Last week the House Energy and Commerce Committee announced it would expand its investigation into lab safety at federal facilities, specifically select agents such as anthrax. Last year there were a number of instances that suggested safety measures were being inconsistently implemented by the Centers for Disease Control (CDC), the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), all of whom have been involved with the facilities at issue. The CDC issued a report, at least one hearing was held, and the Government Accountability Office (GAO) was asked to investigate.
Since last summer there have been additional incidents. The House investigation, according to The Washington Post, has found some apparent irregularities regarding how inspection reports treat the use of cardboard in cold storage rooms. Those irregularities are important to the 2014 discovery of smallpox virus in cardboard boxes.
The House committee has requested the GAO expand its investigation to address questions related to these apparent irregularities. The letter also goes into detail about the various requests to federal agencies and their responses. My review of the letter suggests that the failures to adequately control the inventory of select agents can be attributed – at least in part – to miscommunications due to several agencies using the facilities over time, and a general failure to manage records over decades.
However, my word should be far from the final one on this matter. The ongoing committee and GAO investigations do not have a set end date, but I would think it behooves everyone involved to complete matters by the end of the calendar year, if not by the anniversary this summer of the initial lab incidents.
As occasionally happens both the Presidential Commission for the Study of Bioethical Issues and the President’s Council of Advisers on Science and Technology (PCAST) will meet in the same month. The PCAST meeting is on May 15th, and while I have posted about it already, there is now a draft agenda available for review. The session on business had already piqued my curiosity, and the agenda has only fueled my speculative interest. The panelist named in the agenda, Rebecca Henderson of Harvard Business School, has been working on disruptions to capitalism and how that economic system could manage major transitions.
The Bioethics Commission will meet in Philadelphia on May 27. No agenda is currently available, but the Federal Register notice indicates the meeting will focus on public engagement on bioethics issues (using deliberation) and bioethics education (also involving deliberation).
Deliberative democracy is a research interest of the Commission’s Chair, and ethics education was part of the Commission’s recommendations in its Gray Matters report, so it makes sense that these would be subjects of a Commission meeting. Additionally, the Commission issued this request for comment in April for information relating to both public deliberation and education on bioethical issues. The call will be open until July 20, so you may wish to watch the meeting (in person or online) before submitting your comments.
At the beginning of April, the Japanese government launched the Agency for Medical Research and Development (AMED) (H/T ScienceInsider (subscription required for full article)). This is following through on plans discussed in 2013 (and earlier) and legislation passed last year.
The agency will function as a central manager for government medical research in Japan. Prior to AMED’s launch, medical research was supported through institutes located in three separate ministries. Now AMED is coordinating the effort through 300 staff and a budget of 140 billion yen (roughly $1.2 billion).
Based on reports and a review of the English translation of the website, practical applications of AMED supported (and conducted) research will be practical application. Agency performance indicators for 2015 and 2020 (slide 4 in this 2014 presentation) strike me as aggressive and possibly too output focused (rather than outcomes focused). Should these goals be accomplished, the relative efficiency of the government investment (its annual budget is roughly 4 percent of the National Institutes of Health budget), would be remarkable.
It’s a perfectly understandable to want frequent screenings for various diseases. But to screen for various problems every year can cause problems that, while not as life-threatening as the diseases the tests are designed to detect, have their own costs.
Take the case of cervical cancer. A very preventable form of cancer, increased screenings and earlier detection have decreased its frequency and lethality. But the nature of the disease and the requirements of screening do not make it something worth doing annually – regardless of how many lives are saved by early detection. The U.S. Preventive Service Task Force recommends screenings for cervical cancer every three years for women 21-65 with an average risk for the disease (there are additional recommendations, but I’ll focus on this one).
A best practice review published in Annals of Internal Medicine examined the consequences of more frequent screening for women with low risk of cervical cancer (H/T Popular Science) (The level of risk is important. A family history of any of these diseases would affect the recommendations for screening.) The review indicated that clinical practice does not adhere closely to the recommendations, resulting in more frequent screening and screening of those outside of the recommended age range. As abnormal test results for those younger than 21 does not often result in actual cervical cancer, this leads to increased testing and procedures that are not warranted. This leads to increased costs for tests and unnecessary procedures (and associated trauma) for patients.
So, to repeat the message I’ve tried to communicate in previous posts on preventive screening, it’s important to place these screening tests in a proper context. One’s risk for the disease has to be considered along with the effectiveness of the tests and the consequences of false positive test results.
Yesterday the Supreme Court heard arguments in Glossip v. Gross, a case brought by condemned men in Oklahoma objecting to its method of capital punishment. The argument in this case hinges on the state’s use of midazolam as the sedative in the three-drug cocktail of anesthestic, paralytic and a drug to stop the heart. Death penalty opponents argue that the drug does not adequately sedate the condemned inmate, allowing them to suffer sufficient pain from the subsequent drugs to qualify as cruel and unusual punishment.
