161–170 of 212 posts
From the very little experience I have of rare chromosomal disorders and attending a number of conferences dedicated to my sons specific condition, what i do see is people passionate to understand and improve things for their kids and for others primarily through research into conditions and from the dissemination of information. It is obvious that you are determined to do what it takes. Please do try and make contac…
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Basic research is mainly government funded. Pharma companies build on that research to do the final phases of drug development and they fund the trials in exchange for a monopoly if the drug pays off. Shkreli wasn't interested in funding research. He looked for drugs he could buy and jack up the price of, so he was just a parasite.
This is very interesting. On a tangential note about Shkreli. I took a break and I remembered in the Facebook senate hearing a senator said to Zuck, "Who elected you to make decisions about what stays on the internet and what doesn't?" . This is tangentially related in the matters of Shkreli. Why it would be ethical for Shkreli to take this "moral burden" of doing the right thing and invest in innovation by essential…
In the USA the best of the best are put in charge of tomorrows medicine, and that is why they succeed while the rest of the world is just pitiful in comparison.
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Sounds like you and your colleagues could start your own company, there may be enough people. I wonder if a nonprofit pharma company is viable.
I have been reading a book recently: The Story of Taxol: Nature and Politics in the Pursuit of an Anti-Cancer Drug , and one of the most fascinating parts was the way they discovered this molecule. Long story short, Taxol is a molecule they isolated from the bark of the Pacific Yew. The interesting part for me was learning about the Cancer Chemotherapy National Service Center [1]. They went around collecting samples…
Perhaps the interesting thing would be to ask what particular thing makes the US different to other western countries, rather than cherry picking one particular thing that happens to agree with whatever ideology is popular today.
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If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? I understand that there are no easy choices here, and having to make a choice will always be heartbreaking. May I suggest contacting MacKenzie Scott (Jeff Bezos' ex) who seems to be looking for worthwhile endeavors to finance. (I have no connection to Ms Scott, I just read articles about her charitable activit…
> If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? Both. We're not even close to our limits on research capacity. If we actually were at the limits of research capacity, and we were actually forced to make decisions between livesaving treatments to research where we couldn't do both without sacrificing something else that saves lives, then yes, of course, ch…
Maybe not at your stage of research, but it's my understanding that further down the pipeline (eg clinical trials) you're mich closer to the limits, simply due to financial constraints: given that a trial for something you can sell to millions isn't that mich more expensive than one you can sell to dozens,and given that you need to get payed for your job, i don't see how it doesn't make sense to prioritize the lower hanging fruit. Am i missing something?
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If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? I understand that there are no easy choices here, and having to make a choice will always be heartbreaking. May I suggest contacting MacKenzie Scott (Jeff Bezos' ex) who seems to be looking for worthwhile endeavors to finance. (I have no connection to Ms Scott, I just read articles about her charitable activit…
> If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? Both. We're not even close to our limits on research capacity. If we actually were at the limits of research capacity, and we were actually forced to make decisions between livesaving treatments to research where we couldn't do both without sacrificing something else that saves lives, then yes, of course, ch…
(Full disclosure: close family member works at $bigPharma)
I don't think it's about finding treatments you can "sell to millions", it's that in pharma, like in pretty much every other business, it's about ROI.
You definitely don't need millions of patients, but you do need to cover the R&D costs _and_ have enough left over to keep the shareholders happy.
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> If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? Both. We're not even close to our limits on research capacity. If we actually were at the limits of research capacity, and we were actually forced to make decisions between livesaving treatments to research where we couldn't do both without sacrificing something else that saves lives, then yes, of course, ch…
> We just don't have a good system for funding cures you can't sell to millions (Full disclosure: close family member works at $bigPharma) I don't think it's about finding treatments you can "sell to millions", it's that in pharma, like in pretty much every other business, it's about ROI. You definitely don't need millions of patients, but you do need to cover the R&D costs _and_ have enough left over to keep the sha…
Earlier quoted context omitted.
If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? I understand that there are no easy choices here, and having to make a choice will always be heartbreaking. May I suggest contacting MacKenzie Scott (Jeff Bezos' ex) who seems to be looking for worthwhile endeavors to finance. (I have no connection to Ms Scott, I just read articles about her charitable activit…
> If you could do research that will save 10 lives, vs research that will save 1 life, which would you choose? Both. We're not even close to our limits on research capacity. If we actually were at the limits of research capacity, and we were actually forced to make decisions between livesaving treatments to research where we couldn't do both without sacrificing something else that saves lives, then yes, of course, ch…
Maybe twenty years from now some people with a high degree of knowledge, skills, and some resources will be able to get around these hoops illegally (by the laws of certain jurisdictions) and fix these conditions (or kill themselves or somebody else trying, but so does god/nature and doctors, with different[citation needed] probability distributions).
Community building will be an important part of your foundation and parents. In our case, we joined the SETBP1 support group on Facebook. It is always heartwarming and comforting to read other peoples and to know that you are not the only one that is in a similar situation. The Facebook group keeps us also updated on recent scientific progress and we were able to join a research study on SETBP1.
I know that having a kid that is unable to communicate is hard. Very hard. When our son was younger he was sometimes very frustated because he could not express himself. But communication is more than speech. Our son is now mainly expressing himself with key word signing (in our case the Dutch variant SMOG) and his speech is gradually improving (at a very slow pace). I hope your daughter will also find a way to properly express herself.
[1] https://rarediseases.info.nih.gov/diseases/13379/setbp1-diso...
[2] Morgan A, Braden R, Wong MMK, Colin E, Amor D, Liégeois F, Srivastava S, Vogel A, Bizaoui V, Ranguin K, Fisher SE, van Bon BW. Speech and language deficits are central to SETBP1 haploinsufficiency disorder. Eur J Hum Genet. 2021 Aug;29(8):1216-1225.
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> We just don't have a good system for funding cures you can't sell to millions (Full disclosure: close family member works at $bigPharma) I don't think it's about finding treatments you can "sell to millions", it's that in pharma, like in pretty much every other business, it's about ROI. You definitely don't need millions of patients, but you do need to cover the R&D costs _and_ have enough left over to keep the sha…
This is such a fucked up incentive structure. Literally having the capacity to save lives but not doing it because otherwise people will pull out their money because Roblox is has a better ROI.
It may well be(!), so let's assume you're right, what better incentive structure should we put in place instead?
Increase general taxation and use that to fund more pharamaceutical R&D?