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While watching a TV show, I was sort of introduced to a new term “ORPHAN DRUG”. The program dealt with rare diseases and the drugs being used for the disease. The rare diseases have also being termed as “ORPHAN DISEASE”.

I did some search on the subject and below are some facts about them. They have been reproduced from internet sites including Wikipedia.

The question that comes to my mind is “ can we in the homeopathic community contribute something on this subject”? We have so many medicine in terms of NOSODES, SARCODES, MIASMATIC REMEDIES, MOTHER TINCTURES,ETC.

We also know of many ways of treating patients developed by some eminent homeopaths of the world.

There is Sensation Method, there is Banerji Protocol, Vithulkas classical method, S. K Banerjea method. Facial analysis, and the list goes on.

All these stalwarts must have had the opportunity of facing the so called “ORPHAN DISEASE”, but nothing much seems to have been written on this subject. There is not much literature on this subject.

Will there be answer to these question? The debate remains open. Please share your thoughts on this.

An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity periods, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive. The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.

The Orphan Drug Act (ODA) of January 1983, passed in the United States, with lobbying from the National Organization for Rare Disorders and many other organizations,[2] is meant to encourage pharmaceutical companies to develop drugs for diseases that have a small market. Under the law, companies that develop such a drug (a drug for a disorder affecting fewer than 200,000 people in the United States) may sell it without competition for seven years,[3] and may get clinical trial tax incentives.[3]

Orphan drug designation means that the sponsor qualifies for certain benefits, such as reduced taxes, from the federal government. It does not mean the drug is safe and effective and legal to manufacture and market in the United States

A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.

Most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday.[1] With a single diagnosed patient only, ribose-5-phosphate isomerase deficiency is considered the rarest genetic disease.

No single cutoff number has been agreed upon for which a disease is considered rare. A disease may be considered rare in one part of the world, or in a particular group of people, but still be common in another.


There is no single, widely accepted definition for rare diseases. Some definitions rely solely on the number of people living with a disease, and other definitions include other factors, such as the existence of adequate treatments or the severity of the disease.

In the United States, the Rare Diseases Act of 2002 defines rare disease strictly according to prevalence, specifically "any disease or condition that affects less than 200,000 people in the United States,"[2] or about 1 in 1,500 people. This definition is essentially like that of the Orphan Drug Act of 1983, a federal law that was written to encourage research into rare diseases and possible cures.

In Japan, the legal definition of a rare disease is one that affects fewer than 50,000 patients in Japan, or about 1 in 2,500 people.[3]

However, the European Commission on Public Health defines rare diseases as "life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them."[4] The term low prevalence is later defined as generally meaning fewer than 1 in 2,000 people. Diseases that are statistically rare, but not also life-threatening, chronically debilitating, or inadequately treated, are excluded from their definition.

The definitions used in the medical literature and by national health plans are similarly divided, with definitions ranging from 1/1,000 to 1/200,000

How many rare diseases are there?

There are more than 6,800 rare diseases. Altogether, rare diseases affect an estimated 25 million to 30 million Americans.

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Comment by Dr. Aadil Chimthanawala on August 9, 2014 at 6:51am

Quite a Topic.. Orphan Drugs n Orphan Diseases... Doctors need to visit their past case records a bit frequently to find such diseases. Thanx for highlighting about this topic


Comment by Viktor Kalocsai on August 9, 2014 at 2:37am

Good point Yvonne.

Write a major Blog with Discussions about 'Homeopathic treatment/care of Orphaned Children' and make a part about those children who are in orphanages.

Comment by Yvonne Siblini on August 1, 2014 at 2:12pm

When you mentioned orphan I thought about children... which is a big subject too with many treatments needed as they are lost or displaced, especially with whats happening in the Middle East today

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