Orphan Drug Laws A Pharmacists Perspective

Pharm Exec’s sister organization CBI hosted its annual Orphan Drug Innovation Summit in Philadelphia on July 17-18.  This is a topic that I have professional interest in, as well as a general curiosity.  I was thrilled to find the meeting in my own city since my total transportation cost was a $2.00 subway token (in each direction).  And, as one would imagine, this is not a topic that is the subject of countless seminars, webinars and symposia as is the case these days about health insurance exchanges, HIPAA requirements, value-based pricing and risk remediation.

I left with an array of interesting research questions to be presented to some of my research students here at Temple University.  But of equal importance it was fascinating to learn from the discussion that many countries do not have orphan drug regulations, including some developed countries where one would have expected this to have been addressed decades ago.  In the US, a disease affecting less than 200,000 persons is considered a rare disease and a drug intended to treat that condition is eligible for orphan drug designation. Such status permits a smaller clinical trial, a waiver of some FDA fees, help from FDA staff and a period of seven years of market exclusivity.  Clearly, without these financial incentives, manufacturers would be hesitant to spend the money on R&D and clinical trials for a very small potential market where the manufacturer might never recoup its investment.

We learned about a different system in Canada that nevertheless has identical goals to the US Orphan Drug Act. But what was most illuminating was the extent of grey areas where those in attendance had often vastly different interpretations of the regulations and policies where a second drug for an orphan condition was presented to the FDA.  In essence, on what basis should the FDA review it,  since it is only supposed to do so for a drug with superior clinical results compared to the existing drug on the market?

Despite the thousands of orphan conditions, there have only been about 350 drugs approved since the inception of the US law in 1983.  New regulations go into effect on August 12th of this year that are intended to simplify the process and make modifications for some of the aspects of the regulations causing problems today.  We learned about the European orphan drug program and were able to compare and contrast it to the US program. There were several presentations on commercializing orphan drugs, about developing RNA-based therapeutic strategies for rare diseases, and about an orphan drug “business model” designed to capitalize on the favorable regulatory conditions through market launch and beyond.

Dr. Albert Wertheimer is Professor and Director, Health Services Research, Temple University, Philadelphia, PA.