Interesting National Public Radio Interview

[Rosie]

I was listening to NPR today while doing some errands, and heard an interesting interview with a prominent British scientist (didn’t catch the name for sure, but I think it might be Colin Blakemore) who was/is head of the Medical Research Council which is responsible for deciding what therapies will be covered by their national health insurance. He made a couple of points that are pertinent to our problems with getting doctors to accept the idea that the vast majority of MC patients can help their symptoms tremendously by eliminating gluten and other offending foods from their diet.

His main point is that observation is just as valid a way of coming up with answers in medicine as experimentation with the “gold standard” of randomized controlled trials. He gave the interesting example of Mendel having come up with principles of heredity by experimentation with plants and Darwin having come up with the theory of evolution based solely on careful observation.

His next comment was that in America the medical profession is really hung up exclusively on experimentation to the detriment of patients, and he gave several examples. One of them really is analogous to our issues. There is a drug used to treat one disease that was coincidently observed to also prevent blindness in virtually all patients with a certain type of retinal deterioration in Britain. The drug was then used immediately to treat everyone with this condition in Britain and some other countries. But in the US, it was decided that a clinical trial had to be done before allowing the drug to be used. So during that year, all the control patients went blind, as well as those patients who could have been treated but weren’t because they were waiting for the end of the clinical trial.

And this is just what Dr. Fine is running into, when he tries to get his careful “observational” data on gluten intolerance published. The journals want a rigorous randomized controlled trial before considering publication, with the resultant suffering of those in the “control” group.

Tex, you have often quoted the statistic that about 80% of all the people on the MC Forum have discovered that their symptoms have improved when gluten was removed from their diet, and about 50% also have to get rid of dairy. That’s a huge percentage! Many drugs that are tested require a statistician to show that they have an effect. And my husband who worked in pharmaceutical research for many years often said that if you need a statistician to prove that a drug works, then you don’t have a very good drug, but many of them are marketed anyway.

It’s hard to change entrenched mind sets, but hopefully Dr. Fine and persistent MC patients will be able to make some progress with the medical community over time.

[Polly]

Very interesting, Rosie. Thanks.

Polly

[Lucy]

Rosie,

There are alot of ethical problems that come up in doing research, that's certain.
Think there is a way to suspend a study before it's over, if the results are overwhelmingly positive in a group of test subjects (patients). If that's the case, I think they go ahead and put ALL the patients on the medication in some cases.

Perhaps some of you have had experience with studies like this in which that has been done. Not sure what the procedure is for fast forwarding the use of a medication like that, however.

Yours, Luce

[tex]

Rosie,

That was a very enlightening interview. We definitely have our share of problems, with the FDA. Of course, many of our regulatory agencies in this country are dysfunctional, because they're hopelessly entangled in red tape of their own making. It seems that no matter how much money the legislature throws at them, the situation just continues to decline. The FDA has to be the world's worst, though. On the other hand, I sure wouldn't want their job.

I totally agree with your husband - if they need a statistician to determine the effectiveness of a drug, they're grasping for straws.

Thanks for the information,
Tex

[MaggieRedwings]

Rosie,

Thanks for posting that information and I found it very informative. Sure wish they could fast forward a trial when everything shows that it works.

Love, Maggie

[Rosie]

Lucy and Maggie, there are ways to fast forward a drug, but by the time all the rules are followed, I suspect that the time has passed anyway.

Dr. Fine's research is a great example of why observational data should be acceptable. Normally, clinical trials are done to establish 3 things:

Safety (side affects)
Efficacy (does the treatment work)
Optimization (dose and timing)

In the case of gluten-free diet, safety isn't an issue and optimization isn't an issue, because going gluten free is the only "dosage".

Efficacy can simply be established by determining what percentage of people improve significantly on the gluten free diet. There are a few things to figure out, such as sample size, time frame, and the definition of "significant improvement". But I'll bet that Dr. Fine has already done that. It isn't necessary to have a control group that doesn't go on the gluten free diet, since gluten insensitivity isn't something that just goes away on its own, as many of us with years of problems can testify.

But unfortunately the medical establishment is hung up on the idea that the only way is the randomized, controlled trial.

[tex]

But unfortunately the medical establishment is hung up on the idea that the only way is the randomized, controlled trial.

Also, the medical community doesn't like "upstarts" who try to argue against long-standing, (but unproven), medical "beliefs", and they are not above going out of their way, to try to suppress such efforts. That has happened many times, in the history of medicine.

Tex