72 karma · joined March 14, 2019
I work as a computational biologist in the pharmaceuticals industry, and I can absolutely vouch for the idea that centralization of resources is a massive problem. Pharma companies have to build extremely redundant infrastructure (vivariums, lab space, etc.), and buy equipment that often goes underutilized, in order to repeat experiments that other companies have already completed but not published. From the computational perspective, teams suffer greatly from "not invented here syndrome," and spend massive engineering and technical effort to re-implement YET ANOTHER RNA-seq processing pipeline.
If we, as a field, want to maximize our impact upon the largest patient population possible, we need to develop a decentralized system to do science. If there was a way to publish intermediate results (e.g. "we tried to see if this drug fixes kidney fibrosis and it seems to sort of work"), and subsequently get credit for that finding's role in the creation of a drug down the line, we could massively reduce the amount of experimental redundancy that plagues the field today. I'm not sure if that solution is via a blockchain or not, but I think that vision would yield a better system.
I only have a rudimentary understanding of blockchain technologies, 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.
Another idea would be to completely socialize all biopharmaceutical research, but that type of system would require an extremely radical societal shift.
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 therapeutic development. In fact, drugs that only offer marginal improvements in quality of life for rare genetic disease patients are often fast-tracked by the FDA. Requiring that a drug _works_ shouldn't inhibit drug development. Otherwise, we end up with tragedies like what happened with the use of thalidomide, which prompted this amendment in the first place.
[1] https://clue.io
Obviously this can't work in the current pharma industry configuration; what financial incentive is there for big pharma companies to publish their results for another company to beat them to a new drug? I don't have a solution to this problem, but I hope someday we as a society can find one. This would absolutely revolutionize biopharmaceutical science.
I think that more people studying rare diseases would result in a net gain of lives saved; I don't think it's as zero-sum as "either 1 person's life is saved or 10 are" in this instance.
Research also cross pollinates across disease areas. For example, understanding altered metabolism in cancer can yield insights for non-oncological metabolic disorders. Oftentimes, though, nobody's working on translating that work out of a cancer model, because the financial incentives are not there.
It is a deeply frustrating position to be in, wanting to work on these rare diseases and help this rarified patient population and not being able to, even though me and my colleagues are poised to do so. I often get maligned for being a scientist in pharma; laypeople often assert that I "don't want to treat cancer / rare genetic disease / etc; because then I'd be out of business." I can assure those reading that all of us DESPERATELY would like to work in these indications, and often times it's tragically finance that dictates whether we are able to or not. The system feels broken.
I started to have similar issues, and my optometrist set me up with some reading glasses optimized for how far away I usually sit from my monitor. They have eliminated a lot of the eyestrain issues I'd been dealing with.