PARIS, Feb 20 2015 - Further measures are needed in France, in addition to those incorporated under temporary use authorisations, to allow and regulate more off-label use of drugs for rare diseases, according to a spokesman for the French muscular dystrophy association.
Since their formation in 2012, “there have been very few temporary use recommendations, you can count them on one hand, therefore for rare diseases the results are quick,” AFM-Telethon’s leader of advocacy actions Christophe Duguet told APM on Thursday.
On its website, France’s drug regulator ANSM identifies three drugs with temporary use recommendations (RTUs), two of which are rare disease drugs: Roche’s Roactemra (tocilizumab) in inflammatory Castleman disease (with high CRP levels), not associated with HHV8 virus, and MSD’s Remicade (infliximab) in Takayasu disease, unresponsive to conventional therapies.
“Our fears are confirmed. This is a special system that will take a lot of time before it covers drugs possibly eligible to treat rare diseases,” because it is difficult to prove efficacy at the required level, Duguet stressed during interview at a press conference on rare diseases.
“What we want is to reinforce the current system of RTUs with another regulatory system which secures the use of molecules of interest and, if necessary, stops use of some non authorised drugs. Regarding rare diseases, we need to be able to collect data to achieve an expert consensus. We can certainly call upon reference and competence centres.”
For AFM-Telethon, non authorised drugs could be prescribed by the physicians of these certified centres, according to a formalised process, with an obligation to sign up. “ANSM would have the authority to oppose, not to validate these decisions,” Duguet suggests.
For both rare diseases and paediatric indications, the system must attract investments in the development of known molecules. “The objective is to also overcome uncertainties around reimbursement,” he added.
Duguet now expects advancements from the bill on health to be examined by the National Assembly early April, regretting that no action was taken when the recommendations had been expanded.
In December 2014, at the last meeting of the 2011-2014 rare diseases follow-up and foresight committee, the general health administration (DGA) committed to set up a working group, “but we have no news as of now and there is nobody to implement this measure,” he said.
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