Special Report: Worth the Cost and Benefit? Drugs and Devices–Who Decides?

This is a second of a two part series on health care technology costs, benefits, effectiveness and safety. 

At $152 million dollars, a new cancer proton therapy facility sits on the campus of Northwest Hospital in Seattle, Washington, a joint venture of Fred Hutchinson Cancer Research Center, Seattle Cancer Care Alliance and Seattle Children’s Hospital. Medicare reimburses $32,428 for a course of treatment.  The treatments, however, have not been shown to be more effective on most cancers than the current radiology treatments that cost $18,575. While it sits on the campus of Northwest Hospital which is now part of the publicly funded University of Washington, the facility is owned by a privately held venture capital firm, ProCure.

The public believes that once a drug or device comes to market that it is safe and effective.  That is not always the case.

Johnson and Johnson faces 11,000 lawsuits over its metal on metal artificial hips that ground and scraped and discharged metal into the body.  Johnson and Johnson reportedly put aside one billion dollars to cover lawsuit settlement costs.  The first settlement was for $8.3 million to a retired Montana man. The artificial hip was on the market from 2003 to 2010 here and abroad. There are drug recalls from Fen Phen to Vioxx and Avastin, even after FDA approval.

The cost and efficacy of this facility and the recall of drugs and device raise the question of how drugs and medical devices come to market.  Who decides on their efficiency?  Who reviews their effectiveness in actual practice?  Who determines the costs? Welcome to the Byzantine world of pharmaceutical companies, medical devices, clinical trials, efficacy, effectiveness, cost, safety and venture capital.

How new drugs and medical devices are tested

 Most Americans would be surprised to learn that drugs and medical devices are only tested against placebos, not against other comparable devices or drugs that are on the market.  Not only that, the FDA (Food and Drug Administration) reviews cannot include any economic criteria in their decision-making observes Paul Fishman, PhD, Senior Investigator, Group Health Research Institute, Group Health Cooperative, Seattle, WA.  “They review effectiveness and safety, but only relative to a placebo,” he said. “There is a move toward making decisions on the basis of comparative effectiveness or relative to to existing and effective treatments rather than just a placebo, but even this move does not bring cost into the equation.”

“After regulatory approval by the FDA, the product goes to CMS (Center for Medicare and Medicaid Services). CMS, acting as an insurer, will consider economic considerations, but those decisions are heavily influenced by lobbying,” Fishman observed.

Fishman cited the example of sleep apnea and CPAP machines. CPAP machines cost in the range of $450 to $1,000.  To have a CPAP machine covered by insurance or Medicare, however, requires a diagnosis of sleep apnea.  Sleep apnea can be diagnosed in two ways Fishman noted:  1) in a physician’s office where the physician can test for windpipe blockage or obstruction; or 2) overnight sleep tests in  hospitals.  Up until recently, Fishman indicated, Medicare required an overnight hospital stay vs. the simple physician office test.  Pulmonary physicians said there was no reason for hospital stays except they were cash cows for both the hospital and physician.  But even though Medicare knew the economics, they still required the overnight stay.

“CMS is incredibly risk adverse because of the influence of industry lobbyists on Congress. CMS does things that may not be necessary to keep Congress off their backs. The pressure is very intense.  Medicare is very big business,” Fishman added. “At Group Health, for example, Medicare accounts for a significant amount of Group Health’s business. CMS is a key oversight and pricing agency and drives many health care coverage in pricing decisions.  . The major issue is the systemic and successful effectiveness of the pharmaceutical and medical device industries to limit regulatory intrusions.

“There is a relatively new group called the Patient Centered Outcomes Research Institute (PCORI) which is similar to the National Institute of Clinician the United Kingdom.  NICE is a multi-university group funded explicitly to examine cost-effectiveness, as well as determine whether or not the product is also both effective and safe,” Fishman said. “It can also address the economics. They can ask the question: is the investment worth it? PCORI, however, does not include costs in its outcome evaluations.

Another example Fishman cited was psoriasis. “There are two treatment options for severe psoriasis—biologic medicines, such as Enbrel with little or no out of pocket costs, but the drug costs $15,000 to $20,000 a year.  Or, Ultraviolet therapy 3-4 times which costs significantly less. Patients are often not aware of costs, all they see are the co-payments, not the total cost.  Studies are now starting to do add cost to the equation.

