Latest Drugwonks' Blog
When it comes to Robert F. Kennedy, Jr., I refuse to yell. I refuse to name-call. I refuse to sign FaceBook petitions with cut-and-paste social media tirades. I refuse to predict the end of the world – because I want to make a positive difference. I want my voice to be heard by those in positions of authority and influence. I’ve been around the block enough times to recognize that requires being persistent, patient, persuasive, polite, respectful, and humble. I refuse to reflexively kowtow to my betters. I refuse to be a sock-puppet for science. This doesn’t make me an “enabler.” It makes me a realist.
The mainstream, well-meaning, and devoted public health community has, in very short order, effectively zeroed out any influence they might have had with the new administration by making their attacks judgmental and personal and the media has been all to glad to magnify these lapses in rhetorical judgment. Two words – stop it.
Here’s my message to those who believe they have somehow earned the right to peer down their peer-reviewed noses and at those with fewer letters after their names – get over yourselves. You’re on the outside looking in and your Nobels ring no bells with RFK2. As another Kennedy once said, “Life is unfair.” This is your wake-up call.
I’m not a science-denier. It’s not what “the experts” are saying that’s wrong (although sometimes it is). It’s the way they’re saying it. It’s as useless as trying make a non-English speaker understand you by TALKING LOUDER – and a whole lot more dangerous. Exaggeration is the enemy of belief and trust – and its bad science. Exaggeration is misinformation by another name.
Not everything the new RFK2 crew are doing represents an end to civilization as we know it. The daily hand wringing and predictions of doom-and-gloom by professors and pundits show how little they have learned from the pandemic experience. “It’s settled science, so shut up,” didn’t work during the dark days of Covid-19 – and we continue to reap the whirlwind. Ringing the alarm bells all day every day just makes for annoying ambient noise that becomes increasingly easy to ignore.
It’s time for less atonal cowbell and more determined, cool and collected engagement.
Turning up the volume on pomposity is the height of self-importance and only serves to turn real experts into late-night punchlines. It’s time for America’s public health establishment to get out from behind the sheltered protection of the lectern, and onto a more collegial playing field.
What? Engagement with that bunch of yahoos? Well, first of all, that’s rude – and secondly, that’s right. You may have tenure, but that doesn’t give you much if any policy heft in the world of realpolitik. Otto von Bismarck reminds us, “Politics is the art of the possible.” There’s no ivy on the walls of HHS headquarters. Sometimes you have to eat some crow if you want a seat at the table. Don’t believe me? Ask Volodymyr Zelenskyy.
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.
The mainstream, well-meaning, and devoted public health community has, in very short order, effectively zeroed out any influence they might have had with the new administration by making their attacks judgmental and personal and the media has been all to glad to magnify these lapses in rhetorical judgment. Two words – stop it.
Here’s my message to those who believe they have somehow earned the right to peer down their peer-reviewed noses and at those with fewer letters after their names – get over yourselves. You’re on the outside looking in and your Nobels ring no bells with RFK2. As another Kennedy once said, “Life is unfair.” This is your wake-up call.
I’m not a science-denier. It’s not what “the experts” are saying that’s wrong (although sometimes it is). It’s the way they’re saying it. It’s as useless as trying make a non-English speaker understand you by TALKING LOUDER – and a whole lot more dangerous. Exaggeration is the enemy of belief and trust – and its bad science. Exaggeration is misinformation by another name.
Not everything the new RFK2 crew are doing represents an end to civilization as we know it. The daily hand wringing and predictions of doom-and-gloom by professors and pundits show how little they have learned from the pandemic experience. “It’s settled science, so shut up,” didn’t work during the dark days of Covid-19 – and we continue to reap the whirlwind. Ringing the alarm bells all day every day just makes for annoying ambient noise that becomes increasingly easy to ignore.
It’s time for less atonal cowbell and more determined, cool and collected engagement.
Turning up the volume on pomposity is the height of self-importance and only serves to turn real experts into late-night punchlines. It’s time for America’s public health establishment to get out from behind the sheltered protection of the lectern, and onto a more collegial playing field.
