Next Tuesday and Wednesday (in collaboration with FDA News and United BioSource), the Center for Medicine in the Public Interest is sponsoring the "Risk Management and Drug Safety Summit." Topic A: REMS.
Here's a link to the agenda.
A few thoughts in advance.
Many have looked at the FDAAA language on REMS and seen it as an ill-advised green light for the FDA to inject itself into the practice of medicine.
While I agree that REMS does indeed represent an expansion of the FDA's mission, I do not agree that it is ill advised. REMS is the responsible extension of the FDA's mission of safety and efficacy into the new realm of safe use. As previously discussed in this space ("A Potent FDA Double Feature") the FDA's role in educating both patients and providers on the safe use of approved therapies is a logical and approprite third leg of the agency's mission.
The first real move into this space was the FDA's change in the warfarin label. As Dr. Caroline Wright (Foundation for Genomics and Population Health) wrote:
“Just a month after the label for the blood-thinning drug warfarin was updated to explain that genetic variation in specific genes influences how patients respond to the drug, the US Food and Drug Agency (FDA) has approved the first genetic test for warfarin sensitivity.
Warfarin is the most widely used anti-coagulant medication in the world, prescribed to over 2 million people a year to prevent blood clots, heart attacks and strokes. Patients can display markedly different responses to the drug, so doses vary enormously between individuals. Achieving the correct dose is critical, as patients who receive too high a dose are at risk of severe bleeding, whilst those who receive too low a dose may remain at risk of life-threatening blood clots.
The Nanosphere Verigene Metabolism Nucleic Acids Test detects particular variations in two genes, CYP2C9 and VKORC1, which are involved in the metabolism and mechanism of action of warfarin respectively. Specific variants of these genes are identified from a patient sample by hybridization to sequence specific probes (oligonucleotides) attached to a microarray. These are subsequently detected using the Verigene System which measures light scattering from gold nanospheres tethered to another complementary oligonucleotide. Depending upon the genotype, patients can then divided into slow, fast or normal warfarin metabolisers and their doses adjusted accordingly.
FDA states that it cleared the test based on a broad range of published literature together with the results of a study, conducted by the manufacturer, on hundreds of DNA samples. ‘In a three site study, the test was accurate in all cases where the test yielded a result, although 8% of the tests could not identify which genetic variants were present.’ Although the Nanosphere test is not intended as a stand-alone tool to determine optimum drug dosage but to be used alongside clinical evaluation and other tools to determine the best treatment for patients, this approval underlines the FDA’s ongoing commitment to personalised medicine."
It's important to note that when the FDA announced the warfarin label change the agency (and Larry Lesko in particular) came under attack from critics who asserted that this was the FDA, inappropriately, telling doctors how to practice medicine.
What some see as mission creep, others see as responsibility in our new age of more precise diagnostics.
The concept of "safe use" as an integral part of the FDA's 21st century mission and the REMS concept (as refined via FDAAA) as one of many tactics to achieve better patient care is contentious.
And crucial.
Here's a link to the agenda.
A few thoughts in advance.
Many have looked at the FDAAA language on REMS and seen it as an ill-advised green light for the FDA to inject itself into the practice of medicine.
While I agree that REMS does indeed represent an expansion of the FDA's mission, I do not agree that it is ill advised. REMS is the responsible extension of the FDA's mission of safety and efficacy into the new realm of safe use. As previously discussed in this space ("A Potent FDA Double Feature") the FDA's role in educating both patients and providers on the safe use of approved therapies is a logical and approprite third leg of the agency's mission.
The first real move into this space was the FDA's change in the warfarin label. As Dr. Caroline Wright (Foundation for Genomics and Population Health) wrote:
“Just a month after the label for the blood-thinning drug warfarin was updated to explain that genetic variation in specific genes influences how patients respond to the drug, the US Food and Drug Agency (FDA) has approved the first genetic test for warfarin sensitivity.
Warfarin is the most widely used anti-coagulant medication in the world, prescribed to over 2 million people a year to prevent blood clots, heart attacks and strokes. Patients can display markedly different responses to the drug, so doses vary enormously between individuals. Achieving the correct dose is critical, as patients who receive too high a dose are at risk of severe bleeding, whilst those who receive too low a dose may remain at risk of life-threatening blood clots.
The Nanosphere Verigene Metabolism Nucleic Acids Test detects particular variations in two genes, CYP2C9 and VKORC1, which are involved in the metabolism and mechanism of action of warfarin respectively. Specific variants of these genes are identified from a patient sample by hybridization to sequence specific probes (oligonucleotides) attached to a microarray. These are subsequently detected using the Verigene System which measures light scattering from gold nanospheres tethered to another complementary oligonucleotide. Depending upon the genotype, patients can then divided into slow, fast or normal warfarin metabolisers and their doses adjusted accordingly.
FDA states that it cleared the test based on a broad range of published literature together with the results of a study, conducted by the manufacturer, on hundreds of DNA samples. ‘In a three site study, the test was accurate in all cases where the test yielded a result, although 8% of the tests could not identify which genetic variants were present.’ Although the Nanosphere test is not intended as a stand-alone tool to determine optimum drug dosage but to be used alongside clinical evaluation and other tools to determine the best treatment for patients, this approval underlines the FDA’s ongoing commitment to personalised medicine."
It's important to note that when the FDA announced the warfarin label change the agency (and Larry Lesko in particular) came under attack from critics who asserted that this was the FDA, inappropriately, telling doctors how to practice medicine.
What some see as mission creep, others see as responsibility in our new age of more precise diagnostics.
The concept of "safe use" as an integral part of the FDA's 21st century mission and the REMS concept (as refined via FDAAA) as one of many tactics to achieve better patient care is contentious.
And crucial.
