Latest Drugwonks' Blog
From today’s edition of the East Bay Times …
Patients threatened by new drug coverage limits
Kareem Abdul-Jabbar’s toughest fight wasn’t on a basketball court.
In his early 60s, the six-time NBA champion was diagnosed with leukemia, the deadly blood cancer. Fortunately, Abdul-Jabbar had access to state-of-the-art medications, including the advanced drug Tasigna, which paralyzed his cancer cells and prevented further growth. Today, eight years after his initial diagnosis, Abdul-Jabbar is thriving and cancer-free.
Unfortunately, many of today’s leukemia patients won’t be so lucky. CVS Health, the nation’s second-largest pharmacy benefit manager that oversees 65 million Americans’ drug plans, recently rescinded coverage for Tasigna –— and 130 other specialty drugs.
As a result, millions of people could be denied access to drugs that could save their lives. Instead of prescribing the medicines best-suited to patient needs, physicians will be forced to recommend lower-quality treatments.
Pharmacy benefit managers, or “PBMs” for short, administer the prescription drug plans used by health insurers and employers. In recent years, these organizations have gotten stingy about which drugs they cover.
Back in 2012, the nation’s largest PBM, Express Scripts, excluded no medicines from its list of covered drugs, while CVS Health left off about 30. Today, they exclude more than 200, including an array of popular treatments for arthritis, Hepatitis C, and various skin conditions.
PBMs have also stopped paying for cutting-edge cancer treatments. In addition to Tasigna, CVS won’t cover the revolutionary prostate cancer treatment Xtandi. Meanwhile, Express Scripts just stopped covering Zyclara, a cream that can help prevent skin cancer.
PBMs are restricting drug access in other, more devious ways as well.
CVS is also steering patients away from ultra-complex “biologic” drugs, forcing them to switch to lower-cost treatments the company claims are medically equivalent. But in many cases these less expensive therapies, known as “biosimilars,” aren’t approved by the FDA to be interchangeable with their brand name alternatives.
Consider one study that compared the effectiveness of a Crohn’s disease treatment and its biosimilar. An alarming eight in 10 patients who took the biosimilar required a hospital readmission for additional treatment, compared to only one in 20 who took the original drug.
Despite these disturbing results, PBMs are comfortable forcing patients to use biosimilars and generic medications. That’s because their only concern is bringing down short-term drug spending — even if those savings come at a cost to patients’ well-being.
Ironically, this strategy will end up raising health care costs in the long-run. If doctors can only prescribe less-effective treatments, folks will get sicker, be hospitalized more frequently, and require more expensive care. That demand will drive up overall healthcare costs and overwhelm doctors and hospitals with waves of new patients.
That doesn’t matter to PBMs, though. A dollar saved by avoiding top-notch drugs is a dollar that goes into PBMs’ pockets — even if the patient becomes sicker on less effective treatments and racks up much larger hospital bills for insurers and patients to pay down the road.
PBMs coverage denials are a deadly prescription for America’s patients. By shrinking coverage for cutting-edge treatments, PBMs are forcing sick people to use substandard drugs. It’s about time patients mount a full-court press against this callous behavior.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.
Patients threatened by new drug coverage limits
Kareem Abdul-Jabbar’s toughest fight wasn’t on a basketball court.
In his early 60s, the six-time NBA champion was diagnosed with leukemia, the deadly blood cancer. Fortunately, Abdul-Jabbar had access to state-of-the-art medications, including the advanced drug Tasigna, which paralyzed his cancer cells and prevented further growth. Today, eight years after his initial diagnosis, Abdul-Jabbar is thriving and cancer-free.
Unfortunately, many of today’s leukemia patients won’t be so lucky. CVS Health, the nation’s second-largest pharmacy benefit manager that oversees 65 million Americans’ drug plans, recently rescinded coverage for Tasigna –— and 130 other specialty drugs.
As a result, millions of people could be denied access to drugs that could save their lives. Instead of prescribing the medicines best-suited to patient needs, physicians will be forced to recommend lower-quality treatments.
Pharmacy benefit managers, or “PBMs” for short, administer the prescription drug plans used by health insurers and employers. In recent years, these organizations have gotten stingy about which drugs they cover.
Back in 2012, the nation’s largest PBM, Express Scripts, excluded no medicines from its list of covered drugs, while CVS Health left off about 30. Today, they exclude more than 200, including an array of popular treatments for arthritis, Hepatitis C, and various skin conditions.
PBMs have also stopped paying for cutting-edge cancer treatments. In addition to Tasigna, CVS won’t cover the revolutionary prostate cancer treatment Xtandi. Meanwhile, Express Scripts just stopped covering Zyclara, a cream that can help prevent skin cancer.
PBMs are restricting drug access in other, more devious ways as well.
CVS is also steering patients away from ultra-complex “biologic” drugs, forcing them to switch to lower-cost treatments the company claims are medically equivalent. But in many cases these less expensive therapies, known as “biosimilars,” aren’t approved by the FDA to be interchangeable with their brand name alternatives.
Consider one study that compared the effectiveness of a Crohn’s disease treatment and its biosimilar. An alarming eight in 10 patients who took the biosimilar required a hospital readmission for additional treatment, compared to only one in 20 who took the original drug.
Despite these disturbing results, PBMs are comfortable forcing patients to use biosimilars and generic medications. That’s because their only concern is bringing down short-term drug spending — even if those savings come at a cost to patients’ well-being.
Ironically, this strategy will end up raising health care costs in the long-run. If doctors can only prescribe less-effective treatments, folks will get sicker, be hospitalized more frequently, and require more expensive care. That demand will drive up overall healthcare costs and overwhelm doctors and hospitals with waves of new patients.
That doesn’t matter to PBMs, though. A dollar saved by avoiding top-notch drugs is a dollar that goes into PBMs’ pockets — even if the patient becomes sicker on less effective treatments and racks up much larger hospital bills for insurers and patients to pay down the road.
PBMs coverage denials are a deadly prescription for America’s patients. By shrinking coverage for cutting-edge treatments, PBMs are forcing sick people to use substandard drugs. It’s about time patients mount a full-court press against this callous behavior.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.
As they say, everything you read in the newspaper is true -- except for those things you know about personally.
According to the latest report from the Altarum Institute, “moderate 2016 health spending growth continues a slow downward trend.” Unfortunately this doesn’t fit the narrative of those who want to talk about runaway trains – especially for pharmaceuticals.
Here are the numbers: Hospital spending represents 32% of American healthcare spending, 20% goes to physician and clinical services, 15% goes to “other health spending,” and 10% is for prescription drugs.
If the media had covered this story -- which they have not -- the headline would have been, "Pharmaceuticals represent 10% of American healthcare spending. A dime on the dollar. A smaller percentage than almost every nation in Europe."
And here’s the subhead – Spending on pharmaceuticals is growing at a slower rate (under 4%) than either hospital (just under 5%) or physician costs (just over 5%). This breakdown is based on the Bureau of Economic Analysis monthly spending data, including its most recent update released on August 29th of this year.
