Jerry Avorn has been a long time malignant presence on the health policy scene. Among his contributions has been a frontal assault on one of the most effective drugs developed to treat heart failure in African Americans (BiDil) by claiming it shouldn't be approved because we didn't know the mechanism of action. That brought a swift response from the FDA's Bob Temple who noted that if the FDA only approved drugs where the mechanism of actio was known, a lot of people alive today would be dead.
Now Avorn and part of his crew, Aaron Kesselheim and Jan Myers are arguing that drugs for rare diseases are not studied long enough and don't have enough people in clinical trials AND should be studied in randomized controlled trials.
Avorn, Kesselheim and Myers to kids dying of rare disease: Drop dead while we study you as long as we deem it appropriate. Note to Kesselheim and the rest of the Kevorkian Corp: orphan drug trials are designed to reflect the size and needs of patients, not the use of CER as a rationale for rationing.
Orphan drugs add more years to life than any other class of treatments. Two-thirds of the people who benefit are kids who can now live longer instead of dying painfully. But CER advocates like Avorn and Kesslehim want longer and more complicated trials because they know, as with Avastin , average results will reduce the number of treatments. CER will be a death sentence to many children. People like Kesselheim and Avorn raise the bloody shirt of drug safety to justify their position. Here's my view: let them say no to orphan drugs when it might save the life of their loved ones out of safety concerns and leave the rest of us alone. Please.
Thankfully, Congress is putting it’s foot down in support of innovation and personalized medicine. Sen. Robert Casey, D-Penn., has introduced legislation last March to support this research. The Creating Hope Act of 2011, will provide incentives “to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions." Meanwhile, Senators Jon Kyl R-TX and Mitch McConnell R-KY are sponsoring The Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2011. The PATIENTS Act would bar the federal government from using “comparative effectiveness research” to deny or delay coverage of a health-care treatment….”
Today medical innovatons can use the understanding of how we get sick to prevent disease or death. Such advances lead to longer, better lives, enriching us all. CER, by design, censors these biological insights. It deliberately delays progress by demanding studies that, by ignoring individual differences, conclude no one benefits from medical progress. It is used to justify rationing, not make individuals more sustainable. To save ourselves and children dying of rare diseases we have to pull the plug on CER and it’s adherents. Starting with Kessleheim, Myers and Avorn -- Harvard's Kevorkian Krew -- is a great place to start.
Now Avorn and part of his crew, Aaron Kesselheim and Jan Myers are arguing that drugs for rare diseases are not studied long enough and don't have enough people in clinical trials AND should be studied in randomized controlled trials.
Avorn, Kesselheim and Myers to kids dying of rare disease: Drop dead while we study you as long as we deem it appropriate. Note to Kesselheim and the rest of the Kevorkian Corp: orphan drug trials are designed to reflect the size and needs of patients, not the use of CER as a rationale for rationing.
Orphan drugs add more years to life than any other class of treatments. Two-thirds of the people who benefit are kids who can now live longer instead of dying painfully. But CER advocates like Avorn and Kesslehim want longer and more complicated trials because they know, as with Avastin , average results will reduce the number of treatments. CER will be a death sentence to many children. People like Kesselheim and Avorn raise the bloody shirt of drug safety to justify their position. Here's my view: let them say no to orphan drugs when it might save the life of their loved ones out of safety concerns and leave the rest of us alone. Please.
Thankfully, Congress is putting it’s foot down in support of innovation and personalized medicine. Sen. Robert Casey, D-Penn., has introduced legislation last March to support this research. The Creating Hope Act of 2011, will provide incentives “to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions." Meanwhile, Senators Jon Kyl R-TX and Mitch McConnell R-KY are sponsoring The Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2011. The PATIENTS Act would bar the federal government from using “comparative effectiveness research” to deny or delay coverage of a health-care treatment….”
Today medical innovatons can use the understanding of how we get sick to prevent disease or death. Such advances lead to longer, better lives, enriching us all. CER, by design, censors these biological insights. It deliberately delays progress by demanding studies that, by ignoring individual differences, conclude no one benefits from medical progress. It is used to justify rationing, not make individuals more sustainable. To save ourselves and children dying of rare diseases we have to pull the plug on CER and it’s adherents. Starting with Kessleheim, Myers and Avorn -- Harvard's Kevorkian Krew -- is a great place to start.
[1] http://jama.ama-assn.org/content/305/22/2320.short
