In the largest market for orphan drugs, USA, there was a shortage of adequate therapies for treating many rare diseases. These therapies were not developed as companies did not expect these drugs to be highly profitable. Hence there was a lack of interest and thus investment on the part of pharma companies in the USA. Therefore, the FDA introduced incentives for developing such drugs. This step taken by the FDA was successful in creating a thriving market for orphan drugs.
It was in the USA first that a special law exclusively for governing orphan drugs was framed in the form of the Orphan Drug Act of 1983. This led to an increase in the popularity of orphan drugs. The FDA also has been continuously increasing its efforts to support this market by providing significant financial and non-financial incentives to the pharmaceutical companies to attract them. This has been one of the major drivers of growth for the US orphan drugs market.
The orphan drugs enjoy significant competitive advantages in the market owing to the market exclusivity period after the drug has been authorized. Through this clause, the manufacturer of orphan drugs is given a monopoly status in the market because according to the law, no other company is allowed to market the orphan drugs during the exclusivity period. Additionally, this monopolistic power is further strengthened with the fact that no other alternative health technology exists for many orphan drugs.
A scenario where orphan drugs would be denied coverage is highly difficult to visualize. However it is most likely that the payer scrutiny would increase with new products entering the market and budgets contracting in a weakening economic environment. The plans would be based on costs and would also include payer resources, philosophies, and available benefit design options, as all these factors could affect patient access. A deep and clear understanding of the clinical and economic value of the drugs will play an increasingly important role in decision-making.
“US Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to US orphan drug market: