Latest Drugwonks' Blog
The US is at a critical juncture in biomedical innovation, facing the twin towers of high costs and complexity in research. We’re not alone. China is racing ahead in the development and application of Artificial Intelligence (AI) to drug discovery and development. Beijing is actively supporting AI’s role in healthcare technology development with an aggressive and robust national strategy that includes extensive biodata collection, R&D facilitation, and commercialization of medical AI. Alas, inside-the-Beltway, we’re more-or-less standing still.
Without significant attention to revitalizing our processes and procedures for discovery (Hello NIH!), evaluating (Hello FDA!), and paying for new therapies (Hello CMS!), China will leave us in the dust when comes to developing new therapies. Are you ready for this headline, “Who Lost Healthcare?”
For example, Chinese companies are already at the forefront of integrating AI into various stages of drug development, exemplified by its development of ISM3312, an AI-designed drug targeting COVID-19, which has already entered clinical trials in China. That’s what pandemic preparedness means. Where are we? Who Lost Healthcare?
It’s happening in China and it’s not by accident. China is making significant investments to shift from traditional animal-based preclinical models to advanced AI-driven in silico models and digital twin technologies. This transformation is driven by government initiatives, rapid growth in AI biotech startups, and the need to reduce the ethical, financial, and scientific limitations associated with animal testing. Who Lost Healthcare?
If our public health bureaucrats aren’t paying attention, the private sector certainly is. China's advancements in AI-driven drug development have garnered significant interest from innovative pharmaceutical companies. For instance, AstraZeneca, has entered a $2 billion licensing deal with China's CSPC Pharmaceutical Group to develop a small molecule addressing dyslipidemia, extending their existing collaboration focusing on advanced lung cancer research. Similarly, Merck recently signed a $3.3 billion agreement with China-based LaNova Medicines to develop advanced immuno-therapies targeting PD-1 and VEGF proteins. What’s wrong with this picture? Who Lost Healthcare?
China’s National Medical Products Administration (NMPA) -- the equivalent of our FDA --has been actively reforming its regulatory framework to foster the integration of AI-driven technologies, including virtual models, digital twins, and advanced trial designs. This transformation aligns with China’s broader strategic goals under the “Healthy China 2030” plan and the “New Generation Artificial Intelligence Development Plan.” It is a targeted and thoughtful great leap forward for both healthcare innovation and industrial policy. Matching and then overtaking China in AI-driven healthcare must become a national priority. And it need not be driven by government.
Consider Vial, a California-based start-up whose goal is to significantly reduce clinical trial costs through automation, digitization, and streamlined workflows. Their TrialOS platform structures, digitizes, and automates over 200 discrete trial tasks, cutting both time and expenses. This efficiency is crucial for the U.S. to maintain its competitive edge against China's state-supported, rapidly scaling pharmaceutical infrastructure.
By integrating AI-driven target identification, generative chemistry, and automated preclinical testing, Vial also accelerates the drug development pipeline. Their use of organoid and organ-on-a-chip (OOC) technologies reduces reliance on costly animal models, making preclinical data collection faster, cheaper, and more predictive of human outcomes AI is increasingly being used to mitigate dose-related failures in drug development through advanced applications in pharmacokinetic/pharmacodynamic (PK/PD) modeling, dose-response optimization, individualized dosing, and post-marketing surveillance.
Other companies like Unlearn.AI, Phesi, and ArisGlobal are pushing the boundaries in areas including digital twining, synthetic control arms, and regulatory automation. Another player, Insilico Medicine, reports reductions of up to 70–90% in animal testing during preclinical phases. By eliminating the need for extensive animal studies, in silico models can reduce preclinical R&D costs by up to 40%.
This approach aligns with the strategic need to outpace China in drug development. The ability to launch numerous clinical programs simultaneously could give the U.S. a significant advantage, leveraging scale and speed to dominate globally
The FDA needs update its drug development regulations, and the right place to start is by incorporating AI into its clinical trial protocols. FDA should create an "AI Fast-Track" designation similar to its expedited pathways for AI-driven drug discovery. This would promote advanced technologies, reduce administrative hurdles, and bring therapies to market faster, enhancing U.S. competitiveness and enhancing safety and effectiveness.
By embracing technologies that reduce clinical trial costs and accelerate drug discovery—coupled with regulatory reforms to support AI-driven advancements—the U.S. can not only challenge but potentially surpass China's rapid advances in drug development. The future of global health leadership depends on our ability to integrate these innovations into a cohesive, scalable strategy.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest and a Visiting Professor at the University of Paris School of Medicine.
Robert Goldberg, Ph.D., is co-founder and Vice President of Research at the Center for Medicine in the Public Interest
Without significant attention to revitalizing our processes and procedures for discovery (Hello NIH!), evaluating (Hello FDA!), and paying for new therapies (Hello CMS!), China will leave us in the dust when comes to developing new therapies. Are you ready for this headline, “Who Lost Healthcare?”
For example, Chinese companies are already at the forefront of integrating AI into various stages of drug development, exemplified by its development of ISM3312, an AI-designed drug targeting COVID-19, which has already entered clinical trials in China. That’s what pandemic preparedness means. Where are we? Who Lost Healthcare?
It’s happening in China and it’s not by accident. China is making significant investments to shift from traditional animal-based preclinical models to advanced AI-driven in silico models and digital twin technologies. This transformation is driven by government initiatives, rapid growth in AI biotech startups, and the need to reduce the ethical, financial, and scientific limitations associated with animal testing. Who Lost Healthcare?
