We identified a number of ways to rebuild that trust and hope to share those ideas with you.
icer.org/news-insight...

But significant challenges remain around evidence quality, transparency, and financial incentives. These boil down to a one theme that is now familiar in our political discourse: trust, and the lack thereof.

It was a good moment to learn about what's improved and where we have unfinished business. We found that changes following FDORA have been largely well received - encouraging news that reform to improve performance works!

A lot changed in the 5 years since that report. FDORA gave FDA new enforcement tools. The agency issued updated guidance on surrogate endpoints and confirmatory trials and initiated a number of withdrawals. And the new administration has fundamentally reshaped the regulatory environment.

New from Institute for Clinical and Economic Review (ICER) and Verdant Research: We revisited our 2021 white paper on FDA's accelerated approval pathway.
icer.org/news-insight...

Scoop: Vinay Prasad is out at FDA. HHS spokesperson said "he did not want to be a distraction to the great work of the FDA in the Trump administration."
Story TK in Pink Sheet.

Pharmaceutical Innovation and the IRA: 2024 in Review | Resources for Innovations in Care | ATI Advisory ATI evaluates pharmaceutical innovation in the context of the 2025 implementation of the Part D redesign and political uncertainties.

5. Part D Redesign in the spotlight: While Medicare negotiation got less airtime, companies gave more detail on the effects of the 2025 cost-shift in Part D — up to $2B in projected revenue hits for some firms.

Get more details in our report: atiadvisory.com/resources/ph...

4. China on the Rise: Major players are increasingly licensing directly from Chinese biotechs, especially in oncology and metabolic disease—cutting out US VC and biotech.

3. Small molecule development alive and well: Among the 37 deals struck in 2023 and 2024 that exceeded $1bn, 19 (51%) had small molecules as lead assets. Over the same timeframe, companies discontinued 79 products, of which 36 were small molecules.

2. Deal Size Down, Risk Appetite Up: M&A value fell 68% vs. 2023, with a clear pivot toward early-stage and preclinical assets — with many assets maturing to late stage, companies are turning to smaller, earlier stage opportunities.

1. R&D Momentum Holds Strong: Companies continued to double down on late-stage key assets, with R&D spend growing another 11% yoy. Not surprising – 62% of global sales come from drugs that face loss of exclusivity by 2029. The 25 drugs selected for negotiation accounted for 10% of US sales.

🚨 Top 5 Takeaways from our 2024 Biopharma Wrap-Up 🚨

Every 6 months, my team and I review biopharma R&D and investment decisions. Our latest report examines 2024. Much of the last year was shaped by the ongoing race to fill impending revenue gaps as older drugs face LOE. More below 👇

🩺📊 #medsky

Is U.S. health care in a "gilded age"? A New Yorker article argues that we are in "the gilded age of medicine." So what does that mean for U.S. health care? Dr. Dhruv Khullar, associate professor of health and economics at Weill Cornell Me...

I had a chance to join CBS News and Major Garrett to discuss my recent @newyorker.com piece on the consequences of money in medicine. Is U.S. health care entering a "gilded age"?

Vertex touts pain drug results, but medicine does not outperform placebo in sciatica study A Vertex drug hit its mark in a Phase 2 study in chronic pain, but faces questions about its performance versus placebo.

Vertex $VRTX touts pain drug results, but medicine does not outperform placebo in sciatica study www.statnews.com/2024/12/19/v... via @matthewherper.bsky.social

National Health Spending Explorer - Peterson-KFF Health System Tracker This interactive tool provides up-to-date information on U.S. health spending by federal and local governments, private companies, and individuals. It was developed by KFF using data from the National...

At @kff.org, we’ve been busy updating our interactive with national health spending data released yesterday.

Explore trends in US health care costs from 1960-2023 and make your own charts using this tool:

www.healthsystemtracker.org/health-spend...

Opinion | In This House, We’re Angry When Government Fails The Democratic Party needs to rethink what it means to be the party of institutions.

www.nytimes.com/2024/11/22/o...

Every drug I used when I took care of kids with cancer had serious risks.

The only thing that could justify my using them was that their benefits outweighed their risks.

But without proof of efficacy, there’s no way to make that calculation.

Safety & efficacy are 2 sides of the same coin.

Medicare Pays Wildly Different Prices for the Same Drug Middlemen negotiate prescription costs at thousands of different price points, depending on your insurance plan.

A costly symptom of our complicated drug reimbursement system: People in the same Medicare drug plan face wildly different prices for the same drug

Wow, yeah. Good call. Back to the drawing board!

I‘m so excited to see live-action reporting on this platform. But we need a better moniker!

We are hiring new fellows and faculty in PORTAL. Please reach out if interested! And spread the word.

How to write a scientific paper in four slides.

Full guide here: www.surgeryredesign.com/s/WritingFor...

YouTube format here: youtu.be/fgeahUrXVaY?...

#meded #medsky #research

a cartoon character wearing a hoodie is laughing in front of a fire background . ALT: a cartoon character wearing a hoodie is laughing in front of a fire background .

On the DHHS and CMS news, I will not allow MAHA. I may allow for MWAHA because, sarcasm.

Health journalists and academics, say no to the acronym. First act of resistance. ##healthpolicy #youmustbejoking

RFK Jr. vs. Vivek: Trump world's two paths for pharma regulation Between RFK Jr. and Vivek Ramaswamy, the incoming Trump administration has widely divergent views on regulating the drug industry.

I already have a headache sorting signal from noise and it‘s not even 2025 yet. Gonna be a while before we have a clear picture of what this means for Rx.

www.axios.com/2024/11/19/r...

I’m focused on #drugpricing and would love to be added as well!

Time To Tip the Scales? Humira Biosimilars And The Limitations Of Competition In US Health Care <p>In year two, Humira biosimilar uptake – or the lack thereof – continues to build the case for policy reform to incentivize use of cheaper biologics in the outpatient setting. </p>

"We’ve had members and manufacturers say, OK we will not invest in developing a biosimilar in Medicare Part D or in the PBM space, if the PBM system does not change.” - Juliana Reed of the Biosimilars Forum #healthpolicy

A big congrats to Biden administration and CMMI on moving forward with a novel payment approach for cell and gene therapies! www.cms.gov/newsroom/pre...

Incentive for R&D is always a topic in Pharma policy, but debate usually focuses on investment and development. I fear the pressure to show novelty at the bench is being overlooked. How much money and effort is going into chasing scientific dead ends? www.statnews.com/2024/01/19/d...

For any docs out there who struggle with prior auth and would love to have your biases confirmed, I highly recommend this new study by @michaelannica.bsky.social and Nancy Keating, out yesterday in JCO:

pubmed.ncbi.nlm.nih.gov/38086013/

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