Blog and Whitepapers

Recon takes an analytical look behind select developments in healthcare

Trends in biotech deals in 2022 and 2023: oncology vs. the rest

Summary After challenging conditions in 2022, deal-driven biotech spending appears to have picked up in 2023. To quantify this trend we have systematically reviewed deals announced during this period and examined differences based on therapeutic areas and stage of development. We find that (1) deal counts shrank in oncology compensated by growth in other TAs; (2) the decrease in number of oncology deals was largely driven by less appetite for earlier phase assets, whereas compensated growth in other TAs came from the same earlier stages of development, (3) across the

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An opinionated take on NEJM highlights for the fourth quarter of 2023

A Chinese study uncovers a new use for an old drug Bleeding in the small intestine is often due to vascular malformations and is difficult to diagnose and manage because 1) the area is hard to reach with a scope and 2) bleeding tends to be intermittent.  Existing approaches are cumbersome and far from satisfactory, which makes a recent blinded RCT study conducted in China exploring thalidomide use in those patients valuable – not only did it show a significant decrease in bleeding episodes after a course of 4 months

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Multi-Cancer Early Detection tests – Will they become part of regular clinical care?

PDF here Summary The idea of a single blood-test to detect many cancers is attractive, but is it practical? Can an MCED enhance, or even replace, current screening tests? This paper delves into these questions by reviewing the effectiveness of current cancer screening methods and identifying areas with the most unmet need. We examine results of Grail’s PATHFINDER2 study on Galleri®, one of the first tools designed to detect many cancers in a single test. However, the test’s adoption will depend on cost-effectiveness, which considers price, cancer detection rate, and

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An opinionated take on NEJM highlights for the third quarter of 2023

How the US taxpayer saved 25M+ people A short piece celebrating the 20th anniversary of PEPFAR, a program that few outside of the field of Global Health are aware of. The world is not always nasty and brutish – it’s a shame it’s not said a bit more often. PEPFAR at 20 — A Game-Changing Impact on HIV in Africa   A hypertension trend? A second study of an investigational antihypertensive drug in the Journal this year; are we back in the 80s or something?  Joking aside, cardiac and vascular

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An opinionated take on NEJM highlights for the second quarter of 2023

Preparing for the next pandemic: don’t waste the value of waste During the Covid-19 pandemic, I got in the habit of checking weekly the Boston sewage report, as a reliable, unbiased way of monitoring trends of infectious activity. There is clearly much more information to extract from waste monitoring and a recent report from the National Academies of Sciences, Engineering, and Medicine makes a number or recommendations. The CDC is implementing some of them, but in the end, exploiting this source of information will depend on 100s of local actors

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TEDxBoston AI in Healthcare

What does Socrates have to do with AI? And what’s the Spooky Mountain and why we need to conquer it to get to an AI enabled future?

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Trends and strategy in oncology development

PDF: Trends and strategy in oncology development Summary We review a sample of oncology programs from the past decade and arrive at the following key findings: The use of expansion cohorts in first-in-human (FIH) studies has grown significantly in the last decade, with an accompanying decrease in separate phase 2 studies Commercial-stage sponsors and early-stage companies backed by established VCs are much more aggressive in discontinuing programs quickly compared to other early-stage companies For drugs that achieve approval, the time from FIH to approval is much shorter for big pharma

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An opinionated take on NEJM highlights for the first quarter of 2023

Hypertension?! Antihypertensive drugs are so 80s and 90s (well, except for pulmonary hypertension) to the point that I cannot recall when I last read research in the NEJM about a new blood pressure drug. One of the reason is that we have so many existing drugs (most of them cheap generics) but despite this, a fraction of hypertensive patients are refractory and cannot get to their blood pressure goal despite using multiple agents. This is where baxdrostat (CinCor Pharma) comes in; it is an oral drug blocking the synthesis of

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Is the future of US pharma manufacturing domestic?

