Years ago I ran across some proposed federal legislation that advocated truncating the later stages of clinical trials by using Electronic Medical Records (EMRs) to provide real-time feedback. I can't find that legislation any more - if anyone runs across it please post here.

The idea was instead of the long drawn out, extremely expensive, final phase the keeps everyone waiting for years on end for a result the drug would be placed in much wider circulation and any clinical side effects would be reported immediately. There would still be a much shorter initial human trial that followed earlier studies of expected effects based on testing and computer models.

The main benefit to communities like ours would be that drugs that currently are not brought to market because of the huge expense of full clinical trials. A good example would be ASC-J9. ASC-J9 seems to have some potential to treat SBMA. But the chances of ASC-J9, or any other drug targeted to an orphan disease, going into clinical trails are near zero.

I have heard of some legislation sponsored by disease communities that would exempt promising drugs from needing to go the full clinical trail route. I would suspect that if their attempts succeed money will be directed away from research and into lobbying for each special interest's drug of choice. A much better approach would be legislation that could leverage EMRs to make the alternative trails practically for anyone. KDA doesn't have enough funds to fully sponsor all promising research let alone get into the vote buying business in DC.

So, what to do? Standing pat isn't going to bring any promising drug like ASC-J9 to market if it only aids SBMA patients. Something has to come about to over come the current testing regime in the FDA. I would guess there will be 100's of millions spent lobbying against any change as I'm sure their are folks making billions of the current regime.

How do we break the log jam?

Bob, you make a strong case. I also wrote about some of my frustrations with the current process in my blog. 1 in 40,000 does not provide enough incentive to focus on a treatment or cure for KD. As you mention, the money just is not there. ASC-J9 has done well as a treatment for acne and something else(?). That is probably what keeps it on the table. We hope to hear something positive out of NIH concerning their testing of J9 soon. If the results are positive, perhaps we can move forward to a clinical trial.

The KDA is a member of NORD (National Organization of Rare Disorders). Through NORD, we have lobbied, wrote our congressional leadership, and helped support opportunities to speed up the testing process for those of us living with rare disorders (like KD). NORD is very active at the FDA and congressional levels of government. There are hundreds of non-profits that are members. We speak as one voice through NORD and hope that the numbers provide greater creditability than just one small organization.