Hacker News Board Hacker News, browsed like a classic message board
Live data from Hacker News

Ask HN: How to raise funds for rare disease research?

news.ycombinator.com

Post reply on HN
Print view View original

101–110 of 212 posts

Re: Ask HN: How to raise funds for rare disease research?

#101

Post by not2b »

I'm sorry about your daughter. This is a tough problem.

Even if pharmaceutical company profit weren't an issue, limited resources still would be a problem, so very rare diseases would still suffer from lack of attention, I'm afraid, because if we replaced "what research produces the most profit" with "what research benefits the most people", that still wouldn't help KS sufferers much. Perhaps the kind of fundraising you are doing is the best that can be done. Or maybe some line of research that can benefit sufferers from multiple diseases (say, anything that results in a missing protein) could help a larger number and obtain government funding.

Re: Ask HN: How to raise funds for rare disease research?

#102

Post by codekilla »

biols wrote: post #85
codekilla wrote: post #80

Earlier quoted context omitted.

I've thought about things like the patent/ip problem, the structure of biomedical research, Pharma research, etc. This is an area where I don't actually see competition as a net benefit, however....it's the reality. The only thing I can come up with is a version of 'data rental'. Rather than Pharma companies locking this data away from others indefinitely, is there a way they could profit from it somehow, while still…

I agree that the system is just "reality" right now. I think the idea of a cryptographic data commons is an interesting idea, I'm just trying to imagine how it'd play out in my day-to-day research. If a system would tell me that my hypothesis is correct, but I couldn't look at (and share with colleagues) the raw data being computed, it'd be tough to believe that system. Maybe there's some type of zero-knowledge proof…

Yes, it's admittedly an incredibly tough problem (how do you get those competing to cooperate?), and how to compute on data that is trustworthy. Ultimately, in biotech/pharma you have to ultimately know what an underlying gene or say pathway actually is, it can't be totally obfuscated. Still not sure how to set this up (if it's really even possible) that solves for this use case.

> but a system where pieces of a research puzzle are stored on chain and each user can claim ownership of those findings, a resultant drug's profits could be proportionally split by every entity which contributed to the research.

I think ultimately this is how a research cooperative could work. If distributing and re-allocating fractional ownership is efficient enough, it seems like something like this might be feasible. The idea with multiparty communication (MPC) is that in this setup a research entity would contribute their data in an encrypted fashion, and any parties would be granted access to compute on it based on some set of rules/buy in etc.

This is a really difficult technical approach, as MPC is really only in it's infancy, made only to seem easy by the far more difficult and distant prospect of socializing medicine, which would seem to be of the greatest benefit.

Re: Ask HN: How to raise funds for rare disease research?

#103

Post by not2b »

anyfactor wrote: post #70
biols wrote: post #5

I work at a medium-sized pharmaceutical company as a computational biologist. Diseases like KS sometimes come up as potential repurposing targets (or novel drug targets), but we get a LOT of pushback from finance / leadership because we're unlikely to turn a profit working on ultra-rare indications. It is a deeply frustrating position to be in, wanting to work on these rare diseases and help this rarified patient pop…

I want to preface this by saying I am not providing an opinion rather I am genuinely curious. When Martin Shkreli bought the rights to Daraprim, some of his rhetoric about pharmaceutical industry sounded fair. He said that he is willing to send the drug for free to anyone who wrote to the company and he was essentially making the insurance companies pay the absurd price of the drug. He claimed no patient would ever f…

Insurance companies get their money from people, companies, or governments that pay for insurance. To get him an absurd amount of money, ultimately we (individuals, companies, taxpayers) have to provide it.

Re: Ask HN: How to raise funds for rare disease research?

