Latest Drugwonks' Blog
Yesterday, four former FDA commissioners warned that proposals to allow importation of pharmaceuticals would create serious risks for consumer and patients, and would produce only minimal savings.
While importation proposals are intended to "make lower-cost but genuine, safe and effective drugs available to U.S. consumers," in practice they would "harm patients and consumers and compromise the carefully constructed system that guards the safety of our nation’s medical products," the former commissioners wrote in an open letter to Congress. Robert Califf, Margaret Hamburg, Mark McClellan, and Andrew von Eschenbach signed the letter.
The former commissioners note that in exceptional circumstances, limited importation has been permitted, but wrote that when they were in office, none of them was "able to conclude that a wider policy of routine importation would increase access to safe and effective drugs for the American public."
The letter directly addresses legislation that seeks to assure the safety of imported drugs by limiting importation to drugs made in FDA-inspected plants. "Allowing importation of drugs purported to be manufactured overseas in FDA-inspected facilities and drugs purported to be manufactured domestically for export to other countries and re-imported from those countries to the United States can not meet the requirements under the existing closed drug manufacturing and distribution system because the drugs could not be tracked and certified by the manufacturer."
Sales of illicit, ineffective, or adulterated products are a lucrative business for organized crime, the letter warns. It would be practically impossible to screen and verify the authenticity of massive quantities of imports, the group wrote, adding that "if spot-checking discovered a dangerous or counterfeit product, in the absence of the closed system currently in use, there would be no way to trace that product to its origin or intervene to protect other consumers before irreparable harm occurs."
The letter also challenges assumptions about cost savings from importation, noting that drugs are allotted to countries based on the needs of their populations, leaving little extra inventory for export to the U.S. It states that importation "would likely have only a small, incremental effect on cost and access for drugs in the U.S. market; further, these small savings might not be passed on to patients, even if consumers are able to obtain a legitimate imported drug."
The letter concludes: "We urge Congress and the many others concerned about the cost of drugs to deal directly with the issues driving the cost of medicines and not to place false hope in measures that will place patients who need treatment at risk and jeopardize public health.
While importation proposals are intended to "make lower-cost but genuine, safe and effective drugs available to U.S. consumers," in practice they would "harm patients and consumers and compromise the carefully constructed system that guards the safety of our nation’s medical products," the former commissioners wrote in an open letter to Congress. Robert Califf, Margaret Hamburg, Mark McClellan, and Andrew von Eschenbach signed the letter.
The former commissioners note that in exceptional circumstances, limited importation has been permitted, but wrote that when they were in office, none of them was "able to conclude that a wider policy of routine importation would increase access to safe and effective drugs for the American public."
The letter directly addresses legislation that seeks to assure the safety of imported drugs by limiting importation to drugs made in FDA-inspected plants. "Allowing importation of drugs purported to be manufactured overseas in FDA-inspected facilities and drugs purported to be manufactured domestically for export to other countries and re-imported from those countries to the United States can not meet the requirements under the existing closed drug manufacturing and distribution system because the drugs could not be tracked and certified by the manufacturer."
Sales of illicit, ineffective, or adulterated products are a lucrative business for organized crime, the letter warns. It would be practically impossible to screen and verify the authenticity of massive quantities of imports, the group wrote, adding that "if spot-checking discovered a dangerous or counterfeit product, in the absence of the closed system currently in use, there would be no way to trace that product to its origin or intervene to protect other consumers before irreparable harm occurs."
The letter also challenges assumptions about cost savings from importation, noting that drugs are allotted to countries based on the needs of their populations, leaving little extra inventory for export to the U.S. It states that importation "would likely have only a small, incremental effect on cost and access for drugs in the U.S. market; further, these small savings might not be passed on to patients, even if consumers are able to obtain a legitimate imported drug."
The letter concludes: "We urge Congress and the many others concerned about the cost of drugs to deal directly with the issues driving the cost of medicines and not to place false hope in measures that will place patients who need treatment at risk and jeopardize public health.
Liz Szabo wrote what I predict will be the worst (as in most deceptive and inaccurate) article on prescription drug costs to be published by a so-called mainstream media outlet in 2017.
"As Drug Costs Soar, People Delay Or Skip Cancer Treatments" claims that high drug prices are causing cancer patients to go bankrupt. To make that point distorts and omit facts in ways that let PBMs and health plans off the hook for imposing high out of pocket drug costs on a small group of patients.
Szabo overstates the problem and then fails to support her claim:
"We're talking about huge numbers of patients," says Dr. Scott Ramsey, director of the Hutchinson Institute for Cancer Outcomes Research at the Fred Hutchinson Cancer Center in Seattle. "It's an epidemic. And it's not going away."
While a huge problem for anyone in that situation, the good news is that the percentage of patients that must pay thousands of out of pocket is very small. Nearly 95 percent of cancer patients are not exposed to high out of pocket cancer costs. The other 5 percent often have supplemental insurance or receive cost sharing assistance.
Szabo claims that between 168,000 to 405,000 ‘ration’ their prescription use because of cost. This a sloppy use of the word made even sloppier because Szabo defines rationing as delaying the use of cancer drugs. Moreover, the studies she relies on to generate her guesstimate looks only at the small group of patients that –because of a low income or lack of insurance coverage – cite cost as a barrier to care.
The burden of out of pocket cost is not a small matter to those who must bear it. But to address the problem you have to identify the cause. Szabo strenuously avoids doing so.
She insinuates that the most important factor is the price of medicines. In fact, it is extreme drug cost sharing imposed by PBMs and insurers on the sickest 1 percent of patients with cancer, MS, rheumatoid arthritis, cystic fibrosis and other rare diseases.
Further, PBMs and insurers systematically target people with these conditions particularly in Medicare Part D and commercial plans provided under the Affordable Care Act. In those plans, insurers and PBMs have placed most or all new cancer drugs on the highest cost-sharing tier of drug benefit programs.
Net drug prices have been declining so the excuse that cost sharing has to increasse in step with increasing prices won't wash.
