An FDA (Food & Drug Administration) approved treatment means that the treatment has been reviewed and approved by the FDA for a particular disease and usually for a particular type or stage of the disease. The FDA approval is based on the efficacy and safety of the drug demonstrated in large phase III trials. Since most trials are conducted for a limited period of time, after a drug is approved, the FDA continues to conduct post-approval, also called post-marketing surveillance, to gather more data regarding the safety of the drug.
Currently, nine drugs are approved by the FDA for the treatment of multiple sclerosis based on their ability to change the course of multiple sclerosis by either reducing the frequency of attacks or slowing the progression of the disease or both. These drugs (year of FDA approval) include:
- Betaseron 1993
- Avonex 1996
- Copaxone 1997
- Novantrone 2000
- Rebif 2002
- Tysabri 2006
- Gilenya 2010
- Aubagio 2012
- Tecfidera 2013
An off-label treatment means that the drug has not been officially approved by the FDA for a particular disease, but there is evidence and experience to support the use of that drug for the treatment of that disease.Off-label treatments are very common in medical practice and usually are the result of years of experience and research carried out by doctors and scientists. However, since these drugs have not been formally tested in phase III trials with successful results presented to the FDA, they are considered “off-label”. Nonetheless, from small scale studies, there is reasonable evidence to support their use based on their ability to reduce the frequency of relapses or slow the disease progression or both. Examples of off-label treatments for multiple sclerosis include:
- IVIG (intravenous immunoglobulins)
- Plasmapheresis or plasma exchange
- Autologous hematopoetic stem cell transplant therapy
The above-mentioned list is not complete and there may be other drugs used by doctors in their practices as “off-label” for their multiple sclerosis patients.