For a novel I’m working on, I’ve been thinking a lot about the role of science in capitalism, and the role of capitalism in science. It’s relevant to patient advocacy too.
Something I didn’t fully comprehend before the first time I volunteered for a research study was that the scientists who do basic science (the early work of trying to understand things before there’s a breakthrough discovery that can be developed further) have to be entrepreneurs. They’re mostly academics, who are paid to teach and to provide other services to a university, but 1) they need to also do research and get their findings published in order to keep their academic jobs, and 2) they need to get money from outside the university to pay for the costs of that research.
Since the research is separate from the academic endeavors (even though it’s also required to maintain the academic position), the researchers establish a lab to do the research, and those labs are, in many ways, basically start-up companies that need funding in order to create a “product” (a research study). We think of that funding as being provided by venture capitalists, since banks aren’t going to give money for work that almost never turns a direct profit. Venture capitalists do get involved in later stages of biomedical research, but only once a potentially viable product has come along, not at the stage of basic science (like the work that discovered FGF23), because the likelihood of making a profit is infinitesimally small.
So how do researchers fund their basic science research? Sometimes it’s self-funded, but that gets expensive fast, as I’ll explain below. More often, it’s through grants, either from a pharmaceutical company that thinks the research might have implications for its own products or interests, or from a governmental entity like the National Institutes of Health that has a mission of supporting basic science.
Unfortunately, there just isn’t enough money from either source to do all the basic science research that still needs to be done. It sometimes feels like medical science has all the answers already, but in reality, it’s barely begun to scratch the surface of how our bodies (and minds) work. One example that everyone in the chronic hypophosphatemia community should be aware of is the calcification/enthesopathy process. It happens in a lot of disorders (like XLH and ENPP1 deficiency) and even more widely with aging, but no one in the medical community fully understands the basic science behind it (the biochemistry of how and why it’s regulated). And without that basic science, there can’t be a treatment or cure that works across all the disorders where it occurs.
If you follow the coverage of Rare Disease Week (or attend Rare Disease Day at NIH virtually), you’ll likely see patient advocates and representatives from medical groups saying to politicians, over and over, that we need more money for basic research. For obvious reasons, the common medical conditions like cancer and heart disease and dementia tend to get the bulk of the research money. It’s not enough for them, even so, and then on top of that inadequacy, rare disorders have to scramble for the rest of the money. With some seven thousand identified rare disorders and multiple labs working on each one, you can do the math and realize the competition is fierce, budgets have to be as tight as possible to have any chance of getting funded, and more researchers are denied money than receive it.
Some disorders either have a large enough community or an engaged enough community to raise enough money for the basic research that venture capitalists aren’t interested in. They don’t expect to make a profit, which is why this kind of research grant is called venture philanthropy (and I’ll be talking about that more later in the year). They do it, not for a profit, but because it’s part of their mission, to improve the lives of the relevant patients and caregivers.
Smaller, less well-funded communities can support only the tiniest of basic research studies. It’s not cheap though. Remember that the researcher is basically its own little start-up company, and even if the lab isn’t expected to turn a profit, it needs to cover its expenses.
What are those expenses? In theory, they’d include overhead (rent, utilities) and staff, and in fact a percentage of those costs can go into a grant application (but need to be a small percentage of the total or the grant will be denied). For simplicity’s sake, let’s say the lab is operating out of an already paid-for space and doesn’t have any employees. And it’s going to do the simplest of research, basically just a brief little survey that will be analyzed and reported on
“That can’t cost much,” you think. “A few hundred bucks, and it’s done.”
Ha!
Even if you zero out all the usual expenses that a business has, even if you don’t factor in any kind of stipend for the patients who are responding to the survey or travel reimbursement if it’s an in-person interview, there’s one big expense that can’t be avoided: publicizing the results of the research. The goal of a research lab isn’t (primarily) to make a profit like other types of start-up companies, but to find answers that someone else can then turn into a product (which for drug development costs hundreds of millions of dollars, way beyond what a single researcher can raise funding for). And that means the research and its results need to be publicized so others know about it.
How do you publicize the results? You write an article and get it published in a reputable journal. Easy, right? After all, writing up research is part of what these scientists are trained to do, right?
Unfortunately, it’s not the writing that’s the problem. It’s the challenge of getting it published. (The same is true in fiction-writing, by the way, where I have more direct experience. Writing stories is hair-pullingly difficult at times, but it’s still a million times easier than the work required to find a publisher and then to find readers who are interested in the story.)
Let’s skip over the hurdle of having to convince the editors that your research will be interesting to its audience (which is challenging for research into rare disorders, since editors may think the topic doesn’t have broad enough appeal for their readers). Even after the editor agrees your research is relevant to their readers, and the peer reviewers have confirmed that it’s scientifically acceptable, you still need to cough up some cash to get the article published. And not just a few hundred bucks either.
Since Dr. Glaucomflecken (pseudonym for opthalmologist who’s become well known online for his humorous but based-on-truth social-media commentaries on the U.S. medical system) explains it better (and funnier) than I could, here’s his recent youtube video, “Academic Journals Doing Crime,” on how science journals work. Note that, as the doctor explained on his Twitter feed, he didn’t make up the dollar amount cited in the video. It’s absolutely not inflated for comic effect, as many viewers initially thought.
The bottom line, as the video points out, is that it takes money to get research included in a prestigious journal (and the prestige affects the perception of the research’s credibility). That money needs to be raised by the owner of the research lab as part of the grant application. Which further restricts the amount of research that can be done by the limited pool of grant money. And also places an extra burden on tiny labs working on rare disorders when larger labs working on more common disorders are likely to be better funded, so they don’t have to spend any time on the hard decision about whether they can afford to make the research widely available.
I only know the very basics of the costs of a research lab, but I’ve had some fairly direct experience with the cost of getting a journal article published (more on that later in the year). I’m offering this expense just as an example of how researchers can’t be the carefree stereotypes of the absent-minded professor, happily looking into microscopes, or staring out a window contemplating the building blocks of the human body, insulated from mundane concerns about money. Instead, the real picture is of exhausted but dedicated scientists who are willing to do both the research that they’re obsessed with, and the time-consuming, very-grounded-in-real-world entrepreneurial/administrative/managerial work of raising enough money to pay the costs of that research.
So, if you’re a fan of The Witcher (or even if you’re not), please remember to toss a coin to your researcher every once in a while. That “coin” can be a simple thank you that acknowledges their hard work, or a financial contribution to an organization that provides grants, or volunteering to advocate for more money for basic research. Researchers won’t stop doing the research (as long as they can get at least some funding), even if they don’t get any “coins” from the community, but you’ll make their day if you let them know, by word or action, that you appreciate their hard work.
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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