A Kaiser study recently found that “Payments for deductibles and coinsurance have outpaced increases in costs paid by the health plans themselves. Average payments toward deductibles more than tripled, rising 256 percent, and average payments toward coinsurance more than doubled, rising 107 percent. This is while average payments by health plans themselves only increased 58 percent. “
The real reason for the cost sharing discrimination Szabo ignores is that is where the money is.
The one percent of patients and the 1 percent of the drugs that are dispensed to them generate about $150 billion in drug sales at list price. PBMs and insurers extract $30-40 billion from drug companies in the form of rebatres. These rebates reduce the actual cost of drugs. But those savings are not fully passed on to patients.
Instead, most of these drugs are placed in the highest cost sharing formulary tier and patients then pay up to 50 percent of the list -- not rebated -- price of drugs. On average, the cost sharing is about 35 percent of the retail price of drugs. That's another $45 billion insurers and PBMs collect. Generating $75 billion on sales of $150 billion is a nice haul. But Ms. Szabo seems to think it's not such a big deal.
Did I mention that this practice is systematic?
A recent study found that insurers are increasingly designing benefits to maximize profitability and turn patients into revenue centers. The analysis notes for example that patients being treated for cancer or multiple sclerosis will cost an average of $61,000 but only generate $47,000 in revenue after accounting for the large risk adjustment and reinsurance transfer payments. The difference is made up by squeezing more profits out of drugs.
The researchers said this creates a large incentive to place such drugs on specialty tiers, where patients face high out-of-pocket costs and where drugs generate the most rebates.
This is certainly what has happened to cancer drugs. Since 2011 net price increases have declined for cancer medicines. Yet list prices have increased. The difference between net and list prices goes to rebates pocketed by health plans and insurance companies even as they hike cost sharing.
Further, even as rebates increase, more health plans are putting all cancer drugs in higher cost-sharing tiers, generic or not:
As a recent American Cancer Society study concludes: “Plans continue to place most or all oral chemotherapy medications on the highest cost-sharing tier, creating transparency and cost barriers for patients. The two generic oral cancer drugs we studied regularly appeared on the most expensive tier (41 and 62 percent of the time). The effect may be to inappropriately discourage enrollment by cancer patients.”
This is both unfair and inefficient. the use of new medicines lower treatment costs over time as well as substantially improving health over time.
In some case, insurers have recognized the dynamic contribution new medicines make to health and health systems and have protected cancer patients from high-cost sharing. A study of coverage for targeted drugs treating chronic myeloid leukemia found that their use of “was associated with lower spending on other types of healthcare services. CML patients on such targeted drugs ..”had roughly $12,000 less in nonpharmaceutical medical costs than did patients on alternative forms of therapy. This translated into a decline of more than 30% in medical spending and offset roughly 40% of the cost of the drugs...This result is consistent with prior work that suggested changing generosity for one healthcare service has both short- and long-term implications for spending in other areas.”
Instead, Szabo ignores the qualitative improvement in new cancer drugs and advantage of eliminating cost barriers for the most innovative treatments:
Spending on cancer has remained about 4 percent of health care spending since 1960 because drugs have been displacing hospitalization, which is more expensive. For instance, if people were being hospitalized for cancer in 2014 at the same rate they were in 1995, total hospital costs would be $60 billion a year more.
Additionally, whereas there were only 4 million cancer survivors in 1975, there are 14 million survivors today. The cancer death rate for men and women combined fell 25% from its peak in 1991 to 2014.
New cancer medicines have reduced health care spending and increased well-being and capacity to work. So, my question is: if the use of new medications makes cancer care more effective and affordable, why are health plans and PBMs making them more expensive?
Maybe another reporter will write an article about that.
"As Drug Costs Soar, People Delay Or Skip Cancer Treatments" claims that high drug prices are causing cancer patients to go bankrupt. To make that point distorts and omit facts in ways that let PBMs and health plans off the hook for imposing high out of pocket drug costs on a small group of patients.
Szabo overstates the problem and then fails to support her claim:
"We're talking about huge numbers of patients," says Dr. Scott Ramsey, director of the Hutchinson Institute for Cancer Outcomes Research at the Fred Hutchinson Cancer Center in Seattle. "It's an epidemic. And it's not going away."
While a huge problem for anyone in that situation, the good news is that the percentage of patients that must pay thousands of out of pocket is very small. Nearly 95 percent of cancer patients are not exposed to high out of pocket cancer costs. The other 5 percent often have supplemental insurance or receive cost sharing assistance.
Szabo claims that between 168,000 to 405,000 ‘ration’ their prescription use because of cost. This a sloppy use of the word made even sloppier because Szabo defines rationing as delaying the use of cancer drugs. Moreover, the studies she relies on to generate her guesstimate looks only at the small group of patients that –because of a low income or lack of insurance coverage – cite cost as a barrier to care.
The burden of out of pocket cost is not a small matter to those who must bear it. But to address the problem you have to identify the cause. Szabo strenuously avoids doing so.
She insinuates that the most important factor is the price of medicines. In fact, it is extreme drug cost sharing imposed by PBMs and insurers on the sickest 1 percent of patients with cancer, MS, rheumatoid arthritis, cystic fibrosis and other rare diseases.
Further, PBMs and insurers systematically target people with these conditions particularly in Medicare Part D and commercial plans provided under the Affordable Care Act. In those plans, insurers and PBMs have placed most or all new cancer drugs on the highest cost-sharing tier of drug benefit programs.
Net drug prices have been declining so the excuse that cost sharing has to increasse in step with increasing prices won't wash.
A Kaiser study recently found that “Payments for deductibles and coinsurance have outpaced increases in costs paid by the health plans themselves. Average payments toward deductibles more than tripled, rising 256 percent, and average payments toward coinsurance more than doubled, rising 107 percent. This is while average payments by health plans themselves only increased 58 percent. “
The real reason for the cost sharing discrimination Szabo ignores is that is where the money is.
The one percent of patients and the 1 percent of the drugs that are dispensed to them generate about $150 billion in drug sales at list price. PBMs and insurers extract $30-40 billion from drug companies in the form of rebatres. These rebates reduce the actual cost of drugs. But those savings are not fully passed on to patients.