View the PDF here. The supply chain of pharmaceuticals consumed in the US has become reliant on foreign sources of drug products and intermediates over the last two decades. Growth of generics and biosimilars has driven firms to reduce labor costs, which has led to China and India leading much of upstream supply. In response, the White House has announced a series of funded initiatives aimed at improving the US’s domestic market share, creating “good jobs,” and reducing supply chain risk. However, our analysis of the pharmaceutical manufacturing industry suggests

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Can BiTEs take a bite out of CAR-Ts in hematologic cancers?

Download a PDF of this article here. Hematologic cancers account for ~10% of annual new cancer diagnoses and continue to have some of the poorest overall outcomes, particularly for older adults.[1] However, the last decade has seen major clinical improvements, led by drugs from two new modalities: cell therapy[2] and bispecifics. Here, we discuss the looming competition between these two drug-classes for hematological cancer indications. With recent approvals, and multiple late-stage assets for the same indication, the success of future launches will depend on how treatment paradigms shift, or do

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An opinionated take on NEJM highlights for December 2022

An unusual article on road safety Motor vehicle accidents are a major share of morbidity and mortality in the young, and as such prevention has long been considered a medically adjacent area (e.g. with pediatricians asking about infant car seats etc.).  Still it was a surprise to see an article on an intervention specifically designed to improve road safety in the NEJM, but it applies specifically to teens with an ADHD diagnosis, for whom distraction while driving is a significant risk factor. In the study, 152 teen drivers with ADHD

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Hearts and souls: An opinionated take on NEJM highlights for October and November 2022

A psychedelic for refractory depression While major depression is awful, treatment-resistant depression (as defined not responding to at least two different courses of therapy) is worse with sufferers stuck in a long dark tunnel of anguish without a light at the end of it. Since psilocybin (the psychedelic agent in so-called magic mushrooms) has shown some effectiveness in depression, it seemed reasonable to test its differentiated mechanism of action in this high need patient population.  In this study that was mostly Europe-based but included sites in the US (finally), patients

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Amazon becomes a shopping facilitator for healthcare services (finally…)

Back in 2018, I offered a speculation about what Amazon might do in healthcare as part of its now defunct joint venture with JPMorgan and Berkshire Hathaway.   The focus was on doing what Amazon is remarkably good at: creating simple, consumer-friendly, transparent and highly liquid markets.   Here was my suggested Step 1: “[T]here are several classes of healthcare that many patients are ready to shop for (and payers have been trying to get them to shop for): minor acute care (sniffles and coughs to simple fractures), basic diagnostic

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Some very disappointing failures: An opinionated take on NEJM highlights for August and September 2022

Is Parkinson’s α-synuclein going to be as elusive a target as β-amyloid has been for Alzheimer’s? Alpha-synuclein aggregates are characteristic of Parkinson’s disease and genetic variants of this protein clearly lead to familial forms of Parkinson’s; it was therefore reasonable to target alpha-synuclein with antibodies in the hope of modifying disease progression. Sadly, two well designed placebo-controlled studies have now crushed this hope. Both showed absolutely no impact on the progression of patients with early Parkinson’s over times that extended to 1.5 year of follow-up. If there is silver lining,

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Trends in oncolytic virus therapeutics: then and now

PDF: Trends in oncolytic virus therapeutics: then and now The idea of using pathogens to treat disease is not new; ranging from phage therapy to malariotherapy (resulting in the only Nobel in medicine to a psychiatrist – in 1927), but it was really only with the onset of the biotech revolution in the 1990s that an old concept of using viruses as tumor killing agents started to come into its own.  As the ability to characterize viruses at the genetic level grew, it was followed by the techniques to genetically modify

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Are some of Kaiser’s regional ambitions starting to pay off?

