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

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

#181

Post by bluGill »

fartcannon wrote: post #130
robotresearcher wrote: post #123

Earlier quoted context omitted.

Have you not had a family member or close friend cured of disease that would have killed them a hundred years ago, thanks to modern pharma drug? I sure have.

No, not personally, I do have 2 on life long treatments. But I was being too hyperbolic and I don't doubt your story. I'm just extrapolating incentives. Is there any incentive to cure (invoice once), when they can treat (life long invoices)? Certainly scorn is irrelevant as OPs comment suggests they're unwilling to work on rare diseases. And these companies are public. They have shareholders that expect them to const…

competion provides the incentive, a cure will put all your treatment competitors out of business as you get everything from everyone who doesn't like treatment.

You know many people who would have died of smallpox,measles, polio, and the like 100 years ago, you just have no idea who those people are.

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

#182

Post by hgh »

We had a scare around Cystic Fibrosis. I admire your courage and compassion - my best to you and your family.

The story of how CF treatments developed (Trikafta) is remarkable. KS seems much rarer than CF, but I recommend studying how the CF Foundation evolved. https://www.cff.org/about-us/our-history

There are lots of good suggestions in the thread. It seems you're already doing this, but I recommend connecting with people (e.g. families of those with KS, medical professionals with an interest, marketing volunteers, ...) and keeping in touch. This can help create momentum behind a movement, leverage networks, and mutual support/encouragement. We also very much appreciated connecting with others affected by CF during that time.

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

#183

Post by logifail »

mschuster91 wrote: post #180
newswasboring wrote: post #178

Earlier quoted context omitted.

Can you illustrate for a lay person the difference between research and development in pharma context?

Research = determining a candidate compound, e.g. a new antibiotic, (e.g. by taking and tweaking an existing compound, by basing off of the actual viral/bacterial genetic code such as with the Biontech/Moderna CoV vaccines or by isolating a compound found in a plant, fungus or other natural agent which has been determined to have the effect you want), and initial checking against cell cultures and/or lab animals for…

> Development = taking a lot of candidate compounds

At least one $bigPharma defines "research" as being everything up to and including the first proof of concept in a human [Phase I] trial. After that would be "development".

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

#184

Post by Pawamoy »

This is probably a bit off-topic, but I wonder if you know about the GenIDA project: https://genida.unistra.fr/. It's basically a research website where families can register and answer questionnaires about the everyday life of patients with rare diseases. The data can then be used to discover new things about these rare diseases. They have a Kleefstra cohort if I recall correctly. Maybe you could get in touch with the people in charge, like Jean-Louis Mandel.

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

#185

Post by newswasboring »

MathCodeLove wrote: post #179
newswasboring wrote: post #177

Earlier quoted context omitted.

> Increase general taxation and use that to fund more pharamaceutical R&D? Take the US military budget and give it to life saving research instead of life taking. Edit: Also implement a wealth tax instead of income tax. Discourage hoarding of wealth and concentration of power in the hands of top 1%.

Ah yes so China or Russia can invade and establish a totalitarian regime. Excellent idea.

You don't have to give up all of it. The total budget of US is larger than the next 6 countries combined. India, which is actually surrounded by hostile neighbors, one of which is china manages to defend itself with a tenth of the budget. My guy you are giving military money they didn't even ask for in this year's budget. Please stop repeating this line. American budget it not to stop China or Russia, its to assert their own will on the world.

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

#186

Post by biols »

Blackstone4 wrote: post #168

Instead of asking for donations of money, could you ask companies to work pro bono / donate resources. I.e. biols the computational biologist (top comment) could donate Friday afternoons to research. Pharma/biotech companies could use this as marketing to improve their image.

I'd be very happy to participate in those type of projects. One could even imagine that pharma companies could be _mandated_ to spend a certain number of people-hours on R&D in a research collaborative.

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

#187

Post by vimy »

newswasboring wrote: post #185
MathCodeLove wrote: post #179

Earlier quoted context omitted.

Ah yes so China or Russia can invade and establish a totalitarian regime. Excellent idea.

You don't have to give up all of it. The total budget of US is larger than the next 6 countries combined. India, which is actually surrounded by hostile neighbors, one of which is china manages to defend itself with a tenth of the budget. My guy you are giving military money they didn't even ask for in this year's budget. Please stop repeating this line. American budget it not to stop China or Russia, its to assert t…

> The total budget of US is larger than the next 6 countries combined.

Not in % of gdp. The only metric that matters. The US economy is massive in size so of course the budget is a big number.

> one of which is china manages to defend itself with a tenth of the budget.

Everything is cheaper in China. You can do a lot more with one million there than in the US.

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

#188

Post by newswasboring »

vimy wrote: post #187
newswasboring wrote: post #185

Earlier quoted context omitted.

You don't have to give up all of it. The total budget of US is larger than the next 6 countries combined. India, which is actually surrounded by hostile neighbors, one of which is china manages to defend itself with a tenth of the budget. My guy you are giving military money they didn't even ask for in this year's budget. Please stop repeating this line. American budget it not to stop China or Russia, its to assert t…

> The total budget of US is larger than the next 6 countries combined. Not in % of gdp. The only metric that matters. The US economy is massive in size so of course the budget is a big number. > one of which is china manages to defend itself with a tenth of the budget. Everything is cheaper in China. You can do a lot more with one million there than in the US.

I don't know why you are talking about china while the point I was making was about India defending itself against china. Also indian % of GDP for military budget is 2.8 while for US its 3.75. US has no neighboring countries actively attacking it, India does.

edit: BTW, is there any actual proof that this whole military budget dissuades Russia and China in any way?

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

#189

Post by freen »

halukakin wrote: post #153
freen wrote: post #116

Hmmm… what does the average KS patient cost to their insurance company? What is the likelihood of research finding treatments that will reduce that cost, and by how much? I wonder if a financial instrument could be crafted that captures a potion of future insurance company savings in return for funding research now. Probably would require some concessional, first loss funding, or perhaps a philanthropic evergreen fun…

That's a good question. Several days/hours worth of PT. Hospital visits/stays every few months. Lab work. That should add up to something for the insurance companies.

So, $/patient/year * # of patients = total annual cost to insurance industry.

A therapy would reduce cost per patient by $x/year. Potential payment to investors in therapy could be 0.5x/year. Someone might be willing to buy that future cash flow now.

Hard to pull off: gotta build a consortium of all insurance companies in US, possibly national health systems in the world, get them all to agree on a current cost and value of a cost reduction.

Then have to have a reasonable degree of certainty in the cost to produce a novel therapy, and potential value of said therapy.

However, once you create the process for the first of these financial instruments, subsequent instruments should be much easier.

If there are many such groups of people with rare diseases, banding together to create the process could be a thing.

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

#190

Post by halukakin »

Pawamoy wrote: post #184

This is probably a bit off-topic, but I wonder if you know about the GenIDA project: https://genida.unistra.fr/ . It's basically a research website where families can register and answer questionnaires about the everyday life of patients with rare diseases. The data can then be used to discover new things about these rare diseases. They have a Kleefstra cohort if I recall correctly. Maybe you could get in touch with…

I'm not sure how much we are in contact with them through idefine. We should definitely encourage patients to signup on genida. This increases awareness at many levels. Thank you. I'll study genida's website.
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