Earlier this month, Scott Gottlieb, announced that he will be stepping down from his post as FDA Commissioner to spend more time with his family. Although he has had a relatively short term, serving since [...]
Last week, the FDA launched its new ‘Request to Connect’ portal, an initiative developed by the agency to encourage greater understanding of, and participation in, the FDA's regulatory work. The online portal invites patients, their [...]
Challenge A pharmaceutical company based in North America received EMA marketing approval for its bioequivalent product for an ultra-rare disease. However, because the originator product had been on the market for years previously without any [...]
The Orphan Drug Act (ODA), which was passed in 1983 to incentivize drug development for rare diseases, has come under scrutiny in recent years. There are some within industry who claim that the program has been [...]
Challenge A leading pharmaceutical company wanted to supply its portfolio of treatments for a rare congenital disease into new markets on an unlicensed basis. This required the company to navigate the varied regulatory landscapes across [...]
Challenge WEP Clinical was contacted by a company located outside North America which wanted to access a controlled substance from the US. This was the first time WEP Clinical had received such a request and [...]
The FDA has recognized the important role that real-world evidence (RWE), i.e. evidence that is collected outside the tightly controlled clinical trial setting, can play in furthering drug development and facilitating drug approvals. In line [...]