PDF: Are some of Kaiser’s regional ambitions starting to pay off? We have produced a new study evaluating developments in Kaiser’s regions (outside of California). The report provides an explanation for recent improvements in performance as well as leadership and governance changes. Further, we identify several implications for the future of Kaiser strategy. The Kaiser Permanente model has a heavy infrastructure, and so it requires a lot of local market share and operating scale to deliver a positive net underwriting margin (we roughly size how much using statistical analysis). Yet,

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What should school nurses do (and not do)? A call for further research

About the author: Alyssa Idusuyi is currently a high school senior attending Miss Porter’s School (Connecticut). She spent the summer of 2022 with Recon Strategy as a paid intern assigned a project to research the evolving role of school nurses in student health. Alyssa plans to attend college next year, and is considering a career in healthcare.   School nurses do more than just give students band aids and Tylenol. Since the rise of the COVID-19 pandemic, school nurses have been front and center in taking care of students and

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An auto- or allo- future for cell therapy?

Download a PDF of this article here. Ex vivo gene-therapies have been a game-changer for aggressive hematologic cancers. These products can effectively cure patients who otherwise had very low chance of survival beyond 5 -years.  However, making these products is exceptionally complex and the drugs are priced accordingly (from $370,000 to over $2.5M). While few patients receive these treatments today, clinical development could lead to a much broader population. If 1 in 20 newly diagnosed cancers were optimally treated with these high-price therapies, the cost to health-payers could become untenable

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An innovative sales strategy for a pivotal drug; Novartis trailblazes with Leqvio

After the $9.7B acquisition of The Medicines Company two and a half years ago, Novartis is eager to demonstrate value with a successful launch of the primary target of the deal, Leqvio. This launch is also a key catalyst for new revenue generation at Novartis, due to a $9B patent cliff by 2026. Given the high-stakes nature of Leqvio’s launch, the decision of the Swiss pharma to employ a non-traditional sales strategy in several key countries, including US and England, is noteworthy.   Novartis paves an innovative commercial path for

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An excellent July vintage: an opinionated take on NEJM highlights for July 2022

Tirzepatide in pole position As previously described in this blog, GLP-1 agonists initially designed to treat glycemia in Type-2 diabetes are emerging as powerful weight loss agents in obesity independent of diabetic status. Recently, tirzepatide (Mounjaro, Lilly, approved May 2022) which combines GLP-1 and GIP activity has shown potential to be best in class, and this is supported by a 72-week study that randomized 2539 obese individuals to 3 different tirzepatide doses or placebo. The results are nothing short of spectacular with ~-21% mean change in weight at the optimal

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What’s so special about One Medical in the eyes of Amazon? A few quick thoughts

There isn’t a bricks-and-mortar primary care acquisition out there that is beyond Amazon’s financial reach.  While the announcement regarding One Medical has provoked fresh rounds of speculation about what Amazon might do broadly in primary care (e.g., push Pillpack),[1] our interest here is in why Amazon seems to think One Medical specifically is the right move right now.  Below are some quick thoughts:  Compatible business models One Medical’s legacy commercial business and Amazon (Prime) both operate a fixed annual membership fee plus charge-per-transaction business model.  The membership fee provides customers

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Looking into the crystal ball – What is in store for the future of gene therapy delivery?

Download Here: Troubleshooting GT_Recon Strategy 2022 In the last decade, gene therapies have been a major area of development and interest. What kicked off with the approval of Spark Therapeutics’ Luxturna in 2017 has now blossomed into a robust pipeline including the approval of Zolgensma (Novartis), and a number of cutting-edge therapies in clinical trials (such as Uniqure and CSL Behring’s therapy for hemophilia or Ultragenyx’s therapy for MPS IIIA). However, while the number of gene therapies entering clinical trials has increased, so have the inevitable stumbling blocks in development.

