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.

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Comment by Pat Furlong on December 23, 2008 at 10:45am
Hi Julie,
Sorry for the delay in my response. At the moment, Santhera is still in discussion about next steps for Duchenne. The goal is to have Idebenone approved in the US, though the discussion in Boston actually centered around availability, access and pricing.
Comment by Julie Garcia on December 10, 2008 at 12:28pm

Will Santhera be able to get this insurance approved without a clinical trial drawing existing data from other areas? Do they have other areas they can draw data from to support insurance coverage?

If they answers to the questions above are no, then is Santhera still lanning on a clinical trial to get the evidence for insurance coverage. Do they have have their criteria for the patients for a clinical trial? Where and when do they plan to start the clinical trial and what is the expected length of the clinical trial?



Comment by Pat Furlong on December 9, 2008 at 3:10pm
The idebenone available via the internet is basically the same compound as Catera. Santhera has performed trials and is gathering evidence to prove benefit. Evidence based medicine is essential if insurers are to cover costs of therapy and therefore clinical trials very important.
Warm regards,
Comment by Bains on December 8, 2008 at 8:24am
Thanks for the update. I believe that the idebenone available over the internet is not the same as the Catera/Idebenone they are planning phaseIII trials for duchenne. Could you please comment on this? Thanks. -bain

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