BRUSSELS, Belgium - Progress was made last week at a Brussels workshop on developing partnerships to find new treatments for a range of rare diseases, according to the European biotechnology industry association, EuropaBio.
Industry representatives and academic scientists, together with officials from the European Medicines Agency and the U.S. FDA, jointly examined prospects for cystic fibrosis, Castleman's and Gaucher's diseases, Diamond-Blackfan anemia, epidermolysis bullosa, retinitis pigmentosa, pancreatic carcinoma, multiple myeloma, Wilson's disease, ulcerative colitis, myasthenia gravis and severe combined immunodeficiencies.
Finding industrial partners for scientists and clinicians who want to see their ideas developed into treatments for rare disorders is an essential step toward new therapies, said the group that set up the meeting, the European Platform for Patients' Organizations, Science and Industry. This is the way "to ensure that new science is applied to provide real benefits for patients," it said.
Michael Griffith, chief executive of a patient group known as Fighting Blindness Ireland, chairing the workshop, said it was essential that patient groups work with other stakeholders. "This is an idea whose time has come," he said. "Patients and their families need the hope that scientific progress gives them. They also need the confidence that they and their children will be able to benefit from this progress."
Erik Tambuyzer, of Genzyme Corp., said the workshop "has given a definite boost to the newly adopted European Union regulation on orphan medicinal products," which he described as a vital legal framework so that "industry can meet the needs of families longing for those new treatments and realize the potential market opportunities."
A further meeting is being planned for Oct. 16, 2001, in the Paris Senate to maintain the momentum for development of new medicines in the field.