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An opinionated take on NEJM highlights for June 2022

A cell therapy success against a solid tumor In CAR-T therapies, T-cells are transformed to express an antibody on their surface that allows them to home in on cancer cells and effect killing. This approach has seen remarkable successes in hematological tumors but not so much in solid tumors. However, efforts have been underway to use the intrinsic killing mechanism by T-cells which relies on the native T-cell receptor. The idea is that instead of inserting an antibody construct (i.e., the CAR-T approach), the T-cell receptor is modified to be

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An opinionated take on NEJM highlights for May 2022

A step forward for kidney xenotransplantation A report on the recent, well-publicized, temporary transplantation of kidneys from genetically modified pigs in two deceased individuals at NYU. The kidneys functioned well and there was no sign of acute rejection, but because these experiments took place over only 54 hours, this is not indicative of longer term compatibility of the transplant of kidneys from this particular strain of pigs. A question in my mind is why there have not been kidney xenotransplants in live patients yet – especially given a recent porcine

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Advances in AI for Health in 2021: An Analysis of the Clinicaltrials.gov Registry

The role of artificial intelligence (AI) and machine learning (ML) in healthcare spans a vast range of potential applications. A view of AI development on clinicaltrials.gov helps to focus this landscape, giving a sense of trends and near-term applications. Following last year’s review of AI development, we have updated our analysis to include registered studies starting in 2021.[1] The analysis revealed continued strong growth of studies across the field, especially in patient engagement and research. Within patient engagement, the tools studied (mostly chatbots) have increased in complexity, tracking with advancements

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An opinionated take on NEJM highlights for March-April 2022

The opioid crisis and patient abandonment A perspective from a February issue that I originally missed highlighting the plight of patients who have been on a long-term opioid regimen for chronic pain. They are often stable, but it is when their physician retires or leaves that all hell breaks loose because, in this day and age, they cannot find another physician to continue the regimen. For someone who has been taking large doses of opioid for many years, going cold-turkey, or even tapering, is just not an option. These folks

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The coming site-of-care shock for infusions

There’s a major disruption looming in how the site-of-care for infusions is managed which will have repercussions for plan competition, delivery system economics, PE hunting for healthcare opportunity and biopharma commercialization strategy. Plans have long been frustrated by the growth of infusion care in expensive hospital outpatient department (HOPD) settings. HOPD infusion services can cost health plans on average 70% more than a physician office for the same infusion. But because patients on infusion therapy are often very sick and the therapies hard to tolerate, plans have historically been reluctant

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The growth of small molecule drug classes over time

Modern drug discovery is largely based on the identification and validation of a biochemical target, and then screening and optimizing molecules that engage that target. Over time, this has given rise to an extensive set of small molecule drugs approved for human use with activity at specific biological targets. We wanted to answer the question of how many different targets are addressed by existing agents, and how the rate of addition of newly addressed-target has varied over time. To do this, we reviewed every small molecule drug approved since 1970

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An opinionated take on NEJM highlights for January-February 2021

Gene therapy in beta-thalassemia and sickle cell anemia Beta-thalassemia and sickle cell anemia are common genetic diseases of hemoglobin (Hb) which manifest themselves in the former through transfusion dependence, in the latter through painful vaso-occlusive crises that frequently land patients in the hospital in a pitiful state. BlueBird’s lentiglobin therapy is an ex-vivo gene therapy in which autologous stem cells are harvested, transfected with new HbA gene, and then returned to the patient. Because the HbA is slightly modified via a single amino-acid substitution (HbAT87Q), expression is trackable.  Now in

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Wrapping up 2021: An opinionated take on NEJM highlights for November-December 2021

In search of a diagnosis: deploying genomics at scale A substantial number of children have a disease identified as “rare” without having any kind of causative diagnosis (autism is not a causative diagnosis!). In a UK pilot study, 2,183 proband children were referred for exome sequencing with additional sequencing of family members if warranted. The overall diagnostic yield was surprisingly high, about 25% overall. Of the approximately half of probands who had neurological or sensory disorder, an explanatory diagnosis was reached in almost 40% of the cases. Two thoughts: 1)

