The history, regulations and uses of orphan drugs.

Published: 2019/12/04 Number of words: 1429

Drugs which are developed to treat rare diseases are known as ‘orphan drugs’. In this case, ‘rare diseases’ mean diseases that affect few members of the population. Mostly research is always going on in relation to the commonly produced diseases but no one was carrying research out on the rarely developed diseases. Because of this, in 1983 the Orphan Drug Act was passed to increase the research, development and approval of the drugs in America. Under this Act, financial benefits were provided to companies to produce more orphan drugs. After this Act, more than 100 drugs have been developed, which is beneficial to people who are affected by rare diseases.

History 2, 3, 4
In the 1980’s, a very small number of orphan drugs had been developed because of fewer benefits to companies and small patient populations. Mostly in America, there are large numbers of the population affected by rare diseases. So the United States FDA is responsible for the safety and efficiency of drugs which are developed for diseases. On the basis of this, in 1982 the Orphan Drug Act (ODA) FDA arranged that the Offices of Orphan Product Development (OOPD) focus on drugs which are used for rare disorders and which would not make any profit. P resident Reagan signed the Orphan D rug A ct into law on 3 January 1983. In this A ct, some benefits are provided to the companies or manufacturer s. This A ct provide guarantees to the developer of an orphan drug of seven years of market exclusivity and 50% tax credit for certain rare disease research. Before this A ct, only 41 drugs had been developed, but after this Act, the FDA has designed 375 drugs and 183 of them got approved as new orphan drug products.

In the ODA, various minor improvements were carried which were passed by Congress in 1985, 1988 and 1984. In 1983, this Act (ODA) was to give the direction to the Orphan Product Development (OOPD) to focus on drugs which are used for rare disorders and which would not make any profit. This means they are not considering the recovery cost of that product. But in 1984, the Act defined an orphan drug as one with a potential patient population of less than 200,000 patients affected by rare diseases. This means they are considering the recovery cost of the orphan drug. Again in 1985, the Act was changed, whereby the market exclusivity was extended for both un-patentable and patentable products. In 1988, the application submission for orphan drugs was improved.

Regulations on orphan drug development 2, 5
There are different regulations on orphan drugs in different countries. In the USA, the FDA is responsible for the safety and efficiency of drugs on the market which were established in the regulations for orphan drugs in 1983 (ODA). Singapore also has approved the Orphan Drug Exemption Act 1991. Similarly, Japan passed its own Orphan Drug Act in 1993 and the European Medicines Agency (EMEA) set up regulations for the European Union in 1995. Different regulations have been established by different countries but their aims are the same for all, i.e. to encourage the development and marketing of orphan drugs for rare diseases. There are many different committees which regulate the orphan drugs combined with FDA, EMEA and others. Legislation has been introduced in the 107th Congress to modify marketing exclusivity provisions and to accelerate and expand tax benefits for orphan drug manufacturers. In the 107th Congress, Government modified laws which were more beneficial for manufacturers of orphan drugs.

Incentives for orphan drug development 2
The Orphan D rug Act provided some incentives to induce companies to develop drugs for rare diseases. So to increase the research and development on orphan drugs, this Act provides the following various incentives for the manufacturer :

  1. 50% t ax credit for clinical research on orphan drug development
  2. Guaranteed seven years of market exclusivity to the developer of an orphan drug
  3. Smaller n ew drug application
  4. Federal award grant available to fund orphan drug development
  5. Waivers of drug approved application fees and annual FDA product fees.

From among all the above, the market exclusivity is a very strong incentive to increase the development of orphan drugs. It gives a guarantee to the developer that their drug should not be approved for other developers by the FDA for seven years. Market exclusivity is a more effective incentive than a patent.

Rare diseases and orphan drug development 6, 7, 8, 9, 10
Diseases that affect to 6-8% of the world population are known as rare diseases. The pharmaceutical Manufacturers Association (PMA) and the National Organisation for Rare Disorders (NORD) have estimated that there are 5,000 rare diseases found in 200,000 people . Every year there are hundreds of pathogenic disorders identified and that are of genetic origin. The top seven rare diseases are cancer, metabolic, infectious, neurological, haematological, and pulmonary diseases and those due to poisoning . Cancer is the most prevalent rare diseases such as ovarian cancer or hairy cell leukaemia, followed by metabolic disorders. Before the Orphan D rug Act, only 41 drugs had been developed, but after this Act the FDA has designed 375 drugs and 183 of them got approved as new orphan drug products.

Also there are many drugs approved by FDA for the above mentioned top seven types of rare diseases, i.e. 38 for cancer, 37 for metabolic, 23 for infectious, 18 for n eurological, 16 for haematological, and nine for pulmonary diseases and poisoning.

Following are some rare diseases and approved orphan drugs: 10, 11

Rare diseaseOrphan drug
Wilson’s diseaseZinc acetate dihydrate
Ovarian cancerEpothilone B, Oregovomab
Cystic fibrosisDuramycin, ciprofloxacin
Pancreatic cancer and gastric cancerG17(9) gastrin-diphtheria toxoid conjugate
Follicular lymphomaTositumomab
Cocaine poisoninga-1-acid glycoprotein
Gaucher’s diseaseMiglustat (zavesca)
Fabry’s diseaseGalsidase beta, galactosidase

From all above reports, the cost of product development is the major disadvantage for research on orphan drugs. The regulations increase the research and development on orphan drugs in the pharmaceutical industry. The development of the Orphan Drugs Act gives benefits to society which is suffering from rare diseases. So such types of Acts are required, which solve the problems for both the pharmaceutical industry and patients.


2. M. Angeles Villarreal, Analyst in industrial organization resources, science and industry division, Orphan Drug Act: Background and Proposed Legislation in the 107th Congress, July 25, 2001.



5. Newsline, The Journal of Nuclear Medicine, Agency Matches Investigational Drugs with Commercial Sponsors, FDA ORPHAN DRUG PROGRAM READY TO AID RADIOPHARMACEUTICAL INVESTIGATORS.

6. Aidan Hollis, Drugs for Rare Diseases: Paying for Innovation


8. Andrew W Knight and Timothy P Senior, The common problem of rare disease in general practice, MJA, Volume 185 Number 2, 17 July 2006

9. Marlene E. Haffner, MD, MPH, Adopting Orphan Drugs – Two Dozen Years of Treating Rare Diseases related article, pg. 462.



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