The National Institute of Mental Health (NIMH), one of the National Institutes of Health (NIH), is changing its criteria for funding clinical trials. Director Thomas Insel explained the decision in a blog post late in February (H/T Nature News). It’s a shift in focus that resonates – at least for me – with the NIMH decision last year to not use the DSM-V in how it organizes research on mental illness.
For clinical trials moving forward, NIMH will require that the proposed treatment being tried is not the sole purpose of the trial. The trial must also generate knowledge about the mechanisms underlying the relevant disorder. In other words, it will not be enough for a trial to see whether a drug has the desired outcome (usually controlling symptoms). The trial would also have to increase the knowledge base around the underlying condition. This shift in focus will accompany a set of new standards for efficiency, transparency and reporting. Insel and others at NIMH have looked at current Institute trials and found them wanting.
“Recent performance in our clinical trials program is not acceptable: recruitment is too slow, registration in public databases is not consistent, and reporting takes too long to meet the needs of the public for better treatments. To respond to the public concern that “time matters,” we will be establishing new requirements for timelines, trial registration, publication, and data sharing.”
The new criteria will apply to all new applications of trials, effective immediately. The transition will not be easy, but the prospect of being able to do more than just treat the symptoms of psychological conditions is enough to make the Institute act.
A Food and Drug Administration advisory panel provided some filler for the 24-hour news channels this week. The reason: discussion of mitochondrial replacement as a means of in-vitro fertilization (IVF). It involves a ‘donor’ cell which has its nucleus removed to receive the nucleus of the mother (it bears some resemblance to somatic-cell nuclear transfer, except there is an additional DNA source involved). This method could be used for situations where the mother has mitochondrial defects that could be passed on to the offspring.
The method gets the superficial cable news attention because the resulting offspring would have DNA from all three donors. While the Presidential Commission for the study of Bioethical Issues has not weighed in (it doesn’t meet again until June), the FDA panel discussed the state of science and research on the technique, as well as the design requirements for early-phase clinical trials. Researchers have produced monkeys via this IVF technique, but panel members were reluctant to recommend human trials at this time.
Over on the other side of the Atlantic, the U.K. is further along in regulating the technique. The appropriate advisory bodies started assessing mitochondrial replacement in 2011, and the government announced last year that it was working on regulations. It issued a consultation on Thursday for the draft regulations (questions of interest are on pages 27-28). Responses will be accepted until May 21.
The Food and Drug Administration (FDA) is going to study whether or not drug advertisements on television need to be changed. There are concerns that the current model, where all of the drug’s side effects are supposed to be listed, is giving a realistic picture of the benefits and harms of the advertised drug.
(FWIW, it certainly provides an easy, if sometimes lazy, vehicle for comedy.)
The Federal Register notice describes the possible study, and explains what the FDA hopes to accomplish. The idea is to examine the effects on consumer understanding of providing different levels of risk information in a direct-to-consumer advertisement (while the request is specific to television, I have to think any changes would influence other advertising).
“Our hypothesis is that, relative to inclusion of the full major statement, providing limited risk information along with the disclosure about additional risks will promote improved consumer perception and understanding of serious and actionable drug risks. We will also investigate other questions such as whether overall drug risk and benefit perceptions are affected by these changes.”
As the FDA is at the study phase of this particular regulatory process, it will be months before we have a different form of commercial for comedians to mock.
Two recent actions involving the Food and Drug Administration (FDA) suggest to me that the matter of whether stem cell patents are valid may not be so critical.
The D.C. Circuit Court of Appeals ruled last week that the FDA has jurisdiction over stem cells cultured for therapeutic use (H/T The Scientist). This decision upheld a lower court ruling that considered the act of culturing the cells more than ‘minimal manipulation,’ and therefore subject to FDA drug oversight regulations.
(IANAL, but I think this decision could be used to strengthen the case that stem cell patents – at least for cultures of said cells – would be valid. After all, if there was more than minimal manipulation, wouldn’t that be sufficiently transformative to make the cultures no longer products of nature? Again – I Am Not A Lawyer.)
Aside from the legal matters, there appears to be a big regulatory mismatch that will hinder commercialization of stem cell treatments. In the February 6 edition of Cell Stem Cell researchers note (H/T The Scientist) that differing regulations between the National Institutes of Health (NIH) and the FDA may reduce the number of stem cell lines that could be used in clinical practice.
FDA regulations require that stem cell donors be screened for various diseases (so that treatments derived from those cells cannot infect others). NIH regulations – not focused on commercial applications of stem cell research – do not have this requirement. Now it is possible, as the article from The Scientist notes, for the FDA to allow some treatments to be approved without such a screening, but some alternative measure will likely be needed to mitigate the risk of infection.
Edited to add - As noted in the comments, I managed to conflate the Food and Drug Administration with the Federal Trade Commission. This is perhaps the biggest screwup in the history of this blog, and I apologize. The following text has been edited to reflect that, with strikethroughs and new text in italics. (And I had to take two runs at revising the post title. I was inexcusably sloppy.)
While it may feel like it, the Federal Trade Commission (FTC) is not focusing all of its attention on the genetic testing company 23andMe is not the only genetic testing company getting scrutiny from federal regulators.
(The agency Food and Drug Administration (FDA) issued a warning letter to the company because it considered the marketing of its genetic testing kit to lack necessary approval from the agency. Given how the FTC FDA believes 23andMe was selling the kits to provide medical information, the agency concluded that the tests were subject to agency regulation. Months of negotiation to obtain evidence of the efficacy of 23andMe’s tests did not resolve the matter, which led to the warning letter. )
The Federal Trade Commission (FTC) has brought a lawsuit against GeneLink Biosciences related to a service it provides based on genetic tests. The company will provide nutritional supplements based on a customer’s DNA sample. They claim various health benefits based on the genetic tests, and the FTC is suing the company for what it considers false advertising.