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The road ahead for genomic testing

New molecular diagnostics, especially tests based on next-generation sequencing and gene-panels, could revolutionize how diseases are evaluated. But experience shows that insurers won’t cover these tests just because they’re available – they need to improve clinical care. Here, we explore how researchers and test developers can prove value, and improve their chances at broad clinical uptake. Outside the niche realm of self-pay (where buyers have very different expectations[1]), the business opportunity of new molecular tests is driven by payer-coverage and reimbursement. Payers are beginning to manage[2] high-cost (>$1,000) lab tests

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The ongoing physician affiliation land grab in Pennsylvania’s Capital District

Closed vs. open Hospitals can compete for patient referrals either by exclusively affiliating with a subset of physicians (“closed model”) or by collaborating with as many qualified physicians as possible irrespective of competing affiliations (“open model”). (Both of these strategies are from the perspective of the facility, of course. A population health strategy would still focus on affiliating with physicians to aggregate patients, but with a goal of minimizing (not just directing) facilities based care.) The two strategies are generally incompatible. If most hospitals use the same approach in a

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Optum opening new competitive terrain in Revenue Cycle Management

What is revenue cycle management? Revenue cycle management (RCM) is the process of converting care delivery into cash. At its most comprehensive, services include: patient intake (scheduling/registration, coverage verification and financial counseling) claim submission (charge capture, coding, documentation, submission), and payment capture (payment processing, denials, customer service and collections).   Effective RCM is challenging because of: The variety of plans and benefits designs (what’s covered, patient co-pays, rates, etc.), Ambiguities of payer approval of specific clinical services (prior authorization, etc.), Requirements and opportunities in characterizing the care and patient risk

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Profit engine swap-outs: How UPMC sustained itself after the Highmark decoupling

UPMC’s decoupling from Highmark exposed a critical vulnerability: economic dependence on its Allegheny County hospitals. In FY11-12,[1] these hospitals provided 70% of UPMC’s overall operating margin, an average of ~$270M annually.[2] A few years later, these operating margins had been cut in half and, by FY19, these same hospitals could contribute just $9M to the enterprise.[3] How did this happen? The Highmark dispute created severe economic headwinds for its western Pennsylvania (what we call “Core”)[4] hospitals: Decay in payer mix. As Highmark patients went elsewhere, the overall commercial share of

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Taking on tough indications: An opinionated take on NEJM highlights for October 2021

What makes an indication especially tough? The combination of a lack of understanding about the mechanism of disease and a lack of clarity on what endpoints would be sufficient for approval of a therapy in studies of reasonable duration. This month, two good examples in the NEJM.   A phase 2b success in NASH Non-alcoholic steatohepatitis (NASH) is a common slow-progressing disease and for most affected individuals the timescale to liver-related morbidity is at least 20 years, although some progress much faster. NASH is also a graveyard of clinical programs

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The Puzzling Future of Healthcare

This post is from a talk at the 2021 Link Ventures CEO summit. Original talk: Transcript: The practice of medicine has always been an information problem. Clinicians seek to diagnose an ailment and to prescribe a cure based on an incomplete understanding of what’s happening in a patient’s body as it relates to an incomplete set of knowledge about diagnoses and treatments.  This aspect of medicine has remained the same for centuries even as our collective understanding of medicine has gone through three major phases.  Until the 18th century, much

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From bleeding edge to basics: an opinionated take on NEJM highlights for August and September 2021

The weird world of synthetic biology In biology 101, one learns about the alphabet that translates DNA/RNA code into amino acids sequences ultimately resulting in proteins. Given that there are 64 possible triplet codons and 20 amino acids, multiple codons can map to the same amino acids. But this mapping can be reassigned including incorporating new amino acids – it has been done in E. coli with the promise of generating biologics with novel properties, although applications are not yet clear. A more practical application is to create strains of

