BioWorld International Correspondent
BRUSSELS, Belgium - The need for more partnerships was underlined by European biopharmaceutical industry executives when they met European Union officials here last week for a discussion of orphan drugs.
Their call received an encouraging response from European Research Commissioner Janez Potocnik.
"Collaboration between industry and academia in rare-diseases research is essential to be able to convert an initial scientific idea into a real-life safe and effective medicine for patients," said Carlo Incerti, president of European Biopharmaceutical Enterprises. "This collaboration needs to include other key stakeholders, such as patient organizations and regulators."
The industry urged the EU to foster closer links between patients and industry and between science and the public sector. It also underlined the need for improved access to life-saving orphan medicines resulting from those research partnerships - if necessary, by changes in national prescribing and reimbursement rules. In addition, the industry wants the EU to provide more funding for clinical trials of possible treatments for rare diseases.
Erik Tambuyzer, of Genzyme Corp., said it was cooperation that had made possible the success to date of the European scheme to promote development of orphan medicines, under which 40 treatments have received official backing, and 470 designated orphan products are in development. "Yearly approvals of orphan drugs in the EU now compare in number to those in the U.S.," Tambuyzer said. "It is vital that this cooperation continue at all stages, including ensuring that European patients get equal and real access to authorized treatments in all member states."
The response from Potocnik - the senior figure behind the EU's multibillion-dollar research support program - recognized that more needs to be done on rare-disease research in Europe. He offered a personal commitment to promoting "real progress in the area of rare-diseases research."