While pharmaceutical spending seems to be the only issue of interest to the media and politicians, the Altarum account isn’t the one they’re telling. Almost every story and every speech on the drug sector focuses on price without the context of value, using a few bad actors to represent the entire industry. Unfair? Sure -- but life is unfair. There is, however, no excuse for slanted stories and untrue, accusatory political oration.
Disease is the enemy. Practicing physicians know this, but their professional association – the American Medical Association – seems in need of some education. The AMA’s new program, “truthinrx.org,” is entirely silent on the actual metrics of healthcare spending and the value of pharmaceutical innovation. Ignorance is not bliss.
Healthcare innovation saves lives, saves money, promotes economic growth, and provides hope for hundreds of millions of people (both patients and care-givers) in the United States and around the world. It deserves respect – or at least honest reportage.
Does this sound naïve? Perhaps, but as Schopenhauer said, “All truth passes through three stages. First, it is ridiculed. Second, it is violently opposed. Third, it is accepted as being self-evident.”
The complete Altarum report can be found here.
According to the latest report from the Altarum Institute, “moderate 2016 health spending growth continues a slow downward trend.” Unfortunately this doesn’t fit the narrative of those who want to talk about runaway trains – especially for pharmaceuticals.
Here are the numbers: Hospital spending represents 32% of American healthcare spending, 20% goes to physician and clinical services, 15% goes to “other health spending,” and 10% is for prescription drugs.
If the media had covered this story -- which they have not -- the headline would have been, "Pharmaceuticals represent 10% of American healthcare spending. A dime on the dollar. A smaller percentage than almost every nation in Europe."
And here’s the subhead – Spending on pharmaceuticals is growing at a slower rate (under 4%) than either hospital (just under 5%) or physician costs (just over 5%). This breakdown is based on the Bureau of Economic Analysis monthly spending data, including its most recent update released on August 29th of this year.
While pharmaceutical spending seems to be the only issue of interest to the media and politicians, the Altarum account isn’t the one they’re telling. Almost every story and every speech on the drug sector focuses on price without the context of value, using a few bad actors to represent the entire industry. Unfair? Sure -- but life is unfair. There is, however, no excuse for slanted stories and untrue, accusatory political oration.
Disease is the enemy. Practicing physicians know this, but their professional association – the American Medical Association – seems in need of some education. The AMA’s new program, “truthinrx.org,” is entirely silent on the actual metrics of healthcare spending and the value of pharmaceutical innovation. Ignorance is not bliss.
Healthcare innovation saves lives, saves money, promotes economic growth, and provides hope for hundreds of millions of people (both patients and care-givers) in the United States and around the world. It deserves respect – or at least honest reportage.
Does this sound naïve? Perhaps, but as Schopenhauer said, “All truth passes through three stages. First, it is ridiculed. Second, it is violently opposed. Third, it is accepted as being self-evident.”
The complete Altarum report can be found here.
A recent article in MedPage - Insurers to CMS, Congress: For Lower Rx Costs, Hand Us the Reins – needs a bit of translation to help people under stand what handing insurers the rein over what drugs and how much to spend really means. For instance, the headline should actually read "Insurers to CMS, Congress: We Want More Rx Rebates So Give Us Total Control Over Medicare Drug Benefit"
According to Medpage: “Congress and the administration can help rein in rising drug costs through a few key regulatory and legal fixes, said insurers and pharmacists at AHIP's National Conference on Medicare, Medicaid and Duals on Tuesday.”
"I think plans need to be given a little bit more leeway to truly assess the value of drugs to make a decision: does this drug at the cost really provide any additional value?" said Sarah Marche, PharmD, vice president of pharmacy services at Highmark, a Blue Cross affiliate in Pennsylvania.
Translation: We already limit access to life saving medicines for the most chronically and seriously ill seniors based on our current value assessment. But we want even more control over what patients get. And by ‘additional value’ we mean increasing the 35-40 percent price discounts we get and what consumers are charged. We want more.
Marche noted that not every drug Medicare covers adds value.
"Glumetza [a branded version of metformin] provides no value above and beyond what's available, and if you look at a lot of the commercial formularies, it's blocked," she said. "Nobody's dying. Nobody has any huge issues with that, but we don't have that same flexibility within the Medicare space. So, you're covering a drug that really provides no additional value, just increasing the cost."
Translation: After Valeant got hammered for hiking Glumetza by more than 800% in 2015, the list price of stood at $10,020 for 90 tablets, up from $896 in January 2013,. For the longest time, insurers didn’t complain . Half of Glumetza’s price is given back in discounts, rebates, chargebacks, and the like to wholesalers, managed care organizations, pharmacy benefit managers, federal and state healthcare programs, and others.
Under pressure to reduce prices, Valeant gave Walgreens’s an exclusive deal to sell Glumetza as generic prices. So Express Scripts, the company running our drug benefit decided it could turned around an blocked Glumetza and cutting a deal with a company making a generic version of the drug to make up for the lost rebate revenue. So we would have covered it if had provided additional profit… I mean value to us regardless of cost. It really depends on the rebates Express Scripts can share with us.
Another panelist, Mark Owen, MBA, president of government programs for the the Blue Cross-affiliated Health Care Service Corp., power “to limit beneficiary access to specialty pharmacies.”
While Owen noted that critics of the idea have said such a change could pose access challenges, he argued that allowing plans to direct beneficiaries towards only certain pharmacies could lower costs and increase adherence.
Translation: Limiting access allows us to pocket more money because it ensures patients have no choice but to take the drugs that we give them. That way we make the biggest profit margin whether they like it or not. And we know that charging patients about 40 percent of the retail price of products is a good way to force them to use drugs what give us the biggest margin. We will work really hard to increase adherence, but only with drugs that fatten our bottom line regardless of whether the drug works for that patient or not.
According to Medpage: “Congress and the administration can help rein in rising drug costs through a few key regulatory and legal fixes, said insurers and pharmacists at AHIP's National Conference on Medicare, Medicaid and Duals on Tuesday.”
"I think plans need to be given a little bit more leeway to truly assess the value of drugs to make a decision: does this drug at the cost really provide any additional value?" said Sarah Marche, PharmD, vice president of pharmacy services at Highmark, a Blue Cross affiliate in Pennsylvania.
Translation: We already limit access to life saving medicines for the most chronically and seriously ill seniors based on our current value assessment. But we want even more control over what patients get. And by ‘additional value’ we mean increasing the 35-40 percent price discounts we get and what consumers are charged. We want more.
Marche noted that not every drug Medicare covers adds value.
"Glumetza [a branded version of metformin] provides no value above and beyond what's available, and if you look at a lot of the commercial formularies, it's blocked," she said. "Nobody's dying. Nobody has any huge issues with that, but we don't have that same flexibility within the Medicare space. So, you're covering a drug that really provides no additional value, just increasing the cost."
Translation: After Valeant got hammered for hiking Glumetza by more than 800% in 2015, the list price of stood at $10,020 for 90 tablets, up from $896 in January 2013,. For the longest time, insurers didn’t complain . Half of Glumetza’s price is given back in discounts, rebates, chargebacks, and the like to wholesalers, managed care organizations, pharmacy benefit managers, federal and state healthcare programs, and others.