Instead, most of these drugs are placed in the highest cost sharing formulary tier and patients then pay up to 50 percent of the list -- not rebated -- price of drugs. On average, the cost sharing is about 35 percent of the retail price of drugs. That's another $45 billion insurers and PBMs collect. Generating $75 billion on sales of $150 billion is a nice haul. But Ms. Szabo seems to think it's not such a big deal.
Did I mention that this practice is systematic?
A recent study found that insurers are increasingly designing benefits to maximize profitability and turn patients into revenue centers. The analysis notes for example that patients being treated for cancer or multiple sclerosis will cost an average of $61,000 but only generate $47,000 in revenue after accounting for the large risk adjustment and reinsurance transfer payments. The difference is made up by squeezing more profits out of drugs.
The researchers said this creates a large incentive to place such drugs on specialty tiers, where patients face high out-of-pocket costs and where drugs generate the most rebates.
This is certainly what has happened to cancer drugs. Since 2011 net price increases have declined for cancer medicines. Yet list prices have increased. The difference between net and list prices goes to rebates pocketed by health plans and insurance companies even as they hike cost sharing.
Further, even as rebates increase, more health plans are putting all cancer drugs in higher cost-sharing tiers, generic or not:
As a recent American Cancer Society study concludes: “Plans continue to place most or all oral chemotherapy medications on the highest cost-sharing tier, creating transparency and cost barriers for patients. The two generic oral cancer drugs we studied regularly appeared on the most expensive tier (41 and 62 percent of the time). The effect may be to inappropriately discourage enrollment by cancer patients.”
This is both unfair and inefficient. the use of new medicines lower treatment costs over time as well as substantially improving health over time.
In some case, insurers have recognized the dynamic contribution new medicines make to health and health systems and have protected cancer patients from high-cost sharing. A study of coverage for targeted drugs treating chronic myeloid leukemia found that their use of “was associated with lower spending on other types of healthcare services. CML patients on such targeted drugs ..”had roughly $12,000 less in nonpharmaceutical medical costs than did patients on alternative forms of therapy. This translated into a decline of more than 30% in medical spending and offset roughly 40% of the cost of the drugs...This result is consistent with prior work that suggested changing generosity for one healthcare service has both short- and long-term implications for spending in other areas.”
Instead, Szabo ignores the qualitative improvement in new cancer drugs and advantage of eliminating cost barriers for the most innovative treatments:
Spending on cancer has remained about 4 percent of health care spending since 1960 because drugs have been displacing hospitalization, which is more expensive. For instance, if people were being hospitalized for cancer in 2014 at the same rate they were in 1995, total hospital costs would be $60 billion a year more.
Additionally, whereas there were only 4 million cancer survivors in 1975, there are 14 million survivors today. The cancer death rate for men and women combined fell 25% from its peak in 1991 to 2014.
New cancer medicines have reduced health care spending and increased well-being and capacity to work. So, my question is: if the use of new medications makes cancer care more effective and affordable, why are health plans and PBMs making them more expensive?
Maybe another reporter will write an article about that.
Politico 3-15-17: TODAY: WYDEN INTRODUCES BILL TARGETING PBMs — The ranking member of the Senate Finance Committee will roll out a proposal today to lower drug costs by targeting prescription benefit managers (PBMS) — middlemen who negotiate discounts on drugs with pharma companies and administer prescription drug coverage for health insurance companies.
According to an email obtained by POLITICO, the bill would mandate that patient co-pays or co-insurance for drugs in Medicare Part D are based on the negotiated price of the drug, not the higher list price. The bill also requires more transparency by mandating that PBMs publicly disclose aggregate rebates and the amount of those rebates passed on to health plans, as well as the difference between what a PBM pays a pharmacy for a drug and what the PBM charges a health plan for the drug.
According to an email obtained by POLITICO, the bill would mandate that patient co-pays or co-insurance for drugs in Medicare Part D are based on the negotiated price of the drug, not the higher list price. The bill also requires more transparency by mandating that PBMs publicly disclose aggregate rebates and the amount of those rebates passed on to health plans, as well as the difference between what a PBM pays a pharmacy for a drug and what the PBM charges a health plan for the drug.
Dr. Scott Gottlieb
I have worked with Scott Gottlieb for over a decade on making drug development swifter by making it more scientific and increasing patient access to medical innovations. Scott played an important role in developing the Critical Path Initiative that has been the FDA's roadmap for moving the agency -- and our health care system -- towards predictive, personalized and participatory medicine. None of the advances in expediting approval of important new medicines would have been possible in the absence of The Critical Path Initiative.
I could discuss his accomplishments in the post. But many others will write about Scott's ability and experience in support of his nomination to be FDA commissioner. A handful will attempt to demonize him for that very experience.
So let me tell you what I know about the human being who has been nominated to be FDA commissioner.
I know of Scott's calm courage after his cancer diagnosis, his devotion to his family and his dedication to the practice of medicine. For years (until he had children) he was an internist and hospitalist, seeing and healing patients on weekend, evenings and holidays, all the while performing his duties at FDA and CMS. He loves medicine and that emotion shapes his view about how to best use advances in science to improve the safety and effectiveness of medical technologies. He is not an ideologue. He is data-driven and his views and decisions are informed by the kind of robust discussion and debate that is increasingly rare in our public sphere.
Finally, Scott has never let the intricacies of policy or statistical hair splitting deter him from thinking about the FDA as an institution that has more control over life and death -- as well as how well we live -- than any other agency on earth. He knows that patients and their families have as much a right to weigh in on FDA decisions as any so-called expert. The great medical reformer, William Osler once noted: "The good physician treats the disease; the great physician treats the patient who has the disease."
Scott is a great physician and will be a great FDA commissioner who puts patients first.
At the recent abuse deterrent opioids summit, CDER Deputy Director for Regulatory Programs Dr. Doug Throckmorton presented the FDA’s position. He said, “We must move on from older to newer technologies. Amen. But how?
