"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 companies.
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 status.
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 $169 billion by 2022, growing at a CAGR of 6.8% from 2016 to 2022.
So, besides enacting an
orphan drugs legislation with provisions for incentives for
orphan drug developers, the government needs to create a framework that is conducive for the manufacturing of
orphan drugs in India, according to ORDI.
Orphan Drugs Market, by Disease Type, Revenue (US$ 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.