If our public health bureaucrats aren’t paying attention, the private sector certainly is. China's advancements in AI-driven drug development have garnered significant interest from innovative pharmaceutical companies. For instance, AstraZeneca, has entered a $2 billion licensing deal with China's CSPC Pharmaceutical Group to develop a small molecule addressing dyslipidemia, extending their existing collaboration focusing on advanced lung cancer research. Similarly, Merck recently signed a $3.3 billion agreement with China-based LaNova Medicines to develop advanced immuno-therapies targeting PD-1 and VEGF proteins. What’s wrong with this picture? Who Lost Healthcare?
China’s National Medical Products Administration (NMPA) -- the equivalent of our FDA --has been actively reforming its regulatory framework to foster the integration of AI-driven technologies, including virtual models, digital twins, and advanced trial designs. This transformation aligns with China’s broader strategic goals under the “Healthy China 2030” plan and the “New Generation Artificial Intelligence Development Plan.” It is a targeted and thoughtful great leap forward for both healthcare innovation and industrial policy. Matching and then overtaking China in AI-driven healthcare must become a national priority. And it need not be driven by government.
Consider Vial, a California-based start-up whose goal is to significantly reduce clinical trial costs through automation, digitization, and streamlined workflows. Their TrialOS platform structures, digitizes, and automates over 200 discrete trial tasks, cutting both time and expenses. This efficiency is crucial for the U.S. to maintain its competitive edge against China's state-supported, rapidly scaling pharmaceutical infrastructure.
By integrating AI-driven target identification, generative chemistry, and automated preclinical testing, Vial also accelerates the drug development pipeline. Their use of organoid and organ-on-a-chip (OOC) technologies reduces reliance on costly animal models, making preclinical data collection faster, cheaper, and more predictive of human outcomes AI is increasingly being used to mitigate dose-related failures in drug development through advanced applications in pharmacokinetic/pharmacodynamic (PK/PD) modeling, dose-response optimization, individualized dosing, and post-marketing surveillance.
Other companies like Unlearn.AI, Phesi, and ArisGlobal are pushing the boundaries in areas including digital twining, synthetic control arms, and regulatory automation. Another player, Insilico Medicine, reports reductions of up to 70–90% in animal testing during preclinical phases. By eliminating the need for extensive animal studies, in silico models can reduce preclinical R&D costs by up to 40%.
This approach aligns with the strategic need to outpace China in drug development. The ability to launch numerous clinical programs simultaneously could give the U.S. a significant advantage, leveraging scale and speed to dominate globally
The FDA needs update its drug development regulations, and the right place to start is by incorporating AI into its clinical trial protocols. FDA should create an "AI Fast-Track" designation similar to its expedited pathways for AI-driven drug discovery. This would promote advanced technologies, reduce administrative hurdles, and bring therapies to market faster, enhancing U.S. competitiveness and enhancing safety and effectiveness.
By embracing technologies that reduce clinical trial costs and accelerate drug discovery—coupled with regulatory reforms to support AI-driven advancements—the U.S. can not only challenge but potentially surpass China's rapid advances in drug development. The future of global health leadership depends on our ability to integrate these innovations into a cohesive, scalable strategy.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest and a Visiting Professor at the University of Paris School of Medicine.
Robert Goldberg, Ph.D., is co-founder and Vice President of Research at the Center for Medicine in the Public Interest
GLP-1 drugs (Ozempic, Wegovy, Zepbound, etc.) have important therapeutic potential beyond weight loss. According to a new Pharmacy Practice in Focus article, “Since their original entry into the market, GLP-1 receptor agonists have gained FDA approval and have established significant roles in therapy for cardiovascular disease, obesity, and kidney disease. Interestingly, emerging data suggest that this class of medications may have roles in other—somewhat unrelated—conditions.”
These “unrelated” conditions include Parkinson’s Disease, Alzheimer’s Disease, osteoarthritis, gambling addiction, and chemical dependencies. To say GLP-1 drugs have potential beyond weight loss is an understatement. But there haven’t been any large-scale studies to move beyond the exciting (but limited) anecdotal data – and the plural of anecdote isn’t data. Without solid data, the FDA can’t add any of these potentially groundbreaking secondary indications to the official product label.
(In regulatory parlance, “secondary indications” come after the primary indications granted when a drug is initially approved by the FDA.)
Why does this matter? Because unless these “secondary indications” are more intensely studied, and the data reviewed and vetted by the FDA, physicians will be rightfully skeptical and insurance companies unlikely to reimburse patients for what they consider suspicious off-label use. And rightfully so.
With such exciting potential, why aren’t the expensive large-scale clinical trials that would move these new therapeutic opportunities moving forward at warp speed? One troubling answer is an unintended (but entirely predictable) codicil of the Inflation Reduction Act, specifically, the IRA’s blunt language that says that any “negotiated” price covers all indications current or future. In blunt terms, the IRA is saying to drug developers, “You will not be rewarded for investing in any new important science.” This predictable stifling of innovation cannot and must not stand.
The pharmaceutical industry recognizes this problem. Patients and disease organization do too. And you can add to that physicians who read about the potential – but are left wondering where the data are that will better inform these new and exciting use indications. Everyone is waiting.
Well, not everyone. There’s been a strange and uncomfortable silence from a major player in American health care – the insurance companies. While “Big Pharma” regularly face the wrath of pundits and politicians, “Big Payor” seems to get a pass. And it’s not unreasonable to conclude the reason -- they don’t want to pay for important new GLP-1 indications.
