spasms awareness. 🙂 (
@ PLM_Epilepsy: our journey with infantile spasms: epilepsy walk

  • June 23, 2010: Congressional Hearing on the Food and Drug Administration’s (FDA) Review Process for Rare & Neglected Diseases
  • June 29-30, 2010: Public Hearing Regarding Food and Drug Administration’s Review and Regulation of Articles for the Treatment of Rare Diseases
  • July 1, 2010: Prescription Drug User Fee Act (PDUFA) Reauthorization Stakeholder Meeting