Clinical studies today are funded entirely by the pharma companies, keep that. Selling at cost of production would be something extra, for patients who want the drugs but aren't enrolled in an actual study. The company doesn't get solid data it can use for regulatory approval, so making them donate the drugs in that case seems excessive.
A downside would be that for drugs that don't cost much to produce, patients might be less willing to enroll in the studies, given the chance of getting a placebo. That could be handled by shutting down the informal access while the study is enrolled, for anyone who's eligible. I'm sure there are other wrinkles that would need to be considered too.
Put this way, this seems reasonable. Beyond cell therapy, I don't thin the cost of drug is the motivation for not making it more freely available. 'Misuse' leading to potential liability or unjustified bad outcomes, along with some regulatory burden seems like the issue.
Knowing whether drugs work isn’t trivial. Patients are typically very heterogeneous in their responses to drugs. For example, pembrolizumab (the most successful cancer drug ever) typically only works in, say 30% of patients depending of the cancer type. Just throwing therapeutic ideas out there and letting physicians sort out how to use them and in which patients, isn’t a panacea. Looking at clinical data can be like star gazing even in planned studies. Structured, statistically powered studies, and costly rigorous assays on biomarkers and correlative studies are essential for understanding how and in what patients drugs are working. I’m all for expanding access to drugs, and there is abundant waste and greed in big Pharma and venture, but there are also people doing hard expensive science, medicine and manufacturing. Im not sure I have the answer. A “yelp for medicine” won’t improve immediate outcomes, nor longer term understanding and progress. A great and excruciating read about the tension there is a real-time blog (the story’s story) that was written by Jake Seliger unt he passed in 2023.
Go watch or listen to Plenary Session, and you'll have direct access to his thoughts. My ability to rehash Prasad's arguments doesn't have any bearing on what I wrote.
Slightly different point but many bacteria in us right now also make lipopolysaccharide (LPS). If it were purified and injected iv, the LPS in me could probably kill me 1000 x over.
LPS is a critical component of gram negative bacteria, like E. coli.
We evolved LPS detection so long ago that it's in our innate immune system instead of adaptive immunity. It's so ancient we share this immune function with fruit flies.
LPS detection is so good and immediate because it's tuned to pick up single instances of LPS molecules. Not a few nmol. Single molecules. Detection will trigger inflammation and immune scale up to deal with the problem.
If you go injecting LPS or E coli into your blood stream, of course your own body is going to kill you. It'll freak out and think WW III has started and begin firing the nukes in every direction to stop it.
I agreee with the caution. I am not endocrinologist enough to
guess what may happen and when. Because of the level of variably in all that is being experimented with, my guess is there may be a slower burn rather than explosion of odd toxicities. It does feel like stuff will happen.
Will side effects for hormonal and gene therapy approaches be shaken out in just 3-5 years? For gene therapy, the rare blood cancers associated with car-t or bluebirdbio suggest maybe not. Maybe they remain rare, but as scale and flexibility of use increases, how that may evolve. Hormones are a whole different calculation. With the creative and dosing, combinations, and applications I’m not sure how many from conclusions will be available. I’m not judging good/bad here, I’m just thinking that this “democratization” of medicines (maybe otherwise not available to some) will increase access, with both risks and benefits.
“Viruses” have a very broad feature set-beyond evoking Batman, it seems like a lot of details need to be hammered out here, even residually chlorinated water can be problematic in maintaining titers.
IMO, These days, public health policy (conspiracy?) seems to be a more efficient way to spread pathogens. Not precise targeting tho.
AIDS has been treatable for a few decades, with good progression getting it to developing world and some towards a cure. That said, some of this recent progress has been impeded due to differing beliefs of some current governmental regulatory and research leadership, and beliefs that infectious disease treatments and vaccines are not needed for most people that are 'healty'.
This made me think of the "Institute for one world health" . It came out as a non-profit pharmaceutical company in the mid 2000's. Victoria Hale was the founder-it got her a MacArthur fellowship. It is focuses(focused?) on global health and populations underserved by for-profit models. I think they successfully developed a treatment for leishmaniasis. it's an adorable model and should be pushed but as usual it seems like the philantropy money is limiting.