“Consumer Reports has a lobbying group for evidence based medicine. We are now seeing large employer groups coming together to put some pressure on value and evidence based medicine, such as Buyers Action Group on Health and the Pacific Business Group on Health in San Francisco. Here in the NW, we have the Puget Sound Health Alliance,” Fishman indicated. “The Foundation for Shared Decision-Making in Portland also has some very useful patient-oriented materials.  We need to make some good assessments and then boost communications to help consumers know how to interpret the information we have.”

“There used to be a federal Office of Technology Assessment which like the CBO was an arm of Congress, but that closed 15 years ago,” he indicated.  We used to have a state and federal CON (Certificate of Need) process, but that did not stem the tide. If someone wanted to build a 500 bed hospital, they would ask for a 1,000 bed hospital to make sure they would get the 500 beds. Then suddenly they were getting the 1,000 bed hospital.  We started to see capacity exceed demand.  CON simply did not work,” he said.

Evolution of Drug and Device Development and Perverse Incentives

 “The FDA has had wild swings. There was a long period when the US was one of the last places to start a new drug, after Thalidomide and all the birth deformities in the 1950’s and 60’s,” indicated Eric Larson, MD, MPH, Vice President and Group Health Executive Director and Senior Investigator, Group Health Research Institute.  “But now, the FDA is under tremendous pressure to be more efficient in its review process. The Medicare Part D market is huge. Great pressure has been exerted since the 1990’s for fast track approval of drugs and devices. The industry has overwhelmingly more resources than the FDA, and in our country it has behaved like a cost-plus industry.

“I remember my days at the University of Washington Medical Center. We had lots of sales and marketing representatives trying to sell us everything. Basically their sales pitch was to tell us how much revenue we could earn by new tests, new hospital stays, and so on.  They kept saying now you can do A, B and C which will generate so much new revenue for you.  But,” Larson questioned at one meeting with persons promoting cardiology business lines , “what if there is little to no marginal benefit for those new stays and products?”

Not only is the FDA involved in the drug approval process, it is now engaged in dramatic post-marketing surveillance. Larson noted that it is relatively easy to show safety in a controlled clinical trial because FDA regulations and common practice is to use the medication in highly selected populations, but once a drug is approved there is considerable off-label marketing, as well as on-label in everyday patients who may have added complexity compared with those in trials used to gain drug approval. .  “Often there may be some slight signs of a potential problem that really doesn’t become obvious until it moves into the larger population with hundreds of thousands of people not in controlled situations,” Larson pointed out.  “The approval process is very efficient to get products to market. They are not as efficient with safety.  The non-profit Public Citizen Health Research Group recommends people not use a new drug that is not a real “breakththrough drug, which most new drugs are not, for five years.”

Off label marketing is a huge problem.  AARP recently cited the company Par-Pharmaceuticals that began off-label marketing of Megace ES an FDA approved drug to help treat weight loss in AIDs patients.  Par starting promoting Megace ES for use in nursing homes for non-AIDs patients, even though they had been told twice by the FDA that it had to be tested again for uses other than AIDS. In March, Par faced civil and criminal suits about their off label marketing, and it agreed to pay $45 million in penalties. According to AARP Par had made $11 million from their off label marketing.

“Regrettably, there is very little that the FDA regulates besides verifying the product does what it says it can do,” Larson indicated. “There is no CON other than payers and the liability system. Entrepreneurs can  decide the product and the costs. This is a serious problem for our society and the medical profession. Many years ago there was the debate about which was better the CT scan or the MRI.  Now we have a higher level of sophistication and cost.  There’s not much that can be done when some technology is new. We need to look at how many this technology will help and how it will it need to be used to pay for the investment. Often, that use will be off  label use”

Community Benefit

Larson believes we need to ask the larger question: if we have a for-profit working with non-profit corporations, what is the community benefit? “No one seems to be considering the issue of  community benefit.  In the past Washington State had a rate commission and CON process.  Today,no one is looking at CON or hospital rates in the current political environment,” Larson observed.  “As a society, we have left these cost, effectiveness and safety issues to free market forces which are not driven by  interest in these issues.

“We need to look at comparative effectiveness so we can begin to talk about costs.  We need to question if a new technology or drug is only one percent more effective, is it worth it when the new technology costs three times as much as what is already being used?” Larson suggested.

