Orphan drugs are increasingly being approved for treatment of rare diseases in various regions across the globe

         

                                                         Orphan drugs

Orphan drugs are just chemical substances that are designed to cure certain rare medical conditions that would not be economically profitable for the manufacturer to make without support of the government. For instance, heart disease, it is a condition that is most often associate with the word orphan in medical terms. Yet, orphan drugs are also developed for conditions like typhoid, tuberculosis, Hodgkin’s disease and skin diseases such as eczema and herpes. These pharmaceuticals also include cancer DRUGS, AIDS drugs, and other type is known as RKIP or Restricted Use Information Program drug. But mostly orphan drugs are only used for rare conditions and manufacturing these drugs is often very costly and extremely risky.

Orphan drugs are of two types, one is termed as CVA or Condition-Related Access Program drug and other is designated as RKIP or Restricted Use Information Program drug. CVA is most known and most often used drug in clinical trials. The RKIP has limited uses. As the U.S. Food and Drug Administration (FDA) approval of RKIP is of extreme importance, the U.S. FDA needs manufacturers to submit data that supports their eligibility of RKIP status before allowing them to apply for it. Therefore, orphan drugs are far less available to public health market. However, the incentives to keep these drugs on the market for limited number of diseases make RKIP attractive to drug manufacturers, who can later create some new drugs for rare conditions for people with rare diseases. Recently, in November, 2021, GC Pharma received orphan drug designation (ODD) from European Medicines Agency (EMA) for its Hunterase ICV developed for the treatment of mucopolysaccharidosis type II (Hunter syndrome) in the European Union. GC Pharma had received manufacturing and marketing approval for Hunterase ICV in Japan on January 2021.

Many people question the ethics of clinical trials, particularly those conducted with human subjects. But there are good reasons for conducting them, such as tracking the effects of a new cancer drug over time or testing the effects of an anti-seizure medication. Some pharmaceutical companies have said that they carefully monitor the outcomes of these clinical trials, and the fact that all of the clinical trials of new drugs are double-blind means that there is less for the company researchers to control.