Dustin Moskovitz pours funds into high-risk research
nature.com
nature.com
A notable example is recent efforts to fund a study on the use of corticosteroids and vitamin C to stabilize patients with sepsis [1]- this would be a huge impact on mortality for patients with all sorts of diseases, since sepsis is a common consequence of lowered immune system functioning or post-surgical infections, but since neither corticosteroids or vitamin C are patentable, there's no commercial incentive to fund trials. Another example is the use of MDMA as a psychiatric drug- any highschool dropout can tell you that it works as a powerful mood-elevating chemical with relatively rare and benign side effects (at least by the standards of psychopharmaceuticals,) but clinical trials were only recently begun after decades of agitating from psychiatrists.
The most potent benefits from philanthropic funding tend to be in areas that can't be reached by conventional for-profit commercial work. There's still much more low-hanging fruit out there.
I don't know how Sepsis fits into this, but they specifically do trials that drug companies won't.
Adherence to "evidence based" therapy guidelines is typically enforced via malpractice claims. Obamacare tried to introduce incentives for outcome measurement/tracking and evidence based care by either cutting or supplementing medicare reimbursements, but even that has been difficult to implement nationally across the 5-7 most frequent and costly conditions and procedures (e.g. acute myocardial infarction, COPD, knee replacement, etc).
While the FDA's regulatory approach does slow the pace of innovation, they exist for a reason -- I acknowledge that GP is not calling for any policy change. Snake oil salesmen were a real thing, and still abound in the valley albeit in different form.
So to say that the issue is funding for FDA approval is probably an oversimplification- equally powerful studies without FDA involvement may or may not suffice, that's a question having to do with law and other non-science factors. But the FDA has established what is the broadly recognized "gold standard" of establishing the medical effectiveness of an intervention.
https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAcc...
(My understanding was something along the lines of, the FDA regulates which drugs can be used/sold but not how, so if a drug was approved for any purpose a doctor can use it "off-label," but a new drug still requires FDA approval. Is that accurate?)
For example, nutritional supplements are often recommended by doctors and incorporated in their practices, but are marketed as "promoting general health and wellness" and not treating a particular condition. Here you don't need any evidence of efficacy (or even safety), you only need to be able to show a "history of use". Many "structure-function" claims push the envelope (ex promotes liver health), but are still legal and FDA has been pretty consistent and clear about where they draw line.
Doctors can use anything already FDA approved off label but they don’t... unless there’s compelling evidence.
Beyond that, are you aware of any related notable research, sources, etc. - on the topic you’ve presented for additional reading or action.
I couldn’t find a comprehensive treatment of the broader issue on a quick check- I suspect that the nature of an issue revolving around “what we know vs what we formally know” resists discussion in places like scientific journals. But there’s some overlap with more well-known issues, such as antibiotic development- even though new antibiotics can be patented, the return isn’t enough to stimulate nearly sufficient development (e.g. https://www.pharmaceutical-journal.com/news-and-analysis/fea... .)
Of course, I anticipate a number of replies telling me how wrong I am and that regulations are needed for safety; none of them will mention the trade offs involved.
A growing number of people have voiced objections to the burdens of the FDA's efficacy testing requirements, but it's worth at least thinking about why they're in place. The main issue is that "safety" in the context of pharmaceuticals doesn't mean "perfectly safe" or even "non-lethal" — it really means, "safe enough, given the benefits". Side effects of a chemotherapy drug that can completely eliminate a tumor, for instance, could be acceptably substantial, because it cures an otherwise terminal illness. Side effects of a drug mildly lowering cholesterol levels, by contrast, must be strictly limited, because many Americans may take such a drug for much of their adult lives, sometimes in combination if the effects of a single drug are too weak.
It's hard to know if the benefits outweigh the risks if you don't know what the benefits are. Obviously, this efficacy testing carries a huge time and monetary burden, but without it, contextualizing acceptable risk would be difficult, if not impossible.
To get an idea of the money involved here, his life's fortune is about what is currently spent every 6 months on medical research that no one will ever double check (since it is impossible to do):
"Overall, the team found that poor materials made the largest contribution to reproducibility problems, at 36%, followed by study design at 28% and data analysis at 26%. The team estimates the overall rate of irreproducibility at 53%, but cautions that the true rate could be anywhere between 18% and 89%. That puts the potential economic cost of irreproducibility anywhere from $10 billion to $50 billion per year."
http://www.nature.com/news/irreproducible-biology-research-c...
And irreproducible in principle studies are just the tip of the iceberg. Beyond that there are many problems that can render research useless or misleading (p-hacking, bias due to unblinding, misinterpreting results, incorrect analysis, etc). Just pumping more money into this system rather than trying to change the incentives and practices seems wrong.
This will give you a decent overview of antibiotic resistance:
http://www.who.int/mediacentre/factsheets/antibiotic-resista...
If you like to watch documentaries, Frontline has a few on the topic, though each show is several years old now which is an eon to pathogens
https://www.pbs.org/wgbh/frontline/film/hunting-the-nightmar...
(on youtube https://www.youtube.com/watch?v=gE4VzFz9PPo )
https://www.pbs.org/wgbh/frontline/film/trouble-with-antibio...
If you want to see concrete progress thanks to Dustin and Open Phil, read about Target Malaria, one of the research organizations.
I am seeing firsthand the need in Southeast Asia: an emerging drug-resistant "super-malaria" is spreading along the Mekong and is impossible to treat with standard medicine.
Big U.S. pharma has no answer for this, and the problem is accelerating. I believe Open Phil and similar independent science funding orgs can give us all a major avenue of research to help save many lives.
Due to procurement not having a research background, they often due poorly negotiating prices for basic scientific chemicals and supplies. The result makes it very difficult for researchers to buy from the open market such as Amazon, etc.
Here are 12 tweezers for nearly $700 list price: https://us.vwr.com/store/catalog/product.jsp?catalog_number=...
Procurement feels good getting 45% off of list price across all products, but they do not compare the actual price.
https://www.amazon.com/Tweezees-Precision-Stainless-Steel-Tw...
For example, a contract gives the scientific distributor the right to match a price [1]. Scientists need to make this request through their procurement office or to a sales rep. which takes a couple of days and additional paperwork to complete. These extra steps create enough friction that researchers will often by through contracted providers despite them being more expensive.
[1] examples: https://procurement.fsu.edu/vendors/VWR https://www.dfa.cornell.edu/procurement/news/improved-prefer...
If you know anyone working in this field, it never hurts to contact this org.