Under pressure to reduce prices, Valeant gave Walgreens’s an exclusive deal to sell Glumetza as generic prices. So Express Scripts, the company running our drug benefit decided it could turned around an blocked Glumetza and cutting a deal with a company making a generic version of the drug to make up for the lost rebate revenue. So we would have covered it if had provided additional profit… I mean value to us regardless of cost. It really depends on the rebates Express Scripts can share with us.
Another panelist, Mark Owen, MBA, president of government programs for the the Blue Cross-affiliated Health Care Service Corp., power “to limit beneficiary access to specialty pharmacies.”
While Owen noted that critics of the idea have said such a change could pose access challenges, he argued that allowing plans to direct beneficiaries towards only certain pharmacies could lower costs and increase adherence.
Translation: Limiting access allows us to pocket more money because it ensures patients have no choice but to take the drugs that we give them. That way we make the biggest profit margin whether they like it or not. And we know that charging patients about 40 percent of the retail price of products is a good way to force them to use drugs what give us the biggest margin. We will work really hard to increase adherence, but only with drugs that fatten our bottom line regardless of whether the drug works for that patient or not.
From Medpage Today ...
FDA, Industry Focus on Abuse-Deterrent Opioids
No consensus immediately apparent after 2-day meeting
FDA officials and representatives from both the generic and branded drug industries spent two days hashing out next steps for development of abuse-deterrent opioids.
The two topics on the table: reviewing a draft guidance for the development of generic versions of abuse-deterrent opioids, and developing standard in vitro testing methods to characterize a drug's abuse-deterrent properties.
The meeting was in line with FDA's strategic plan for mitigating the opioid epidemic, which it released in February 2016, said Douglas Throckmorton, MD, deputy director of regulatory programs at the FDA's Center for Drug Evaluation and Research. That includes incentivizing the development of "progressively better" abuse-deterrent opioids and supporting a transition to these products, he said.
Throckmorton acknowledged the tension between generic and branded developers, but advocated working together to come up with a solution that benefits all parties.
"If you all came up with a single approach that suits the best product development, we would be delighted and take it very seriously," Throckmorton said. "It would move the field a great deal. It would require careful collaboration, but it's been successful in other fields, like drug-eluting stents, which had a similar challenge but the industry pulled together and made suggestions we all make use of."
FDA released its draft guidance for evaluating generic abuse-deterrent formulations last March, with the goal of ensuring that a generic is no less abuse-deterrent than the brand-name opioid. No generic abuse-deterrent opioids are currently approved.
In general, an ANDA applicant isn't required to provide independent evidence of the safety and effectiveness of the generic drug; instead it relies on the FDA finding that the previously approved product is safe and effective and must demonstrate that the generic is "bioequivalent" to the reference drug, primarily on the basis of pharmacokinetic studies.
Robert Lionberger, PhD, director of the office of research and standards in the FDA CDER's office of generic drugs, noted that the ANDA for any generic abuse-deterrent opioid would cover all routes of abuse: oral, nasal, injected, and smoked. It would also evaluate the drug in comparative in vitro studies and, in some cases, in relevant pharmacokinetic or other studies to show it is no less abuse-deterrent.
But some industry voices -- particularly from the branded industry -- said these assessments did not go far enough.
"I am concerned that this draft guidance is not sufficient to ensure that generic versions will be no less abusable than the reference drugs," said Alexander Kraus, PhD, of Grunenthal USA in Morristown, N.J. "It does not do enough to ensure that the generic meets therapeutic equivalence. It can't be based solely on in vitro testing. I encourage the FDA to require not only Category I in vitro tests, but Category II pharmacokinetic and Category III human reference studies as well."
Those Category I in vitro tests for abuse-deterrent opioids have their own issues to be worked out, which was why they were also a focus of the meeting.
Xiaoming Xu, PhD, a senior staff fellow at the FDA's division of product quality, said in vitro testing for abuse-deterrent opioids has not been standardized, making both comparisons with other drugs and overall assessments challenging.
Ideally, that testing should assess each potential route of abuse -- oral, injectable, nasal, and smoking -- starting with simple and gentle mechanical and chemical manipulations, progressing to complex and more destructive manipulations. It should also address mechanisms by which abusers can be expected to attempt to overcome abuse-deterrent properties as well as the ways that patients may alter the formulation to change the amount of of drug that gets released, Xu said.
The challenge is that the design of these experiments is complex: any single product can have more than a dozen possible methods to achieve a desired manipulation; scores of different solvents; different reactions to various temperature conditions; different volumes released, and so on. Indeed, the number of experiments can run into the thousands, Xu added.
The problem garnered unique observations about the automobile industry and coffee grinders. Richard Lostritto, PhD, acting associate director in the office of policy for pharmaceutical quality at FDA's CDER, noted that the auto industry standardized the type of hammer it would use in safety tests; Karsten Lindhardt, PhD, of Egalet Corporation, noted that he's had to buy several different types of coffee grinders to do proper in vitro testing.
"You end up with each material behaving differently," he said. "One grinder may be optimal for one drug but not for another."
Throckmorton acknowledged the tension between developing standardized versus individual tests for these types of analyses: "We've seen that even small changes in some formulations can have a large effect on product performance."
Although it was not a point of the meeting, critics at past FDA advisory committee meetings for abuse-deterrent opioids have called for epidemiologic data on whether or not these formulations can actually reduce abuse and the more serious consequences of addiction, overdose, and death.
Also, the meeting did not address patent issues, which generally fall outside FDA's jurisdiction. Although the major opioid drug compounds are now off-patent, it may be many years before patents expire on the specific abuse-deterrence technologies now in use.
FDA, Industry Focus on Abuse-Deterrent Opioids
No consensus immediately apparent after 2-day meeting
FDA officials and representatives from both the generic and branded drug industries spent two days hashing out next steps for development of abuse-deterrent opioids.
The two topics on the table: reviewing a draft guidance for the development of generic versions of abuse-deterrent opioids, and developing standard in vitro testing methods to characterize a drug's abuse-deterrent properties.
The meeting was in line with FDA's strategic plan for mitigating the opioid epidemic, which it released in February 2016, said Douglas Throckmorton, MD, deputy director of regulatory programs at the FDA's Center for Drug Evaluation and Research. That includes incentivizing the development of "progressively better" abuse-deterrent opioids and supporting a transition to these products, he said.
Throckmorton acknowledged the tension between generic and branded developers, but advocated working together to come up with a solution that benefits all parties.
"If you all came up with a single approach that suits the best product development, we would be delighted and take it very seriously," Throckmorton said. "It would move the field a great deal. It would require careful collaboration, but it's been successful in other fields, like drug-eluting stents, which had a similar challenge but the industry pulled together and made suggestions we all make use of."
FDA released its draft guidance for evaluating generic abuse-deterrent formulations last March, with the goal of ensuring that a generic is no less abuse-deterrent than the brand-name opioid. No generic abuse-deterrent opioids are currently approved.
