Friday 6 January 2012


I. What are Orphan Drugs?

In the USA, Orphan Drugs are defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but it is not expected to recover the costs of developing and marketing a treatment drug”.

In the EU, Orphan Drugs are defined as “those that are intended for diagnosis, prevention or treatment of diseases that affect fewer than 5 in 10,000 people across the EU.”

In simple words “Orphan Drugs are those, which are intended for the treatment of rare diseases.”

The Companies which are involved in the research and development of Orphan Drugs are provided incentives by the Governments of various countries which are discussed below.

II. What is the need for designation of drugs used in the treatment of rare diseases as orphan drugs and providing incentives to pharmaceutical companies which are involved in developing orphan drugs?

For the development and commercialization of new medicines, lot of money and time is involved and moreover, the success rate of new medicines is less. Hence, we cannot expect a pharmaceutical company to work on medicines used for the treatment of rare diseases, since the market for rare diseases is very less.  Hence there is a need for providing incentives in order to encourage pharmaceutical companies which are involved in developing orphan drugs.

The following may be the general incentives provided by governments of various countries:
  • Tax incentives.
  • Enhanced patent protection and marketing rights.
  • Clinical research financial subsidization.
  • Creating a government-run enterprise to engage in research and development

III. Significant Legislations related to Orphan Drugs in USA and EU-

a) Orphan Drug Act of 1983-
  • It is a law passed in the United States designed to facilitate the development and commercialization of drugs to treat rare diseases, called Orphan Drugs.
  •  Orphan drug designation does not indicate that the therapeutic is either safe and effective or legal to manufacture and market in the United States.
Important features of Orphan Drug Act-
  • The companies developing Orphan Drugs have marketing exclusivity for 7 years.
  • The companies may get clinical trial tax incentives.
  • The companies may have reduced taxes from the federal government.
b)  Rare Diseases Act-
This legislation amended the Public Health Service Act to establish the Office of Rare Diseases in USA. It also increased funding for the development of treatments for patients with rare diseases.

c) REGULATION (EC) No 141/2000 of European Union-

Features of REGULATION (EC) No 141/2000-
  • This legislation lays down a Community procedure for the designation of medicinal products as orphan medicinal products.               
  •  It provides incentives for the research, development and placing on the market of designated orphan medicinal products.          
  •  It sets up a Committee for Orphan Medicinal Products (COMP) within the Agency
  • Orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval
In addition to the United States and the European Union, legislation has been implemented by Japan, Singapore, and Australia which  have all passed legislation that offers subsidies and other incentives to encourage the development of drugs that treat orphan diseases.

IV.Some of the Organizations/offices of Regulatory Agencies associated with Orphan Drugs:

a)      FDA Office of Orphan Product Development (OOPD)-

The OOPD has following roles-
  • Evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare disease and to further advance scientific development of such promising medical products.
  • It provides incentives for sponsors to develop products for rare diseases.

b)       National Organization for Rare Disorders (NORD)-
  • It is a NGO based in USA, involved in providing information, advocacy, research, and patient services to help all patients and families affected by rare diseases.
  • It was involved in lobbying along with other organizations for passing Orphan Drug Act in USA.

C ) Committee for Orphan Medicinal Products (COMP)-
  • It is one of the scientific committee of EMA that is responsible for reviewing applications from people or companies seeking orphan medicinal product designation.
  • It is also responsible for advising the European Commission on the establishment and development of a policy on orphan medicinal products in the EU, and assists the Commission in drawing up detailed guidelines and liaising internationally on matters relating to orphan medicinal products

d)  European Organization for Rare Diseases (EURORDIS)-

It is an NGO based in Europe, dedicated towards improving quality of life of all people living with rare diseases in Europe.

V.Significant results due to passage of legislations in support of Orphan Drugs-
  • Many orphan drugs have been developed to treat rare diseases like Hodkins Lymphoma, congenital Factor XIII deficiency. glioma, multiple myeloma, cystic fibrosis,  phenylketonuria, snake venom poisoning, neoplastic meningitis etc.
  • Before enactment of ODA in USA, only 38 drugs were approved to treat orphan diseases. From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds, which is a significant achievement.
  • Although the European Medicines Agency grants market access to its 27 member states, medicines only reach the market when each member state decides that its national health system will reimburse for the drug. For example, 35 orphan drugs reached the market in Belgium, 44 in the Netherlands, and 28 in Sweden in 2008. 35 such drugs reached the market in France and 23 in Italy in 2007.
Orphan Drugs Approved by the FDA in the year, 2011 (until November, 2011)-

Name of Drug
Adcetris(brentuximab vedotin)
For treatment of Hodgkin’s lymphoma and ALCL (systemic anaplastic large celllymphoma).
Anascorp(Centruroides [Scorpion] Immune F(ab’)2 [Equine])
For treatment of clinical signs of scorpion envenomation.
Caprelsa (vandetanib)
To treat adult patients with metastatic (late-stage ) medullary thyroid cancer who are ineligible for surgery and who have disease
that is growing or causing symptoms.
Corifact(Factor XIII Concentrate
For routine prophylactic treatment of congenital Factor XIII deficiency.
For treatment of acute attacks of a rare condition called hereditary angioedema (HAE) in people ages
18 years and older.
Xalkori (crizotinib) and companion genetic  test
To treat certain patients with late stage (locally advanced or metastatic), non-small cell lung
cancers (NSCLC) who express the abnormal anaplastic lymphoma kinase (ALK) gene.
Yervoy (ipilimumab)
To treat patients with metastatic melanoma (late-stage skin cancer).
Zelboraf (vemuranfenib)
with companion diagnostic
To treat patients with metastatic (late-stage) or unresected (cannot be removed by surgery) melanoma (skin cancer) in patients whose tumors express a gene mutation called BRAF V600E.
Nulojix (belatacept)
To prevent organ rejection in adult patients who have had a kidney transplant, in combination with
other immunosuppressants.
Spherusol(Coccidioides immitis
Spherule-Derived Skin Test Antigen)
For detection of delayed type hypersensitivity to C. immitis in individuals, 18-64 years of age,
with a history of pulmonary coccidioidomycosis

Keywords- Orphan Drugs, rare diseases, Orphan Drug Act of 1983, Rare Diseases Act, Regulation (EC) No 141/2000, OOPD, NORD, COMP, EURORDIS.


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