Per Dr. Throckmorton, “Ongoing and planned activities reflect the commitment by FDA to integrate the use of all of our available tools to achieve our goals related to the safe use of prescription opioids.” Two crucial aspects of moving forward are smart policy initiatives and regulatory clarity. We are inching our way forward on both. We can and must do better.
A key part of the FDA’s Opioid Action Plan is “Expand access to abuse-deterrent formulations (ADFs) to discourage abuse.” In order to achieve this worthy goal, the agency has issued numerous guidance’s – but actions speak louder than words – and the FDA’s actions have been, at times, confusing. When it comes to the development of new therapies to prevent opioid abuse and addiction, predictability is power in pursuit of the public health.
One of the FDA’s stated goals is to “Incentivize the development of opioid medications with progressively better AD properties and support their widespread use.” Bravo. But the devil is in the details. One area of regulatory clarity that could be improved is the agency’s views on differentiation between extended-release and long-acting (ER-LA) and immediate release (IR) opioids and how new products are tested for abuse-deterrent properties. This is a critical concern as IR opioids, with over 240 million scripts annual, represent nearly 96% of the entire opioid market. They are the “gateway” drug of prescription opioid abuse and do not have a single approved ADF formulation.
Believe it or not, abuse deterrence is largely defined and determined by how admitted opioid abusers “like” the product. (Abuse of IR opioid drugs is attractive to abusers because bypassing intestinal absorption and metabolism can lead to a higher Cmax and faster Tmax resulting in a more intense and more immediate high.)
Not surprisingly, abusers prefer immediate release products because they get their highs faster. But, as far as regulatory review is concerned, that also means that IR products under review by the FDA are determined to be less abuse-deterrent.
It’s an apples-to-oranges comparison that is resulting in an uneven ER-LA/IR regulatory playing field. And the unfortunate result is that it’s harder for immediate release opioids to pass FDA muster. Since IR opioids are designed to release “immediately” and last for a short duration (3-4 hours), measurements of drug exposure and “liking” should be taken at earlier, more IR-relevant intervals (when measured against existing, non-abuse deterrent comparator drugs). Measuring IR products by ER-LA standards is not a real world proposition. (When ER-LA’s are compared to IR’s by abusers – of course they like the immediate “high” better). The “clinic” experience needs to be reflective of real-world abuse.
Regulatory ambiguity and common sense are not allies. In fact, the agency has identified one of its major challenges as assessing the impact of individual formulations. Admitting the problem is important. Addressing it is urgent.
It’s time for the FDA to reflect on a more nuanced approach in measuring and determining appropriate standards for demonstrating abuse-deterrent properties of immediate release opioid. Small differences can lead to big benefits in the real world of deterrence. The key question remains not unanswered, but unaddressed – are the endpoints suggested in FDA’s “Guidance for Industry: Abuse-Deterrent Opioids—Evaluation and Labeling” the most relevant for demonstrating abuse-deterrent properties of an IR opioid?
While the public health imperative must drive the regulatory agenda, another important issue is how agency actions impact continued robust research and development. Minus more up-to-date and predictable FDA review criteria for abuse deterrent opioids, investment in their development is already becoming less attractive. Can we really afford to leave these decisions in the hands of admitted drug addicts? Actions (or inactions) have consequences.
As Dr. Throckmorton said at the recent meeting of the Agency’s Science Board: “FDA will act within its authorities in support of our public health mission to help defeat the epidemic of opioid abuse through a science-based and continuously evolving approach by improving the use of opioids through careful and appropriate regulatory activities, improving the use of opioids through careful and appropriate policy development, improving the treatment of pain through improved science, and improving the safe use of opioids through communication, partnership and collaboration.”
The time to focus on this issue is now. Lives are at stake.
Per Dr. Throckmorton, “Ongoing and planned activities reflect the commitment by FDA to integrate the use of all of our available tools to achieve our goals related to the safe use of prescription opioids.” Two crucial aspects of moving forward are smart policy initiatives and regulatory clarity. We are inching our way forward on both. We can and must do better.
A key part of the FDA’s Opioid Action Plan is “Expand access to abuse-deterrent formulations (ADFs) to discourage abuse.” In order to achieve this worthy goal, the agency has issued numerous guidance’s – but actions speak louder than words – and the FDA’s actions have been, at times, confusing. When it comes to the development of new therapies to prevent opioid abuse and addiction, predictability is power in pursuit of the public health.
One of the FDA’s stated goals is to “Incentivize the development of opioid medications with progressively better AD properties and support their widespread use.” Bravo. But the devil is in the details. One area of regulatory clarity that could be improved is the agency’s views on differentiation between extended-release and long-acting (ER-LA) and immediate release (IR) opioids and how new products are tested for abuse-deterrent properties. This is a critical concern as IR opioids, with over 240 million scripts annual, represent nearly 96% of the entire opioid market. They are the “gateway” drug of prescription opioid abuse and do not have a single approved ADF formulation.
Believe it or not, abuse deterrence is largely defined and determined by how admitted opioid abusers “like” the product. (Abuse of IR opioid drugs is attractive to abusers because bypassing intestinal absorption and metabolism can lead to a higher Cmax and faster Tmax resulting in a more intense and more immediate high.)
Not surprisingly, abusers prefer immediate release products because they get their highs faster. But, as far as regulatory review is concerned, that also means that IR products under review by the FDA are determined to be less abuse-deterrent.
It’s an apples-to-oranges comparison that is resulting in an uneven ER-LA/IR regulatory playing field. And the unfortunate result is that it’s harder for immediate release opioids to pass FDA muster. Since IR opioids are designed to release “immediately” and last for a short duration (3-4 hours), measurements of drug exposure and “liking” should be taken at earlier, more IR-relevant intervals (when measured against existing, non-abuse deterrent comparator drugs). Measuring IR products by ER-LA standards is not a real world proposition. (When ER-LA’s are compared to IR’s by abusers – of course they like the immediate “high” better). The “clinic” experience needs to be reflective of real-world abuse.
