But the availability of the product remains restricted. It can be used in France under its named patient compassionate use
program. But it cannot be used in any other member state that does not allow similar use. And that has provoked interest that
goes much wider than the liver disease community. This unprecedented refusal of a marketing authorization by the commission—in
the face of strong support for the product from the EMA and most member states—has revealed a hitherto unsuspected weakness
in the European Union's regulatory procedures.
The commission has justified its refusal on the basis of inadequate testing and insufficient evidence of established use.
Questions have been raised about the wisdom of this justification. A product for such a deadly disease in such a limited population
may not be appropriate for the sort of wide-ranging clinical testing required for an anti-rheumatic or an anti-fungal, it
has been suggested. And the commission has also chosen to measure established use only from the time that CTRS took the product
over in 2007—ignoring its use since 1993. These are not considerations that the EMA felt were sufficient to invalidate the
There are systematic questions that arise from the case. Is the commission determined to over-rule scientific opinion, even
though it lacks the scientific expertise that the EMA (and each national authority in the member states) possesses? The member
states are—via the council —the official holders of power, and they only delegate power to the commission, which then exercises
it under the supervision of the member states. In theory, member states are then able to block the commission's proposed regulations
by a qualified majority vote. Can the commission restart a procedure for a second time, when it has already been defeated?
Can it reinitiate a vote indefinitely in this sort of case, until it gets the result it wants? Recent changes to the EU's
mechanisms, introduced by a new treaty—the Lisbon Treaty—in 2010, are still playing out. Orphacol has become a leading case
in this drama.
There are other questions that have been raised. Why did a US company try to get in on the act? Asklepion Pharmaceuticals,
owned by the Seventh Day Adventists Church in the United States, which tried to support the commission in the first court
hearing, has submitted a marketing authorization to the EMA for what it claims is a competing product in this field. Why did
the commission take such a determined—and apparently perverse—stance on Orphacol? By a strange coincidence, both the key commission
officials in the Orphacol case are no longer in post. Dalli was forced to resign as health commissioner last autumn as allegations
swirled around him of being too close to the tobacco industry he was supposed to be regulating. Patricia Brunko, the head
of the commission's pharmaceuticals unit that handles authorizations, has retired as of the end of 2012. Their motivations
for such firmness have never been elucidated.
Much now hangs on the outcome of Orphacol's second case, still before the EU court. The judgement will be awaited with more
than normal interest by European pharmaceutical executives and everyone with an interest in how the EU really works.
Reflector is Pharm Exec anonymous columnist, a commentator so close to the action in Europe that his identity must be secret.