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Summer catch-up: an opinionated take on NEJM highlights for May-July 2021

Cancer and mutational complexity Probably the biggest news in cancer treatment this year is the approval of sotorasib (Lumkras, Amgen) which received accelerated approval in lung cancer with mutated KRAS (G12C) and has potential for use in other cancers (see here). Mirati is hot on the trail with adagrasib, but unusually for a drug in the approval process, their paper in the NEJM is a deep dive into the mechanisms of resistance to KRAS G12C inhibition that lead to treatment failure. They present very detailed in vitro studies in which

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Iora Agonistes: High-touch primary care in Medicare Advantage is no sure bet after all

Iora Health was one of the original primary care transformers offering a clinically capable, engagement-focused, and accountability-grounded care model.  After an initial foray into commercial, Iora pivoted in 2014 to Medicare Advantage (MA), an alliance with HUM and a global capitation-oriented strategy broadly similar to Oak Street or ChenMed. MA – whose members often have chronic conditions that respond to management and with a payment model that rewards quality (via stars) and patient intimacy (via risk coding) – is fertile ground for Iora’s high touch primary care.  And that terrain

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Four plausible rationales for incumbent hospitals to embrace hospital@home despite the potential to divert “heads from beds”

We identify four different business strategy rationales for hospital@home depending on each hospital’s specific market situation and each with clear predictive implications for local markets.  These are: on-demand capacity expansion, bed capacity rationalization, competitive matching and system consistency.  Almost all participants in CMS’s current Hospital@home program could find one of these four rationales applicable.  If these rationales become compelling business cases, the future of hospital@home is bright and it is time to start the healthcare ecosystem to prepare for its disruptive consequences. The cannibalization challenge Hospital@home uses innovative protocols, technology,

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WMT’s MeMD acquisition: too small a play to make the difference

Last week, WMT acquired MeMD, a virtual provider in Arizona. Per the press release, MeMD will “allow Walmart Health to provide access to virtual care across the nation including urgent, behavioral and primary care, complementing our in-person Walmart Health centers.”  Can MeMD rescue Walmart’s struggling clinic strategy?  I am skeptical. Virtual is a great complement to the clinics Adding virtual to the clinics could be a very good idea: Walmart’s clinics had the extraordinarily bad luck of rolling out during a pandemic.  More importantly, they are weighed down with unresolved

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Clinicaltrials.gov and AI-related studies: a brief status check in April 2021

Healthcare is by nature conservative; despite a huge level of interest for artificial intelligence (AI) and machine learning (ML) techniques, significant penetration in standard workflows of care will depend on demonstrations of safety and efficacy. As a result, it is today’s pipeline of clinical studies using AI/ML that will determine mainstream usage tomorrow.  In order to better understand this pipeline, we have mined clinicaltrials.gov for characteristics and trends of registered studies making use of AI/ML. Unsurprisingly we see strong growth, but starting from a very small baseline. Although most of

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Remodel, rewire, repopulate: an opinionated take on NEJM highlights for April 2021

Remodeling to health? Chronic disease progression is often due to a maladaptive tissue response which leads to remodeling – such as cardiac hypertrophy with hypertension.  While most therapeutics focus on tamping down the injury, sotatercept (Acceleron Pharma) in pulmonary arterial hypertension (PAH) breaks new ground by focusing on the remodeling. This modulator of the TGF-beta superfamily looks to inhibit the remodeling of the pulmonary vasculature that worsens the PAH over time, and eventually leads to the demise of most patients. In a 24-week placebo-controlled phase 2 trial (N=106), the pulmonary

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Balancing the humors: an opinionated take on NEJM highlights for March 2021

A new approach to schizophrenia Antipsychotic agents treat schizophrenia by manipulating the dopaminergic system. While they are effective at treating psychosis, they can have major side effects and they lack the ability to address so called negative symptoms e.g. apathy, lack of social connection, poverty of thought.  Enter the muscarinic cholinergic system which is tricky to manipulate in the CNS without untoward systemic effects as Lilly found out with their M1/4 agonist xanomeline.  Karuna Therapeutics has now licensed the molecule and cleverly combined with it with trospium, an antagonist with

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How much are Humana’s value-based care models really taking out of utilization?