Regulatory ambiguity and common sense are not allies. In fact, the agency has identified one of its major challenges as assessing the impact of individual formulations. Admitting the problem is important. Addressing it is urgent.
It’s time for the FDA to reflect on a more nuanced approach in measuring and determining appropriate standards for demonstrating abuse-deterrent properties of immediate release opioid. Small differences can lead to big benefits in the real world of deterrence. The key question remains not unanswered, but unaddressed – are the endpoints suggested in FDA’s “Guidance for Industry: Abuse-Deterrent Opioids—Evaluation and Labeling” the most relevant for demonstrating abuse-deterrent properties of an IR opioid?
While the public health imperative must drive the regulatory agenda, another important issue is how agency actions impact continued robust research and development. Minus more up-to-date and predictable FDA review criteria for abuse deterrent opioids, investment in their development is already becoming less attractive. Can we really afford to leave these decisions in the hands of admitted drug addicts? Actions (or inactions) have consequences.
As Dr. Throckmorton said at the recent meeting of the Agency’s Science Board: “FDA will act within its authorities in support of our public health mission to help defeat the epidemic of opioid abuse through a science-based and continuously evolving approach by improving the use of opioids through careful and appropriate regulatory activities, improving the use of opioids through careful and appropriate policy development, improving the treatment of pain through improved science, and improving the safe use of opioids through communication, partnership and collaboration.”
The time to focus on this issue is now. Lives are at stake.
Some snippets from an excellent article in the Pink Sheet.
Gottlieb Nomination As US FDA Chief Could Signal Changes To Generic Approval Process
Michael Cipriano
Former deputy commissioner will also likely push increased use of biomarkers and more flexibility in off-label communication, among other reforms. President Donald Trump's choice of Scott Gottlieb to head the US FDA could signal efforts to bring reforms among a variety of fronts, perhaps none more notable than how the agency approves generic drugs.
Peter Pitts, president of the Center for Medicine in the Public Interest and a former FDA associate commissioner, touted Gottlieb as "a great choice." Pitts, who worked with Gottlieb for nearly two years at the agency, tells the Pink Sheet that he "knows the people, he knows the process, and he understands how to make both work harder and smarter."
"He understands the need to bring generic drugs to the market faster," Pitts said. "He understands the importance of addressing the single-source generic issue from a competitive perspective. He also understands, probably most importantly, the need not just for things happening faster, but things happening with greater predictability."
Public Citizen, however, was not so rosy about Gottlieb's selection, citing his close ties to industry, as it similarly did when Robert Califf was tapped to head the agency under President Obama.
In a statement, Michael Carome, director of Public Citizen's Health Research Group, noted that Gottlieb is currently serving or has recently served on the boards of five pharmaceutical companies, including that of GlaxoSmithKlein, and that he received at least $413,000 from multiple drug and medical device companies for mainly consulting and speaking fees. Carome called on the Senate to reject his nomination.
"When Gottlieb served as FDA deputy commissioner, he was recused from many key meetings and decisions due to his close relationship with industry," Carome said. "If the Senate does not reject Gottlieb, he will have to be recused from key decisions time and time again, otherwise there is no way to be sure he will put the public’s health over industry profits."
Pitts, however, contended that close ties to industry "are incredibly important."
"FDA has to be both a regulator of and ally with industry," Pitts said. "And I think Scott is the right guy to practice that nuanced relationship."
Gottlieb Nomination As US FDA Chief Could Signal Changes To Generic Approval Process
Michael Cipriano
Former deputy commissioner will also likely push increased use of biomarkers and more flexibility in off-label communication, among other reforms. President Donald Trump's choice of Scott Gottlieb to head the US FDA could signal efforts to bring reforms among a variety of fronts, perhaps none more notable than how the agency approves generic drugs.
Peter Pitts, president of the Center for Medicine in the Public Interest and a former FDA associate commissioner, touted Gottlieb as "a great choice." Pitts, who worked with Gottlieb for nearly two years at the agency, tells the Pink Sheet that he "knows the people, he knows the process, and he understands how to make both work harder and smarter."
"He understands the need to bring generic drugs to the market faster," Pitts said. "He understands the importance of addressing the single-source generic issue from a competitive perspective. He also understands, probably most importantly, the need not just for things happening faster, but things happening with greater predictability."
Public Citizen, however, was not so rosy about Gottlieb's selection, citing his close ties to industry, as it similarly did when Robert Califf was tapped to head the agency under President Obama.
In a statement, Michael Carome, director of Public Citizen's Health Research Group, noted that Gottlieb is currently serving or has recently served on the boards of five pharmaceutical companies, including that of GlaxoSmithKlein, and that he received at least $413,000 from multiple drug and medical device companies for mainly consulting and speaking fees. Carome called on the Senate to reject his nomination.
"When Gottlieb served as FDA deputy commissioner, he was recused from many key meetings and decisions due to his close relationship with industry," Carome said. "If the Senate does not reject Gottlieb, he will have to be recused from key decisions time and time again, otherwise there is no way to be sure he will put the public’s health over industry profits."
Pitts, however, contended that close ties to industry "are incredibly important."
"FDA has to be both a regulator of and ally with industry," Pitts said. "And I think Scott is the right guy to practice that nuanced relationship."
Governor Cuomo’s budget aims to control drug pricing through price controls . While this is a good political sound bite, it’s bad public policy with dangerous unintended consequences – the worst of being that it won't lower prices but will reduce patient choice.
Have a look at this interview I did yesterday on Capital Tonight for a robust conversation on PBMs, the value of innovation, the role of the FDA in lowering drug costs, and other timely topics.
Have a look at this interview I did yesterday on Capital Tonight for a robust conversation on PBMs, the value of innovation, the role of the FDA in lowering drug costs, and other timely topics.
Finally, some bipartisan sanity on healthcare transparency.
Representatives Doug Collins (R-GA), Buddy Carter (R-GA), Dan Loebsack (D-IA), John Sarbanes (D-MD) and John Duncan (R-TN) have introduced the Prescription Drug Price Transparency Act.