In general, an ANDA applicant isn't required to provide independent evidence of the safety and effectiveness of the generic drug; instead it relies on the FDA finding that the previously approved product is safe and effective and must demonstrate that the generic is "bioequivalent" to the reference drug, primarily on the basis of pharmacokinetic studies.
Robert Lionberger, PhD, director of the office of research and standards in the FDA CDER's office of generic drugs, noted that the ANDA for any generic abuse-deterrent opioid would cover all routes of abuse: oral, nasal, injected, and smoked. It would also evaluate the drug in comparative in vitro studies and, in some cases, in relevant pharmacokinetic or other studies to show it is no less abuse-deterrent.
But some industry voices -- particularly from the branded industry -- said these assessments did not go far enough.
"I am concerned that this draft guidance is not sufficient to ensure that generic versions will be no less abusable than the reference drugs," said Alexander Kraus, PhD, of Grunenthal USA in Morristown, N.J. "It does not do enough to ensure that the generic meets therapeutic equivalence. It can't be based solely on in vitro testing. I encourage the FDA to require not only Category I in vitro tests, but Category II pharmacokinetic and Category III human reference studies as well."
Those Category I in vitro tests for abuse-deterrent opioids have their own issues to be worked out, which was why they were also a focus of the meeting.
Xiaoming Xu, PhD, a senior staff fellow at the FDA's division of product quality, said in vitro testing for abuse-deterrent opioids has not been standardized, making both comparisons with other drugs and overall assessments challenging.
Ideally, that testing should assess each potential route of abuse -- oral, injectable, nasal, and smoking -- starting with simple and gentle mechanical and chemical manipulations, progressing to complex and more destructive manipulations. It should also address mechanisms by which abusers can be expected to attempt to overcome abuse-deterrent properties as well as the ways that patients may alter the formulation to change the amount of of drug that gets released, Xu said.
The challenge is that the design of these experiments is complex: any single product can have more than a dozen possible methods to achieve a desired manipulation; scores of different solvents; different reactions to various temperature conditions; different volumes released, and so on. Indeed, the number of experiments can run into the thousands, Xu added.
The problem garnered unique observations about the automobile industry and coffee grinders. Richard Lostritto, PhD, acting associate director in the office of policy for pharmaceutical quality at FDA's CDER, noted that the auto industry standardized the type of hammer it would use in safety tests; Karsten Lindhardt, PhD, of Egalet Corporation, noted that he's had to buy several different types of coffee grinders to do proper in vitro testing.
"You end up with each material behaving differently," he said. "One grinder may be optimal for one drug but not for another."
Throckmorton acknowledged the tension between developing standardized versus individual tests for these types of analyses: "We've seen that even small changes in some formulations can have a large effect on product performance."
Although it was not a point of the meeting, critics at past FDA advisory committee meetings for abuse-deterrent opioids have called for epidemiologic data on whether or not these formulations can actually reduce abuse and the more serious consequences of addiction, overdose, and death.
Also, the meeting did not address patent issues, which generally fall outside FDA's jurisdiction. Although the major opioid drug compounds are now off-patent, it may be many years before patents expire on the specific abuse-deterrence technologies now in use.
The November issue of Lancet Oncology focuses on pharmacovigilance and drug safety. The lead editorial drives home some key points:
At a time when the number of biological agents due to come off patent is increasing, and in the face of a market fuelled by escalating drug prices and pressure from pharmaceutical companies and patient groups alike for expedited drug approval, issues surrounding the safety and efficacy of agents such as biosimilars and generics are paramount.
Substantial variation exists between high-income and low-to-middle-income countries with regards to manufacturing and supply chain regulation of generic drugs, despite countries such as India providing a large market share of generic drugs worldwide. Moreover, bioequivalence—a key consideration when comparing generic formulations with their trademarked counterparts—can vary substantially, making appropriate regulation particularly important. Issues of safety and regulation are further compounded when approving biosimilars: despite nearly 10 years’ of experience in dealing with biosimilar agents, regulators still need to streamline and expedite approval processes, and improve ways of reducing cost. Thus, taken together, the importance of pharmacovigilance has never been greater.
Given that multiple health-care systems encourage or enforce generic and biosimilar prescribing, sometimes without physician knowledge or consent, coupled with further potential complications created by generic or biosimilar switching during a course of treatment, pharmacovigilance needs to evolve beyond merely the uncovering, monitoring, and reporting of adverse events, to continual pre-marketing and post-marketing surveillance.
Although a focus on regulatory and policy issues is key to monitoring safety and efficacy, especially for newer agents, there are other ways in which drug safety can be improved.
The November issue also includes my new paper, 21st century pharmacovigilance: efforts, roles, and responsibilities.
Here’s the abstract:
In an era when the number of expedited and conditional review pathways for newly available brand-name drugs and biosimilar medicines to treat serious and life-threatening diseases is increasing, defining pharmacovigilance has never been more crucial. 21st century pharmacovigilance is not merely about uncovering, reporting, and addressing adverse events associated with already approved and marketed agents, but can be described as the systematic monitoring of the process of pre-market review and post-market surveillance, which includes the use of medicines in everyday practice. Pharmacovigilance identifies previously unrecognised adverse events or changes in the patterns of these effects, the quality and adequacy of drug supply, and should ensure effective communication with the public, health-care professionals, and patients about the optimum safety and effective use of medicines. In this paper, the first in a Series of three about drug safety in oncology, we discuss evolving challenges in the purview, roles, and responsibilities of the US Food and Drug Administration and the European Medicines Agency with respect to pharmacovigilance efforts, with a special emphasis on oncology treatment.
If you’d like a copy of this article, please contact me at ppitts@cmpi.org.
At a time when the number of biological agents due to come off patent is increasing, and in the face of a market fuelled by escalating drug prices and pressure from pharmaceutical companies and patient groups alike for expedited drug approval, issues surrounding the safety and efficacy of agents such as biosimilars and generics are paramount.
Substantial variation exists between high-income and low-to-middle-income countries with regards to manufacturing and supply chain regulation of generic drugs, despite countries such as India providing a large market share of generic drugs worldwide. Moreover, bioequivalence—a key consideration when comparing generic formulations with their trademarked counterparts—can vary substantially, making appropriate regulation particularly important. Issues of safety and regulation are further compounded when approving biosimilars: despite nearly 10 years’ of experience in dealing with biosimilar agents, regulators still need to streamline and expedite approval processes, and improve ways of reducing cost. Thus, taken together, the importance of pharmacovigilance has never been greater.
Given that multiple health-care systems encourage or enforce generic and biosimilar prescribing, sometimes without physician knowledge or consent, coupled with further potential complications created by generic or biosimilar switching during a course of treatment, pharmacovigilance needs to evolve beyond merely the uncovering, monitoring, and reporting of adverse events, to continual pre-marketing and post-marketing surveillance.
Although a focus on regulatory and policy issues is key to monitoring safety and efficacy, especially for newer agents, there are other ways in which drug safety can be improved.
The November issue also includes my new paper, 21st century pharmacovigilance: efforts, roles, and responsibilities.