That’s craven. The Big Insurance Bailout must end – and a good place to start is by amending the IRA’s inexcusable, unnecessary, and dangerous innovation-penalty.
These “unrelated” conditions include Parkinson’s Disease, Alzheimer’s Disease, osteoarthritis, gambling addiction, and chemical dependencies. To say GLP-1 drugs have potential beyond weight loss is an understatement. But there haven’t been any large-scale studies to move beyond the exciting (but limited) anecdotal data – and the plural of anecdote isn’t data. Without solid data, the FDA can’t add any of these potentially groundbreaking secondary indications to the official product label.
(In regulatory parlance, “secondary indications” come after the primary indications granted when a drug is initially approved by the FDA.)
Why does this matter? Because unless these “secondary indications” are more intensely studied, and the data reviewed and vetted by the FDA, physicians will be rightfully skeptical and insurance companies unlikely to reimburse patients for what they consider suspicious off-label use. And rightfully so.
With such exciting potential, why aren’t the expensive large-scale clinical trials that would move these new therapeutic opportunities moving forward at warp speed? One troubling answer is an unintended (but entirely predictable) codicil of the Inflation Reduction Act, specifically, the IRA’s blunt language that says that any “negotiated” price covers all indications current or future. In blunt terms, the IRA is saying to drug developers, “You will not be rewarded for investing in any new important science.” This predictable stifling of innovation cannot and must not stand.
The pharmaceutical industry recognizes this problem. Patients and disease organization do too. And you can add to that physicians who read about the potential – but are left wondering where the data are that will better inform these new and exciting use indications. Everyone is waiting.
Well, not everyone. There’s been a strange and uncomfortable silence from a major player in American health care – the insurance companies. While “Big Pharma” regularly face the wrath of pundits and politicians, “Big Payor” seems to get a pass. And it’s not unreasonable to conclude the reason -- they don’t want to pay for important new GLP-1 indications.
That’s craven. The Big Insurance Bailout must end – and a good place to start is by amending the IRA’s inexcusable, unnecessary, and dangerous innovation-penalty.
Said it before and I’ll say it again, Take. A. Breath.
Consider the headline from STAT, “FDA purges material on clinical trial diversity from its site, showing stakes of Trump DEI ban .” True.
Now the subhead, “The scrubbing could affect the ways researchers and companies test drugs and medical devices.” Key word, “could.” The great journalistic wiggle word.
Alas, science is about using clear language. Conditional phraseology isn’t always helpful. Aliens could land their flying saucers atop FDA headquarters tomorrow -- but what’s the likelihood? It’s not War of the Worlds but War of the Words.
And words matter. Let’s put the President’s DEI executive order in context. Here’s the actual reality: It is VERY important to understand that, website “scrubbing” notwithstanding, clinical trial diversity guidance development and related FDA initiatives relative to more representative patient participation are unchanged and on-track. Full stop. No conditional wording required.
While it is the media’s job to generate important debate, high velocity “what ifs” show (with respect to Matt Herper and Lizzy Lawrence – two of the best reporters in this space) a lack of finesse. There isn’t anything factually wrong with their reporting, but the article implies a lot that is neither helpful nor likely. Does the executive order presage other possibilities? It could, but is that helpful, prescient reporting – or click bait? Correlation is not causation.
Whether you're a DEI diehard or otherwise, don't let your positions on that ideology or phraseology take your eyes off the prize - clinical trials that are more representative of the population (more than half of whom are women) and of any given medical technology’s specific disease focus.
Political rhetoric (regardless of where you are on the linguistic pronoun spectrum) should play zero role is advancing 21st century regulatory science.
As Rudyard Kipling wrote, “Words are the most powerful drug used by mankind.”
Onward!
Consider the headline from STAT, “FDA purges material on clinical trial diversity from its site, showing stakes of Trump DEI ban .” True.
Now the subhead, “The scrubbing could affect the ways researchers and companies test drugs and medical devices.” Key word, “could.” The great journalistic wiggle word.
Alas, science is about using clear language. Conditional phraseology isn’t always helpful. Aliens could land their flying saucers atop FDA headquarters tomorrow -- but what’s the likelihood? It’s not War of the Worlds but War of the Words.
And words matter. Let’s put the President’s DEI executive order in context. Here’s the actual reality: It is VERY important to understand that, website “scrubbing” notwithstanding, clinical trial diversity guidance development and related FDA initiatives relative to more representative patient participation are unchanged and on-track. Full stop. No conditional wording required.
While it is the media’s job to generate important debate, high velocity “what ifs” show (with respect to Matt Herper and Lizzy Lawrence – two of the best reporters in this space) a lack of finesse. There isn’t anything factually wrong with their reporting, but the article implies a lot that is neither helpful nor likely. Does the executive order presage other possibilities? It could, but is that helpful, prescient reporting – or click bait? Correlation is not causation.
Whether you're a DEI diehard or otherwise, don't let your positions on that ideology or phraseology take your eyes off the prize - clinical trials that are more representative of the population (more than half of whom are women) and of any given medical technology’s specific disease focus.
Political rhetoric (regardless of where you are on the linguistic pronoun spectrum) should play zero role is advancing 21st century regulatory science.
As Rudyard Kipling wrote, “Words are the most powerful drug used by mankind.”
Onward!
As Robert F. Kennedy Jr. undergoes Senate scrutiny for the position of Secretary of Health and Human Services, the stakes – and opportunities -- for America's public health infrastructure couldn't be higher.