It is designed to:
* Safeguard patient information collected by a PBM (currently used to steer patients to PBM-owned/preferred outlets)
* Prohibit PBMs from requiring patients to utilize a PBM-owned pharmacy (including specialty pharmacy)
* Require maximum allowable cost transparency
* Apply these standards to both Tricare and FEHBP (the Federal Employee Health Benefit program).
In sum, the bill would establish a far more competitive marketplace for brand and generic products and lessen the monopsony of the large PBMs.
Transparency means looking at the whole ecosystem.
Stay tuned.
Representatives Doug Collins (R-GA), Buddy Carter (R-GA), Dan Loebsack (D-IA), John Sarbanes (D-MD) and John Duncan (R-TN) have introduced the Prescription Drug Price Transparency Act.
It is designed to:
* Safeguard patient information collected by a PBM (currently used to steer patients to PBM-owned/preferred outlets)
* Prohibit PBMs from requiring patients to utilize a PBM-owned pharmacy (including specialty pharmacy)
* Require maximum allowable cost transparency
* Apply these standards to both Tricare and FEHBP (the Federal Employee Health Benefit program).
In sum, the bill would establish a far more competitive marketplace for brand and generic products and lessen the monopsony of the large PBMs.
Transparency means looking at the whole ecosystem.
Stay tuned.
To import or not to import? Lawmakers push another bill to combat high drug prices
By ED SILVERMAN @Pharmalot
FEBRUARY 28, 2017
A group of lawmakers is introducing yet another bill that would allow Americans to import prescription medicines from Canada. And in a bid to quell long-standing criticism of the notion, the latest effort includes several provisions designed to address concerns that medicines bought from online pharmacies in other countries may not be safe.
Known as the Affordable and Safe Prescription Drug Importation Act, the bill would instruct the US Secretary of Health and Human Services to issue regulations allowing wholesalers, licensed US pharmacies, and individuals to import prescription drugs from licensed Canadian sellers. And the drugs would have to be made at facilities inspected by the US Food and Drug Administration.
After two years, however, the HHS secretary would have authority to permit importation from countries in the Organization for Economic Cooperation and Development that “meet specified statutory or regulatory standards that are comparable to US standards,” according to the bill. Nearly three dozen countries are members of the OECD.
“In 2014, the US spent about 40 percent more on prescriptions per person than Canada, twice as much as the average major industrialized country,” according to talking points that were distributed with the bill. “… In order to get the medicine they need, millions of people are buying their prescription drugs from other countries.”
The legislation — which is being spearheaded by Senator Bernie Sanders (I-Vt.) and Representative Elijah Cummings (D-Md.), who have conducted various probes into drug pricing — comes amid escalating national angst over the cost of prescription medicines. Poll after poll finds Americans want the federal government to take action, although there has been little movement on the national level.
As many as 77 percent of Americans reported last fall that drug costs are unreasonable, up from 72 percent a year earlier, according to a poll by the Kaiser Family Foundation. At the time, 71 percent favored allowing Americans to buy prescription drugs imported from Canada, among other measures they would like the federal government to pursue to lower their bills.
Although President Trump recently complained about “astronomical” prices,” he has not released any plan. But his remarks have placed drug makers on the defensive. A few pledged to limit annual price hikes to single-digit increases, while others have released data to argue their price increases have been reduced by rebates to middlemen that act on behalf of insurers.
Meanwhile, a bill introduced in the House is gaining support because it would presumably address some price gouging by providing incentives to drug makers to develop generics when there is a lack of competition or a shortage exists. A somewhat similar piece of legislation was recently introduced in the Senate, but it would also temporarily permit prescriptions drugs to be imported in order to mitigate shortages.
Importation is hardly a new idea. In fact, this is only the latest attempt by various lawmakers to find a way for Americans to import medicines from Canada. But given that President Trump has previously voiced support for the idea — and also accused drug makers of “getting away with murder” — they apparently see an opening to renew the call.
Whether the effort can succeed, however, is unclear. Under a 2003 law, the FDA can issue waivers for individuals to import medicines for personal use. But importation is not otherwise permitted until the HHS secretary certifies that importation would not pose a health risk and could lower consumer costs. And ensuring safety of imported drugs has been cited by both drug makers and regulators as a concern. Nearly all Republicans have also dismissed the idea and this latest bill is being pushed by a mix of Democrats and Independents.
“I’ve seen this bill in various iterations many different times and they never really address two major concerns — safety and pricing,” said Peter Pitts, a former FDA associate commissioner who heads the Center for Medicine in the Public Interest, a think tank that is funded, in part, by the pharmaceutical industry.
“We exist in a closed regulatory system and when you bring in drugs from outside FDA control, I don’t care how you phrase it, it’s still caveat emptor. The products can’t be guaranteed, because there are holes [in the legislation], such as not being able to guarantee that product labeling is correct,” he told us.
And no drug from Canada is going to cost less than a co-pay, which is $20 or $30 for many people, so it’s highly unlikely that it will do anything for the majority of Americans with health insurance. So it offers a largely useless alternative. I think this is a good political talking point, but won’t deliver what it promises. Importation is a vampire issue — you can drive a stake through its heart, but it won’t die.”
To assuage safety concerns, the bill states that legally imported drugs must be purchased from an FDA-certified foreign seller and have the same active ingredient, route of administration, and strength as drugs approved in the US. And certain types of drugs, such as certain biologics, could be imported only by wholesalers or pharmacies.
In order to be a so-called certified foreign seller, the bill states that the operation must be a wholesale distributor or licensed foreign pharmacy that is current with applicable registration fees and sells only qualifying prescription drugs. There is a list of criteria to be met, which you can read here.
Americans can buy medicines only from pharmacies licensed in Canada, and only for personal use in quantities that do not exceed a 90-day supply. They must also have a valid prescription issued by a health care practitioner licensed to practice in the US. Any pharmacy selling a counterfeit drug is subject to a $250,000 penalty and 10 years imprisonment. And the HHS is required to ask the US Government Accountability Office to run a report after 18 months to assess the impact.
