On July 9th, 2012 President Obama signed The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187, an important bipartisan piece of legislation that hopefully will encourage pharmaceutical and biotech companies to invest further in the development of medications for rare diseases. Without a doubt, further investment certainly is needed. In the United States, approximately 30 million Americans (10% of the population) are suffering from nearly 7,000 different rare diseases including multiple system atrophy (MSA). However, there are fewer than 400 FDA-approved treatments for these rare diseases. Hopefully, FDASIA will provide the incentive needed to generate a much greater number of medications for rare diseases. A key component of FDASIA is designed to encourage the FDA to more broadly and consistently use the accelerated approval pathway with drugs for rare disorders. The accelerated approval pathway can be used by the FDA to approve treatments based on clinical study data that might not be sufficient in the normal approval process, but because an urgent need exists the data is promising enough to place the drug on the market. When drugs receive accelerated approval further studies are still required to fully prove clinical benefit, but the studies are done after the product is placed on the market. This is important because clinical trials for rare diseases are very difficult to conduct due to the small numbers of patients. To date, however, the FDA has largely reserved the accelerated approval pathway for cancer and HIV drugs, even though most rare diseases have an urgent need for new or better treatments. With FDASIA now signed into law pharmaceutical companies can proceed more confidently with developing treatments for rare diseases, knowing the have a faster and more reasonable path to market. The MSA Support Group, as a member of the rare disease community, is pleased with the passage of FDASIA and hopes to see it quickly implemented and used swiftly to benefit patients with rare disease. Hopefully, one day soon, the multiple system atrophy community will have medicines that slow, halt or cure MSA itself.
FDASIA Offers New Hope For Rare Disease Treatments
Jul 10, 2012 | MSA-Blog