Blog Archive - Page 2 of 15

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

Marc Herant August 1, 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

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

Tory Wolff and Thomas Uhler July 27, 2022

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

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

Jason Brauner and Anja Schempf July 18, 2022

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.

An opinionated take on NEJM highlights for June 2022

Marc Herant July 1, 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

An opinionated take on NEJM highlights for May 2022

Marc Herant June 2, 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

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

Irina Ahmad June 2, 2022

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

An opinionated take on NEJM highlights for March-April 2022

Marc Herant May 1, 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

The coming site-of-care shock for infusions

Jacob Wiesenthal and Tory Wolff April 26, 2022

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

STAT News: Incentives to spur repurposing of existing generics for novel uses would open a new paradigm in drug R&D

Nikhil Bhojwani April 13, 2022

The growth of small molecule drug classes over time

Marc Herant April 8, 2022

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

An opinionated take on NEJM highlights for January-February 2021

Marc Herant March 7, 2022

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

Harvard Business Review: Countries Will Need a More Nimble Response to the Next Pandemic

Nikhil Bhojwani February 14, 2022

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

Marc Herant January 6, 2022

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)

The road ahead for genomic testing

Sarah Dolman December 24, 2021

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

New York Magazine: The Great American Testing Crunch How it happened, what we can do about it, and when it might end.

Nikhil Bhojwani December 22, 2021

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

Jacob Wiesenthal and Tory Wolff December 21, 2021

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

Optum opening new competitive terrain in Revenue Cycle Management

Tory Wolff November 21, 2021

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

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

Jacob Wiesenthal and Tory Wolff November 2, 2021

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

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

Marc Herant October 29, 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

The Puzzling Future of Healthcare

Nikhil Bhojwani October 10, 2021

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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