What? Engagement with that bunch of yahoos? Well, first of all, that’s rude – and secondly, that’s right. You may have tenure, but that doesn’t give you much if any policy heft in the world of realpolitik. Otto von Bismarck reminds us, “Politics is the art of the possible.” There’s no ivy on the walls of HHS headquarters. Sometimes you have to eat some crow if you want a seat at the table. Don’t believe me? Ask Volodymyr Zelenskyy.
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.
When speaking this past week at the White House, Oracle founder Larry Ellison said that the newly announced ‘Stargate’ – an artificial intelligence infrastructure project – will pave the way to the United States developing a ‘cancer vaccine.” Well, as my grandmother used to say, “From his mouth to God’s ears.”
In the past, such advances have led our country to longer, healthier lives, to save premature babies, and irradicate devastating diseases. Innovators should be rewarded for propelling the future of care and treatment options for the millions of Americans. Instead, the unfortunate reality is that innovators now stand to be punished due to outdated policies governing Medicare administered by third party middlemen with financial incentive to deny access to innovative diagnostic testing.
Crucially, finding a way to eradicate cancers, one of our planet’s most devastating diseases, will be in vain without advancements in diagnostic testing. However, this comes with an urgent caveat -- this future will not be fulfilled if healthcare middlemen continue to have their way.
The 68 million Americans covered by Medicare (including me) have their claims processed by private insurers known as Medicare Administrative Contractors (MACs). Last month, two of those MACs – Novitas Solutions and First Coast Service Options (FCSO) – issued a Local Coverage Determination (LCD) which ended the coverage of nine groundbreaking cancer diagnostic tests. It’s a warning shot across the bow to precision medicine and investment in innovation.
These decisions must not stand. Early detection is a critical step of prevention, especially for cancer patients. Tests like these allow doctors to intervene and understand their patients’ care far earlier into their diagnosis, enabling them to develop more personalized and cost-efficient treatment plans that, most importantly, improve outcomes. When such care is delayed or inaccessible, there are often greater problems associated with treatment, such as more intensive intervention options like biopsies or radiation therapy or potentially missed and undertreated disease. This comes alongside higher costs of care and, unsettlingly, a lower chance of survival.
There also must be high alert for “regulatory mission creep.” That’s why the 21st Century Cures Act included additional guidelines on the MACs’ LCD process and determination timeline. MACs delaying their decision and blatantly diverting from the Cures Act is a flagrant breaking of the law that has become the norm for MACs – not an exception – and certainly not an exemption.
Cancer patients are not the only ones negatively impacted by these actions. In 2023, the MAC Palmetto GBA, published an article to the CMS website, changing the coverage of two tests for organ transplant patients --one of which could predict rejection problems with transplanted organs weeks or even months ahead of clinical signs of damage.
Luckily, CMS stepped in and coverage for the blood tests was restored. That was not without struggle and hardships for patients and their loved ones. In the process, it also came to light that the MAC ignored the recommendations of medical experts when making the decision to deny coverage.
The bad news is that CMS is limiting access to cutting-edge diagnostics for those on Medicare. This negligence is unacceptable. It is regulatory malpractice. CMS and MACs must be held accountable and reduce uncertainty for patients, healthcare providers, and innovators alike instead of allowing critical coverage decisions to cause greater uncertainty in the future of American healthcare. Additional Congressional oversight should push CMS and their MAC henchmen to reexamine their coverage decisions that impact seniors’ access to novel healthcare and increase taxpayer burden.
If we are to more fully and rapidly realize a world where we can detect cancer, organ failure, and a host of diseases before they occur, we must establish the foundations and incentives to develop testing and treatments. This is the goal of Mr. Ellison’s Stargate initiative – and it is one worth pursuing aggressively.
Peter J. Pitts is president and co-founder of the Center for Medicine in the Public Interest and a former associate commissioner of the U.S. Food and Drug Administration.
In the past, such advances have led our country to longer, healthier lives, to save premature babies, and irradicate devastating diseases. Innovators should be rewarded for propelling the future of care and treatment options for the millions of Americans. Instead, the unfortunate reality is that innovators now stand to be punished due to outdated policies governing Medicare administered by third party middlemen with financial incentive to deny access to innovative diagnostic testing.