The new Choosing Wisely Campaign of the ABIM Foundation with Consumer Reports, the Puget Sound Health Alliance, and now larger employers are trying to put value into the equation. “Here at Group Health,” Larson noted, “we have an Evidence Review Committee which has been very effective in reviewing evidence and value.  We tend to be later adapters in terms of drugs, but consequently we have avoided drugs that turned out to be toxic. We also participate with Kaiser Permanente in Northern California in an  orthopedic device registry to help identify the devices with the best track record.

“I have a strong belief in our profession, but we really need to educate the public about the tremendous consequences of our expensive, cost-plus system. We have great medical research, but we have to figure out a way to have an efficient and equitable system,” Larson stressed.  “If we are going to bring in 30 to 40 million more people into the health care system.  We can’t afford the waste we have now.”

Comparative Effectiveness

Noting that there is little evidence-based approach to the evaluation and review of drugs and medical devices, Denise Boudreau, Associate Scientific Investigator with the Group Health Research Institute noted that the FDA does not require any comparative testing of drugs or devices.  Clinical trials are only compared to placebos and monitored for safety, but they are not compared to standards of care. “These standards are not cut and dried, however,” Boudreau observed.  “Some medical groups write standard guidelines, but there is considerable geographic variation in their use of drugs and technologies and variations by clinical settings and types of providers.

“Even after a drug comes to market there can be trials and observational studies by groups such as Group Health, academic medical settings, PhRMA, Agency for Health Care Research and Quality and the new PCORI (Patient-Centered Outcomes Research Institute) and foundations. The real push now is with PCORI which is now just a few years old,” Boudreau said.

There are also various layers of review. “Health plans determine what drugs they will pay for on their formularies, what devices they will cover.  They look at efficacy, safety and cost.  Efficacy and safety are usually the top considerations,” Boudreau observes. “Group Health, for example, did not cover Vioxx.  Group Health looked at the data, and we were not convinced that data were there. There were too many questions.”

Limits of Clinical Trials and Safety Evaluation

One of the limits of randomized clinical trials, Boudreau observed, is they tend to be for a short period of time—usually one to three years.  “That is not nearly enough time to determine cumulative effects or long-term efficacy and safety. Clinical trials tend to be composed largely of healthy men, and not women of child bearing age or children. The sample size is limited to a few thousand people.  But when a drug goes to market, it goes out to the public at large, with hundreds of thousands of people with different health conditions now taking a specific drug,” she said.

The FDA now has more active surveillance. It has a new sentinel program that has patient data on over 100 million patients over the past four years.

“There was a push to give the FDA more teeth. When Congress was pushed to look at the FDA.  They found that the FDA had no money to have teeth. When it looked at the FDA’s budget, Congress found the vast majority of employees were on the approval side, not the surveillance side,” Boudreau indicated.

As noted earlier, the US was not the first to bring a new drug to market for a long time after Thalidomide. “But in the late 1980’s and early 1990’s because of the AID’s epidemic, there was tremendous pressure to fast track drug trials. That has flipped the drug development equation. Now the US is the first to bring drugs to market.  More drugs are tested here now but,” Boudreau observed, “there is still not enough safety surveillance.”

How the State Decides What to Cover

Leah Hole-Curry is the Medical Administrator in the Department of Labor and Industries (LNI).  Prior to that, she was the first head of the Health Technology Assessment (HTA) Program in the Health Care Authority (HCA) from 2006 to 2011.  The HTA conducts clinical literature reviews and then asks a panel of practicing doctors to make a decision about whether the state should invest public funds in the new drug or device.  LNI (as well as Medicaid and the Public Employee Benefits program) by law, must implement the decisions coming out of the HTA about the use of a technology.

Unlike drugs, for a device to receive FDA approval, it goes through either a Pre-Market Approval or the exemption (510K) process within the FDA.  For a 510K approval, the manufacturer need only show it is similar to something already approved.  Most medical devices go through the exemption process because it is an easier standard.  Neither process requires the manufacturer to demonstrate a better outcome for patients than what is currently available.