#106

Post by JoshTriplett »

WalterBright wrote: post #48
biols wrote: post #5

I work at a medium-sized pharmaceutical company as a computational biologist. Diseases like KS sometimes come up as potential repurposing targets (or novel drug targets), but we get a LOT of pushback from finance / leadership because we're unlikely to turn a profit working on ultra-rare indications. It is a deeply frustrating position to be in, wanting to work on these rare diseases and help this rarified patient pop…

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, choose the thing with the highest number of predicted lives saved. But we're not even close to needing to make such decisions yet.

We just don't have a good system for funding cures you can't sell to millions. That's not anywhere close to "heartbreaking decisions" territory; that's "societal coordination problem" territory.

Re: Ask HN: How to raise funds for rare disease research?

#107

Post by anyfactor »

not2b wrote: post #103
anyfactor wrote: post #70

Earlier quoted context omitted.

I want to preface this by saying I am not providing an opinion rather I am genuinely curious. When Martin Shkreli bought the rights to Daraprim, some of his rhetoric about pharmaceutical industry sounded fair. He said that he is willing to send the drug for free to anyone who wrote to the company and he was essentially making the insurance companies pay the absurd price of the drug. He claimed no patient would ever f…

Insurance companies get their money from people, companies, or governments that pay for insurance. To get him an absurd amount of money, ultimately we (individuals, companies, taxpayers) have to provide it.

How does near-welfare state do innovation and research in pharmaceuticals? How do countries with universal medical care performs in terms of research and innovation and treating rare diseases?

Shkreli's ideology was when it comes to innovation in Pharma and America's patent driven capitalist nature towards it is the reason why America leads the way in innovation.

Re: Ask HN: How to raise funds for rare disease research?

#108

Post by Coding_Cat »

csee wrote: post #100
biols wrote: post #81

Earlier quoted context omitted.

I think the idea would have been that Pfizer and Moderna could have pooled resources and made a single optimal vaccine faster, though I'll admit I'm not sure it could have happened any faster than it did from my perspective. The Kefauver Harris Amendment you refer to was immensely important towards the development of safe and efficacious drugs -- I do not see the connection between that act and rare disease therapeut…

Competition is an incredible driver of outcomes, e.g. moon landing. It's also better from a risk perspective since it decorrelates efforts and we only need 1 to succeed. It also allows evolution to operate, where the incompetent and broken and corrupt die off and the productive are given more resources, which tends to lead to overall improvement. Sure, competition also creates waste, which is your main point here, bu…

It is possible to maintain the benefits of competition without the level of duplication and profit motives that we struggle with today.

Large scale collaborative scientific endavours like CERN show us that it is possible to both publically share knowledge and still explore multiple avenues and competing designs. There's also no financial profit motive and while CERN receives a lot of public funding, it has to pump that funding back into the economies of the funding countries so it serves more like a high-tech industry stimulus and technological incubator.

I see no reason why a similar aproach for the development of (specific) therapeutics could not work.

Re: Ask HN: How to raise funds for rare disease research?

#109

Post by not2b »

anyfactor wrote: post #107
not2b wrote: post #103

Earlier quoted context omitted.

Insurance companies get their money from people, companies, or governments that pay for insurance. To get him an absurd amount of money, ultimately we (individuals, companies, taxpayers) have to provide it.

How does near-welfare state do innovation and research in pharmaceuticals? How do countries with universal medical care performs in terms of research and innovation and treating rare diseases? Shkreli's ideology was when it comes to innovation in Pharma and America's patent driven capitalist nature towards it is the reason why America leads the way in innovation.

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.

Re: Ask HN: How to raise funds for rare disease research?

#110

Post by pabs3 »

In countries with single-payer health care, it seems reasonable to use some funding from taxpayers on diseases that aren't considered to be profitable by the existing for-profit medical research companies. i.e. the medical research market is not providing what is needed by the population, so supplement that market using taxpayer funding. In the USA though, that doesn't seem feasible, but perhaps you could reach out to patients in Canada, the UK, Europe etc and through them get taxpayer funding from outside the USA.
Post reply on HN