Remember when I asked you to sign an online petition in support of expanded access (aka compassionate use) to promising treatments for ALS? Well, in a bit of cruel irony, the writers of the original petition (dubbed ALS Treat Us Now) have sadly succumbed to the disease. However, their spirit lives on…In 2012, with your Read more about ALS-ETF[…]

Rare Diseases, Rarely Affordable Drugs

The Orphan Drug Act of 1983 was designed to encourage pharmaceutical manufacturers to develop drugs for diseases that affect less than 200,000 Americans (ALS affects an estimated 30,000 Americans). “While many industries serve niche markets without special government encouragement, few face the extraordinary regulatory barriers that pharmaceutical companies face,” Bill Frezza for Bio-IT World. These barriers Read more about Rare Diseases, Rarely Affordable Drugs[…]

Factoid Friday #111

The IMS Institute for Health Informatics report, The Global Use of Medicines: Outlook Through 2016, forecasts that global launches for new molecular entities (NMEs) will rebound to 32 to 37 per year through 2016 – up from fewer than 30 in four of the last seven years.

Future Treatments for ALS

According to IMS Health, the number of drugs to treat orphan diseases is set to rise, yet in some of these disease areas pipelines are dry and launches scarce. One orphan disease in particular that is unlikely to feature prominently among new molecular entities (NMEs) is amyotrophic lateral sclerosis (ALS). The most common type of Read more about Future Treatments for ALS[…]

We Need the FDA to be Faster

Last Monday, leaders from nearly every major ALS organization in the United States, in addition to patients, caregivers, physicians, scientists and other concerned individuals, addressed a panel of representatives from the U.S. Food and Drug Administration (FDA) as the agency conducted its first-ever public hearing specific to amyotrophic lateral sclerosis (ALS).The day’s agenda can be Read more about We Need the FDA to be Faster[…]

FDA ALS Public Hearing (cont’d)

Pat Wildman, Director of Public Policy for the ALS Association, plans to call for expanded access and accelerated development and approval of ALS therapies at next week’s FDA ALS Public Hearing. He also will advocate for the use of biomarkers and surrogate endpoints β€œto speed development and let us know sooner whether something is working or not working,” Read more about FDA ALS Public Hearing (cont’d)[…]

FDA ALS Public Hearing

The Food and Drug Administration (FDA) has announced a public hearing to obtain input on their regulation of drugs for the treatment and/or management of amyotrophic lateral sclerosis (ALS). The FDA is holding this public hearing to allow patients, caregivers, advocates, health care providers, academia, industry, and other interested persons to give their perspectives. The input Read more about FDA ALS Public Hearing[…]