"The three most important things for companies tackling rare diseases to get right are regulatory affairs, regulatory affairs, and regulatory affairs," says Tim Coté, former director of the FDA's Office of Orphan Products and signatory to decision letters for over 1,400 orphan designation applications. - PharmaTimes, Nov 2016
The reason Mr. Coté points this out is that while the science is important for overall drug development, Orphan Designations are, both in the US and EU, mainly a Regulatory Pathway. There are specific requirements for population in both zones. In the EU there is a little bit of flexibility as the requirement is 5 patients or less out of every 10,000. To contrast, in the US, the number is set at less than 200,000 patients. What can this mean? This can mean that while you get an orphan in one area, you might not get it in both.
As I have stated earlier on this blog, the best thing to do here is seek expertise. You may have it in house as you may have done a number of them, but don't be afraid to find a consultant experienced in Orphan indications. The trick here is to view it as a regulatory agency would view the submission, and that is both when and where a good Regulatory Consultant can be invaluable.
#FDA #Regulatory #Biotechnology #Pharmaceuticals #Consulting #EMA