Worldwide collaboration for orphan drug designation

From promising molecules to orphan drugs: Early clinical drug development

“…For some designated orphan drugs such as olipudase (designated by the Food and Drug Administration on 08MAR2000) a within-patient dose-escalation strategy was required (11,12). In a sample of 605 candidate orphan drugs designated by the European Medicines Agency (EMA) between 2002 and 2012 only 110 (18 percent) (13) were in phase 1 and in another sample of 1406 EMA orphan drug applications between 2000 and 2014 only 183 (13 percent) (14) were in phase 1. The GlaxoSmithKline Clinical Data Sharing System contains 17 phase 1 trials with an orphan designation for rare neurological disorders, rare cancers and rare autoimmune diseases (15).…”

Future of Rare Diseases Research 2017–2027: An IRDiRC Perspective

“…To cope with an increased volume of applications and requests of protocol assistance, a number of changes will be necessary: streamlining the approval process, creating collaborative review processes between regulators from different jurisdictions, increasing human resource and training programs, and potentially updating regulations to assist in accelerating therapy development. Efforts are already under way to streamline and align regulatory processes across jurisdictions; IRDiRC aims to aid and foster such efforts, as they will ultimately contribute to the development of new rare disease therapies. In addition to coordinating research efforts, data sharing, and patient engagement, it is also vital to promote changes to the drug development landscape such as new models of risk and incentive sharing between public and private partners, systematic repurposing of existing agents, and developing a more flexible regulatory framework.…”

Progress in Rare Diseases Research 2010–2016: An IRDiRC Perspective

“…In the past two decades, several countries have introduced policies dedicated to orphan drug development and others are currently following suit with specific policies for their jurisdictions . To better grasp the number of therapies available for rare disease patients, IRDiRC tracks the cumulative number of approvals of medicinal products with a marketing authorization and orphan designation since 2010 via data from the European Medicines Agency (EMA) and the FDA.…”