Here’s the abstract:
In an era when the number of expedited and conditional review pathways for newly available brand-name drugs and biosimilar medicines to treat serious and life-threatening diseases is increasing, defining pharmacovigilance has never been more crucial. 21st century pharmacovigilance is not merely about uncovering, reporting, and addressing adverse events associated with already approved and marketed agents, but can be described as the systematic monitoring of the process of pre-market review and post-market surveillance, which includes the use of medicines in everyday practice. Pharmacovigilance identifies previously unrecognised adverse events or changes in the patterns of these effects, the quality and adequacy of drug supply, and should ensure effective communication with the public, health-care professionals, and patients about the optimum safety and effective use of medicines. In this paper, the first in a Series of three about drug safety in oncology, we discuss evolving challenges in the purview, roles, and responsibilities of the US Food and Drug Administration and the European Medicines Agency with respect to pharmacovigilance efforts, with a special emphasis on oncology treatment.
If you’d like a copy of this article, please contact me at ppitts@cmpi.org.
ICER released a report claiming that at retail prices, immunotherapies (Tecentrig, Keytruda and Opdivo) for non small cell lung cancer (after a first line of treatment) are not worth using compared to a 20 year old drug (docetaxel) that is 3-4 times less likely to help patients and when it does work, is half as effective in extending life and generating a treatment response. The table below shows the significant clinical benefits of new drugs that block PDL-1 and PD-1 protein expression,
Source: Institute for Clinical and Economic Review, Evidence Report - Non-Small Cell Lung Cancer, Table 6 page 36 and Table 10 page 45
ICER determined that the maximum amount of money to be spent on an additional year of life in perfect health -- a quality adjusted life year (QALY) is $150K. Which means that most of the new drugs developed and to extend the life of lung cancer patients over the past ten years would not be used.
Applying the ICER QALY calculations to lung cancer patients over the past ten years, I found that if ICER had its way 10 years ago, and denied access to new lung cancer drugs, today the death rate from lung cancer would be 23 percent higher. And the cost of care will be more expensive, increasing hospital related costs by $3 billion a year.
ICER ignores indirect costs, such as lost productivity and caregiver salaries or the productive possibilities of being able to make plans to marry, raise kids, compose music, travel, go to school. ICER ignores the value of two additional years of life to a patient who has run out of options.
But the worst element of the ICER framework is how it devalues the additional months and years of life people with advanced lung cancer can get from newer medicines.
At face value ICER’s QALY standard of value is arbitrary. Patients who live twice as long as those taking a generic drug should be credited with the total years of extended life. ICER believes that an additional year of life for a lung cancer patient is really worth two-thirds of an additional year of life for a healthy person.
Where has this metric been used before? Article 1, Section 2, Paragraph 3 of the United States Constitution, which allowed slave states and their collective slave drivers to count each of their slaves as 3/5 of a white man.
To mind the use of a QALY in determining access and drug prices violates the spirit, if not the letter of the Equal Protection Clause.
Worse (perhaps)ICER uses that same discriminatory rationale by assigning a lower value to patients and a higher value to insurers. When it comes to patients, ICER uses the retail price of the drug to determine a QALY When it comes to payors, ICER uses the price or cost of the drug LESS any cost savings generated.
ICER is the worst example of how economic analysis has been applied to health care. In the end, it is not about numbers. It is about people and the additional years and vitality medical innovation generates. ICER is not alone in failing to factor in the economic value of hope or the virtue of forward looking, of having a project.
Modern medicines are extending lives, improving lives and saving lives. Setting an arbitrary limit on what that is worth is simply closing the door on medical progress, increasing the death rate and driving up costs at the same time. And it also forecloses the possibilities of life that, while intangible or uncounted, adds more to our well-being than consumption. As the great economist Irving Fisher noted: The true “wealth of nations” is the health of its individuals. And greater health through the diffusion of medical innovation is the most important way to eliminate inequality and eliminate barriers to people battling disease. ICER's value framework diminishes the value of longer life as part of an effort to show most new drugs are NOT cost effective. In doing so, it is violating the civil rights of people because they have lung cancer.
| Drug | Ratio of Patients Likely to Benefit from Immunotherapy Compared to Docetaxel | Expected Survival in Months | Differnce in Duration of Response in Months |
|---|---|---|---|
| Docetaxel | 8.5 | ||
| Acentriq | 3.4 | 14.8 | 7.2 |
| Opdivo | 4.0 | 11.1 | 11.6 |
| Keytruda | 3.8 | 19.4 | Not reached |
ICER determined that the maximum amount of money to be spent on an additional year of life in perfect health -- a quality adjusted life year (QALY) is $150K. Which means that most of the new drugs developed and to extend the life of lung cancer patients over the past ten years would not be used.
Applying the ICER QALY calculations to lung cancer patients over the past ten years, I found that if ICER had its way 10 years ago, and denied access to new lung cancer drugs, today the death rate from lung cancer would be 23 percent higher. And the cost of care will be more expensive, increasing hospital related costs by $3 billion a year.
ICER ignores indirect costs, such as lost productivity and caregiver salaries or the productive possibilities of being able to make plans to marry, raise kids, compose music, travel, go to school. ICER ignores the value of two additional years of life to a patient who has run out of options.
But the worst element of the ICER framework is how it devalues the additional months and years of life people with advanced lung cancer can get from newer medicines.
At face value ICER’s QALY standard of value is arbitrary. Patients who live twice as long as those taking a generic drug should be credited with the total years of extended life. ICER believes that an additional year of life for a lung cancer patient is really worth two-thirds of an additional year of life for a healthy person.
Where has this metric been used before? Article 1, Section 2, Paragraph 3 of the United States Constitution, which allowed slave states and their collective slave drivers to count each of their slaves as 3/5 of a white man.
To mind the use of a QALY in determining access and drug prices violates the spirit, if not the letter of the Equal Protection Clause.
Worse (perhaps)ICER uses that same discriminatory rationale by assigning a lower value to patients and a higher value to insurers. When it comes to patients, ICER uses the retail price of the drug to determine a QALY When it comes to payors, ICER uses the price or cost of the drug LESS any cost savings generated.
| Immunotherapy | Payer Cost per patient net of drug generated savings |
Cost used for patient QALY | |
|---|---|---|---|
| Tencentriq | 39000 | 102519 | |
| Keytruda | 73900 | 108841 | |
| Opdivo | 44500 | 180489 |
ICER is the worst example of how economic analysis has been applied to health care. In the end, it is not about numbers. It is about people and the additional years and vitality medical innovation generates. ICER is not alone in failing to factor in the economic value of hope or the virtue of forward looking, of having a project.
Modern medicines are extending lives, improving lives and saving lives. Setting an arbitrary limit on what that is worth is simply closing the door on medical progress, increasing the death rate and driving up costs at the same time. And it also forecloses the possibilities of life that, while intangible or uncounted, adds more to our well-being than consumption. As the great economist Irving Fisher noted: The true “wealth of nations” is the health of its individuals. And greater health through the diffusion of medical innovation is the most important way to eliminate inequality and eliminate barriers to people battling disease. ICER's value framework diminishes the value of longer life as part of an effort to show most new drugs are NOT cost effective. In doing so, it is violating the civil rights of people because they have lung cancer.
