Rare Disease Drugs Companies Require to Undergo Rigorous Testing for Approval and Launch of Drugs

Rare disease drugs, also known as orphan drugs, are simply drugs developed with no connection to any previous medical use in humans, and thus would not be profitable enough to make any profits from manufacture without governmental support. However, the conditions for obtaining these drugs are quite demanding. This is because the companies that develop them are generally those specializing in research and development of medicines for rare diseases. Thus, in order to receive financing from the government for the research and development of these rare drugs, these companies must prove beyond reasonable doubt that their drugs can be useful for humans.

Rare Disease Drugs

 

The companies attempting to market rare disease drugs require to undergo rigorous testing. This testing procedure can take many months or even years, depending on the condition with which the company is seeking to treat and its profitability level. Often, it is the long duration of testing that hampers the development of drugs intended for rare diseases.

Thus, it is not surprising that pharmaceutical companies are less than enthusiastic about rare disease drugs. One reason for this is that these drugs may have numerous side effects and this makes them less likely to bring about any benefit when they are used in patients. Another reason for the reluctance of the companies to develop drugs intended for orphan diseases is the sheer complexity of the drug itself.

It requires great expertise to be able to manufacture rare disease drugs and thus it takes time before such drugs are released into the market. It is also important to realize that some drugs intended for treating rare diseases are also highly effective in the treatment of common diseases. Hence, it is possible for pharmaceutical firms to release drugs that have low potentials of causing side effects and yet render other benefits to patients suffering from common diseases. Such drugs are more profitable as compared to drugs intended for rare conditions.

There are a number of pharmaceutical companies in the U.S that are involved in the production of rare disease drugs. For instance, in June 2020, TFF Pharmaceuticals, Inc. received the U.S. Food and Drug Administration’s orphan drug designation to Tacrolimus Inhalation Powder for prophylaxis of lung allograft rejection.

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