"This review concludes that two of every three orphan drugs
approved show the FDA's flexibility in its review of effectiveness data on orphan drug
therapies," says Sasinowski.
To further accelerate the development of orphan drugs
by the pharmaceutical and biotechnology industries, the NCOD recently asked Congress to approve greater incentives for orphan drug
But companies that produce them under the orphan drug
designation are offered several advantages, including research grants, tax benefits, protocol assistance, marketing exclusivity (seven years in the United States) and regulatory challenges (such as a smaller number of clinical trials) that are less daunting than those faced by other new medicines.
The PMA's commission on drugs for rare diseases is encouraging the development of orphan drugs
, and finding corporate sponsors for promising therapies.
The Orphan Drug
Act of 1983 has worked well in achieving the purpose for which it was enacted--to provide incentives that encourages orphan drug
development, says PMA president Gerald Mossinghoff.
Genentech's human growth hormone, used to treat dwarfism, and Amgen's EPO, used to treat anemia in kidney dialysis patients, would retain their orphan drug
Some 36 orphan drugs
have been approved by the FDA under the Orphan Drug
Act and are available to patients.
According to a new report published by Allied Market Research, titled, "Orphan Drugs
Market by Disease Type and Indication: Global Opportunity Analysis and Industry Forecast, 2014-2022," the global orphan drugs
market was valued at $106 billion in 2015, and is projected to reach $169 billion by 2022, growing at a CAGR of 6.8% from 2016 to 2022.
So, besides enacting an orphan drugs
legislation with provisions for incentives for orphan drug
developers, the government needs to create a framework that is conducive for the manufacturing of orphan drugs
in India, according to ORDI.
Market, by Disease Type, Revenue (US$ Bn) 2015-2025
Seven of the top ten companies by orphan drug
sales are global industry players."
For those interested in attending Orphan Drugs
2016, registration is live online at http://www.orphandrugs-event.com/prcom.