Orphan Drugs Essay Research Paper The term

Orphan Drugs Essay, Research Paper The term orphan drug refers to a product that treats a rare disease affecting fewer than 200,000 Americans. Orphan drugs help the companies that manufacture them, under the Orphan drug act. Under the act a small company can pick up a product that would be worth anywhere from $5 million to $20 million a year.

Orphan Drugs Essay, Research Paper

The term orphan drug refers to a product that treats a rare disease affecting fewer than 200,000 Americans. Orphan drugs help the companies that manufacture them, under the Orphan drug act. Under the act a small company can pick up a product that would be worth anywhere from $5 million to $20 million a year. The orphan drug act has helped in the development of products to treat drug addiction, leprosy, hemophilia, and rare cancers, as well as diseases most people have never heard of, such as cryptosporidiosis (an infection caused by a protozoan parasite found in animals’ intestines that causes diarrhea, fever, weight loss, and lymph node enlargement) and neurocysticerosis. In the past the FDA under the drug act has approved few years ‘ 41 orphan drugs. One of the premier examples of how well the Orphan Drug Act can work came with the approval of the drug PEG-ADA (adenosine deaminase and enzyme the body usually produces on its own). “This was a breakthrough” FDA’s Haffner said of PEGnology, the enzyme replacement process developed by Enzon. “If it works the way it’s thought it will, this technology will change the way we can provide drugs to the patients”.

The history of the Orphan Drug Act

FDA set up the offices of Orphan Product Development in 1982 to focus on drugs, medical devices, foods for medical purposes, and biologics such as immune globulin for rare disorders. President Reagan signed the Orphan Drug Act into law on JAN. 3 1983. It guarantees the developer of an orphan drug seven years of market exclusivity and 50 percent tax credit for certain clinical research expenses. Imagine Pepsi or Coke having a monopoly on each other’s soft drink, that would be the equivalent on that. Initially, the act applied to only patient populations when it could be shown there was little or no hope of recovering development cost from sales in the United States. A later amendment to the act defined an orphan product as one with a potential patient population of fewer than 200,000 people.

Under the Orphan Drug Act, the FDA makes grants for drug development, assists the drug developer in the designing the clinical studies required for marketing and can speed up the drug approval process.

“A lot of these drugs are for very serious and life threatening disorders, in some cases affecting only a few hundred people. You have the smaller NDA (new drug application) and a group of very needy people, and that’s what expedites it. But the safety and the efficacy requirements are the same” Marlene Haffner FDA’s director of orphan product development.

Since the enactment of the Orphan Drug Act, 41 drugs for rare diseases have been developed and brought onto the market. Activity in orphan products, which, in addition to drugs, can include biologics, medical devices, and foods marketed for medical purposes, is researching an all time high. According to Haffner, FDA has designated as orphans 375 drugs and at least 150 are being actively developed or are going through the approval process. The federal award grant available to fund orphan drug development have increased steadily from $500,000 in 1983 to $7.5 million in 1990. However hardly any of the federal grants go to private organizations such as NORD.

Today’s more common definition of Orphan Drug”.

Another definition of “Orphan Drug” refers to a drug that will serve so few patients that it will be commercially impractical for a manufacturer to sponsor it. Orphan drug also refers to compounds in the public domain for which there can be no patent protection once a company goes through expensive efficacy test and gets FDA approval, so that a competitor could immediately begin producing a generic copy at a fraction of the cost. The phrase was coined in a 1968 editorial in American Journal of Pharmacy, “Homeless or Orphan Drugs.”


The Orphan Drug Act has been a major success and a model federal program. The FDA has designated Fifteen years ago after its passage, more than 150 orphan drugs have been approved for marketing in the United States, and 800. Additionally, Japan, Singapore, and Australia have enacted orphan drug legislation, and the European Union is expected to enact a similar law in 1999. Approximately 85% of orphan drugs are treatments for diseases affecting fewer than 50,000 people in the United States, But there are about 200,000 people in the united states that are currently taking orphan drugs.

The Problems:

The major obstacle to development of many new orphan drugs is the lack of research funding available to the academic scientists who are studying orphan diseases. Congressional funding for the orphan drug research grants have hardly been sufficient to fund the number of worthy grant applications submitted by researchers to the FDA (as you will see later on the application is not very easily completed). In an effort to provide “seed money” to academic scientists, NORD is raising funds from the private sector to fund worthy clinical research projects to enhance development of new orphan therapies. At the same time, educational efforts aimed at familiarizing pharmaceutical manufacturers about orphan diseases are underway. Nevertheless, congress has appropriated only $12 million per year to the FDA’s Orphan Drug Grant Program, which is hardly enough to fund research on 5,000 rare disorders.

