Student Dissertation

Agency : Market Opportunity; Orphan Indications; Pharmaceutical Companies

Objective : To evaluate the Orphan flagged Pharmaceutical companies and the amount of work done by ZS with these companies and priortize them.

Background : More than 25 years of orphan drug regulations have yielded several new treatments for patients with rare diseases. Orphan diseases are typically those that are having no commercial incentives to research and develop actual therapies. To boost the pharmaceutical companies to invest in the development of orphan drug, various countries, beginning with the USA have announced their own regulations to provide suitable motivations. There is a significant degree of similarity in the incentives provided from one nation to another and this variety from market exclusivity for the product in its proposed indication (the most important incentive) to tax credits and reduction or waivers of fees. The World Health Organization gives the idea about Orphan or Rare diseases as; it is the pathological conditions that affect 0.65-1 out of every 1000 population. The EU defines a rare disease that affecting 5 peoples out of 10,000 Europeans; The USA accept the condition as a Rare disease as it affecting less than 200,000 peoples or the condition that affects less than 1 out of 5,000 in the general population; Japan has the limit up to 50,000 Japanese patients and Australia accepts the disease as an Orphan disease, that affects less than 1 at 2000 Australian patients. There are approximately 6,000 Orphan diseases, out of which 80% are genetic. As Pharmaceutical companies generally invest many millions of dollars to develop single new drug, the interest of companies were diverted towards the development of drugs that are used for the large patient population (Non-Orphan drugs).

Methodology : Type of study: Exploratory Secondary Research Study subjects: Orphan/Ultra-orphan indications and products

Findings : Shire, Novartis and Pfizer were observed to be investing the maximum for Orphan since they had maximum of number of drug products in development and in market for Orphan indications. So the opportunity of working with these companies would reap maximum benfits compared to other 450 companies identified.                                                                                                                    Recommendations : Maximum amount of work done by ZS with these Companies was in the area of sales, therefore the Practice Area of Marketing could be emphasized upon more in order to maintain an equilibrium between the two.


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