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FEATURE clinical trial participants ’ data : “ the threat to patient privacy and the ability to conduct cancer clinical trials .”
“ De-identifying data is actually quite hard ,” Drs . Sekeres and Bolwell , both from the Cleveland Clinic , Taussig Cancer Institute , explained , and some smaller centers might not have the resources to meet these mandates . “ Introducing a requirement that clinical trial data be made publicly available requires infrastructure in the form of databases , computer servers , and personnel , which adds to the price tag for these studies . At a certain financial inflection point , studies may not be conducted , even when they ask important research questions for patients who desperately need new therapies .”
Adding public disclosure of data to the informed-consent process also puts patients in an unfair position , they added , since it will require clinical trial participants to “ opt-in ” to sharing their data . “ Our patients would face what amounts to a Faustian bargain : agree to allow your data to be made public , or you cannot enroll on a clinical trial ,” Drs . Sekeres and Bolwell wrote . “ This is not the sort of choice we want to force people to make when they are about to undergo treatment for their cancer .”
“ While we appreciate the scientific rationale for the proposal to make clinical trial data publicly available ,” they concluded , “ in the end it cannot trump the rights of our cancer patients to maintain their privacy , have a full range of clinical trials available , and make treatment decisions free of conflict .” 5
will allow the participants to donate their data at the click of a button , essentially . People can ‘ opt in ’ at whatever level they want to and with whatever project they want to .”
Despite the fervent reactions that the “ research parasites ” argument elicited in people , everyone we spoke with agreed that it raises important questions about data sharing – and all members of the research community will have to work together to find the answers .— By Jill
Sederstrom ●
When Treating Acquired Hemophilia A
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REFERENCES 1 . Longo DL , Drazen JM . Data Sharing . N Engl J Med . 2016 ; 374:276-7 . 2 . Bloom T , Ganley E , Winker M , PLOS Data Group . “ Data access for the open access literature : PLOS ’ s data policy .” Accessed April 5 , 2016 from https :// www . plos . org / data-access-for-the-open-access-literature-ploss-data-policy /.
3 . Blood . “ Author Guide - Editorial Policies for Authors .” Accessed April 5 , 2016 from http :// www . bloodjournal . org / page / authors / author-guide / Editorial-policies-forauthors ? sso-checked = true # data _ share .
4 . Taichman DB , Backus J , Baethge C , et al . Sharing clinical trial data : a proposal from the International Committee of Medical Journal Editors . N Engl J Med . 2016 ; 374:384-6 .
5 . Sekeres MA , Bolwell BJ . Will cancer patients be the next victims of the data privacy debate ? FoxNews . com . Accessed April 19 , 2016 from http :// www . foxnews . com / opinion / 2016 / 02 / 27 / are-cancer-patients-next-victim-dataprivacy-blurred-lines . html .
ABOUT
The first recombinant porcine sequence factor VIII replacement treatment 1
RESPONSE
Fast-acting : 95 % ( 19 / 20 ) response seen at 8 h ; 100 % ( 18 / 18 ) at 16 h ; 100 % ( 28 / 28 ) at 24 h after initial dosing 1
EFFICACY
94 % ( 16 / 17 ) of patients treated first-line achieved overall treatment success 1
SAFETY
Safety profile established in the adult population in a clinical trial 1
MEASURABILITY
The ability to measure factor VIII levels helps find the right dosing balance 1
Opening Up the Dialogue About Open Data
The authors of the NEJM editorial had a different opinion on how best to motivate investigators to gather data : co-authorship . It ’ s concept of “ how data sharing should work ” includes “[ reporting ] the new findings with relevant co-authorship to acknowledge both the group that proposed the new idea and the investigative group that accrued the data that allowed it to be tested .” 1
Others feel that co-authorship isn ’ t the answer . “ It ’ s essentially people expecting to do one thing and get double-credit for it . I think that ’ s potentially unethical and , frankly , kind of ridiculous ,” Dr . Hoffman said .
Dr . Shaywitz proposed another possible solution : returning the power of the data to the patients themselves , allowing them to decide where the data should be used .
“ One of the exciting aspects of the National Institutes of Health ’ s Precision Medicine Initiative is its goal of empowering study participants with their own data ,” he said . “ They are trying to create the functionality that
The efficacy ( N = 28 ) and safety ( N = 29 ) of OBIZUR were studied in the first interventional , prospective , clinical trial for acquired hemophilia A ( AHA ) patients . Patients were treated with OBIZUR until resolution of bleeding , or dosing was continued at the physician ’ s discretion according to the clinical assessment . 1
Indication
OBIZUR , Antihemophilic Factor ( Recombinant ), Porcine Sequence , is a recombinant DNA derived , antihemophilic factor indicated for the treatment of bleeding episodes in adults with acquired hemophilia A .
Limitations of Use :
• Safety and efficacy of OBIZUR has not been established in patients with baseline anti-porcine factor VIII inhibitor titer greater than 20 BU
• OBIZUR is not indicated for the treatment of congenital hemophilia A or von Willebrand disease
Detailed Important Risk Information CONTRAINDICATIONS
OBIZUR is contraindicated in patients who have had life-threatening hypersensitivity reactions to OBIZUR or its components ( including traces of hamster proteins ).
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions can occur with OBIZUR . OBIZUR contains trace amounts of hamster proteins . Early signs of allergic reactions , which can progress to anaphylaxis , include angioedema , chest-tightness , dyspnea , hypotension , wheezing , urticaria , and pruritus . Immediately discontinue administration and initiate appropriate treatment if allergic or anaphylactic-type reactions occur .
Inhibitory Antibodies
Inhibitory antibodies to OBIZUR have occurred . Monitor patients for the development of antibodies to OBIZUR by appropriate assays .
Visit www . OBIZUR . com
If the plasma factor VIII level fails to increase as expected , or if bleeding is not controlled after OBIZUR administration , suspect the presence of an anti-porcine factor VIII antibody . If such inhibitory antibodies to anti-porcine factor VIII are suspected and there is a lack of clinical response , consider other therapeutic options .
Monitoring Laboratory Tests
• Perform one-stage clotting assay to confirm that adequate factor VIII levels have been achieved and maintained
- Monitor factor VIII activity 30 minutes and 3 hours after initial dose
- Monitor factor VIII activity 30 minutes after subsequent doses
• Monitor the development of inhibitory antibodies to OBIZUR . Perform a Nijmegen Bethesda inhibitor assay if expected plasma factor VIII activity levels are not attained or if bleeding is not controlled with the expected dose of OBIZUR . Use Bethesda Units ( BU ) to report inhibitor levels
ADVERSE REACTIONS
Common adverse reactions observed in greater than 5 % of subjects in the clinical trial were development of inhibitors to porcine factor VIII .
Please see accompanying page for Brief Summary .
Reference : 1 . Obizur [ Prescribing Information ]. Westlake Village , CA : Baxter Healthcare Corporation ; October 2014 .
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Baxalta and Obizur are trademarks of Baxalta Incorporated . USBS / MG114 / 16-0003
A Confident Approach .