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Posts by Program On Regulation, Therapeutics, And Law (PORTAL)

International Reference Pricing for Prescription Drugs in the United States: Administrative Limitations and Collateral Effects Many countries use international (or external) reference pricing—benchmarking prices against those in other countries—to manage spending on prescription drugs. By contrast, the United States (US) allo...

Lots of talk about international reference pricing today. With that in mind, sharing this great article by @leah-rand.bsky.social and @akesselheim.bsky.social from @portalresearch.org!

www.valueinhealthjournal.com/article/S109...

11 months ago 1 1 0 1

@akesselheim.bsky.social @benro.me @wbfeldman.bsky.social @leah-rand.bsky.social @araymakers.bsky.social @kellyanderson.bsky.social @harvardmed.bsky.social

11 months ago 2 0 0 0
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Home Truth, Power, and the Drugs You Take

⭐Out now from Dr. Jerry Avorn— RETHINKING MEDICATIONS: Truth, Power, and the Drugs You Take

In his new book, Dr. Avorn explains why some of the medications we take are poorly evaluated, overpriced, or pose unwarranted risks—and how we fix that.

📖 Grab a copy today: rethinkmeds.info

11 months ago 6 5 1 1

@akesselheim.bsky.social @benro.me @wbfeldman.bsky.social

11 months ago 0 0 0 0
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Opinion | It’s not hard to see through RFK Jr.’s ‘radical transparency’ pledge So far, what’s been released is little more than tactics in pursuit of Kennedy’s own agenda.

New op-ed with @drjoshs.bsky.social and Peter Lurie about the agenda-driven reality of 'radical transparency' -- www.washingtonpost.com/opinions/202...

11 months ago 4 2 0 1

View his full testimony before the Health Subcommittee: waysandmeans.house.gov/event/health...

Read his written testimony: waysandmeans.house.gov/wp-content/u...

1 year ago 1 0 0 0
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And, on strategies to improve the biosimilar market, including by implementing policies to facilitate biosimilar interchangeability, clear patent thickets, and preventing biosimilar rebate gaming by PBMs.

1 year ago 2 2 1 0
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On protecting Medicaid - “Cutbacks planned for the Medicaid program will force states to severely limit their drug coverage, which will inevitably lead to patient deaths.”

1 year ago 1 1 1 0
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WATCH: @akesselheim.bsky.social testifies before @waysmeanscmte.bsky.social

On NIH cuts - "...the next generation will look back at this moment & ask what we were doing while the Trump administration systematically disposed of 1000s of research projects that could've helped patients."

1 year ago 11 9 1 1
Health Subcommittee Hearing on Lowering Costs for Patients: The Health of the Biosimilar Market
Health Subcommittee Hearing on Lowering Costs for Patients: The Health of the Biosimilar Market YouTube video by Ways and Means Committee Republicans

At 10am Eastern Time, watch as our Editor-in-Chief, @akesselheim.bsky.social , gives testimony to the House Ways and Means Committee on "Lowering Costs for Patients: The Health of the Biosimilar Market." www.youtube.com/watch?v=x_rh...

1 year ago 0 2 0 0

@waysmeanscmte.bsky.social

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Health Subcommittee Hearing on Lowering Costs for Patients: The Health of the Biosimilar Market
Health Subcommittee Hearing on Lowering Costs for Patients: The Health of the Biosimilar Market YouTube video by Ways and Means Committee Republicans

Tune in TODAY at 10am ET as PORTAL Director Aaron Kesselheim @akesselheim.bsky.social testifies before the House Ways & Means Health Subcommittee:

He'll discuss the biosimilar market, threats to US biomedical research funding, & the need to protect & expand Medicare drug price negotiation.

1 year ago 0 0 1 0
Perspective 
"Without access to accurate and timely data, scientists’ work will become more difficult, and we will understand less about the world." 

"Disappearing Data at the U.S. Federal Government" by Janet Freilich, J.D., W. Nicholson Price II, J.D., Ph.D., and Aaron S. Kesselheim, M.D., J.D., M.P.H.

Perspective "Without access to accurate and timely data, scientists’ work will become more difficult, and we will understand less about the world." "Disappearing Data at the U.S. Federal Government" by Janet Freilich, J.D., W. Nicholson Price II, J.D., Ph.D., and Aaron S. Kesselheim, M.D., J.D., M.P.H.

Starting in January 2025, various government health datasets were removed, restored, manipulated, or not posted as planned. The authors of a new Perspective write that the consequences of these changes could be far-reaching. Read the full Perspective: nej.md/4bYvujL

#MedSky #HealthPolicy

1 year ago 27 19 1 2
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Just out @jama.com

Led by Dr Hwang we show when oncology drugs withdrawn due to negative trials are still accessible off-abel, prescribing plummets anyway

Romidepsin a notable exception, unsurprising to lymphoma docs who use it in TFH lymphomas

jamanetwork.com/journals/jam... @portalresearch.org

1 year ago 5 1 0 0
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PORTAL Post | April 2025 Skinny labeling, 340B expansion, patent games, and more analysis from the PORTAL team.

Check out the latest research from the PORTAL team in the April edition of our newsletter, featuring analysis on generic drug 'skinny labeling,' the expansion of the 340B program, a new form of product hopping, and much more! 🛟🧪

mailchi.mp/57ccd5e0b342...

1 year ago 0 1 0 1
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Disappearing Data at the U.S. Federal Government | NEJM Starting in January 2025, various government health datasets were removed, restored, manipulated, or not posted as planned. The consequences of these changes could be far-reaching.