As I’ve written previously, creative disruption within federal health agencies can lead to much-needed reform. Increased transparency and accountability in vaccine research and policy could rebuild trust in public health institutions. However, this disruption must not extend to the foundational elements of our health system like vaccines.
Vaccines are arguably the greatest achievement in public health history. The development of the polio vaccine in the 1950s virtually eradicated a devastating disease that once paralyzed thousands of children annually. Today, the Centers for Disease Control and Prevention (CDC) continues to recommend it as an essential immunization.
President-elect Donald Trump has voiced support for vaccines, praising their role in eradicating diseases like polio. Senators must ensure that Kennedy aligns with this view. A retreat from vaccination would not advance Trump's agenda of strengthening the nation but would instead undermine decades of progress, leading to preventable illnesses and deaths.
This is a defining moment for public health in America. Vaccine advocates must work tirelessly and with transparency to promote health literacy and counter misinformation, and senators must fulfill their constitutional advice and consent obligation when it comes to nominees overseeing the American healthcare system. This vetting process transcends partisan politics; it concerns the health and safety of every American.
There’s been a troubling rise in vaccine skepticism, fueled by misinformation. Given the high stakes for America's public health, it is imperative to scrutinize the positions of public figures, especially those whose past stances on medically proven vaccines could jeopardize foundational elements of our healthcare system.
Senators should, respectfully but forcefully, question Mr. Kennedy on his stance on issues such childhood vaccines – and press for clarity. It is crucial to seek clear commitments to preserving access to childhood immunizations and maintaining vaccine schedules essential for public health. Senators should ask Mr. Kennedy about how he would promote trust in science rather than exacerbate skepticism. Finally, RFK Jr.’s potential to use policy levers to alter FDA approval standards, school vaccine mandates, and funding for immunization programs deserve to be honestly and aggressively examined.
Mr. Kennedy's confirmation hearing mustn’t dwell on the past, but on how he plans to safeguard our future. A potential rollback of vaccination programs could lead to outbreaks of preventable diseases like measles, polio, and whooping cough, with devastating consequences for public health. Vaccines are not merely a medical achievement; they are the foundation of a healthy society. The Senate has an opportunity—and an obligation—to ensure they remain protected.
Secretary-Designate Kennedy’s past comments on vaccines should be front and center during his confirmation hearing. They are what everyone is waiting for. It is the sharp tip of the spear for those who support his nomination and for those contrary. The question is whether Mr. Kennedy will be skewered by the spear or use it himself to advance into the corner office at HHS headquarters.
David Brooks, in a recent New York Times op-ed, invented a resonant phrase, “the redistribution of respect.” And that’s applicable on both macro and micro levels. When it comes to the FDA, it can mean a significant magnification and integration of the patient voice, a focus on facilitating innovation, and working with industry to drive enhanced competitiveness.
Over the last decade the agency that regulates nearly a third of the US economy has been lending a more regular and critical ear to the real-life experiences of patients and caregivers and trying (although not always with equal enthusiasm across centers and divisions) to address the many thorny issues relative to clinical trial design, endpoints and biomarkers, safety and efficacy and Phase IV studies. But the status quo is a harsh commandant.
What will the Trump FDA look like? Well, for starters, it will look a lot like it currently does. The FDA has only a handful of political appointees (including the Commissioner) and none reside in any of its regulatory review centers. But, with a new sheriff in town we can expect – and should embrace -- more targeted, regular, and collegial friction. And that’s a good thing. Meaningful change, respectful change can help to grease the skids of 21st century regulatory change. A little creative destruction can go a long way.
What will the Trump FDA look like? Well, it’s not even early days yet, but when I attend JP Morgan early next year, one of my messages will be, “Ladies and Gentlemen – fasten your seatbelts and start your engines.”
Over the last decade the agency that regulates nearly a third of the US economy has been lending a more regular and critical ear to the real-life experiences of patients and caregivers and trying (although not always with equal enthusiasm across centers and divisions) to address the many thorny issues relative to clinical trial design, endpoints and biomarkers, safety and efficacy and Phase IV studies. But the status quo is a harsh commandant.
What will the Trump FDA look like? Well, for starters, it will look a lot like it currently does. The FDA has only a handful of political appointees (including the Commissioner) and none reside in any of its regulatory review centers. But, with a new sheriff in town we can expect – and should embrace -- more targeted, regular, and collegial friction. And that’s a good thing. Meaningful change, respectful change can help to grease the skids of 21st century regulatory change. A little creative destruction can go a long way.
What will the Trump FDA look like? Well, it’s not even early days yet, but when I attend JP Morgan early next year, one of my messages will be, “Ladies and Gentlemen – fasten your seatbelts and start your engines.”
As the old saying goes, “Take care of the pennies and the pounds will take care of themselves.” So true. But, when it comes to smart use and aggressive reimbursement for GLP-1 receptor agonists, the adage must be reversed, “Take care of the pounds and the pennies will take care of themselves.”
A new study in the Proceedings of the National Academy of Science, led by researchers at Yale School of Public Health and the University of Florida, demonstrates that it’s time for Uncle Sam step up and to remove the barriers that are hindering appropriate access to effective weight loss treatments. Simply stated, expanding access to GLP-1s such as Ozempic and Wegovy, and dual gastric inhibitory polypeptide and GLP-1 (GIP/GLP-1) receptor agonists, such as tirzepatide could prevent more than 40,000 deaths a year in the United States. This estimate includes approximately 11,769 deaths among individuals with type 2 diabetes — a group particularly vulnerable to the complications of obesity. Even under current conditions of limited access, the researchers project that around 8,592 lives are saved each year, primarily among those with private insurance.