Crucially, finding a way to eradicate cancers, one of our planet’s most devastating diseases, will be in vain without advancements in diagnostic testing. However, this comes with an urgent caveat -- this future will not be fulfilled if healthcare middlemen continue to have their way.
The 68 million Americans covered by Medicare (including me) have their claims processed by private insurers known as Medicare Administrative Contractors (MACs). Last month, two of those MACs – Novitas Solutions and First Coast Service Options (FCSO) – issued a Local Coverage Determination (LCD) which ended the coverage of nine groundbreaking cancer diagnostic tests. It’s a warning shot across the bow to precision medicine and investment in innovation.
These decisions must not stand. Early detection is a critical step of prevention, especially for cancer patients. Tests like these allow doctors to intervene and understand their patients’ care far earlier into their diagnosis, enabling them to develop more personalized and cost-efficient treatment plans that, most importantly, improve outcomes. When such care is delayed or inaccessible, there are often greater problems associated with treatment, such as more intensive intervention options like biopsies or radiation therapy or potentially missed and undertreated disease. This comes alongside higher costs of care and, unsettlingly, a lower chance of survival.
There also must be high alert for “regulatory mission creep.” That’s why the 21st Century Cures Act included additional guidelines on the MACs’ LCD process and determination timeline. MACs delaying their decision and blatantly diverting from the Cures Act is a flagrant breaking of the law that has become the norm for MACs – not an exception – and certainly not an exemption.
Cancer patients are not the only ones negatively impacted by these actions. In 2023, the MAC Palmetto GBA, published an article to the CMS website, changing the coverage of two tests for organ transplant patients --one of which could predict rejection problems with transplanted organs weeks or even months ahead of clinical signs of damage.
Luckily, CMS stepped in and coverage for the blood tests was restored. That was not without struggle and hardships for patients and their loved ones. In the process, it also came to light that the MAC ignored the recommendations of medical experts when making the decision to deny coverage.
The bad news is that CMS is limiting access to cutting-edge diagnostics for those on Medicare. This negligence is unacceptable. It is regulatory malpractice. CMS and MACs must be held accountable and reduce uncertainty for patients, healthcare providers, and innovators alike instead of allowing critical coverage decisions to cause greater uncertainty in the future of American healthcare. Additional Congressional oversight should push CMS and their MAC henchmen to reexamine their coverage decisions that impact seniors’ access to novel healthcare and increase taxpayer burden.
If we are to more fully and rapidly realize a world where we can detect cancer, organ failure, and a host of diseases before they occur, we must establish the foundations and incentives to develop testing and treatments. This is the goal of Mr. Ellison’s Stargate initiative – and it is one worth pursuing aggressively.
Peter J. Pitts is president and co-founder of the Center for Medicine in the Public Interest and a former associate commissioner of the U.S. Food and Drug Administration.
Here’s the good news: Medicare Advantage is one of our nation’s most successful public-private partnerships, providing efficient and effective access for many millions of American seniors to cost-effective healthcare coverage. The bad news is that Medicare Advantage is under threat from a Biden-era proposal that will spell disaster for the Medicare Advantage Program if nothing is done.
For the last two decades, the Centers for Medicare and Medicaid Services (CMS) has used a single normalization factor as a basis to calculate payment rates for both Medicare Advantage Prescription Drug plans (MA-PDs) and standalone Part D plans (PDPs – the Medicare Prescription Drug Benefit). (A normalization factor is used to account for changes in the health status and demographics of traditional Medicare beneficiaries.)
Without going into the heavy math – let’s suffice it to say this makes sense. A single normalization factor allows both MA-PDs and PDPs to compete in the marketplace on a level-playing field and resulting competition has led to lower out-of-pocket costs and more coverage options for millions of American seniors.
Rather than leaving well enough alone, the Biden Administration and Democrats in Congress (always suspicious of public/private partnerships – even successful ones) opted to separate the normalization factors for MA-PDs and PDPs, effectively lowering the payments made to MA-PDs to ensure PDPs got more money -- a shell game aimed at stabilizing the PDP market at the expense of MA-PD beneficiaries. The new normalization factor methodology will result in inaccurate risk scores. What does this mean? Artificial risk scores divorced from reality. In 2025, the difference in normalization factors was 12.4% higher for MA-PDs, dragging down the risk scores. That jump is 34.6% in 2026, nearly tripling.