NEW in @nejm.org: Janet Freilich & couthors discuss the sudden removal of critical health datasets by US federal health agencies—undermining research, public health, & policy-making. Though lawsuits have restored some data, the authors call for broader action to keep taxpayer-funded data public. 🛟

1 year ago 2 1 1 0

@akesselheim.bsky.social @nicholson.bsky.social

1 year ago 0 0 0 0
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The Moral Injury of Inhaler Prescribing | NEJM Many inhalers that are vital for patients with asthma or COPD are associated with societal harms that can worsen the very diseases the products are designed to treat. How can this dilemma be resolved?

Our latest in @nejm.org on the dilemmas of treating patients with chronic lung disease.

www.nejm.org/doi/full/10....

1 year ago 6 2 3 1
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They conclude: "If the FDA is going to continue to exercise maximal flexibility in considering when to approve highly promising drugs for patients in desperate need of treatment options, we believe policymakers should also protect patients from the economic implications of those decisions."

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Out of Pocket Getting Out of Hand — Reducing the Financial Toxicity of Rapidly Approved Drugs | NEJM The FDA often exercises flexibility in deciding whether to approve highly promising drugs for patients in desperate need of treatment options. But it doesn’t consider a drug’s likely financial toxi...

New perspective in @nejm.org from Peter Ubel, Astrid Grouls, & @akesselheim.bsky.social on the financial toxicity patients face from high-cost drugs with uncertain clinical benefits, & the remedies available to policymakers to mitigate financial harms while drugs undergo further review.

1 year ago 0 0 1 0
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Spending After Sacubitril-Valsartan vs Renin-Angiotensin System Blockers for Heart Failure This cohort study assesses health care costs after initiating sacubitril-valsartan compared to an angiotensin-converting enzyme inhibitor or an angiotensin II receptor-blocker in Medicare patients bei...

Analysis from former @portalresearch.org fellow Catherine Hwang and @benro.me showing the extent of out-of-pocket costs associated with starting brand-name Entresto over ACEI/ARB for heart failure and implications for overall spending: jamanetwork.com/journals/jam...

1 year ago 2 1 0 0
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Physician Experiences With and Perspectives on Clozapine Prescribing This survey study examines physician-prescriber experiences with and perspectives on the clozapine risk evaluation and mitigation strategy.

Survey of clozapine prescribers shows satisfaction with the REMS but desire for more educational info, led by @asarpatwari.bsky.social & team at @portalresearch.org in collab with FDA, where world class scientists are sadly being censored and forced out of their jobs: jamanetwork.com/journals/jam...

1 year ago 3 2 0 0
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Association Between Industry Payments and Prescription of Inhaled Medications - PubMed Association Between Industry Payments and Prescription of Inhaled Medications

Physicians who receive money from inhaler manufacturers prescribe more inhalers in the future. New findings from Hasan Nadeem, @wbfeldman.bsky.social &co:

pubmed.ncbi.nlm.nih.gov/39937634/

1 year ago 3 2 0 0
Glucose-Lowering Medications and Risk of Chronic Obstructive Pulmonary Disease Exacerbations in Patients With Type 2 Diabetes, JAMA Internal Medicine.

Glucose-Lowering Medications and Risk of Chronic Obstructive Pulmonary Disease Exacerbations in Patients With Type 2 Diabetes, JAMA Internal Medicine.

🔎 New in JAMA IM: Avik Ray, @wbfeldman.bsky.social & colleagues assess the comparative effectiveness of diabetes drugs in COPD patients.

They find GLP-1s & SGLT-2s are associated with lower risks of moderate or severe COPD exacerbations vs. DPP-4s during follow-up: jamanetwork.com/journals/jam...

1 year ago 2 1 0 0
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PORTAL Post | February 2025 Protecting health agency independence, drug repurposing, academic detailing, and more from the PORTAL team.

Check out the February edition of the PORTAL Post, featuring analysis on:

- the promise and risk of drug repurposing,
- drug importation to address shortages,
- use of subgroup analysis in cost-effectiveness studies
- biosimilar patent litigation,
- and much more!

#medsky #healthpolicy

1 year ago 5 2 0 1

New from Chris Cai, @akesselheim.bsky.social and @benro.me in JAMA:

Despite having gone off patent decades ago, repurposing the antidepressant doxepin for insomnia has led to persistent high prices even though generics are available, highlighting the need for policy reform in drug repurposing.

1 year ago 3 2 0 0
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How to protect HHS, FDA, NIH, and other health agencies from political interference When politicians subordinate scientific institutions to electoral interests, they undermine immediate public health efforts and inflict lasting damage on institutional credibility.

New piece led by Anushka Bhaskar @portalresearch.org in STAT First Opinion on protecting public health agencies from damaging political interference -- even the threat of which can greatly affect people's behavior: www.statnews.com/2025/01/31/p...

1 year ago 6 5 0 1
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Trials Preceding FDA Approval of Novel Psychiatric Drugs This cross-sectional study reports on the study design characteristics of clinical trials for novel psychiatric drugs approved by the US Food and Drug Administration.

From Rosa Ahn-Horst @portalresearch.org -- psychiatric drug innovation over the last decade shows widespread use of clinical endpoints but few new mechanisms of action and 3/16 drugs for which < half the trials submitted to FDA were positive (in JAMA Network Open): jamanetwork.com/journals/jam...

1 year ago 4 2 0 0
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🚨 Just out today (online)! 🚨

Our results show that a common argument against #QALYs (based on the use of subgroup analyses) is not justified with empirical data.

With co-authors: @leah-rand.bsky.social , @wbfeldman.bsky.social, & @akesselheim.bsky.social from @portalresearch.org.

1 year ago 10 7 1 0

📣 Check out the latest in @nejm.org from PORTAL and collaborators at @cfr.org on the role of importation in alleviating generic drug shortages in the short term, particularly for generic sterile injectable drugs.

1 year ago 1 0 0 0