In both cases the agency involved believes that genetic testing companies are making claims that they either have not or cannot provide evidence. If the claims are considered to be ahead of relevant science (or absent same), the FTC (or the FDA) will act.
GeneLink and the
agency FTC are negotiating a settlement agreement. The final agreement will include provisions to improve the security of collected information.
Over three years since I’ve posted it, this item on the (in)efficacy of hand sanitizer is among the top ten posts on this blog (by page views).
Turns out there could be similar concerns about so-called ‘antibacterial’ soap. On Monday the Food and Drug Administration (FDA) announced that it would be taking a closer look at these soaps. Specifically, it has issued a proposed rule that would require manufacturers to provide additional safety data to the agency and demonstrate a clinical benefit of their product(s). Comments are accepted until June 16, 2014.
Why? Because there does not appear to be any evidence that ‘antibacterial’ soaps are any more effective at preventing illness than regular soap and water. Additionally, there are concerns that the use of antibacterial soaps could contribute to the ongoing emergence of antibiotic-resistant bacteria.
For the record, the CDC recommends regular soap and water for handwashing. If you don’t have immediate access to soap and water, and must use a hand sanitizer, use one with at least 60 percent alcohol. (Though, as I posted back in the day, some drug-resistant bacteria don’t mind the alcohol.)
When I’ve posted about trends in antibiotic resistant bugs, it’s usually to join the chorus who argue that antibiotics are overused. But this item from Gregory Daniel at the Brookings Institution helped remind me that matters of antibiotic demand are not the only economic forces at play.
Daniels uses a recent Centers for Disease Control (CDC) report on antibiotic resistance to outline several proposals for addressing the challenges facing antibiotic drug development. The CDC report identifies 18 different bacteria that pose varying levels of threat due to their resistance to certain antibiotics. Daniels focuses on two major thrusts – adjustments to the drug development process and changes in the business model. Both were address during a February 2013 Brookings workshop on the topic. The first is familiar to many who have followed drug development policy, and the second was new to me.
But, frankly, I should have seen it. I’ve noted at length about how production capacity for a variety of minerals, drugs and elements dried up over the last few decades. Similar forces appear to have been at play for antibiotics. Whether swayed by excessive development costs, or what was falsely seen as a limitless future for certain antibiotics, I don’t know. But we have yet another item that we are ill-equipped to manage when it comes time to either make more, or make a necessary substitute. Advances in technology have, perhaps perversely, made us worse in certain respects, about managing resources (or at least their supply chains).
The President’s Commission for the Study of Bioethical Issues will meet in Philadelphia on August 19 and 20th, the fourteenth meeting in its current form. While there is no agenda on the Commission website right now, the Federal Register notice linked to mentions two major items.
The Commission will continue its work on incidental findings – research results derived from human subjects research on unrelated matters (which poses ethical issues given standard practices regarding consent for research). I would anticipate hearing during the meeting when the Commission may release a report related to incidental findings.
It will also engage with the BRAIN Initiative. While that was only a well-educated guess when the initiative was rolled out in April, the President made it official in July. Per the President’s request the Commission will not limit itself to considerations of proper ethical research practices in neuroscience, but also to consider relevant applications of this research and the possible ethical consequences.
Last week The U.S. Circuit Court of Appeals for the D.C. Circuit ruled in a case involving the Food and Drug Administration (FDA). The FDA was subject to a lawsuit on the issue of whether or not the agency should allow the importation of drugs that it cannot (or will not) test for safety.
The drug at issue is sodium thiopental, a drug often used in a three drug combination to execute prisoners in the United States. The drug is an anesthetic, which is why there is still a demand for the product outside of prisons. As I have posted about in 2011 and 2012, supplies of the drug in the United States have dried up through a combination of factors. Domestic manufacturing capacity has disappeared, foreign suppliers have started refusing to export to the United States, and the demand to execute has not diminished.
The appellate court decision affirms the lower court decision in part, that the FDA did not act in accordance with the law to allow the import of sodium thiopental – which it has not approved for use in executions – into the country. The court argued that the agency has other means at its disposal to address such a drug shortage. The appeals court did reverse part of the District court’s decision and will allow states to retain possession of their existing stocks of the drug.
The FDA may still appeal the decision.
Last week the Food and Drug Administration (FDA) announced that it would examine the trend of food products that included caffeine. This has already prompted one company to pause production on a caffeinated gum. The announcement follows action in 2010 to withdraw caffeinated alcoholic beverages from the market. Part of the rationale behind that effort was that the addition of caffeine masked the usual cues people receive from alcohol consumption. (So those arguing the latest action marks a new intrusion aren’t paying close attention.)
Currently FDA regulations allow the addition of caffeine to products if it meets safety standards and gets included in the ingredients list. It has only approved the addition of caffeine to products once – for colas in the 1950s (perhaps because it was a replacement for coca).
James Hamblin and others at The Atlantic has been studying the effects of caffeine and its regulatory history. It would appear that the FDA is focused in part on caffeine in products that are or could be marketed to children. Michael Taylor, the FDA deputy commissioner that was interviewed for the announcement, appears to suggest that things like energy drinks and breakfast foods with caffeine are the kinds of things the agency is particularly concerned about. The FDA hasn’t set a level for children, but it recommends a maximum of 400 milligrams for healthy adults.
While it is possible that the recent announcement would be enough to cut back on the number of caffeinated food products, I suspect the FDA will pursue further study. Whether that ultimately leads to regulation is uncertain, in part because it would not likely be a blanket prohibition. While implementation of any kind of caffeine regulation will be problematic, focusing on certain products and a specific audience will require more time to develop.