FEATURE
How will the OCE handle postmarketing surveillance and the monitoring of drug safety ?
The lifecycle of a new product does not end with approval , and post-marketing surveillance will remain a key feature of ongoing safety reviews . The FDA has mechanisms in place at the time of approval to ensure patient safety , such as an accurate description in labeling of the adverse reactions observed during pivotal clinical trials .
If a safety signal arises , CDER will follow these issues through the Document Archiving , Reporting , and Regulatory Tracking System ( DARRTS ) module . DARRTS was established in January 2007 as a centralized system that ensures a team of safety experts weighs in on the significance of new safety signals , shares recommendations for regulatory action ,
FACTOR REPLACEMENT MIRRORS THE PROTECTION WITHIN and more . Again , because the OCE brings together various centers across the FDA , the OCE director will be able to manage the disposition of resources to address the review of new safety signals .
Additional studies or registries may be required to address areas of concern that regulators have identified , and these requirements can be found in the approval letter .
Are there special considerations unique to the review and approval of hematology products compared with products for solid-tumor oncology and other diseases ?
The clinical trial considerations for hematology products are slightly different than for other disease states because most hematologic disorders are rare – making large , randomized trials difficult to conduct . Therefore , occasionally , non-randomized , controlled trials are used in the approval process . In these situations , FDA reviewers may also rely on patient-reported outcomes data to make approval decisions – as was the case in the 2015 approval of ruxolitinib for the treatment of myelofibrosis .
“[ We ] work in close coordination to help patients get faster access to safe and effective cancer treatments .”
For your patients with Hemophilia A , Factor treatment has a long history of results . 1 It temporarily replaces what ’ s missing and is regulated by the natural hemostatic process to form a proper clot . 2-5
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References : 1 . Franchini M , Mannucci PM . The history of hemophilia . Semin Thromb Hemost . 2014 ; 40:571-576 . 2 . Peyvandi F , Garagiola I , Young G . The past and future of haemophilia : diagnosis , treatments , and its complications . Lancet . 2016 ; 388:187-197 . 3 . Lenting PJ , van Mourik JA , Mertens K . The life cycle of coagulation factor VIII in view of its structure and function . Blood . 1998 ; 92 ( 11 ): 3983-3996 . 4 . Antovic A , Mikovic D , Elezovic I , Zabczyk M , Hutenby K , Antovic JP . Improvement of fibrin clot structure after factor VIII injection in haemophilia A patients treated on demand . Thromb Haemost . 2013 ; 111 ( 4 ): 656-661 . 5 . Hvas AM , Sørensen HT , Norengaard L , Christiansen K , Ingerslev J , Sørensen B . Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A . J Thromb Haemost . 2007 ; 5:2408-2414 .
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Hematologic malignancies also are often treated with combination chemotherapy , particularly in patients with newly diagnosed disease . Drug development , however , frequently emphasizes the use of single agents in patients with relapsed or refractory disease , after other standard treatments have failed . So , in the case of hematologic malignancies , researchers and regulators must consider the complete lifecycle of the investigational agent and that it will eventually be used as part of combination therapy .
The field of hematology has long been at the forefront of scientific discovery and uncovering the mechanistic basis of disease processes . With technology advances such as chimeric antigen receptor T-cell therapy , checkpoint inhibitors , and other forms of cell and molecular immunotherapy , the treatment landscape and development pathway for newer agents will inevitably evolve .
With the greater ability to collect genetic data – from several initiatives launched with the Cancer Moonshot
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