For example, a pain pump is a device that required FDA approval. It is implanted and programmed to release pain medication into the body, rather than taking pain pills. The claim is that it controls pain better with less medication by the direct release of medication (rather than a pill that must go through the blood stream.  The FDA, Hole-Curry indicated looks at whether the device can be physically implanted without rejection and that it can be implanted in the right place and will actually release medicine as programmed.  “LNI, however, evaluates the patient outcomes of using the device – is it safe (surgical complications, revisions, and removals were common experiences for injured workers), do patients function better, have fewer side effects, take fewer oral pain medicines, and return to work and productivity? The FDA does not look at comparative-effectiveness, LNI wants to ensure that it gets value for its public dollars and  wants to have evidence that the device is better than something already on the market.  LNI also looks at cost-effectiveness – does the product provide good value?  Because good quality evidence comparing products is so limited, this is rarely a black and white outcome,” Hole-Curry said.

In the case of the cancer proton therapy facility, it went through the CON process in the Department of Health for new hospital facilities.  Three state agencies—Medicaid, LNI and PEBB (Public Employee Benefits Board) all are payers, and will be required to decide whether and how much public funds should be used to pay for this new treatment.  As for device pricing, the State uses Medicare for pricing guidelines.  Given the very high cost compared to alternatives, the question for public agencies is how can we invest in the treatments that provide the best value and benefit to the most people.  Proton beam therapy has very limited evidence that it is safer or more effective, yet because the facility is now built, the market may drive the outcome unless coverage goes through some type of evidence review process like the HTA. The decision to allow this facility,” Hole-Curry said, “was not insignificant in terms of costs—and once there is one, then others will want it as well.”

What both the scientists at Group Health Research Institute and Hole-Curry agree on is the entire review and approval process is market driven, not medically driven, and that while safety is considered, relatively little consideration is given to whether the treatment will result in better health outcomes than using current available options..  It is often difficult for the payer to say no, and it is even harder for a public payer to say no because there is a lot of pressure to get a new product on the market.

“There is a mismatch between what the FDA does, the scope of its authority and what the public thinks it does.  The public thinks FDA review and approval means a product or drug is safe and effective.  We really need to be clear about what an FDA review really means,” Hole-Curry stresses.

What this boils down to is that we need a better educated public that does not believe every ad on TV. We need to assure safety and efficacy of new drugs and technologies that are based on medicine, not the marketplace.  And we need teeth to protect the American public.

Kathleen O’Connor © May 19, 2013; updated June 5, 2013

 

 

 

 

 

 

 

 

 

 

 

Kathleen O’Connor © May 19, 2013

 

 

 

 

 

 

 

 

At $152 million dollars, a new cancer proton therapy facility sits on the campus of Northwest Hospital in Seattle, Washington, a joint venture of Fred Hutchinson Cancer Research Center, Seattle Cancer Care Alliance and Seattle Children’s Hospital. Medicare reimburses $32,428 for a course of treatment.  The treatments, however, have not been shown to be more effective on most cancers than the current radiology treatments that cost $18,575. While it sits on the campus of Northwest Hospital which is now part of the publicly funded University of Washington, the facility is owned by a privately held venture capital firm, ProCare LLC.

The public believes that once a drug or device comes to market that it is safe and effective.  That is not always the case.

Johnson and Johnson faces 11,000 lawsuits over its metal on metal artificial hips that ground and scraped and discharged metal into the body.  Johnson and Johnson reportedly put aside one billion dollars to cover lawsuit settlement costs.  The first settlement was for $8.3 million to a retired Montana man. The artificial hip was on the market from 2003 to 2010 here and abroad. There are drug recalls from Fen Phen to Vioxx and Avastin, even after FDA approval.

The cost and efficacy of this facility and the recall of drugs and device raise the question of how drugs and medical devices come to market.  Who decides on their efficiency?  Who reviews their effectiveness in actual practice?  Who determines the costs? Welcome to the Byzantine world of pharmaceutical companies, medical devices, clinical trials, efficacy, effectiveness, cost, safety and venture capital.

How new drugs and medical devices are tested. 

 Most Americans would be surprised to learn that drugs and medical devices are only tested against placebos, not against other comparable devices or drugs that are on the market.  Not only that, the FDA (Food and Drug Administration) reviews cannot include any economic criteria in their decision-making observes Paul Fishman, PhD, Senior Investigator, Group Health Research Institute, Group Health Cooperative, Seattle, WA.  “They review effectiveness and safety, but only relative to a placebo,” he said. “There is a move toward making decisions on the basis of comparative effectiveness or relative to to existing and effective treatments rather than just a placebo, but even this move does not bring cost into the equation.”

“After regulatory approval by the FDA, the product goes to CMS (Center for Medicare and Medicaid Services). CMS, acting as an insurer, will consider economic considerations, but those decisions are heavily influenced by lobbying,” Fishman observed.