Unfortunately for these denizens of Big Government, Part D drug plans have been far less efficient when it comes to delivering drug benefits for enrollees compared to Medicare Advantage; monthly premiums for standalone PDPs average $45 compared to just $7 for MA-PDs. These ill-considered (but not unpredictable) policy decisions, baked into the Inflation Reduction Act, resulted in disastrous effects on both the Medicare Advantage and Part D markets. Predictably, premiums went through the roof and many Part D plans went away entirely.
Make no mistake, if allowed to happen, split normalization factors will lead to less choice and higher costs for beneficiaries enrolled in Medicare Advantage. CMS’s separate normalization factor policy favors less-efficient standalone Part D plans over more-efficient MA-PD plans. By harming Medicare Advantage in order to bolster Part D, CMS is robbing Peter to pay Paul.
With a new administration now in the White House and the Biden-era receding into history, it is imperative that we not allow the policy failures of the Biden administration to undermine the integrity of the successful Medicare Advantage program. Leaders in Congress must take action to stop separate normalization factors from becoming the new normal. We mustn’t allow the bureaucrats at CMS to normalize Biden-era mistakes while no one is looking.
It's time for some of what HHS Secretary Kennedy has called, “radical transparency.”
For the last two decades, the Centers for Medicare and Medicaid Services (CMS) has used a single normalization factor as a basis to calculate payment rates for both Medicare Advantage Prescription Drug plans (MA-PDs) and standalone Part D plans (PDPs – the Medicare Prescription Drug Benefit). (A normalization factor is used to account for changes in the health status and demographics of traditional Medicare beneficiaries.)
Without going into the heavy math – let’s suffice it to say this makes sense. A single normalization factor allows both MA-PDs and PDPs to compete in the marketplace on a level-playing field and resulting competition has led to lower out-of-pocket costs and more coverage options for millions of American seniors.
Rather than leaving well enough alone, the Biden Administration and Democrats in Congress (always suspicious of public/private partnerships – even successful ones) opted to separate the normalization factors for MA-PDs and PDPs, effectively lowering the payments made to MA-PDs to ensure PDPs got more money -- a shell game aimed at stabilizing the PDP market at the expense of MA-PD beneficiaries. The new normalization factor methodology will result in inaccurate risk scores. What does this mean? Artificial risk scores divorced from reality. In 2025, the difference in normalization factors was 12.4% higher for MA-PDs, dragging down the risk scores. That jump is 34.6% in 2026, nearly tripling.
Unfortunately for these denizens of Big Government, Part D drug plans have been far less efficient when it comes to delivering drug benefits for enrollees compared to Medicare Advantage; monthly premiums for standalone PDPs average $45 compared to just $7 for MA-PDs. These ill-considered (but not unpredictable) policy decisions, baked into the Inflation Reduction Act, resulted in disastrous effects on both the Medicare Advantage and Part D markets. Predictably, premiums went through the roof and many Part D plans went away entirely.
Make no mistake, if allowed to happen, split normalization factors will lead to less choice and higher costs for beneficiaries enrolled in Medicare Advantage. CMS’s separate normalization factor policy favors less-efficient standalone Part D plans over more-efficient MA-PD plans. By harming Medicare Advantage in order to bolster Part D, CMS is robbing Peter to pay Paul.
With a new administration now in the White House and the Biden-era receding into history, it is imperative that we not allow the policy failures of the Biden administration to undermine the integrity of the successful Medicare Advantage program. Leaders in Congress must take action to stop separate normalization factors from becoming the new normal. We mustn’t allow the bureaucrats at CMS to normalize Biden-era mistakes while no one is looking.
It's time for some of what HHS Secretary Kennedy has called, “radical transparency.”
Peter J. Pitts, a former FDA Associate Commissioner, and member of the United States Senior Executive Service, is President of the Center for Medicine in thePublic Interest.
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.
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.