In a recent tweet, Senator Bernie Sanders made it clear he’s upset about high drug prices. Less clear is that his righteous wrath will put patients in harm’s way. He proved yet again he’s uninformed about the facts and unconcerned about the unintended consequences of his actions.
The target of Senator Sanders terrible tweet was a small innovative pharmaceutical company (Ariad Pharmaceuticals) and its innovative drug Iclusig (for chronic myeloid leukemia treatment). The company raised the price – and Senator Sanders can’t see why. He should open his eyes.
A few facts that are worth sharing. The first is that Iclusig serves a population of approximately 1,000 to 2,000 patients. And these patients have limited options. Ariad Pharmaceuticals, works to ensures that no patient is prevented from treatment due to price. (They provide a robust support program for patients who have accessibility and affordability concerns.)
Second, these types of ultra-orphan disease cancer patient population programs require large investments and face tremendous odds. Ariad sure doesn’t look profitable. They’ve invested more than $1.3 billion in R&D and accumulated losses of approximately $1.4 billion since the company was founded. But they’re betting on innovation. In 2015, Ariad generated $119 million in total revenue and invested $171 million, or 143% of revenue, in R&D alone.
Third and most importantly, Iclusig works. It significantly increases 10-year survival rates for patients with no other hope or option except for expensive stem cell transplants and draconian radiation treatments.
Attention Senator Sanders. It’s not just a question of an ecosystem-driven price. It’s also about investment and value – precisely the rationale behind the Orphan Drug Act -- to encourage investment in treatments for small and in this case, tiny, populations. “Facts,” as John Adams quipped, “are pesky things.
The target of Senator Sanders terrible tweet was a small innovative pharmaceutical company (Ariad Pharmaceuticals) and its innovative drug Iclusig (for chronic myeloid leukemia treatment). The company raised the price – and Senator Sanders can’t see why. He should open his eyes.
A few facts that are worth sharing. The first is that Iclusig serves a population of approximately 1,000 to 2,000 patients. And these patients have limited options. Ariad Pharmaceuticals, works to ensures that no patient is prevented from treatment due to price. (They provide a robust support program for patients who have accessibility and affordability concerns.)
Second, these types of ultra-orphan disease cancer patient population programs require large investments and face tremendous odds. Ariad sure doesn’t look profitable. They’ve invested more than $1.3 billion in R&D and accumulated losses of approximately $1.4 billion since the company was founded. But they’re betting on innovation. In 2015, Ariad generated $119 million in total revenue and invested $171 million, or 143% of revenue, in R&D alone.
Third and most importantly, Iclusig works. It significantly increases 10-year survival rates for patients with no other hope or option except for expensive stem cell transplants and draconian radiation treatments.
Attention Senator Sanders. It’s not just a question of an ecosystem-driven price. It’s also about investment and value – precisely the rationale behind the Orphan Drug Act -- to encourage investment in treatments for small and in this case, tiny, populations. “Facts,” as John Adams quipped, “are pesky things.
As seen in the Daily Caller
Fact Checking Hillary Clinton On Drug Pricing
Both Donald Trump and Hillary Clinton get a lot of flak for lying. But in Secretary Clinton’s latest speech on healthcare reform, she didn’t lie — she just got all her facts wrong.
Clinton used her speech to demonize pharmaceutical companies. She argued that greedy firms are gouging consumers and that government-imposed price controls are needed to protect us from them. Her rhetoric ignores reality and her proposals would harm the patients she wants to help.
According to Mrs. Clinton, Americans are “paying the highest price” for medicines, compared to citizens of other developed nations. She implied that drugcompanies are overcharging Americans just because they can. It’s not so simple.
The real reason medicines are more expensive in the United States is that the socialized medicine systems in other countries cap prices on innovator drugs, while also rationing their use. Many foreign governments threaten to break ‘drug patents if firms don’t agree to sell their products at below-market rates. As a result, American consumers shoulder a disproportionate share of the world’s research and development burden. That’s “free-riderism” and it’s not fair. It’s also important to note that generic drugs, which account for 85 percent by volume of all the medicines used in the United States, are cheaper here than in Europe or Canada.
But the answer isn’t to impose our own price caps. That would only discourage research into new medicines. Here’s a fact that Clinton didn’t mention — America invents more than half of new medicines in the world. Stifling U.S. research would lead to vastly fewer medicines here and across the globe. It’d be smarter economically, and better for patients, to negotiate stronger trade protections to prevent other nations from freeloading off American investments.
Secretary Clinton told her audience that their tax dollars fund drug safety evaluations. They do not. All of the complex and costly clinical trials that must be done to bring a new medicine to market are fielded and funded — 100 percent — by the pharmaceutical industry. They are then reviewed by the Food and Drug Administration. And industry pays for that privilege through “user fees” the FDA collects from pharmaceutical companies.
She also slammed the Medicare Part D drug benefit, touting a doctor’s claim that he “can’t prescribe certain drugs that [his] patients need” because government health programs won’t pay for them. But when it comes to Medicare, that assertion is simply false. Medicare’s prescription drug plans cover, on average, 191 of the 200 medicines most used by seniors. That’s more than most Obamacare exchange options.
In her assault on capitalism and private enterprise, Secretary Clinton singled out the price of hepatitis C drugs. She claimed that makers of these cures — which are vastly more effective than previous therapies — are gouging Americans.
The truth is radically different – and highly documented. Hepatitis C drugs are now cheaper in the United States than in Western Europe, thanks to a price war between competing manufacturers. Clinton inadvertently picked an example that proves the free market yields better, cheaper medicines than socialist systems and fix prices and ration care.
Bashing the companies that research and produce the world’s most groundbreaking medicines might give Clinton a bump in the polls. But her reality-free rhetoric has dangerous consequences.
Clinton’s attacks on drug makers have prompted a sell-off of biotech stocks multiple times over the past year. If companies can’t raise funding from investors, they’ll have to limit new research projects. That means fewer drugs down the road. What’s political expediency worth?
And if Clinton reaches the White House and actually implements price controls, it’s statistically certain Americans will lose out on lifesaving drugs. Price controls in other countries depress research spending by up to $8 billion each year — the equivalent of three or four new drugs, according to a Department of Commerce study.
For a super policy wonk, Secretary Clinton got an awful lot wrong in her recent speech. If she really wants to help patients, perhaps she should pay a little less attention to her focus groups and a little more attention to the facts.
Peter J. Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest.
Fact Checking Hillary Clinton On Drug Pricing
Both Donald Trump and Hillary Clinton get a lot of flak for lying. But in Secretary Clinton’s latest speech on healthcare reform, she didn’t lie — she just got all her facts wrong.
Clinton used her speech to demonize pharmaceutical companies. She argued that greedy firms are gouging consumers and that government-imposed price controls are needed to protect us from them. Her rhetoric ignores reality and her proposals would harm the patients she wants to help.
According to Mrs. Clinton, Americans are “paying the highest price” for medicines, compared to citizens of other developed nations. She implied that drugcompanies are overcharging Americans just because they can. It’s not so simple.