Fishman cited the example of sleep apnea and CPAP machines. CPAP machines cost in the range of $450 to $1,000.  To have a CPAP machine covered by insurance or Medicare, however, requires a diagnosis of sleep apnea.  Sleep apnea can be diagnosed in two ways Fishman noted:  1) in a physician’s office where the physician can test for windpipe blockage or obstruction; or 2) overnight sleep tests in  hospitals.  Up until recently, Fishman indicated, Medicare required an overnight hospital stay vs. the simple physician office test.  Pulmonary physicians said there was no reason for hospital stays except they were cash cows for both the hospital and physician.  But even though Medicare knew the economics, they still required the overnight stay.

“CMS is incredibly risk adverse because of the influence of industry lobbyists on Congress. CMS does things that may not be necessary to keep Congress off their backs. The pressure is very intense.  Medicare is very big business,” Fishman added. “At Group Health, for example, Medicare accounts for a significant amount of Group Health’s business. CMS is a key oversight and pricing agency and drives many health care coverage in pricing decisions.  . The major issue is the systemic and successful effectiveness of the pharmaceutical and medical device industries to limit regulatory intrusions.

“There is a relatively new group called the Patient Centered Outcomes Research Institute (PCORI) which is similar to the National Institute of Clinician the United Kingdom.  NICEis a multi-university group funded explicitly to examine cost-effectiveness, as well as determine whether or not the product is also both effective and safe,” Fishman said. “It can also address the economics. They can ask the question: is the investment worth it? PCORI, however, does not include costs in its outcome evaluations.

Another example Fishman cited was psoriasis. “There are two treatment options for severe psoriasis—biologic medicines, such as Enbrel with little or no out of pocket costs, but the drug costs $15,000 to $20,000 a year.  Or, Ultraviolet therapy 3-4 times which costs significantly less. Patients are often not aware of costs, all they see are the co-payments, not the total cost.  Studies are now starting to do add cost to the equation.

“Consumer Reports has a lobbying group for evidence based medicine. We are now seeing large employer groups coming together to put some pressure on value and evidence based medicine, such as Buyers Action Group on Health and the Pacific Business Group on Health in San Francisco. Here in the NW, we have the Puget Sound Health Alliance,” Fishman indicated. “The Foundation for Shared Decision-Making in Portland also has some very useful patient-oriented materials.  We need to make some good assessments and then boost communications to help consumers know how to interpret the information we have.”

“There used to be a federal Office of Technology Assessment which like the CBO was an arm of Congress, but that closed 15 years ago,” he indicated.  We used to have a state and federal CON (Certificate of Need) process, but that did not stem the tide. If someone wanted to build a 500 bed hospital, they would ask for a 1,000 bed hospital to make sure they would get the 500 beds. Then suddenly they were getting the 1,000 bed hospital.  We started to see capacity exceed demand.  CON simply did not work,” he said.

Evolution of Drug and Device Development and Perverse Incentives

 “The FDA has had wild swings. There was a long period when the US was one of the last places to start a new drug, after Thalidomide and all the birth deformities in the 1950’s and 60’s,” indicated Eric Larson, MD, MPH, Vice President and Group Health Executive Director and Senior Investigator, Group Health Research Institute.  “But now, the FDA is under tremendous pressure to be more efficient in its review process. The Medicare Part D market is huge. Great pressure has been exerted since the 1990’s for fast track approval of drugs and devices. The industry has overwhelmingly more resources than the FDA, and in our country it has behaved like a cost-plus industry.

“I remember my days at the University of Washington Medical Center. We had lots of sales and marketing representatives trying to sell us everything. Basically their sales pitch was to tell us how much revenue we could earn by new tests, new hospital stays, and so on.  They kept saying now you can do A, B and C which will generate so much new revenue for you.  But,” Larson questioned at one meeting with persons promoting cardiology business lines , “what if there is little to no marginal benefit for those new stays and products?”

Not only is the FDA involved in the drug approval process, it is now engaged in dramatic post-marketing surveillance. Larson noted that it is relatively easy to show safety in a controlled clinical trial because FDA regulations and common practice is to use the medication in highly selected populations, but once a drug is approved there is considerable off-label marketing, as well as on-label in everyday patients who may have added complexity compared with those in trials used to gain drug approval. .  “Often there may be some slight signs of a potential problem that really doesn’t become obvious until it moves into the larger population with hundreds of thousands of people not in controlled situations,” Larson pointed out.  “The approval process is very efficient to get products to market. They are not as efficient with safety.  The non-profit Public Citizen Health Research Group recommends people not use a new drug that is not a real “breakththrough drug, which most new drugs are not, for five years.”

