The European Commission on Tuesday launched a targeted consultation to provide input on a revision of its guideline on the content and format of applications for orphan drug designation, as well as the transfer of designations between sponsors.
"The comments received will be taken into account by the European Commission in the finalization of the guideline on the format and content of application for designation as orphan medicinal products and on the transfer of designations from one sponsor to another," the Commission writes. Stakeholders are invited to submit their comments by 28 April 2019.
The current revision of the guideline was drafted in July 2018 to address the European Medicines Agency's (EMA) new online portal for orphan drug submissions
, as the previous version
of the guideline was adopted prior to the online portal's launch. The new version of the guideline also makes numerous changes for clarity and to consolidate recommendations.
When defining the proposed orphan condition, the revised guideline now instructs sponsors to provide the MedDRA term "most closely matching the orphan condition," whereas the previous version of the guideline called for sponsors to refer to an international recognized classification system, such as the World Health Organization's International Classification of Disease (ICD), and called for sponsors to include the Anatomical Therapeutic Chemical (ATC) classification system code for the drug if one had been assigned.
The revised guideline also specifies that the authorized point of contact for the sponsor to EMA should provide their telephone and email address within the EU and no longer lists fax as necessary contact method.
Additionally, the revised guideline removes two recommendations for sponsors to consult a 2010 document, EMA/COMP/15893/2009
, on elements to support the medical plausibility and the justification of significant benefit for an orphan designation.
When estimating prevalence of an orphan condition, the revised guideline emphasizes that "the specific epidemiological measure for disease frequency should be stated and justified" and for indirect calculations to estimate prevalence, "all assumptions need to be justified by referring to appropriate scientific literature."
For products where pharmacy compounded (magistral) or hospital preparations of medicines are available to treat an orphan indication, sponsors are now expected to demonstrate why those treatments "are not considered as 'satisfactory methods.’”
The section on transferring orphan designations between sponsors has also been revised to account for changes to such requests as those requests may now be handled via EMA's online portal rather than via email.
, Revised Guideline