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In recent years, orphan drugs have become a pharmaceutical gold mine — and the latest data underscore the unbridled enthusiasm among drug makers.

Last year, there were 582 requests by companies for an orphan designation by the Food and Drug Administration, a stunning 23 percent increase from 2015 and triple the number of requests that were made a decade ago.


The race to win orphan designation reflects the high price tags that can be charged. Two recent examples illustrate the point. Biogen is charging $750,000 for the first year of treatment, and $375,000 each year thereafter, for a new drug for spinal muscular atrophy. Sarepta Therapeutics is charging $300,000 or more, depending upon patient weight, for a drug to combat Duchenne muscular dystrophy.

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  • Pharma has not and will not generate new drugs for masses. Even if they do, they will be high priced and unaffordable to masses in the developed countries in spite of mutually subsidized healthcare systems. Look at PCSK9 inhibitors, they are expensive and their value is being questioned. Orphan drugs cost six figures and generally do not cure the disease. Either patients die or go bankrupt.

    For IMS projections of $1.3 trillions to come true sales have to go up by about $75 billion per year, a highly improbable situation.

    Drug companies are generally catering to 20% of the global population. This population pays about $0.85 per day per year per patient whereas rest of the world pays about $0.05 for the same duration. Obviously they are not buying expensive drugs when they are challenged to pay for two meals per day. HCV drug is expensive in the developed countries and proportionately priced in the developing countries.

    Pharma and its lobby created a bill for orphan and neglected diseases in 1983. They are benefiting from it. Taxpayers got short end of the stick. However, this will not last forever. Golden goose will be slaughtered soon with as DJT is saying drugs have to be affordable. Patients are clamoring for the same. No one is listening. World has been brainwashed that billions per drug is the norm. Someone has to ask what are they doing to lower their costs and eliminate inefficient methods. So far they have done nothing. Even the generics have followed brand path by of inefficiency.

    A new principle has come and he will hit knuckles if class continues to misbehave. Let’s see what the new teacher can do to rein in the class as well the teachers. Irony is most politicians/teachers who can make a difference have been bought. Fun times are here.

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