BioWorld International Correspondent
BRUSSELS, Belgium - As patient representatives met at the European Medicines Agency in London on March 4 to discuss how the European Union's orphan medicine rules are functioning, the European biotechnology industry revealed ideas as to how the system could be improved.
In a rare show of unity, both the European industry associations with a claim to represent the sector - EuropaBio and the Emerging Biopharmaceutical Enterprises group - presented a common position.
Top of the list of industry demands is for the European Commission to take the lead on ensuring full application of the regulation, which, the industry position underlines, is still not fully applied in the EU's 25 member states five years after it officially came into force. Since then, 254 products have been designated as orphan medicines and, of those, 20 have been granted marketing authorization in the EU. But the industry says it is still too soon to judge the measure fully.
"The application of the EU's orphan medicine rules and spirit is still in its infancy across the EU," said Andrea Rappagliosi, chair of EuropaBio's orphan medicines group.
It also wants action among health insurance agencies to ensure that patients suffering from rare diseases get access to the treatments they need. It urges an end to what it sees as "regional inequalities and disparities in patient diagnosis, access and reimbursement."
But industry also is concerned about the operation of the underlying principle of the rules - the economic incentive for companies to take on the risk of developing orphan medicines. It warned that Europe cannot expect other parts of the world to create and supply incentives for the development of rare diseases, while at the same time benefiting from the availability of therapies for such diseases at low prices. EBE and EuropaBio said there is continuing confusion over how those incentives operate. There is particular concern over how they might be limited if current EU reflections on the incentives result in a reduction of the market exclusivity that constitutes one of the principal inducements to develop orphans.
Above all, warned the industry position paper, there can be no legal justification for cutting the 10-year exclusivity of an orphan product on the grounds that it has become sufficiently profitable if its initial designation as an orphan was based on the low incidence of the condition it was developed to treat.
"This confusion should be avoided because it may negatively affect orphan medicine development in Europe by removing the incentives needed to foster investment and potentially creating a less supportive climate in the field," the industry said.
WTO Documents Reveal EU GMO Doubts
The European Union has admitted to reservations over the safety of biotechnology. Documents submitted by the EU to the World Trade Organization, and leaked in advance of key negotiations in Geneva, acknowledge that because of the evolution of the science, "new risk considerations sometimes arise spontaneously and change the scope of the risk assessment."
The admission has been seized upon by biotechnology opponents, who accuse the EU both of subjecting EU citizens to undue risk, and of confusion and even hypocrisy in their policy. Friends of the Earth Europe alleged that the public and the environment are at threat as a result of that inconsistency, since the EU is deploying its authorization procedures to oblige member states to accept new genetically modified products into their market despite their concerns.
The documents, which form part of Europe's defense in the trans-Atlantic trade dispute at the WTO, note that anxieties about antibiotic-resistant genes and secondary effects on beneficial insects are "legitimate scientific concerns," and that sovereign states should be able to determine their own level of protection. In addition, while within the European context the EU depends heavily on the opinions of its scientific committees, such as the European Food Safety Authority, its evidence in the WTO dispute remarked that "the opinions of the EC scientific committees do not overrule other scientific opinions."