But the Pew Charitable Trusts raised concerns and argued that it may be difficult to enforce the requirement that medicines must be purchased from an FDA-certified foreign seller and there is no mechanism to make it possible for medicines to have the same electronic security and tracking system, which is used in the US to weed out counterfeits.
“This poses a safety risk with respect to imported product, but also undermines the entire system,” wrote Allan Coukell, who is senior director of the Pew health programs, in a letter to Sanders. And while he acknowledged that competition from imports could prompt drug makers to lower prices in the US, he also speculated that foreign governments might eventually limit imports to the US if domestic supplies become strained.
One backer of importation called it a “step in the right direction.” Gabriel Levitt, who is the president of PharmacyChecker.com, which vets online pharmacies, wrote us that “if passed, the new bill … simply instructs the FDA to finally help Americans do what they already do, purchase lower cost medication from safe, international online pharmacies.”
We should note that one of the lawmakers who is co-sponsoring the bill is Sen. Cory Booker (D-NJ), who last month was one of 13 Democratic Senators who voted against an amendment to a budget procedure that would have allowed imports from Canada. His vote angered progressive Democrats. New Jersey is home to a few large drug makers and numerous smaller ones. The amendment was introduced by Sanders and Sen. Amy Klobuchar (D-Minn).
By ED SILVERMAN @Pharmalot
FEBRUARY 28, 2017
A group of lawmakers is introducing yet another bill that would allow Americans to import prescription medicines from Canada. And in a bid to quell long-standing criticism of the notion, the latest effort includes several provisions designed to address concerns that medicines bought from online pharmacies in other countries may not be safe.
Known as the Affordable and Safe Prescription Drug Importation Act, the bill would instruct the US Secretary of Health and Human Services to issue regulations allowing wholesalers, licensed US pharmacies, and individuals to import prescription drugs from licensed Canadian sellers. And the drugs would have to be made at facilities inspected by the US Food and Drug Administration.
After two years, however, the HHS secretary would have authority to permit importation from countries in the Organization for Economic Cooperation and Development that “meet specified statutory or regulatory standards that are comparable to US standards,” according to the bill. Nearly three dozen countries are members of the OECD.
“In 2014, the US spent about 40 percent more on prescriptions per person than Canada, twice as much as the average major industrialized country,” according to talking points that were distributed with the bill. “… In order to get the medicine they need, millions of people are buying their prescription drugs from other countries.”
The legislation — which is being spearheaded by Senator Bernie Sanders (I-Vt.) and Representative Elijah Cummings (D-Md.), who have conducted various probes into drug pricing — comes amid escalating national angst over the cost of prescription medicines. Poll after poll finds Americans want the federal government to take action, although there has been little movement on the national level.
As many as 77 percent of Americans reported last fall that drug costs are unreasonable, up from 72 percent a year earlier, according to a poll by the Kaiser Family Foundation. At the time, 71 percent favored allowing Americans to buy prescription drugs imported from Canada, among other measures they would like the federal government to pursue to lower their bills.
Although President Trump recently complained about “astronomical” prices,” he has not released any plan. But his remarks have placed drug makers on the defensive. A few pledged to limit annual price hikes to single-digit increases, while others have released data to argue their price increases have been reduced by rebates to middlemen that act on behalf of insurers.
Meanwhile, a bill introduced in the House is gaining support because it would presumably address some price gouging by providing incentives to drug makers to develop generics when there is a lack of competition or a shortage exists. A somewhat similar piece of legislation was recently introduced in the Senate, but it would also temporarily permit prescriptions drugs to be imported in order to mitigate shortages.
Importation is hardly a new idea. In fact, this is only the latest attempt by various lawmakers to find a way for Americans to import medicines from Canada. But given that President Trump has previously voiced support for the idea — and also accused drug makers of “getting away with murder” — they apparently see an opening to renew the call.
Whether the effort can succeed, however, is unclear. Under a 2003 law, the FDA can issue waivers for individuals to import medicines for personal use. But importation is not otherwise permitted until the HHS secretary certifies that importation would not pose a health risk and could lower consumer costs. And ensuring safety of imported drugs has been cited by both drug makers and regulators as a concern. Nearly all Republicans have also dismissed the idea and this latest bill is being pushed by a mix of Democrats and Independents.
“I’ve seen this bill in various iterations many different times and they never really address two major concerns — safety and pricing,” said Peter Pitts, a former FDA associate commissioner who heads the Center for Medicine in the Public Interest, a think tank that is funded, in part, by the pharmaceutical industry.
“We exist in a closed regulatory system and when you bring in drugs from outside FDA control, I don’t care how you phrase it, it’s still caveat emptor. The products can’t be guaranteed, because there are holes [in the legislation], such as not being able to guarantee that product labeling is correct,” he told us.
And no drug from Canada is going to cost less than a co-pay, which is $20 or $30 for many people, so it’s highly unlikely that it will do anything for the majority of Americans with health insurance. So it offers a largely useless alternative. I think this is a good political talking point, but won’t deliver what it promises. Importation is a vampire issue — you can drive a stake through its heart, but it won’t die.”
To assuage safety concerns, the bill states that legally imported drugs must be purchased from an FDA-certified foreign seller and have the same active ingredient, route of administration, and strength as drugs approved in the US. And certain types of drugs, such as certain biologics, could be imported only by wholesalers or pharmacies.
In order to be a so-called certified foreign seller, the bill states that the operation must be a wholesale distributor or licensed foreign pharmacy that is current with applicable registration fees and sells only qualifying prescription drugs. There is a list of criteria to be met, which you can read here.
Americans can buy medicines only from pharmacies licensed in Canada, and only for personal use in quantities that do not exceed a 90-day supply. They must also have a valid prescription issued by a health care practitioner licensed to practice in the US. Any pharmacy selling a counterfeit drug is subject to a $250,000 penalty and 10 years imprisonment. And the HHS is required to ask the US Government Accountability Office to run a report after 18 months to assess the impact.
But the Pew Charitable Trusts raised concerns and argued that it may be difficult to enforce the requirement that medicines must be purchased from an FDA-certified foreign seller and there is no mechanism to make it possible for medicines to have the same electronic security and tracking system, which is used in the US to weed out counterfeits.