Per Alison P. Galvani, one of the study's authors and the Burnett and Stender Families Professor of Epidemiology (Microbial Diseases) at the Yale School of Public Health, “Expanding access to these medications is not just a matter of improving treatment options but also a crucial public health intervention. Our findings underscore the potential to reduce mortality significantly by addressing financial and coverage barriers."
The study highlights a critical disparity in drug access. Medicare, for example, doesn’t cover these drugs for weight loss and Medicaid coverage varies widely by state. Expanding access to these medications is not just a matter of improving treatment options but also a crucial public health intervention.
The researchers also considered the impact of socioeconomic factors on the effectiveness of expanded drug access. They adjusted their estimates to account for income disparities, finding that even with these adjustments, the potential for lives saved remains significant.
The study also explored how expanded access could affect different regions and socioeconomic groups. States with high obesity and diabetes rates, such as West Virginia, Mississippi, and Oklahoma, stand to benefit the most from increased medication availability. In these areas, expanding access could lead to the largest per capita reductions in mortality.
According to Dr. Burton H. Singer, PhD, another author of the study and adjunct professor of mathematics at the Emerging Pathogens Institute at the University of Florida. "Addressing these challenges requires a multifaceted approach," "We need to ensure that drug prices are more aligned with manufacturing costs and increase production capacity to meet demand. At the same time, we must tackle the insurance and accessibility issues that prevent many people from getting the treatment they need."
A new study in the Proceedings of the National Academy of Science, led by researchers at Yale School of Public Health and the University of Florida, demonstrates that it’s time for Uncle Sam step up and to remove the barriers that are hindering appropriate access to effective weight loss treatments. Simply stated, expanding access to GLP-1s such as Ozempic and Wegovy, and dual gastric inhibitory polypeptide and GLP-1 (GIP/GLP-1) receptor agonists, such as tirzepatide could prevent more than 40,000 deaths a year in the United States. This estimate includes approximately 11,769 deaths among individuals with type 2 diabetes — a group particularly vulnerable to the complications of obesity. Even under current conditions of limited access, the researchers project that around 8,592 lives are saved each year, primarily among those with private insurance.
Per Alison P. Galvani, one of the study's authors and the Burnett and Stender Families Professor of Epidemiology (Microbial Diseases) at the Yale School of Public Health, “Expanding access to these medications is not just a matter of improving treatment options but also a crucial public health intervention. Our findings underscore the potential to reduce mortality significantly by addressing financial and coverage barriers."
The study highlights a critical disparity in drug access. Medicare, for example, doesn’t cover these drugs for weight loss and Medicaid coverage varies widely by state. Expanding access to these medications is not just a matter of improving treatment options but also a crucial public health intervention.
The researchers also considered the impact of socioeconomic factors on the effectiveness of expanded drug access. They adjusted their estimates to account for income disparities, finding that even with these adjustments, the potential for lives saved remains significant.
The study also explored how expanded access could affect different regions and socioeconomic groups. States with high obesity and diabetes rates, such as West Virginia, Mississippi, and Oklahoma, stand to benefit the most from increased medication availability. In these areas, expanding access could lead to the largest per capita reductions in mortality.
According to Dr. Burton H. Singer, PhD, another author of the study and adjunct professor of mathematics at the Emerging Pathogens Institute at the University of Florida. "Addressing these challenges requires a multifaceted approach," "We need to ensure that drug prices are more aligned with manufacturing costs and increase production capacity to meet demand. At the same time, we must tackle the insurance and accessibility issues that prevent many people from getting the treatment they need."
Yes, once again we’re talking about Bernie Sanders. Here’s the full quote from MacBeth:
“Life's but a walking shadow; a poor player, that struts and frets his hour upon the stage, and then is heard no more: it is a tale told by an idiot, full of sound and fury, signifying nothing.”
That’s the “Scottish Play,” but equally tragic (and a lot more comic) is the way the Senator from Ben and Jerry’s thinks that by talking tough, he’ll be taken seriously. Nope. All syrup, no maple tree.
Since, according to Senator Sanders, Novo Nordisk won’t testify about its GLP-1 agonist products, his committee will issue a subpoena. The game’s afoot. Let’s call it the “Danish Play.”
(Reality check: The Health Committee hasn’t issued a subpoena in more than 40 years.)
Per the Green Mountain State’s favorite son, the Senate Health Committee has “reached out time and time again to schedule Novo Nordisk’s voluntary appearance at a hearing … Unfortunately, despite all of our efforts, they have repeatedly denied our requests.” Nope.
Per a written Novo Nordisk clarification, “The company has responded to every request Sanders has made, and said the company is “committed to a hearing that aligns with the Chairman’s established committee practices … On multiple occasions, we have communicated our CEO’s willingness to testify and offered several dates for a hearing. Based on our continued cooperation, we feel that issuing a subpoena is unnecessary.”
But not nearly as much sturm und drang.
It's also interesting that only one manufacturer is being summoned to testify – and not a single Pharmacy Benefit Manager (PBM) has received a similar honor.
If Senator Sanders wants to be taken seriously, he should recognize that his current approach is … much ado about nothing. Just ask Vermont’s favorite fictional representative, Senator Ortolan Finistirre.
For more, see this excellent reporting by STAT.
“Life's but a walking shadow; a poor player, that struts and frets his hour upon the stage, and then is heard no more: it is a tale told by an idiot, full of sound and fury, signifying nothing.”