All that effort for only 0.4% savings Since 2014, HUM has reported on the performance of primary care in value-based arrangements (“VBA”) vs. traditional contracts (“non-VBA”)[1] in Medicare Advantage (MA).  One statistic regarding total medical expenditures (TME) is, at first blush, a stunner.  The most recent data (2019) shows PMPM TME of VBA members measured only 0.4% less than for non-VBA members.  Given HUM’s long-standing strategic commitment to value-based care, commentators are puzzled: is the whole value-based enterprise in vain? does healthcare transformation require quasi-geologic timeframes to deliver meaningful cost

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All immunity: an opinionated take on NEJM highlights for February 2021

We can cure Hep C but not immunize against it Sovaldi and other drugs have made curing chronic Hepatitis C a routine, if expensive, proposition. Still, given how common Hep C infection is, and how it can irreversibly damage the liver without overt signs, a vaccine would be highly valuable. Unfortunately, an NIH sponsored trial with a GSK vaccine in 548 IV drug users at high risk of contracting the disease failed to show any protection: 28 participants developed chronic Hep C evenly divided between the placebo and treatment groups. 

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Walmart Health: Will the fifth attempt at clinics be the charm?

Eighteen months ago, WMT opened its first Walmart Health clinic attached to a supercenter in Dallas, GA.  The operation combined multiple services – primary/urgent care, dentistry, behavioral health, optometry, and audiology – with WMT’s Every Day Low Price philosophy (for example, $30 total for a primary care check-up, $40 for a sick visit) in one ~11K sq. ft. site. Boosted by typical supercenter traffic (~5K visits a day is our estimate), and a scarcity of primary and dental care in the local area[1], the clinic got to 2.3K visits-per-month within

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While we wait for our shot(s); an opinionated take on NEJM highlights for January 2021

Incremental progress in the fight to treat heart failure After a decade or more of relative quiescence in the 2000s, a volley of new therapies have come to the forefront in the pages of the NEJM: sacubitril valsartan (2014 – Novartis’ Entresto), various SGLT2 inhibitors such as dapagliflozin (2019, AstraZeneca’s Farxiga), vericiguat (2020 – Merck’s Verquvo), and now a 2021 entry from Amgen in the form of the cardiac myosin activator omecamtiv mecarbil. None of those are a panacea, and of the four listed, omecamtiv has the lowest efficacy profile

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Closing a tough year: An opinionated take on NEJM highlights for December 2020

Cardiovascular health – go big or go narrow? A follow up on the development of evinacumab (Regeneron), an inhibitor of ANGPLT3 (see this opinionated take from 2017), confirming the safety and LDL reduction effect in a population of individuals with cholesterol levels refractory to other therapies (including PCSK9 inhibitors).  Given the history of PCSK9 inhibitors, the development path of this asset will be interesting to follow. Of the 6 trials listed on clinicaltrials.gov not one has anything close to a clinical endpoint – it’s all biomarker based. That will probably

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We are back and catching-up: An opinionated take on NEJM highlights for Sep, Oct, and Nov 2020

Small but real progress in ALS: Despite enormous public attention and significant effort, ALS remains a disease for which the development of new therapies has been challenging. Animal models showed potential activity against ALS by sodium phenylbutyrate and taurursodiol, both old (generic) molecules. Now, their effect in combination therapy has been confirmed in a randomized controlled trial.  What’s especially interesting is that the effect at 24 weeks (the original duration of the trial) was a modest though statistically significant improvement in functional scores. However, the trial was extended with all

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