How can the problems be solved?

One or multiple combinations of the following items can solve the problems. Most people don’t know anything about orphan drugs. Maybe if the FDA or NORD made themselves more prominent in the eyes of the public about orphan drugs and the diseases that they treat, then NORD would get more money to give to the researchers. Since NORD and the FDA are already spending $12 million per year in 5,000 rare disorders being researched, maybe they could get up to an additional $1-2 million dollars. Although that may not be enough, but it was a little bit more than what they started off with. I never heard of the term orphan drugs until I was given this paper to write. I’ll bet you if you asked people (not including medical doctors) “What an orphan drug is?” they would probably look at you like you had 3 heads or something. NORD is struggling a lot with the $ factor as I said in the beginning. When I called NORD and when I received information from NORD, in both cases they asked me if I wanted to donate any money. Granted that orphan diseases only effect less than 50,000 people in the United States, but they are 50,000 lives that you might know, or one of your friends may know. To think that the only chance that one of the 50,000 people have to live is with an orphan drug that either cost to much, or it was abandoned because NORD or the FDA didn’t have the money to finish research on the disease to make a drug. NORD is a private based company, that hardly gets any money from the government, and relies mostly on grants from colleges and universities for help. You know how you hear people dying every single day because of organ donors couldn’t be found. It’s the same thing over here. People are dying for stupid reasons, because our great government couldn’t give an organization that has already made drugs for orphan diseases such as Parkinson’s disease a few million dollars but would rather spend it on a brand spanking new jet fighter for a war that will never happen. Even if a war was to happen, two words NUCLEAR WARHEAD. Can’t stop them from coming, and if people really wanted all of us dead it will happen.

How to apply for a designation as an Orphan Product

A. A sponsor that submits a request for an orphan drug designation of a drug for a specified rare disease or condition shall submit each request in the form and containing the information required in paragraph (B) of this section. A sponsor may request orphan drug designation of a previously unapproved drug, or of a new orphan indication for an already marketed drug. In addition, a sponsor of a drug that is otherwise the same drug as an already approved orphan drug may seek and obtain orphan drug designation for the subsequent for the same rare disease or condition if it can be present or plausible hypothesis that its drug may be clinically superior to the first drug. More than one sponsor may receive orphan drug designation of the same drug for the same rare disease or condition, but each sponsor seeking orphan drug designation must file a complete request for the designation as provided in paragraph (B).

B. A sponsor shall submit 2 copies of a completed, dated, and signed request for a designation that contains the following:

1. A statement that the sponsor requests orphan drug designation for a rare disease or condition, which shall be identified with specifically.

2. The name and address of the sponsor; the name of the sponsor’s primary contact person and/or resident agent including title, address, and telephone number; the generic and trade name, if any, of the drug or drug product; and the name and address of the source of the drug if it is not manufactured by the sponsor.

3. A description of the rare disease or condition for which the drug is being or will be investigated, the proposed indication/s for use of the drug, and the reasons why such therapy is needed.

4. A description of the drug and a discussion of the scientific rationale for the use of the drug for the rare disease or condition, including all data from non-clinical laboratory studies, clinical investigations, and other relevant data that are available to the sponsor, whether positive, negative, or inconclusive. Copies of the pertinent unpublished and published papers are also required.

5. Where the sponsor of a drug that is otherwise the same drug as an already approved orphan drug seeks the designation for the drug for the same conditions, an explanation of why the proposed variation may be clinically superior to the first.

6. Where a drug is under the development for only a subset of people with a specific disease or condition, a demonstration that the subset is medically plausible.

7. A summary of the regulatory status and marketing history of the drug in the United States and in foreign countries.

8. Documentation, with appended authoritative references, to demonstrate that: The disease or condition for which the drug is intended affects fewer than 200,000 people in the United States or, if the drug is a vaccine, diagnostic drug, or preventive drug, the people who the drug will be given to inn the United States are fewer than 200,000 people.

9. A statement to whether the sponsor submitting the request is the real party in interest of the development and the intended or actual production and sales of the product.



The U.S. FDA web site

Docket NO. 85n-0483 Orphan Drug Regulations.

Emery J. Sturniole


Program Overview

Alliance of Genetic Support Groups


Rare Disorder Network of General Clinical Research Centers


National Organization for Rare Disorders


Excerpts of Statues Relevant to the Development and Approval of Drugs, Medical Devices, & Medical Foods for Rare Diseases or Conditions.

Orphan Drug Amendments of 1992

Orphan Disease Update newsletter volume 7 and 10 ed. 3

National Organization for Rare Disorders

Rare Disease Database Report