The real reason medicines are more expensive in the United States is that the socialized medicine systems in other countries cap prices on innovator drugs, while also rationing their use. Many foreign governments threaten to break ‘drug patents if firms don’t agree to sell their products at below-market rates. As a result, American consumers shoulder a disproportionate share of the world’s research and development burden. That’s “free-riderism” and it’s not fair. It’s also important to note that generic drugs, which account for 85 percent by volume of all the medicines used in the United States, are cheaper here than in Europe or Canada.
But the answer isn’t to impose our own price caps. That would only discourage research into new medicines. Here’s a fact that Clinton didn’t mention — America invents more than half of new medicines in the world. Stifling U.S. research would lead to vastly fewer medicines here and across the globe. It’d be smarter economically, and better for patients, to negotiate stronger trade protections to prevent other nations from freeloading off American investments.
Secretary Clinton told her audience that their tax dollars fund drug safety evaluations. They do not. All of the complex and costly clinical trials that must be done to bring a new medicine to market are fielded and funded — 100 percent — by the pharmaceutical industry. They are then reviewed by the Food and Drug Administration. And industry pays for that privilege through “user fees” the FDA collects from pharmaceutical companies.
She also slammed the Medicare Part D drug benefit, touting a doctor’s claim that he “can’t prescribe certain drugs that [his] patients need” because government health programs won’t pay for them. But when it comes to Medicare, that assertion is simply false. Medicare’s prescription drug plans cover, on average, 191 of the 200 medicines most used by seniors. That’s more than most Obamacare exchange options.
In her assault on capitalism and private enterprise, Secretary Clinton singled out the price of hepatitis C drugs. She claimed that makers of these cures — which are vastly more effective than previous therapies — are gouging Americans.
The truth is radically different – and highly documented. Hepatitis C drugs are now cheaper in the United States than in Western Europe, thanks to a price war between competing manufacturers. Clinton inadvertently picked an example that proves the free market yields better, cheaper medicines than socialist systems and fix prices and ration care.
Bashing the companies that research and produce the world’s most groundbreaking medicines might give Clinton a bump in the polls. But her reality-free rhetoric has dangerous consequences.
Clinton’s attacks on drug makers have prompted a sell-off of biotech stocks multiple times over the past year. If companies can’t raise funding from investors, they’ll have to limit new research projects. That means fewer drugs down the road. What’s political expediency worth?
And if Clinton reaches the White House and actually implements price controls, it’s statistically certain Americans will lose out on lifesaving drugs. Price controls in other countries depress research spending by up to $8 billion each year — the equivalent of three or four new drugs, according to a Department of Commerce study.
For a super policy wonk, Secretary Clinton got an awful lot wrong in her recent speech. If she really wants to help patients, perhaps she should pay a little less attention to her focus groups and a little more attention to the facts.
Peter J. Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest.
The Wikileaks disclosure of John Podesta's emails reveals that the Clinton campaign considered attacking FDA Commissioner Robert Califf for ties to drug companies as part of a broader effort to divert attention from Secretary Clinton's email scandal.
FDA Commissioner and Clinton Campaign Target Robert Califf, MD
It start with an email from long-time Clinton adviser Mandy Grunwald to Clinton policy adviser Ann O Leary. The email contains a link to a NY Times article on acting FDA Commissioner Rob Califf who had been nominated by President Obama to become the full time commish.
On Sep 21, 2015, at 10:33 PM, Mandy Grunwald sent an email to Clintion policy advierse Jake Sullivan and Ann O'Leary, Joel Benenson a strategist and pollster for the presidential campaign., press secretary Brian Fallon and Communications Director Jennifer Palmieri and asks: Do we want to weigh in on this?
Jake Sullivan responds: What do you think?
On Mon, Sep 21, 2015 Mandy Grunwald replies: I don't know anything about the guy. If we weren't hitting the Administration with Keystone this week, I might be tempted, but I think that probably makes it a bad idea. Lets keep an eye on it and see about those Pharma ties.
Then Anne O Leary: Interesting. I'll do some asking around to see what folks in the public health world think.
On Sat, Sep 26, 2015 at 12:53 PM, Brian Fallon : Any update on this? As we consider fights that fit into the larger themes we are trying to promote, this seems like a good fight to have. Plus, the VP would be in a box of having to support this nominee.
On Sat, Sep 26, 2015 at 12:59 PM, Jake Sullivan wrote: This is ultimately a political call, not a policy call. I don't really like the idea of bashing this White House's nominees, knowing their vetting process and standards. But if you guys want to do it, I cannot identify a policy reason not to -- you all know the facts. Ann, any other intel?
On September 26, 2015 at 1:13 pm Anne O'Leary wrote:
Califf the Obama nominee does have real ties to the drug industry - Chris Jennings is calling a few people for me to learn more so we don't tip our hand directly. We are clean on Clinton Admin FDA Commissioner - it was David Kessler, an academic who had run a teaching hospital - and best known for taking on big tobacco. We could certainly signal that we want someone willing to stand up to Pharma (in the same way Kessler stood up to Tobacco). BUT - I want to do a little more digging and due diligence before we hit this guy. Having been through a nomination fight with my husband (in which he lost), this is personal and messy and horrible on the person nominated and their families - so I don't take attacking this guy lightly. Do you want to do it on Meet the Press?
That was follwed by an email from Podesta on Sep 26, 2015, at 2:27 PM, who wrote: " I think we will pay a huge price with the WH on this one. Worries me."
Nothing transpired. But the back and forth about whether to trash Califf came down to politics, not policy differences. And it had NOTHING to do with Califf as a person, a physician, a researcher and public servant. It was clear that Team Clinton was interested only in dirt that was newsworthy. It was simply a matter of whether attacking Califf on Meet The Press would be worth the headache of taking on an Obama nominee. How lovely.
Rob Califf should remain FDA commissioner regardless of who is elected president because of his qualifications and commitment to accelerating access to safe and effective medicines. I am concerned that the Clinton team -- which exults about going to war against Pharma -- will seek to replace Dr. Califf. That will trigger a prolonged political war that will undermine the FDA.
The emails reveal a Team Clinton eager to find ways to smash up people for short term political gain without tipping their hand that they are behind the hit job. Like it's war on pharma, it has less to do with policies and more to do about the benefits of creating and attacking enemies.
FDA Commissioner and Clinton Campaign Target Robert Califf, MD
It start with an email from long-time Clinton adviser Mandy Grunwald to Clinton policy adviser Ann O Leary. The email contains a link to a NY Times article on acting FDA Commissioner Rob Califf who had been nominated by President Obama to become the full time commish.
On Sep 21, 2015, at 10:33 PM, Mandy Grunwald sent an email to Clintion policy advierse Jake Sullivan and Ann O'Leary, Joel Benenson a strategist and pollster for the presidential campaign., press secretary Brian Fallon and Communications Director Jennifer Palmieri and asks: Do we want to weigh in on this?
Jake Sullivan responds: What do you think?
On Mon, Sep 21, 2015 Mandy Grunwald replies: I don't know anything about the guy. If we weren't hitting the Administration with Keystone this week, I might be tempted, but I think that probably makes it a bad idea. Lets keep an eye on it and see about those Pharma ties.
