On Wednesday, Dec. 3 I was invited to participate in an Expert Roundtable meeting with Santhera, discussing Idebenone. Idebenone is a synthetic CoQ10 and suggested to provide cardiac benefit. It is available via the internet. Santhera's goal is to perform clinical trials in an attempt to have Idebenone approved as a therapy. While it is by no means a magic wand, it is potentially one element in the 'DMD cocktail' for treatment.
We discussed a range of issues from evidence based medicine, clinical trials, pricing treatments in rare disease and access. What is most amazing about all of this is the fact that we ARE now discussing these issues, when just a few short years ago, there were few treatments on the horizon. Discussions such as this one held by Santhera pave the way for future discussions about other therapies. This is just the beginning, but the fact that we were sitting around a table talking about cost, insurance coverage, co-pay, fair pricing and access is pretty exciting.