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social, health, political imagery through the lens of George J Huba PhD © 2012-2019

Posts tagged rare and orphan disease

I frequently tweet about neurological diseases, sending out links to US government and major foundation web sites. These tweets are among the most retweeted and favorited of those I distribute.

As you may have inferred as you look at the fact sheets distributed, there are commonalities among many of these diseases above and beyond the fact that these are all diseases of the nervous system.

Very few of these diseases have treatments. Most of these diseases are rare and often not detected by primary care physicians or even related specialists like psychiatrists. Medications are frequently used off-label for controlling symptoms like depression, anger, tremor, and many others but these treatments are rarely effective for a long time, if at all, for most patients. Because these are rare diseases and neurological research itself is quite expensive, a small portion of the US medical research budget is spent looking for cures or effective symptom control.

The following mind map shows some of the commonalities among the neurological diseases. Click on the image to expand it.

neurological diseases features common to many conditions

The next mind map is identical to that above. The formatting has been changed so that you (and I) can judge if an alternate format is more useful for certain audiences.

neurological diseases features common to many conditions2

stop making publicizing your disease your end goal. You and the other 350 or 3,000 or 25,000 or 199,999 people with the disease will hardly be heard above the shouts of those advocating for funds for cancer, coronary disease, diabetes, HIV/AIDs and other diseases affecting many medically and/or politically.

And in the current system of new drug development, Big Pharma is going to be more interested in developing treatments for gastroenterological disease (heartburn), STDs (avoidable), erectile dysfunction, safer birth control, cancer, heart disease, and obesity.

Get smart.

Your 5,000 sufferers should collaborate with the 350 individuals with another disease and the 199,999 with another and all of the rest of them to be a large and huge advocacy group for encouraging change. Your illness group may not be the first to get attention if changes are made, but somebody will be and as treatments are developed for one rare disease they might also be applicable to other related rare diseases.

This is clearly a situation in whch cooperating with those with other rare diseases will ultimately yield better results for all than screaming ME FIRST on the Internet in social media.

The existing laws and administrative rules probably do not go far enough in encouraging drug companies to develop pharmaceuticals for rare and orphan diseases. Advocate for better incentives and decreased bureaucracy for developing new pharmaceuticals to treat a few thousand. Maybe even the staid Nobel Prize committee will even make an award to somebody who makes a huge research contribution that advances the development of treatments for a rare disease and top research universities will create endowed professorships for high talented physicians and others who study a rare disease.

Click image to expand.

ENCOURAGE MORE RESEARCH  ON RARE AND ORPHAN DISEASES