Then Anne O Leary: Interesting. I'll do some asking around to see what folks in the public health world think.
On Sat, Sep 26, 2015 at 12:53 PM, Brian Fallon : Any update on this? As we consider fights that fit into the larger themes we are trying to promote, this seems like a good fight to have. Plus, the VP would be in a box of having to support this nominee.
On Sat, Sep 26, 2015 at 12:59 PM, Jake Sullivan wrote: This is ultimately a political call, not a policy call. I don't really like the idea of bashing this White House's nominees, knowing their vetting process and standards. But if you guys want to do it, I cannot identify a policy reason not to -- you all know the facts. Ann, any other intel?
On September 26, 2015 at 1:13 pm Anne O'Leary wrote:
Califf the Obama nominee does have real ties to the drug industry - Chris Jennings is calling a few people for me to learn more so we don't tip our hand directly. We are clean on Clinton Admin FDA Commissioner - it was David Kessler, an academic who had run a teaching hospital - and best known for taking on big tobacco. We could certainly signal that we want someone willing to stand up to Pharma (in the same way Kessler stood up to Tobacco). BUT - I want to do a little more digging and due diligence before we hit this guy. Having been through a nomination fight with my husband (in which he lost), this is personal and messy and horrible on the person nominated and their families - so I don't take attacking this guy lightly. Do you want to do it on Meet the Press?
That was follwed by an email from Podesta on Sep 26, 2015, at 2:27 PM, who wrote: " I think we will pay a huge price with the WH on this one. Worries me."
Nothing transpired. But the back and forth about whether to trash Califf came down to politics, not policy differences. And it had NOTHING to do with Califf as a person, a physician, a researcher and public servant. It was clear that Team Clinton was interested only in dirt that was newsworthy. It was simply a matter of whether attacking Califf on Meet The Press would be worth the headache of taking on an Obama nominee. How lovely.
Rob Califf should remain FDA commissioner regardless of who is elected president because of his qualifications and commitment to accelerating access to safe and effective medicines. I am concerned that the Clinton team -- which exults about going to war against Pharma -- will seek to replace Dr. Califf. That will trigger a prolonged political war that will undermine the FDA.
The emails reveal a Team Clinton eager to find ways to smash up people for short term political gain without tipping their hand that they are behind the hit job. Like it's war on pharma, it has less to do with policies and more to do about the benefits of creating and attacking enemies.
Written by Rafael Fonseca MD and Robert Goldberg PhD
Two recent articles in the NEJM (1) e) and Annals of Internal Medicine (2) propose that restrictions or eliminations of co-pay assistance programs by pharmaceutical companies are needed since they distort the market and therefore, while few benefit, many suffer in the form of higher healthcare expenditures. They are factually correct but the conclusions point to a larger problem; the third party payer system in health care. When consumers (in this case patients) are detached from the price consequence of transactions they will be less careful in their selections. However, the proposal presented in these articles are, at least in the short-term, patient unfriendly and immediate implementation of their recommendations would limit, severely, access; particularly for patients with serious or life-threatening conditions. The Annals article is notorious in that it fails to mention the increased cost-sharing pressure that patients are facing from the payers.
So what are the solutions? Ideally, healthcare should be divided between the routine care and the catastrophic care. Routine care should involve much more “skin in the game.” In my view, regular care should not be covered under traditional heath insurance but be bought directly, with compassionate support tools for those in need. In this type of care I could envision more direct pressure in the selection of older medications for which many alternative exist. There are many options for anti-hypertensive medications, statins, etc. In the absence of a third party payer system the Epipen would never cost $600. This is where consumer pressure, with co-pay requirement as a discriminator, should be employed; vigorously, if you may. I would not disagree that if an equivalent generic exists, a true equivalent, then having the patient paying more for a branded product is fine. This is not to say that branded products and generics are always the same. Sometimes production quality may suffer or sometimes an alternative may not be the same as the original, similar and yet not the same product. But if you want the branded statin maybe you should pay a bit more. Who could argue with that?
However, for more expensive medications, medications used for serious, chronic severe or catastrophic illnesses the pressure seems misguided. For instance preventing co-pay assistance for patients with cancer seems inhumane. Dusetzina and colleagues have documented that lack of supplemental insurance delays initiation of treatment for CML (3). Should we disincentivize the use of medications that can be lifesaving for a cancer patient so they consider inferior treatments? Should we place additional burdens in someone who is at high risk for bankruptcy? Someone who cannot work or whose caregiver cannot work? That seems inappropriate. Furthermore, for many of these newer and expensive medications there are no suitable alternatives (4). Dr. Bach and colleagues were able to reduce the price of a competing monoclonal antibody, but that is a rarity in oncology and more of an exception. Should myeloma patients forgo lenalidomide in favor or thalidomide and face certain peripheral neuropathy? Or should a diabetic myeloma patient be forced to be treated with bortezomib instead of carfilzomib and again face neuropathy? To create a disincentive as this for people facing serious medical illness appears to me as a fundamental violation of medicine’s stance of benevolence. Rather this has the appeal of altruism, sacrifice the few for the benefit of the many.
Lastly, government regulations that eliminate this co-pay assistance are demonstrably patient unfriendly. Commercially insured patients can receive this directly and in another study by Dusetzina she shows that the average co-pay for specialty medications is only $35 (5). In another study it was shown that 90% of myeloma patients pay less than $100 per month for lenalidomide, a backbone for the treatment of this disease. In the meantime, Medicare beneficiaries struggle to find indirect support with funds provided by the pharmaceutical companies to third parties. I’ve written about this topic before, but again is a built-in disincentive to lessen the use of medications (6). What if these medications save dollars by preventing hospitalizations and decreasing expenses elsewhere? Once again it is pound foolish to only concentrate on the price of drugs.
References
1. Dafny LS, Ody CJ, Schmitt MA. Undermining Value-Based Purchasing - Lessons from the Pharmaceutical Industry. The New England journal of medicine. 2016. Epub 2016/10/13. doi: 10.1056/NEJMp1607378. PubMed PMID: 27732125.
2. Ubel PA, Bach PB. Copay Assistance for Expensive Drugs: A Helping Hand That Raises Costs. Annals of internal medicine. 2016. Epub 2016/10/11. doi: 10.7326/M16-1334. PubMed PMID: 27723893.
3. Winn A, Keating N, Dusetzina S. Factors Associated With Tyrosine Kinase Inhibitor Initiation and Adherence Among Medicare Beneficiaries With Chronic Myeloid Leukemia. Journal of Clinical Oncology. 2016;10.1200/JCO.2016.67.4184.
4. Fonseca R. 2016. Available from: https://tmblr.co/Z0_1wo2CgPnw1.
5. Dusetzina SB. Share Of Specialty Drugs In Commercial Plans Nearly Quadrupled, 2003-14. Health Aff (Millwood). 2016;35(7):1241-6. Epub 2016/07/08. doi: 10.1377/hlthaff.2015.1657. PubMed PMID: 27385240.
6. Fonseca R. 2016. Available from: https://tmblr.co/Z0_1wo2D7lHbj.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
