Thursday, March 14, 2013

F.D.A. Plans Looser Rules on Approving Alzheimer’s Drugs

There is news on the pharma front regarding medications for the treatment of Alzheimer's.  A parent of mine suffered through the horror of this disease, and the family suffered, watching the shell that my parent became in the end of her life.  So, I watch news of this horrible disease and the drugs being developed to combat it.  The cause and progression of Alzheimer's disease are not well understood. Research indicates that the disease is associated with plaques and tangles in the brain. I could spend pages discussing this disease, but I am able to simply say - if you have seen it - you know how horrible it is.   

According to the NY Times, whose headline I shamelessly copied, here is the news:

Drugs in clinical trial would qualify for approval if people at very early stages of the disease subtly improved their performance on memory or reasoning tests, even before they developed any obvious impairments. Companies would not have to show that the drugs improved daily, real-world functioning.

From JAMA: 

The focus of drug development in Alzheimer's disease has increasingly been earlier disease stages, before overt dementia. This refinement of focus, however, raises important new challenges because the subtleties of cognitive impairment in patients with early-stage Alzheimer's can be difficult to assess. Moreover, the range of focus must extend to healthy people who are merely at risk for the disease but could benefit from preventive therapies. In recognition of these shifting challenges, the FDA has developed guidance for the design and execution of clinical trials involving patients who do not present with dementia. 

Despite our growing understanding of the relationship between various disease-based biomarkers and the clinical course of Alzheimer's disease, it remains unclear whether the effect of a drug on one or more such biomarkers can actually predict a meaningful clinical benefit

So the question is - would speeding up the approval process for new drugs to be used at very early stages make sense? Having seen the disease myself every day, I would say yes. What is the downside? Side effects and their severity would (in my opinion) be hashed out early in the approval process, as they always are. 

The key to me is the statement - a drug that is administered before the impairments become obvious - that holds promise. Unfortunately, the impairments are gradual, and if like me you are many miles from a family member and the visits are not frequent, by the time you notice the impairments, it's too late to slow the progression with medications that may work.  

There are more than 1000 trials involving medications to combat this disease. If there is an expedited approval process, perhaps one or more could help. 

Someone tell me the downsides. email me at galawyerblog(at)