That’s the “Scottish Play,” but equally tragic (and a lot more comic) is the way the Senator from Ben and Jerry’s thinks that by talking tough, he’ll be taken seriously. Nope. All syrup, no maple tree.
Since, according to Senator Sanders, Novo Nordisk won’t testify about its GLP-1 agonist products, his committee will issue a subpoena. The game’s afoot. Let’s call it the “Danish Play.”
(Reality check: The Health Committee hasn’t issued a subpoena in more than 40 years.)
Per the Green Mountain State’s favorite son, the Senate Health Committee has “reached out time and time again to schedule Novo Nordisk’s voluntary appearance at a hearing … Unfortunately, despite all of our efforts, they have repeatedly denied our requests.” Nope.
Per a written Novo Nordisk clarification, “The company has responded to every request Sanders has made, and said the company is “committed to a hearing that aligns with the Chairman’s established committee practices … On multiple occasions, we have communicated our CEO’s willingness to testify and offered several dates for a hearing. Based on our continued cooperation, we feel that issuing a subpoena is unnecessary.”
But not nearly as much sturm und drang.
It's also interesting that only one manufacturer is being summoned to testify – and not a single Pharmacy Benefit Manager (PBM) has received a similar honor.
If Senator Sanders wants to be taken seriously, he should recognize that his current approach is … much ado about nothing. Just ask Vermont’s favorite fictional representative, Senator Ortolan Finistirre.
For more, see this excellent reporting by STAT.
Abraham Lincoln said that the patent system adds “the fuel of interest to the fire of genius.”
Alas, when it comes to the majority of innovative healthcare technologies, that “fire of genius” rarely comes from Inside the Beltway. And having Senator Bernie Sanders hold forth on pharmaceutical research, development, and manufacturing costs is about as useful and thought-provoking as listening to my 13-year-old Golden Retriever parse the allegorical vicissitudes of Milton’s Paradise Lost.
Senator Sanders, it’s important to remember, doesn’t even believe in patents, but rather in the failed fantasy land of “innovation prizes." Well, in the words of the late Senator Daniel Patrick Moynihan, “Everyone is entitled to his own opinion, but not to his own facts."
In the past, Senator Sanders introduced a bill that would replace our current patent system for pharmaceuticals with a “Medical Innovation Prize Fund. It’s not a new idea. The prize model has been used in the past by the old Soviet Union – and it didn’t work. The Soviet experience was characterized by low levels of monetary compensation and poor innovative performance.
The US experience isn’t much better. The federal government paid Robert Goddard (the father of American rocketry) $1 million as compensation for his basic liquid rocket patents. A fair price? Not when you consider that during the remaining life of those patents, US expenditures on liquid-propelled rockets amounted to around $10 billion. It’s certainly not what Schumpeter had in mind when he wrote about a “spectacular prize thrown to a small minority of winners.” There’s a difference between “Creative destruction” and destroying medical innovation.
As Joe DiMasi (Tufts University) and Henry Grabowski (Duke University) have argued, under a prize program, pharmaceutical innovators would lack the incentive to innovate. To quote DiMasi and Grabowski, “The dynamic benefits created by patents on pharmaceuticals can, and almost surely do, swamp in significance their short-run inefficiencies.”
As DiMasi and Grabowski presciently observed in 2004, “The main beneficiaries in the short-term would-be private insurers and public sector purchaser of pharmaceuticals. Governments and insurers are focused myopically on managing health care costs. They are not likely to be strong advocates for funding new drug development that can increase individual quality of life and productivity."
Let’s take a break from the effervescent political bloviation and look at the facts. It’s time to put the “intellectual” back in “intellectual property. Pursuing misguided policies that siphon funding from the groundbreaking medical research happening in the biopharmaceutical industry will have devastating consequences for patients and society.
When Senator Sanders begins to wonder why GLP-1 agonists can’t be radically reduced in price, it’s because he doesn’t fully understand or appreciate the ecosystem.
Allow me to draw your attention to a recent US Chamber blog post highlighting how the private sector is providing positive and cost-effective solutions for diabetes patients. You can find that blog here: Conquering Diabetes with Cost-Effective Solutions for Patients | U.S. Chamber of Commerce.
Two key points:
* Free enterprise creates competition in the marketplace to keep costs down. Contrary to critics, innovation in diabetes is being pioneered by private sector companies.
* New treatments are possible because of private-sector innovation. America’s life science companies are advancing diabetes care by developing more effective treatments to help people manage their condition.
What’s that? Did you think that medical innovation only comes from the NIH? Consider the facts:
A study in Health Affairs by Bhaven N. Sampat and Frank R. Lichtenberg (“What Are The Respective Roles of the Public and Private Sectors in Pharmaceutical Innovation?”) puts the issue in a data-driven perspective that gives the NIH its due — but in the proper frame of reference.
Per Sampat and Lichtenberg, less than 10 percent of drugs had a public sector patent, and drugs with public-sector patents accounted for 2.5 percent of sales, but the indirect impact was higher for drugs granted priority review by the FDA. (Priority review is “given to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.”)
“478 drugs in our sample were associated with $132.7 billion in prescription drug sales in 2006. Drugs with public-sector patents accounted for 2.5 percent of these sales, while drugs whose applications cited federally funded research and development or government publications accounted for 27 percent.”
The NIH plays a vital role in basic research and early discovery, but is robbing Productive Peter to pay Government Paul the best bang for the buck when it comes to advancing public health? The answer is a clear "no."