“This poses a safety risk with respect to imported product, but also undermines the entire system,” wrote Allan Coukell, who is senior director of the Pew health programs, in a letter to Sanders. And while he acknowledged that competition from imports could prompt drug makers to lower prices in the US, he also speculated that foreign governments might eventually limit imports to the US if domestic supplies become strained.
One backer of importation called it a “step in the right direction.” Gabriel Levitt, who is the president of PharmacyChecker.com, which vets online pharmacies, wrote us that “if passed, the new bill … simply instructs the FDA to finally help Americans do what they already do, purchase lower cost medication from safe, international online pharmacies.”
We should note that one of the lawmakers who is co-sponsoring the bill is Sen. Cory Booker (D-NJ), who last month was one of 13 Democratic Senators who voted against an amendment to a budget procedure that would have allowed imports from Canada. His vote angered progressive Democrats. New Jersey is home to a few large drug makers and numerous smaller ones. The amendment was introduced by Sanders and Sen. Amy Klobuchar (D-Minn).
Megan and John Crowley (Courtesy of the Crowley Family)
Last night President Trump was attacked for speaking about Megan Crowley a 20-year-old freshman at Notre Dame who was diagnosed at birth with an orphan illness called Pompe Disease.
The reason? The President used the opportunity to call for more medicines to keep young women like Megan alive.
Here’s what the President said:
"Megan was diagnosed with Pompe Disease, a rare and serious illness when she was 15 months old. She was not expected to live past 5.
On receiving this news, Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan's life. Today she is 20 years old -- and a sophomore at Notre Dame.
Megan's story is about the unbounded power of a father's love for a daughter.
But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need.
If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan."
He had not even finished his speech when cynics on Twitter and in the media, belittled the Crowley family’s courage because, in their view, they were tools for what they hysterically call Trump’s reckless gutting of the FDA. (Note that they never specify what would be gutted but assure us that if Trump got his way, the FDA would turn America into The Walking Dead.
Of all the sniping and snide observations, Former FDA commissioner David Kessler and Washington Post reporter Carolyn John stand out as the most obnoxious and condescending.
For his part, David Kessler posted on Twitter @DavidAKesslerMD
Trump mischaracterizes Pompe drug approval process. Approved in 9 months based on 39 patients. Not "slow and burdensome."
First, Trump never even mentioned the Pompe drug (Myozyme, the drug Crowley’s company developed) approval.
Second, “approval” refers to the FDA’s review of all the other studies and data the agency requested. The FDA is required by law to review such completed applications in 180 days. Not. Nine. Months.
Meanwhile, it took about 6 years to complete the studies used to establish the drug’s safety and efficacy. That’s a lot less than the 10 years it takes to evaluate drugs for larger groups of patients.
But third, and most important, Crowley was in the audience because if the FDA was using its current regulatory approach to orphan drugs in 2001, the drug still wouldn’t be approved. Nor is Crowley is calling for gutting the FDA as critics hysterically claim. As Crowley points out in an op-ed in the New York Observer: “the FDA must always put patient safety first.”
The problem is that despite having a decade of experience in dealing with rare pediatric conditions like Pompe or Fabry, the FDA now often requires more information than ever before to approve orphan drugs. As a result, they take as much time and money to get through the FDA as do medicines more common conditions,
But the award for the most sneering and fact-free hit piece goes to the Washington Post’s Carolyn Johnson:
“The actual drug, that saved Megan’s life is manufactured in Belgium and was developed by a biotech company founded by a Dutch immigrant — a company that is today owned by a French firm. The drug was invented through a scientific experiment that couldn’t have happened without international collaboration. And a president who has said he wants to bring down drug prices just held up as a shining example of innovation a drug that costs an average of $298,000 a year, per patient.”
Key omissions:
The company was Genzyme, a US biotech company founded in Boston – not Europe -- to bring orphan drugs to market. Genzyme was sold to Sanofi, which invests most of its R&D in the United States.
Moreover, the cheap shot about drug prices (“a shining example of innovation” Johnson snarks) ignores the fact that the average price of hard to manufacture medicines for very small groups of patients has declined since Genzyme developed Ceredase for Gaucher’s disease in 1990 for a list price of $360000. (That’s $558000 in 2016 dollars.)
The sneering continues:
"It’s also unclear whether the Crowley’s inspiring story and the development of the drug Myozyme (also called Lumizyme) is really an example of how “our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” as Trump described.
Other companies have pointed to Myozyme as an example of the agency’s flexibility in getting drugs approved quickly, based on small amounts of data.
Briefing materials prepared by the drug company Sarepta Therapeutics to support the approval of their drug last year cited Myozyme as an “approval precedent” that they hoped to follow.
Last April, at a hearing in support of Sarepta’s drug, one of the researchers involved in Myozyme’s approval testified to the advisory committee that its passage through the regulatory process as a helpful parallel to consider.
Against the recommendation of its advisory committee, which wanted to see more evidence, regulators approved that drug."
Omission: In fact, FDA reviewers virtually told the FDA advisory committee not to approve the Saretpa drug because it followed the Myozyme approach.
No one is suggesting that Myozyme was delayed by the FDA or that the agency in the last couple of years has been quicker in reviewing applications for drug approval.
The issue is whether the FDA could move more quickly by requiring data that has less to do with establishing the safety and benefit of medicines and more to do with employees demanding an increasingly higher degree of statistical certainty that has nothing to do with those goals
The issue is whether the Amicus drug for Fabry Disease – approved in Germany, Japan, and Great Britain – requires another randomized study and two more years of data to determine whether patients are more likely to have diarrhea.
The issue is whether patients and their families should have as much to say about the drugs that shape whether they live or die as well how they live, as the naysayers who claim that the experts know better.
The smug elite – like Kessler and Johnson -- realize their power over the FDA review process is slipping away and they are beside themselves that Donald Trump is determined to wrest their control away. How can you tell? Because they turned a courageous young woman and her father’s decade-long struggle to save her life into objects of their rage against the new President.
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.
