Clinical Trials of Orphan Drugs for Cancer

Some Numbers behind Canada’s Decision to Adopt an Orphan Drug Policy: US Orphan Drug Approvals in Canada, 1997–2012

“…While rare disease patients desire timely access to orphan drugs, knowledge about the safety and effectiveness of such drugs tends to be limited at the time of market approval. Owing to the small number of patients afflicted with a given rare disease, “alternative trial designs” (e.g., lacking randomization and blinding) are frequently employed in pre-market studies, which “can lead to identifying benefits that are not real or missing risks that are” ( Kesselheim and Avorn 2011 : 1546). In addition, drugs that address unmet patient needs, which orphan drugs should target almost by definition, are usually fast-tracked through review by regulators.…”

Clinical Translation and Regulations of Theranostics

“…Because of the small numbers of patients who suffer from each disease, FDA often allows nontraditional approaches to establishing safety and effectiveness, for example, the use of smaller, nonrandomized, unblended trials and use of surrogate endpoints in orphan cancer drugs [45,46]. Also, the FDA has several mechanisms that can facilitate development by allowing flexibility during clinical development and review and approval of new drugs that address unmet needs and treat serious and life-threatening disorders or conditions [52].…”