Per the US Chamber report, “Incredible innovative treatments are currently on the market to treat Type 2 diabetes, one of the most serious chronic conditions, which impacts an estimated 35 million Americans. But contrary to what some critics may say, America’s innovative life science companies are advancing diabetes care by developing more effective treatments to help people better manage their condition so that they don’t develop serious complications, including damage to the heart, blood vessels, eyes, kidneys, and nerves.”
Further, “Effective glucose and weight management are key aspects of diabetes care, but only 50% of people with diabetes in the United States achieve their glucose level goals (measured by the HbA1c test). Glucagon-like peptide 1 drugs (GLP-1) have been proven to reduce a person’s A1c by approximately .8 to 1.6%. This is a tremendous innovation for an unpredictable disease that can cause complications like amputations, heart failure, gum disease, and vision loss. “
Diabetes has a major impact on national economies by reducing productivity and life expectancy while increasing disability and health care costs. People with diagnosed diabetes now account for one of every four health care dollars spent in the U.S.
The Economic Report, which is published every five years by the American Diabetes Association, found that the total annual cost of diabetes in 2022 was $412.9 billion, including $306.6 billion in direct medical costs and $106.3 billion in indirect costs.
The Chamber calls it like it is. “Unfortunately, just as millions of Americans are benefiting from these lifesaving and life-altering diabetic treatments, some members of Congress and activist groups are pushing price control policies that will kill future life-science innovation and undermine the ability of patients to access the newest treatments and cures.”
Research from the Chamber shows that in countries with strict price control regimes, patients receive access to fewer cures and have longer wait times than their peers in free market and free enterprise systems like the United States. For the millions of American patients with diabetes, the idea of waiting longer for fewer cures and treatments is simply unacceptable.
The government should get out of the way and let the market drive innovation, to add the fuel of interest to the fire of genius Doing so will not only lead to the newest generation of treatments and cures but will also lead to more options and choices, improving therapeutic outcomes, and lowering costs for patients and taxpayers.
Alas, when it comes to the majority of innovative healthcare technologies, that “fire of genius” rarely comes from Inside the Beltway. And having Senator Bernie Sanders hold forth on pharmaceutical research, development, and manufacturing costs is about as useful and thought-provoking as listening to my 13-year-old Golden Retriever parse the allegorical vicissitudes of Milton’s Paradise Lost.
Senator Sanders, it’s important to remember, doesn’t even believe in patents, but rather in the failed fantasy land of “innovation prizes." Well, in the words of the late Senator Daniel Patrick Moynihan, “Everyone is entitled to his own opinion, but not to his own facts."
In the past, Senator Sanders introduced a bill that would replace our current patent system for pharmaceuticals with a “Medical Innovation Prize Fund. It’s not a new idea. The prize model has been used in the past by the old Soviet Union – and it didn’t work. The Soviet experience was characterized by low levels of monetary compensation and poor innovative performance.
The US experience isn’t much better. The federal government paid Robert Goddard (the father of American rocketry) $1 million as compensation for his basic liquid rocket patents. A fair price? Not when you consider that during the remaining life of those patents, US expenditures on liquid-propelled rockets amounted to around $10 billion. It’s certainly not what Schumpeter had in mind when he wrote about a “spectacular prize thrown to a small minority of winners.” There’s a difference between “Creative destruction” and destroying medical innovation.
As Joe DiMasi (Tufts University) and Henry Grabowski (Duke University) have argued, under a prize program, pharmaceutical innovators would lack the incentive to innovate. To quote DiMasi and Grabowski, “The dynamic benefits created by patents on pharmaceuticals can, and almost surely do, swamp in significance their short-run inefficiencies.”
As DiMasi and Grabowski presciently observed in 2004, “The main beneficiaries in the short-term would-be private insurers and public sector purchaser of pharmaceuticals. Governments and insurers are focused myopically on managing health care costs. They are not likely to be strong advocates for funding new drug development that can increase individual quality of life and productivity."
Let’s take a break from the effervescent political bloviation and look at the facts. It’s time to put the “intellectual” back in “intellectual property. Pursuing misguided policies that siphon funding from the groundbreaking medical research happening in the biopharmaceutical industry will have devastating consequences for patients and society.
When Senator Sanders begins to wonder why GLP-1 agonists can’t be radically reduced in price, it’s because he doesn’t fully understand or appreciate the ecosystem.
Allow me to draw your attention to a recent US Chamber blog post highlighting how the private sector is providing positive and cost-effective solutions for diabetes patients. You can find that blog here: Conquering Diabetes with Cost-Effective Solutions for Patients | U.S. Chamber of Commerce.
Two key points:
* Free enterprise creates competition in the marketplace to keep costs down. Contrary to critics, innovation in diabetes is being pioneered by private sector companies.
* New treatments are possible because of private-sector innovation. America’s life science companies are advancing diabetes care by developing more effective treatments to help people manage their condition.
What’s that? Did you think that medical innovation only comes from the NIH? Consider the facts:
A study in Health Affairs by Bhaven N. Sampat and Frank R. Lichtenberg (“What Are The Respective Roles of the Public and Private Sectors in Pharmaceutical Innovation?”) puts the issue in a data-driven perspective that gives the NIH its due — but in the proper frame of reference.
Per Sampat and Lichtenberg, less than 10 percent of drugs had a public sector patent, and drugs with public-sector patents accounted for 2.5 percent of sales, but the indirect impact was higher for drugs granted priority review by the FDA. (Priority review is “given to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.”)