Off label marketing is a huge problem.  AARP recently cited the company Par-Pharmaceuticals that began off-label marketing of Megace ES an FDA approved drug to help treat weight loss in AIDs patients.  Par starting promoting Megace ES for use in nursing homes for non-AIDs patients, even though they had been told twice by the FDA that it had to be tested again for uses other than AIDS. In March, Par faced civil and criminal suits about their off label marketing, and it agreed to pay $45 million in penalties. According to AARP Par had made $11 million from their off label marketing.

“Regrettably, there is very little that the FDA regulates besides verifying the product does what it says it can do,” Larson indicated. “There is no CON other than payers and the liability system. Entrepreneurs can  decide the product and the costs. This is a serious problem for our society and the medical profession. Many years ago there was the debate about which was better the CT scan or the MRI.  Now we have a higher level of sophistication and cost.  There’s not much that can be done when some technology is new. We need to look at how many this technology will help and how it will it need to be used to pay for the investment. Often, that use will be off  label use”

Community Benefit

Larson believes we need to ask the larger question: if we have a for-profit working with non-profit corporations, what is the community benefit? “No one seems to be considering the issue of  community benefit.  In the past Washington State had a rate commission and CON process.  Today,no one is looking at CON or hospital rates in the current political environment,” Larson observed.  “As a society, we have left these cost, effectiveness and safety issues to free market forces which are not driven by  interest in these issues.

“We need to look at comparative effectiveness so we can begin to talk about costs.  We need to question if a new technology or drug is only one percent more effective, is it worth it when the new technology costs three times as much as what is already being used?” Larson suggested.

The new Choosing Wisely Campaign of the ABIM Foundation with Consumer Reports, the Puget Sound Health Alliance, and now larger employers are trying to put value into the equation. “Here at Group Health,” Larson noted, “we have an Evidence Review Committee which has been very effective in reviewing evidence and value.  We tend to be later adapters in terms of drugs, but consequently we have avoided drugs that turned out to be toxic. We also participate with Kaiser Permanente in Northern California in an  orthopedic device registry to help identify the devices with the best track record.

“I have a strong belief in our profession, but we really need to educate the public about the tremendous consequences of our expensive, cost-plus system. We have great medical research, but we have to figure out a way to have an efficient and equitable system,” Larson stressed.  “If we are going to bring in 30 to 40 million more people into the health care system.  We can’t afford the waste we have now.”

Comparative Effectiveness

Noting that there is little evidence-based approach to the evaluation and review of drugs and medical devices, Denise Boudreau, Associate Scientific Investigator with the Group Health Research Institute noted that the FDA does not require any comparative testing of drugs or devices.  Clinical trials are only compared to placebos and monitored for safety, but they are not compared to standards of care. “These standards are not cut and dried, however,” Boudreau observed.  “Some medical groups write standard guidelines, but there is considerable geographic variation in their use of drugs and technologies and variations by clinical settings and types of providers.

“Even after a drug comes to market there can be trials and observational studies by groups such as Group Health, academic medical settings, PhRMA, Agency for Health Care Research and Quality and the new PCORI (Patient-Centered Outcomes Research Institute) and foundations. The real push now is with PCORI which is now 2 years old,” Boudreau said.

There are also various layers of review. “Health plans determine what drugs they will pay for on their formularies, what devices they will cover.  They look at efficacy, safety and cost.  Efficacy is usually the top consideration,” Boudreau observes. “Group Health, for example, did not cover Vioxx.  Group Health looked at the data, and we were not convinced that efficacy and safety data were there. There were too many questions.”

Limits of Clinical Trials and Safety Evaluation

One of the limits of randomized clinical trials, Boudreau observed, is they tend to be for a short period of time—usually one to three years.  “That is not nearly enough time to determine cumulative effects or long-term efficacy and safety. Clinical trials tend to be composed largely of healthy men, and not women or children. The sample size is limited to about 1,000 people.  But when a drug goes to market, it goes out to the public at large, with hundreds of thousands of people with different health conditions now taking a specific drug,” she said.

