8 ms·

F.D.A. Approves Second Gene-Altering Treatment for Cancer

69 points by sethbannon 9y ago

lawlessone 9y ago
375K, would insurance cover that?
- refurb 9y ago
  Yes, although your doctor might have to jump through some hoops to make it clear you're a good candidate for it.
- lloyd-christmas 9y ago
  Insurance covers it most of the time. That's not typically the issue. Doctors themselves (not any of their employees) need to call the insurance company and get it approved. You know when you call your ISP and get put on hold for 30 minutes? It's exactly that. On the other side are people, frequently with no medical knowledge, that are there to do nothing more than waste your time for a bit before you ask for their manager. How are you supposed to treat all your patients when you spend 3 hours on the phone for one of them?
- RcouF1uZ4gsC 9y ago
  The cost is actually inline with what would be expected from a cancer cure. The NHS uses 20000 - 30000 pounds/quality adjusted life year https://www.nice.org.uk/advice/lgb10/chapter/judging-the-cost-effectiveness-of-public-health-activities https://www.nice.org.uk/advice/lgb10/chapter/judging-the-cos... which would be about $40,000/quality adjusted life year. If this drug provides on average 10 quality years of life, then this would fall under the guidelines as cost effective.
- copperx 9y ago
  That's a drop in the bucket, and if the treatment results in cure, it pays for itself in a few months. Targeted chemotherapy pills are about $20k/month. It's financially smart for insurers to cover the treatment.
nonbel 9y ago
This is the full text of the study? http://onlinelibrary.wiley.com/doi/10.1002/hon.2437_7/full http://onlinelibrary.wiley.com/doi/10.1002/hon.2437_7/full Is that really it? Shouldn't they have to publish something substantive before approval? It doesn't have to be in a journal, but somewhere on the FDA site, etc.
- nonbel 9y ago
  Actually, not even "have to" do that. That makes it seem like the people coming up with these treatments should be an adversary to patients. Shouldn't they want to share as much info as possible? That would seem necessary to convince possible patients (and/or any advisers) that this will help them.
  - refurb 9y ago
    Kite is a start-up in a very competitive field. It wouldn't surprise me that publishing data wasn't that high of a priority vs. getting FDA approval. That said, it's very common for biotechs to publish after the phase 3 is complete as they can put much more information in the paper vs. what their sales reps can say to physicians.
    - nonbel 9y ago
      >"It wouldn't surprise me that publishing data wasn't that high of a priority vs. getting FDA approval." I see, so the problem is physicians/patients are willing to act on very sparse information, so why bother providing it.
      - refurb 9y ago
        You clearly don't understand the pace of work at a biotech start-up. If you could only do one or the other, which would you choose? 1. All hands on deck to get the FDA submission completed so the drug can get to patients sooner. 2. Peel off a bunch of headcount to get a handful of papers published, knowing that will result in a delay in getting the drug to patients?
jfarlow 9y ago
And congratulations once again. This kind of therapy is a big deal because of just how different it is from standard 'drugs'. This is a precise and intentional genetic change to a patient's own immune cells to provide it a new capability. This new capability permits one's own immune system to detect a cancer with precision, clearing it from the body without harming other non-cancerous cells. The same protein doing the work here can be easily tuned to become more and more precise, and the scaffold of the technique can be ported to other targets relatively easily - again very much unlike most small molecule drugs. In this way it is nothing like most any drug of the 20th century. And there are many more to come. There are >100 in the FDA pipeline right now for various types of cancer [1]. And the concepts used here to introduce DNA encoding proteins that confer NEW functions can be used to introduce any number of new functions. So in less than 2 months, the FDA has approved 3 gene therapies: 1) Cure for Leukemia (Novartis' CAR) 2) Cure for Blindness (Spark's hRpe65) 3) Cure for Lymphoma (Gilead's (nee Kite) CAR) Here's our description of the designed protein in case you want to learn what it does at a molecular level, or want to design your own: https://serotiny.bio/notes/proteins/car19/ https://serotiny.bio/notes/proteins/car19/ [1] (paywalled CAR trails list): https://www.ncbi.nlm.nih.gov/pubmed/27613725 https://www.ncbi.nlm.nih.gov/pubmed/27613725

