The FDA is not holding back effective drugs

It’s going unnoticed amidst the news of the rolling disaster that is the incoming Trump administration, but our lame duck Congress has just passed a major piece of legislation called the 21st century cures act. Scientists are happy about the extra $5 billion this bill gives to the NIH – sort of. That money has to go to specific programs, like the Precision Medicine Initiative and Biden’s Moonshot program, rather than being put into the general funds of the NIH, meaning that Congress, and not the NIH, is deciding what specific research to fund. That’s generally not a good idea, but more money toward broad research and translational initiatives like cancer and precision medicine is still a net win.

More controversial are the FDA provisions of this bill. The bill pushes the FDA to take into account other, often less rigorous types of clinical studies when it decides whether or not to approve a new drug. Some worry that this means drug companies will have more leeway to push unsafe or ineffective drugs on the market. I’m more ambivalent – there are cases (drugs for rare diseases) when double blind randomized clinical trials may not be right, and the FDA should have the flexibility to demand the best evidence appropriate to each case. If – and this is a big if as we look ahead – we trust that the FDA can stand up to industry pressure, than giving them more flexibility to follow best scientific practices is the way to go.

My bigger problem with the FDA provisions are that the premise is flawed. As I write in Pacific Standard this week, the bill’s sponsors argue that, by cutting regulations and red tape at the FDA, we’ll free new cures that are just waiting to be put into the hands of patients. That’s wrong – the FDA is not the rate limiting step here. There is no backlog of effective new drugs just waiting to be approved.

Go check out my piece for the details. The rate limiting step is the science. Medical science is hard, and diseases are understood imperfectly. If you want more effective drugs faster, we need to invest more in research.

Filed under: Follies of the Human Condition, This Mortal Coil, Uncategorized Tagged: drugs, FDA, science funding

Standing Up For Basic Research

I don’t like getting drawn into partisan fights when it comes to science, because federally funded research has generally had broad bipartisan support in Congress for more than a half-century. But there is no point in denying that the funding bill that just passed the House is a blatantly partisan effort by GOP leaders to impose their political ideologies — which include climate change skepticism — on our research agenda.

As I discuss in my recent Pacific Standard column, curiosity, and not partisanship should drive our scientific agenda when it comes to basic research. That’s generally been the rule at our basic science agencies, and the current leadership of the House of Representatives doesn’t like it.

This week, the House passed a highly partisan funding bill that would dramatically and disastrously reshape our science agencies along the lines of GOP preferences. Among the mischief attempted by these lawmakers is an push to slash funding for earth sciences at NASA and the NSF, in a poorly disguised effort to dial back climate change research. Representative Bill Foster (D-IL), a physicist, put it best: this bill reflects “the majority’s efforts to impose their own personal beliefs and ideologies on the process of scientific discovery.”

Fortunately, the White House has issued a veto threat. And its Office of Science and Technology Policy is pushing back against the GOP leadership’s misleading claims that they’re just trying to get the NSF and NASA to focus on “core science” or research that’s “in the national interest.” The OSTP has started a Twitter campaign under the hashtag #BasicResearch:

The Value of Basic Research

People’s appreciation of game-changing new technologies frequently ignores the long, often twisting path that transforms an idea from fundamental discovery to practical application. Those who pay for the national research agenda may not always be aware of the early and fundamental work that makes today’s technologies possible…

But basic science has long been under fire. Between 1975 and 1987, the “Golden Fleece Award” was established and bestowed upon projects they deemed “the biggest, most ridiculous or most ironic example of government spending or waste.” Often, the “winners” were Federally funded scientific research projects taken out of context and cited without explanation…

The heyday of the Golden Fleece Award has passed, but misunderstanding of the value of basic research and its ties to valuable applications, products, and knowledge survives today…

The road to many of the next great scientific or technological advances will start with basic science. I encourage you to share your favorite examples of basic research that led to unexpected insights or game changing applications on social media using #BasicResearch.

Over at Pacific Standard, I review one example of how this plays out: CRISPR/Cas9 editing, a technology that is quickly becoming as important as other key biotechnologies like restriction enzymes, PCR, GFP, and high-throughput sequencing. This game-changing invention came from incredibly obscure origins.

The U.S. has been deliberately investing basic research for a half century for a good reason: it works. The payoff is enormous. And that means, as I wrote, “Letting scientists, rather than politicians, define important scientific questions is definitely in our national interest.”

Filed under: Follies of the Human Condition Tagged: science funding, Society and Science

Fun(d) with Science

Many researchers will tell you that financing their work–writing grants, securing funding, and budgeting for varying funding levels year to year–is the least rewarding part of life in academia, but there’s no escaping the simple fact that science costs money. … Continue reading »

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The paradox of more science funding, less research… we’ve seen this before

Does this sound familiar?

Since 19XX, overall federal research funding in all fields has shown a steady increase, resulting in greater than 40 percent growth (adjusted for inflation) from 19XX to 19XX. University-based researchers have been the primary beneficiaries of this growth. Although the data are harder to come by, relevant Figures from [Agency X] and several universities indicate that the growth in funding for XXX research has been comparable to these overall trends.

However, these figures lump together many different kinds of projects and funders. For example, one element of xxx funding is the base-funded (or core) program, which is the primary source of support for small science endeavors. This report looks at base-funded programs at both NSF and [Agency X] and finds, contrary to the trends described above, that they have not even kept up with inflation and have certainly not been able to keep pace with the explosion in grant requests. As a result, grant sizes have decreased, and the percentage of proposals accepted has dropped. A rough calculation shows that researchers must now write two to four proposals per year to remain funded, up from one or two in 19XX. Of course, increasing the time spent searching for support means that less time is spent on productive research. Rising university overhead and fringe benefit costs, that consume more and more of each grant dollar exacerbate this problem. Clearly, the base-funded program has not participated proportionately in the overall XXX research funding increase. Although we do not attempt to quantify the effect this has had on the quality of science produced, we do find that the core program has become much less efficient during the past decade. We also infer that the lion’s share of new funding has gone into project-specific funding, most of which involves big science efforts.

I’ve blanked out a few things… can you guess what area of research and what time period this refers to? The answer is below the fold.

This is from the executive summary of the National Academies’ report A Space Physics Paradox: Why Has Increased Funding Been Accompanied by Decreased Effectiveness in the Conduct of Space Physics Research?, written in 1994 and detailing trends from 1975-1990. Agency X is NASA.

Biomedical scientists are now learning the same lessons:

Big Science vs. Little Science: How Scientific Impact Scales with Funding

Research efficiency: Perverse incentives

I’m not sure what the answer is, but just adding more funding is not the answer.