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New Focus on Specialty Disease Drugs by Pharmaceutical Companies

Here in the United States, rare diseases are categorized as such if they affect less than roughly 650 patients per million people of this country’s total population. Over in Europe in the relatively newly formed European Union, a rare disease is noted or categorized as being rare if it affects less than 500 patients per million of its population. Even rarer diseases yet, which are referred to as ultra-rare, are assigned that distinction when less than 20 patients per million suffer from them. Sometimes known as a specialty disease, rare and ultra rare diseases are quickly becoming the focus of pharmaceutical companies looking to produce niche drugs.

The Math Behind the Numbers
To break it down for you, while in the U.S. very common or prevalent diseases may affect upwards of millions of people, rare diseases will typically touch the lives of less than 200,000 individuals while ultra-rare diseases will be responsible for affecting 6,000 people at the utmost and often times far less. Many of these diseases currently designated as rare and ultra-rare are termed as chronic and progressive. They are usually marked by ongoing pain and severe disability and often accompanied by high rates of mortality.

The Debate Rages On
With the seemingly endless list of diseases to contend with, the debate rages on about the decision by many pharmaceutical companies to pursue the present course of specialty disease drugs for such extremely rare conditions such as aHUS, or atypical hemolytic uremic syndrome, which is an ultra-rare genetic disorder characterized as being extremely debilitating and life threatening. Take Alexion Pharmaceuticals. Alexion has discovered and developed Soliris, aka eculizumab, which has been touted as the first treatment for individuals suffering from paroxysmal nocturnal hemoglobinuria or PNH and aHUS. Both of these horrific and debilitating diseases have far-reaching consequences.

While both of these disease’s sufferers no doubt count their blessings every day for the scientific research and advancements that have led to the development of these break-through drugs, many people are asking the question: Why aren’t cures for diseases that impact the masses like cancer and Alzheimer’s and many other more commonly known diseases that plague the world being more aggressively pursued? This is not to begrudge the patients with far less common maladies. People are almost as happy for them as the sufferers are themselves at the prospect of the relief. No moral and compassionate being would begrudge another relief from pain and suffering.

The Crux of the Situation and Moral Implications
So it isn’t a matter of sour grapes that’s compelling the grumblings. It’s the perceived loss of ground in the advancements for cures for what some have termed the “bigger picture” diseases, those highly prevalent diseases that are taking us out every year in staggering numbers. It’s prompted many to accuse drug companies of tailoring cures for those niche groups that can be charged exorbitant sums of money for drugs that are as rare as the diseases they treat. Let’s face it: once a disease that affects the masses has been cured, the revenue in connection with these diseases would eventually dry up, making it in essence a non-lucrative endeavor for pharmaceutical companies.

After all, the companies themselves will only benefit as long as there’s a demand for the remedy. Once that demand is no longer in need or perceived to be in need and the challenge has been met an end to the profits are surely in sight. The argument has been espoused for decades that there’s no money in finding a cure for cancer because the long-term treatment of it is far too lucrative. Let’s hope that’s not the true motivation behind the lack of a cure for it and the dozens of other life-threatening diseases out there that continue to plague mankind. We’d hate to think of the pharmaceutical companies as being purely for profit with their own agendas as the driving forces behind them.