69 points by sethbannon 9y ago

lawlessone 9y ago

375K, would insurance cover that?

refurb 9y ago
Yes, although your doctor might have to jump through some hoops to make it clear you're a good candidate for it.
lloyd-christmas 9y ago
Insurance covers it most of the time. That's not typically the issue. Doctors themselves (not any of their employees) need to call the insurance company and get it approved. You know when you call your ISP and get put on hold for 30 minutes? It's exactly that. On the other side are people, frequently with no medical knowledge, that are there to do nothing more than waste your time for a bit before you ask for their manager. How are you supposed to treat all your patients when you spend 3 hours on the phone for one of them?
RcouF1uZ4gsC 9y ago
The cost is actually inline with what would be expected from a cancer cure. The NHS uses 20000 - 30000 pounds/quality adjusted life year https://www.nice.org.uk/advice/lgb10/chapter/judging-the-cost-effectiveness-of-public-health-activities https://www.nice.org.uk/advice/lgb10/chapter/judging-the-cos... which would be about $40,000/quality adjusted life year. If this drug provides on average 10 quality years of life, then this would fall under the guidelines as cost effective.
copperx 9y ago
That's a drop in the bucket, and if the treatment results in cure, it pays for itself in a few months. Targeted chemotherapy pills are about $20k/month. It's financially smart for insurers to cover the treatment.

nonbel 9y ago

This is the full text of the study? http://onlinelibrary.wiley.com/doi/10.1002/hon.2437_7/full http://onlinelibrary.wiley.com/doi/10.1002/hon.2437_7/full Is that really it? Shouldn't they have to publish something substantive before approval? It doesn't have to be in a journal, but somewhere on the FDA site, etc.

nonbel 9y ago
Actually, not even "have to" do that. That makes it seem like the people coming up with these treatments should be an adversary to patients. Shouldn't they want to share as much info as possible? That would seem necessary to convince possible patients (and/or any advisers) that this will help them.
- refurb 9y ago
  Kite is a start-up in a very competitive field. It wouldn't surprise me that publishing data wasn't that high of a priority vs. getting FDA approval. That said, it's very common for biotechs to publish after the phase 3 is complete as they can put much more information in the paper vs. what their sales reps can say to physicians.
  - nonbel 9y ago
    >"It wouldn't surprise me that publishing data wasn't that high of a priority vs. getting FDA approval." I see, so the problem is physicians/patients are willing to act on very sparse information, so why bother providing it.
    - refurb 9y ago
      You clearly don't understand the pace of work at a biotech start-up. If you could only do one or the other, which would you choose? 1. All hands on deck to get the FDA submission completed so the drug can get to patients sooner. 2. Peel off a bunch of headcount to get a handful of papers published, knowing that will result in a delay in getting the drug to patients?

jfarlow 9y ago

And congratulations once again. This kind of therapy is a big deal because of just how different it is from standard 'drugs'. This is a precise and intentional genetic change to a patient's own immune cells to provide it a new capability. This new capability permits one's own immune system to detect a cancer with precision, clearing it from the body without harming other non-cancerous cells. The same protein doing the work here can be easily tuned to become more and more precise, and the scaffold of the technique can be ported to other targets relatively easily - again very much unlike most small molecule drugs. In this way it is nothing like most any drug of the 20th century. And there are many more to come. There are >100 in the FDA pipeline right now for various types of cancer [1]. And the concepts used here to introduce DNA encoding proteins that confer NEW functions can be used to introduce any number of new functions. So in less than 2 months, the FDA has approved 3 gene therapies: 1) Cure for Leukemia (Novartis' CAR) 2) Cure for Blindness (Spark's hRpe65) 3) Cure for Lymphoma (Gilead's (nee Kite) CAR) Here's our description of the designed protein in case you want to learn what it does at a molecular level, or want to design your own: https://serotiny.bio/notes/proteins/car19/ https://serotiny.bio/notes/proteins/car19/ [1] (paywalled CAR trails list): https://www.ncbi.nlm.nih.gov/pubmed/27613725 https://www.ncbi.nlm.nih.gov/pubmed/27613725