Targeted therapies in the treatment of germ cell tumors: The need for new approaches against “orphan” tumors

DIA Patient Organization/ Advocate Track - Drug Information Association - conference notice Rare Diseases/Orphan Products Oct 11-13th Washington, Dc

Patient Organization/ Advocate Track
Patient organizations/patients are invited to attend the US Conference on Rare Diseases and Orphan Products at a reduced registration fee of $400. Please submit a registration form to +1-215-442-6199 or Carrie.Dunn@diahome.org.

Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer, June 8, 2011 JAMA

Objectives To define characteristics of orphan cancer drugs and their pivotal clinical trials and to compare these with nonorphan drugs.

.......More treated patients had serious adverse events in trials of orphan drugs vs trials of nonorphan drugs (48% vs 36%; odds ratio, 1.72; 95% confidence interval, 1.02-2.92; P = .04).

Conclusion Compared with pivotal trials used to approve nonorphan cancer drugs, pivotal trials for recently approved orphan drugs for cancer were more likely to be smaller and to use nonrandomized, unblinded trial designs and surrogate end points to assess efficacy.

Aeterna Zentaris Receives Positive Opinion for Orphan Medicinal Product Designation for AEZS-108 for the Treatment of Ovarian Cancer from the Committe

media: Aeterna Zentaris Receives Positive Opinion for Orphan Medicinal Product Designation for AEZS-108 for the Treatment of Ovarian Cancer from the Committee for Orphan Medicinal Products of the European Medicines Agency