The FDA now has more active surveillance. It has a new sentinel program that has patient data on over 100 million patients over the past four years.

“There was a push to give the FDA more teeth. When Congress was pushed to look at the FDA.  They found that the FDA had no money to have teeth. When it looked at the FDA’s budget, Congress found the vast majority of employees were on the approval side, not the surveillance side,” Boudreau indicated.

As noted earlier, the US was not the first to bring a new drug to market for a long time after Thalidomide. “But in the late 1980’s and early 1990s because of the AIDs epidemic, there was tremendous pressure to fast track drug trials. That has flipped the drug development equation. Now the US is the first to bring drugs to market.  More drugs are tested here now but,” Boudreau observed, “there is still not enough safety surveillance.

How the State Decides What to Cover

Leah Hole-Curry is the Medical Administrator in the Department of Labor and Industries (LNI).  Prior to that, she was the first head of the Health Technology Assessment (HTA) Program in the Health Care Authority (HCA) from 2006 to 2011.  The HTA conducts clinical literature reviews and then asks a panel of practicing doctors to make a decision about whether the state should invest public funds in the new drug or device.  LNI (as well as Medicaid and the Public Employee Benefits program) by law, must implement the decisions coming out of the HTA about the use of a technology.

Unlike drugs, for a device to receive FDA approval, it goes through either a Pre-Market Approval or the exemption (510K) process within the FDA.  For a 510K approval, the manufacturer need only show it is similar to something already approved.  Most medical devices go through the exemption process because it is an easier standard.  Neither process requires the manufacturer to demonstrate a better outcome for patients than what is currently available.

For example, a pain pump is a device that required FDA approval. It is implanted and programmed to release pain medication into the body, rather than taking pain pills. The claim is that it controls pain better with less medication by the direct release of medication (rather than a pill that must go through the blood stream).  The FDA, Hole-Curry indicated looks at whether the device can be physically implanted without rejection and that it can be implanted in the right place and will actually release medicine as programmed.

“LNI, however, evaluates the patient outcomes of using the device – is it safe (surgical complications, revisions, and removals were common experiences for injured workers), do patients function better, have fewer side effects, take fewer oral pain medicines, and return to work and productivity?  The FDA does not look at comparative-effectiveness, LNI wants to ensure that it gets value for its public dollars and  wants to have evidence that the device is better than something already on the market.  LNI also looks at cost-effectiveness – does the product provide good value?  Because good quality evidence comparing products is so limited, this is rarely a black and white outcome,” Hole-Curry said.

In the case of the cancer proton therapy facility, it went through the CON process in the Department of Health for new hospital facilities.  Three state agencies—Medicaid, LNI and PEBB (Public Employee Benefits Board) all are payers, and will be required to decide whether and how much public funds should be used to pay for this new treatment.  As for device pricing, the State uses Medicare for pricing guidelines.  Given the very high cost compared to alternatives, the question for public agencies is how can we invest in the treatments that provide the best value and benefit to the most people.  Proton beam therapy has very limited evidence that it is safer or more effective, yet because the facility is now built, the market may drive the outcome unless coverage goes through some type of evidence review process like the HTA. The decision to allow this facility,” Hole-Curry said, “was not insignificant in terms of costs—and once there is one, then others will want it as well.

What both the scientists at Group Health Research Institute and Hole-Curry agree on is the entire review and approval process is market driven, not medically driven, and that while safety is considered, relatively little consideration is given to whether the treatment will result in better health outcomes than using current available options..  It is often difficult for the payer to say no, and it is even harder for a public payer to say no because there is a lot of pressure to get a new product on the market.

“There is a mismatch between what the FDA does, the scope of its authority and what the public thinks it does.  The public thinks FDA review and approval means a product or drug is safe and effective.  We really need to be clear about what an FDA review really means,” Hole-Curry stresses.

What this boils down to is that we need a better educated public that does not believe every ad on TV. We need to assure safety and efficacy of new drugs and technologies that are based on medicine, not the marketplace.  And we need teeth to protect the American public.

Kathleen O’Connor © May 19, 2013; revised June 5, 2013

 

 

 

 

 

 

 

 

 

 

 

Kathleen O’Connor © May 19, 2013

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

About Kathleen

Kathleen O’Connor: 30+ year health care consumer advocate, non-profit executive and author. For more information about Kathleen, please see "About" on the main content bar above.
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