Orphan Drugs and their Promotion

Definition of Orphan Drug:

Orphan drugs are defined as the drugs which are used in the disease or condition which occurs so infrequently (rare disease) in the population that there is no reasonable expectation that the cost of developing and making a drug for such disease or condition will recovered from sales of such drugs.

According to the World Health Organization there are around 6,000 rare diseases. They affect a small part of the population. Tropical diseases are also consider as the rare disease because the patients are poor and cannot spend the money on the treatment. Therefore, the availability of economic incentives is required to encourage pharmaceutical and biotechnological industry to develop orphan drugs.

Following table shows the designation criteria for orphan disease based upon the disease prevalence. 

Country	No. of affected individuals	Prevalence (per 10000 population)
United States	< 200000	7.5
Japan	< 50000	4.0
Australia	< 2000	1.1
European Union	< 215000	5.0
United Kingdom	< 1000	0.18

Promotion of Orphan Drugs: 

In the last 20 years, orphan drug legislation has been adopted in several countries around the world (USA, Japan, Australia, and the European Union) and has successfully promoted R&D investments to develop new pharmaceutical products for the treatment of rare diseases. Guaranteed financial return on the product developed for the rare disease by guaranteed purchase agreement.

To assist and encourage the identification, development, and availability of safe and effective products (drugs, biologics, medical devices and medical foods) for people with rare diseases/disorders by offering following incentives.

• Fast track approval process and also priority is given for the review of the for the orphan drug application.
• The long market exclusivity offered through the orphan drug designation is considered a very powerful pull mechanism and a strong incentive for R&D investment. Regulatory authority cannot approve another marketing application for the same drug for the same orphan use. The exclusivity applies only to the specific orphan indication. If another firm develops the same drug for a common-disease indication or for a different orphan indication, approval will also be granted to that firm.
  
  

  Country	Market exclusivity
  Europe	10 years
  USA	7 years
  Japan	10 years
  Korea	6 years
  Singapore	10 years
  Taiwan	10 years

  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
  
• Exemption or reduction in the registration fees are also awarded to sponsor of the orphan drug.
• Research grants are also available to pharmaceutical company or academic institutes for conducting clinical studies as clinical trials require large financial investments.
• Assistance for the clinical trial protocol and study design as clinical trials are often difficult to organize and manage in poor countries.
• Tax credits up to 50 % of the clinical and non-clinical research costs.
• Several non-profit entities have been developed to fund and develop new drugs and vaccines for orphan diseases. Public–private collaboration between pharmaceutical companies and traditional public sector organizations leading to the evaluation of drug candidates developed by pharmaceutical company in the field of rare diseases. Such as European Rare Diseases Therapeutic Initiative (ERDITI).
  
  
  Rare diseases day is celebrated at last day of February every year to spread the awareness for orphan diseases. By providing such incentives Governments, Medicine Regulatory Authority, Non-profit organizations are encouraging the Pharmaceutical Industry to do research and bring the new medicine as a new hope for the patient.