The ice bucket challenge was a good example of a marketing action that collected money.
Definitely! I have a few creative agencies in mind to get in touch with.
Ask HN: How to raise funds for rare disease research?
151–160 of 212 posts
Re: Ask HN: How to raise funds for rare disease research?
#152Email sent
Received it. Thank you Trey.
Re: Ask HN: How to raise funds for rare disease research?
#153Hmmm… 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.
Re: Ask HN: How to raise funds for rare disease research?
#154Earlier quoted context omitted.
On the other hand, competition has given us what, 6 covid vaccines of varying effectiveness, and in record time. What if only the least effective one was developed, and took 18 months? The 1962 FDA effectiveness mandates have had the side effect of increasing drug development costs enormously, and that shuts down development of treatments for rare disorders.
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…
"Safety" is quite a wide concept. I heard a professional drug researcher say that aspirin wouldn't pass the trials today, and certainly not as an over-the-counter drug. Too many side effects.
If we can use a software analogy, mess like Windows 95 wouldn't see the light of the day. But they were useful nonetheless.
Re: Ask HN: How to raise funds for rare disease research?
#155I wondered, could there be scope for a commercial or not for profit partnership with a University offshoot research team.
Re: Ask HN: How to raise funds for rare disease research?
#156Australia has "Rare Voices Australia" which is for rare genetic diseases that face this same issue. I have met the co-founder who is an impressive and emphatic person. Similar probably exists where you are. https://rarevoices.org.au/
Thank you for sharing this. I think we should add such organizations' links to idefine website. In this case, I'm guessing not all Australian parents would know about this org.
Re: Ask HN: How to raise funds for rare disease research?
#157This probably isn't any help but as a father to a severely disabled autistic girl it feels the entire medical system is broken. There's so much future squandered in the hopes of short term profit paid for at the cost to the next generation. I know people say "Autism can't be cured" etc. But having met other families in this process and visiting group homes and the potential for a life of happiness for kids like ours…
Thank you for sharing this. We share the same thoughts&feelings every day. You are not alone.
Re: Ask HN: How to raise funds for rare disease research?
#158I wondered, could there be scope for a commercial or not for profit partnership with a University offshoot research team.
Once we reach a significant funding level I'm guessing Universities will be where these research will be conducted.
Re: Ask HN: How to raise funds for rare disease research?
#159From 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 contact with Dr. Jannine Cody[1] who when faced with a not too dissimilar position to yours pretty much single handedly set about doing what she had to in order to understand and improve her daughters condition and in the process has created a worldwide network of families that meet at conferences and fund research.
Good luck, know that you do not stand alone. It might seem like you do, but you'll be surprised.
[1]: https://www.chromosome18.org/chromosome18-team/jannine-cody/
Re: Ask HN: How to raise funds for rare disease research?
#160Earlier quoted context omitted.
Thank you! Thanks to Ethan and Julia's weekly meetings on clubhouse, I've heard about some of these. Alok's DAO-based funding system seems very promising. I hope that gets traction. I thought I knew more about RareBase, but now I think I'll need to study their site in more depth.
Ethan Perlstein uses ethnic stereotypes to market his company. He opportunistically uses rare disease kids to shield against complaints. I would steer clear.
I met with Ethan once to discuss potential drug repurposing avenues. Obviously, I understand he is doing this for profit and I hope his company becomes a unicorn one day. His weekly clubhouse meetings are on par with phd level courses, they feel like information coming out of a firehose. His contribution to the community is incredible.