I don’t want to get on the CAR-T

So happy to provide a Monty Python clip about a cart. Consider this an update, and perhaps a precursor, to cell and gene therapy products. It seems Novartis’ breakthrough CAR-T therapy Kymriah has a few things on its plate. Quick recap; approved in May 2017, costs $400,000 with ~$40,000 in post-care treatments, has a REMS program to ensure treatment for severe side effect, sales are under-performing, partly due to manufacturing “variability,” and UnitedHealthcare has formally asked the CMS for a National Coverage Determination. CMS convened last Wednesday on the matter in a Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) meeting. FYI, that was a new acronym for us. The result? Wait for it (because you will) … the “Proposed Decision Memo Due Date” is Feb 16th of 2019. Sigh. If you really want to, you can track the progress here.

Real-Time Oncology Review

Earlier this week, FDA granted its first approval as a part of two new pilot programs that aim to make the development and review of cancer drugs more efficient. The drug is Novartis’ Kisqali and the two programs are the Real-Time Oncology Review (allows for the FDA to review much of the data earlier, after the clinical trial results become available and the database is locked) and the Assessment Aid Pilot Project (used by sponsors to organize their submission into a structured format to facilitate FDA’s review of the application). FDA Commissioner Gottlieb says, “With today’s approval, FDA used these approaches to allow the review team to start analyzing data before the submission of the application and help guide sponsor’s analysis of the top-line data.” Looking for a heavyweight fight? Watch sales of Pfizer Ibrance vs. Novartis Kisqali.

There’s an app for that

Novartis recently launched an app that enables patients taking part in ophthalmic clinical trials to self-report data, which could potentially speed the development of new therapies. Here’s lookin’ at you kid. According to a wonderful report from Deloitte, “the number of apps produced by pharma more than tripled from 2013 to 2016, but the year-over-year growth rate of downloads slowed from 197% between 2013 and 2014 to just 5% between 2015 and 2016.” Maybe pharma lies outside the circle of trust? The Deloitte report also states, “pharma apps are trusted by 32% of consumers, compared to 76% for apps developed by patient communities.” Look for more pharma-association collaborations like the Quitter’s Circle from Pfizer and the American Lung Association. Is it better that the Novartis app is for clinical trials? Hear InsightCity’s take on this article with this short podcast. (It’s our first one. We’re not pros. Don’t be mean.)

“But wait, there’s more”

Order now and we’ll ship you the Thighmaster…and if you’re not satisfied, just return it within 14 days for a full refund. Money-back guarantees are almost a necessity for TV products, but what about for medications? Certainly not the norm, there have been some value-based contracts popping up in the pharma world. There’s a very interesting Forbes article written by former Pfizer global R&D President John LaMattina that lobbies for two major trade organizations (BIO and PhRMA) to pass resolutions that money-back guarantees become a formal practice with member companies. Wow, that would Shake Weight things up. LaMattina points to Amgen’s Repatha (LDL-C lowering) and Novartis’ new gene therapy Kymriah (CAR-T therapy), priced at $475,000/patient. Keep this on your radar, few things could change the healthcare landscape like widespread adoption of value-based pricing.

Trade wars don’t include cancer drugs

US stock markets have been roiling over a possible trade war between the world’s largest economies, the US and China. American President Donald Trump proposed nearly $50B in tariffs on Chinese goods, leading Chinese leaders to propose some of their own, but one product they’re not interested in taxing is foreign cancer drugs. China does have the world’s largest population of cancer patients after all. Drugmakers like Roche, Novartis and AZ should be pretty happy with the zero-tariff arrangement, while Chinese leadership hopes the move will push local pharma to improve their technological capabilities. Why can’t we go back to the good ol’ days when both the Chinese and Americans could ask “War, HUH, yeah, what is it good for?”

When he speaks, please listen

With all due apologies to EF Hutton (the commercial will make you feel older than you might want to), when Vasant Narasimhan, Global Head, Drug Development and CEO of Novartis speaks we should all probably listen. In a recent article he outlined three areas that will change medicine in 2018, and spoiler alert, they all have to do with “big” data. The three areas he outlines include; the Internet of things, AI and machine learning, and emerging data platforms. He also introduced InsightCity (and maybe you) to a new term: data lakes (def. virtual warehouses holding immense amounts of raw data in their native form.) Look for “data lakes” on an upcoming IC Buzzword Bingo. FYI, a great 60-minute precision medicine panel discussion from the World Economic Forum/Davos can be found here.

Swab, Spit, or Blood

We’ve all seen the Ancestry.com ads for DNA tests that reveal your ethnic mix. Harmless enough. But what if a similar test indicated you had a low risk for coronary heart disease? Would you start swallowing these 2,000+ calorie bombs at every meal? Not harmless. Consumers are increasingly embracing genetic health risk (GHR) testing to understand their individual risk for developing diseases and the FDA recently released an update to streamline the development and review pathway for GHR tests. These tests can be valuable for drug development (see how Novartis is using genetic testing in an Alzheimer’s trial) and treatment decisions and the CDC has some thoughts on the topic.

You give $1M and you give $1M and you give $1M (say it like Oprah would)

And soon it starts to add up. This week the NIH announced a pretty cool partnership with the private pharma industry. The Partnership for Accelerating Cancer Therapies (PACT) is a five-year public-private research collaboration totaling $215 million as part of the Cancer Moonshot project. 11 pharma companies (AbbVie, Amgen, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Genentech, Gilead Sciences, GlaxoSmithKline, Johnson & Johnson, Novartis, Pfizer) will contribute $1M each for five years. The NIH will contribute $160 million. The goal: “to identify, develop and validate robust biomarkers — standardized biological markers of disease and treatment response — to advance new immunotherapy treatments.” Sweet. If you want to read what pharma PR folks have been up to you can read their responses here. For the top 10 private-public immuno-oncology collaborations, look here.