EU Seeks Hematology Guidance

April 1, 2008

Applied Clinical Trials

Applied Clinical Trials, Applied Clinical Trials-04-01-2008, Volume 0, Issue 0

Europe aims to create new regulations regarding hematological malignancies.

The EMEA is proposing to create new guidance on hematological malignancies. The agency's idea arises from the recent increased activity in developing new treatments for hematological malignancies, including rare disorders and small target populations. This has led to a rise in applications for marketing authorization, but above all to numerous requests for scientific advice from the agency, prior to submitting an application.

The new guidance will deal with special considerations such as allogeneic stem cell transplantation and its consequences in relation to designation of primary endpoints. It will also take account of the use of molecular techniques in response evaluations, treatments administered with curative or palliative intent, and progression or relapse off-therapy. It will focus on the design of confirmatory studies, and will provide separate discussion of major diagnoses such as the acute leukemias, myelodysplastic syndromes, low and high grade lymphomas, and malignant myeloma. Other, rarer conditions will be covered conceptually. Most of the work will be carried out during this year—and anyone wanting to give their views to the agency is invited to do so until the end of this month.—Peter O'Donnell