The Agora in Athens, 1983
I will start this blog with a scolding. My last blog was designed to induce you to
read a Wall Street Journal Op Ed by an M.D. in Bozeman, Montana who – if not
our answer to Mother Teresa – is a close second. But, it turns out, the WSJ (profit-grubbing capitalist
swine that they are) will not let you read their articles on line unless you
are a subscriber. I have concocted a way
around this impasse and have asked those of you who want to read about Dr. Bob
to let me know by email. Google tells me
that over 20 of you already have looked at that particular blog. However, only two of you have asked for the
article! More proof that most of you
simple look at the picture of Linda and then flip down to the next cute baby or
scenery picture. Fie! My email is myrlbeck@msn.com. I expect some action.
The topic of this blog is “orphan drugs”: what they are and
how they are created. We have talked
about medical economics at excessive length many times
before. Swell, here’s more. An orphan drug is a drug designed to treat a
very rare disease. If it is
truly rare, there is no way that Big Pharma or anyone else can develop and
market a drug specific to that disease, and turn a profit. It may
surprise you to learn that Big Pharma has no soul, and so is extremely unlikely to deliberately
lose money just for the sake of the humanitarian buzz it may provide Thus, to generate orphan drugs, the public must
intervene. Short of setting up a
government owned and operated “Crown Corporation” to do the work, inducements
needs must be applied to get Big Pharma rumbling. These include tax breaks, extended patent
limits, simplified approval procedures, and probably lots of other little
tricks. Anyway, these “tricks” seem to
be successful, because there are some orphan drugs already on the market, and more in
the pipeline. One of these is “enadenotucriev”. This
unspellable and unpronounceable drug (soon to be renamed something short and
catchy, you can bet) is being developed by a smallish company in Oxford,
England. It is a treatment for
epithelial ovarian cancer. May it work
wonders!
More on orphan drugs: A European outfit named Apria has obtained “orphan drug status” for something they call APR-246, apparently a concoction that restores the function of the gene p53, which we have discussed earlier. Defective p53 impairs the ability of cells to die when they ought to have done so, thus contributing to cancerous growth, sometimes. APR-246 fixes that, or at least Apria fervently hopes it does. In this case, orphan drug status – which will greatly accelerate the testing process – was granted by the EMA, which codes for the European Medicines Agency. EMA apparently is equivalent to our FDA, although seemingly it is considerably more nimble. This drug is for use in ovarian cancer, in accompaniment to “standard care”.
ReplyDeleteThis article speculates that, because defective p53 is found in many other cancers, APR-246 may have wide application in oncology.
Read it yourself: http://www.dddmag.com/news/2015/01/apreas-ovarian-cancer-drug-gains-orphan-drug-designation