While it is rarely a guarantee that oral arguments indicate exactly how the justices will rule, it can be suggestive. Reports from the Court indicate that the justices questions focused primarily on two threads. The liberal justices were concerned about the effects of midazolam, while four of the conservative justices focused on what they consider a judicial end around. As I’ve tracked on this blog, manufacturers and others have put drugs used in executions out of reach of states, forcing them to choose alternative drugs, and in some cases expand their execution options.
The justices’ questions about these tactics suggest they are upset at their decisions (about the Constitutionality of the death penalty) being subverted by what could be seen as technical, rather than political, means. While I’d argue that the campaign to get execution drugs pulled is definitely political, I can see where the justices that support the death penalty see this effort as a turf battle.
Again, the oral arguments are more of a guide than a predictor of the court’s vote (much less the specifics of its opinions). But it would seem that this case is unlikely to change the status of the death penalty. And while some justices object to the movement to make certain drugs inaccessible for executions, there is little they can do. In March an Illinois manufacturer of midazolam requested the State of Oklahoma return its supply of the drug (for a refund, of course). The company, Akron Pharmaceuticals, will also take steps to make sure its supplies of midazolam and hydromorphone (a narcotic pain reliever some states have expressed interest in using) cannot be used for executions. Similar letters have been sent to other states. Should other manufacturers follow suit, it may not matter what the Court says when it rules in this case.
Part of the Precision Medicine Initiative is the establishment of a research cohort. Intended to be a million strong, the voluntary cohort would contribute a breadth of data to help extend the understanding of diseases.
As part of this process the National Institutes of Health (NIH) is seeking information from the public about the cohort. Comments are due on May 7. Specific questions the NIH is interested in are:
“A. General topics on the development and implementation of this large U.S. cohort.
1) The optimal study design and sample size for a large U.S. precision medicine cohort.
2) Data to be collected at baseline and follow-up, including mode of collection and frequency and length of follow-up.
3) Potential research questions that could be uniquely or more efficiently and effectively pursued in a large U.S. precision medicine cohort.
4) Any other suggestions for NIH to consider in the development and implementation of such a research cohort.
B. Suggestions for existing or potentially new research entities (a health care system, research network, cohort study or consortium, or other entities such as longitudinal studies using digital-based platforms) that might be combined into a large U.S. cohort. Providing the following information would be useful when suggesting research entities.
1) The capability of the existing or potentially new research entity to efficiently identify and follow 10,000 or more participants who are likely to consent to providing their medical and other health-related data, biospecimens, and genomic data for broad research use, including in sub-group analysis that could help assess various treatment effects and outcomes. It would also be useful to provide the rationale that potential participants are likely to consent, as well as experience with and ability to participate in central IRB and a master contract agreement to streamline enrollment of the precision medicine cohort.
2) The capability for the research entity to provide individual-level participant data, particularly those from electronic health data (including both electronic health record and payer data), that can be integrated into a standard format to create a combined large longitudinal precision medicine cohort.
3) The capability for the research entity to track and retain the participants for several years of follow up. The race/ethnic composition, sex, and age distribution of participants from the research entity likely to consent, by standard U.S. Census categories, would also be helpful. The NIH especially seeks information about studies of populations underrepresented in research and those with phenotypes or disorders of high public health and human impact. Additional information that would be of use includes: for health care systems, the current patient turnover rate and efforts that can be made to capture longitudinal data from clinical visits outside of the system and to continue follow participants who leave the system entirely; and for ongoing cohort studies, the retention rate to date.”
This strikes me as uncharted territory, depending on how many recruits can be found to participate in this cohort. Should it truly be a million strong, there will be new questions that we don’t necessarily have answers for right now. They include how to manage the study(ies), maintain the anonymity and privacy of the medical information, and simply how to analyze such massive amounts of data. There’s a lot that can go wrong here, with unclear benefits on the horizon. Personally, I consider this a risk worth taking. Hopefully it will be remembered that there are risks involved.
The next meeting of the President’s Council of Advisers on Science and Technology (PCAST) will take place in Washington, D.C. on May 15th. As is usually the case, the public session will take place at the National Academies main building from 9 a.m.-noon on a Friday (no respect for possible West Coast webcast viewers.).
The draft agenda is not yet available. However, this Federal Register notice suggests the main themes for the meeting.
“PCAST is scheduled to hear from speakers about the Quadrennial Energy Review and about the Precision Medicine Initiative. The Council will discuss and hear remarks about reimagining business roles to address significant societal challenges.”
I’ve posted about the Precision Medicine Initiative and the Quadrennial Energy Review in the past. Both are new programs, though PCAST has called for a Quadrennial Energy Review since at least 2010. I can only guess, and guess badly, at what PCAST has in mind about reimagining business roles, and how that might be connected to the group’s science and technology portfolio.
More information, particularly the panelists for each topic, should be available via the PCAST website as May 15th approaches.