“478 drugs in our sample were associated with $132.7 billion in prescription drug sales in 2006. Drugs with public-sector patents accounted for 2.5 percent of these sales, while drugs whose applications cited federally funded research and development or government publications accounted for 27 percent.”
The NIH plays a vital role in basic research and early discovery, but is robbing Productive Peter to pay Government Paul the best bang for the buck when it comes to advancing public health? The answer is a clear "no."
Per the US Chamber report, “Incredible innovative treatments are currently on the market to treat Type 2 diabetes, one of the most serious chronic conditions, which impacts an estimated 35 million Americans. But contrary to what some critics may say, America’s innovative life science companies are advancing diabetes care by developing more effective treatments to help people better manage their condition so that they don’t develop serious complications, including damage to the heart, blood vessels, eyes, kidneys, and nerves.”
Further, “Effective glucose and weight management are key aspects of diabetes care, but only 50% of people with diabetes in the United States achieve their glucose level goals (measured by the HbA1c test). Glucagon-like peptide 1 drugs (GLP-1) have been proven to reduce a person’s A1c by approximately .8 to 1.6%. This is a tremendous innovation for an unpredictable disease that can cause complications like amputations, heart failure, gum disease, and vision loss. “
Diabetes has a major impact on national economies by reducing productivity and life expectancy while increasing disability and health care costs. People with diagnosed diabetes now account for one of every four health care dollars spent in the U.S.
The Economic Report, which is published every five years by the American Diabetes Association, found that the total annual cost of diabetes in 2022 was $412.9 billion, including $306.6 billion in direct medical costs and $106.3 billion in indirect costs.
The Chamber calls it like it is. “Unfortunately, just as millions of Americans are benefiting from these lifesaving and life-altering diabetic treatments, some members of Congress and activist groups are pushing price control policies that will kill future life-science innovation and undermine the ability of patients to access the newest treatments and cures.”
Research from the Chamber shows that in countries with strict price control regimes, patients receive access to fewer cures and have longer wait times than their peers in free market and free enterprise systems like the United States. For the millions of American patients with diabetes, the idea of waiting longer for fewer cures and treatments is simply unacceptable.
The government should get out of the way and let the market drive innovation, to add the fuel of interest to the fire of genius Doing so will not only lead to the newest generation of treatments and cures but will also lead to more options and choices, improving therapeutic outcomes, and lowering costs for patients and taxpayers.
Somewhere Bernie Sanders is smiling.
But he shouldn’t be.
A recent study (published in JAMA) found that "estimated cost-based prices" for a range of common diabetes drugs were "substantially lower than current market prices" -- and not just in developed countries like the United States, but also in developing ones like India and Bangladesh.
This finding, while technically true, is worse than uninformative. It is misinformative.
One doesn't need a PhD in economics to understand why the literal cost of manufacturing an already invented medicine is low -- or why it's irrelevant to the medication's much higher development costs and risk premium necessary to incentivize development in the first place. By the authors' logic, the price of a Microsoft Office subscription ought to be a nickel annually (instead of $150) because the software code already exists and the prorated electricity cost for cloud computing is minimal.
The authors further suggest that "robust generic and biosimilar competition could reduce prices" -- and that such competition could be facilitated by "a range of policy tools" including "price controls," "compulsory licensing," and a general weakening of intellectual property protections.
Given that the study finds that prices are supposedly unjustifiably high even developing countries with comparative weak IP protections and mandated price controls, the authors are implicitly endorsing a global IP and price control regime that's more aggressive than even India's and South Africa's.
That would eliminate any incentive to ever invest in developing a new drug again. Wiping out the global drug industry is hardly a good way to "enable expansion of diabetes treatment globally."
And, jeez – sloppy peer review!
But he shouldn’t be.
A recent study (published in JAMA) found that "estimated cost-based prices" for a range of common diabetes drugs were "substantially lower than current market prices" -- and not just in developed countries like the United States, but also in developing ones like India and Bangladesh.
This finding, while technically true, is worse than uninformative. It is misinformative.
One doesn't need a PhD in economics to understand why the literal cost of manufacturing an already invented medicine is low -- or why it's irrelevant to the medication's much higher development costs and risk premium necessary to incentivize development in the first place. By the authors' logic, the price of a Microsoft Office subscription ought to be a nickel annually (instead of $150) because the software code already exists and the prorated electricity cost for cloud computing is minimal.
The authors further suggest that "robust generic and biosimilar competition could reduce prices" -- and that such competition could be facilitated by "a range of policy tools" including "price controls," "compulsory licensing," and a general weakening of intellectual property protections.
Given that the study finds that prices are supposedly unjustifiably high even developing countries with comparative weak IP protections and mandated price controls, the authors are implicitly endorsing a global IP and price control regime that's more aggressive than even India's and South Africa's.
That would eliminate any incentive to ever invest in developing a new drug again. Wiping out the global drug industry is hardly a good way to "enable expansion of diabetes treatment globally."
And, jeez – sloppy peer review!
Excellent piece by Ed Silverman (as always) on the increased usage of Hyrimoz. A few issues that need to be discussed in greater detail: (1) Does this bring us back to the discussion of "biobetters?" If so, should we differentiate between de novo patients and those already on treatment? (2) Will we only see an uptick in biosimilar prescriptions if PBMs can aggressively monitize them? What about putting patients first and not disintermediating healthcare providers? (3) Why is the FDA changing course on its interchangeability guidences AFTER President Biden called for such a course change? Whether or not the science supports such a move, the optics stink of political interference. Asking for a friend. #anotherfdaprocessfoul
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.
