Orphan Drugs........

ORPHAN DRUGS

Orphan drugs are those drugs the pharmaceutical and other biotech companies are constantly researching and developing new medications to treat a rare medical condition, the condition itself is referred to as an Orphan disease. 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.

Orphan Disease:

A rare disease is also referred to as an “Orphan Disease”, is any disease which affects a small percentage of the population. Most of the rare diseases are genetic in nature,so these are present in a person throughout in his/her life,even if the symptoms do not appear immediately. Many rare diseases appear early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday. A rare disease occurs in less than 200,000 individuals in the United States, or less than 5 per 10,000 individuals in the European Union. Government regulatory agencies in the United States and the European Union have thus taken steps to reduce this disparity.

Orphan drug Legislation:

        Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy.

The intervention by government on behalf of orphan drug development can take a variety of forms:

 Tax incentives.

 Enhanced patent protection and marketing rights.

 Clinical research financial subsidization.

 Creating a government-run enterprise to engage in research and development.

1983 US Orphan Drug Act:

The Orphan Drug (ODA) of January 1983 was passed in US with the lobbying from National Organisation of Rare Disorders and many other organisations is meant to encourage phamaceutical companies to develop drugs for diseases that have a small market.

Under the law the companies who develop the drugs are able to sell them without competition for seven years and may get clinical trial tax incentives.

These incentives include:

 Federal tax credits for the research done (up to 50% of costs) to develop an orphan drug

 A guaranteed 7-year monopoly on drug sales for the first company to obtain FDA marketing approval of a particular drug. This applies only to the approved use of the drug. Another application for a different use could also be approved by the FDA, and the company would have exclusive marketing rights for the drug for that use as well.

 Waivers of drug approval application fees and annual FDA product fees.

  

The US office of Orphan Product Development:

The U.S Food and Drug Administration(FDA) is responsible for ensuring the safety and efficacy of medications on the market in the United States. The FDA established the Office of Orphan Product Development (OOPD) to facilitate the development of orphan drugs (and other medical products for rare disorders), including offering research grants.

Orphan drugs, like other medications, still have to be proven that they are safe and effective through research and clinical trials before the FDA will approve them for marketing.

Currently, 350 drugs have been approved for sale under the Orphan drugs in U.S.

Some of the Orphan Drugs are:

1) Trade name: Alphanate
    Generic name: Anti hemophilic factor (human)

· Approved for surgical and/or invasive procedures in individuals with von Willebrand disease   in whom desmopressin is either ineffective or contraindicated.

2) Trade name: Ceprotin
    Generic name: protein C concentrates.

·       Approved for treatment of congenital Protein C deficiency to prevent and treat blood clots in the veins and Purpura fulminas.