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Posts Tagged ‘clinical trials

Science Policy Around the Web September 30th, 2019

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By Allison Dennis B.S.

Image by Gerd Altmann from Pixabay 

23andMe, moving beyond consumer DNA tests, is building a clinical trial recruitment business

Clinical trials are often criticized for poor patient recruitment that inadequately represents patients from minorities, e.g., women of reproductive age, racial minorities, underserved patient populations and people with rare genetic diseases. To fight this, 23andMe feels that its vast database of patients comfortable enough to trade their genetic information and a small bit of spit to learn more about their genetic predisposition to disease and heritage could be a much-needed recruiting tool for those enrolling patients in clinical trials, and one they would be willing to pay for. 

23andMe can mine the database of their 8 million potential research participants to identify which customers fit the needed demographics and genetic profiles relevant for studying a particular drug. For example, 23andMe found about 7,500 carriers of a rare mutation in the gene LRRK2, a potential target being pursued by GlaxoSmithKline to prevent the progression of Parkinson’s disease. In patient populations, this mutation is only found in one in a thousand people, meaning it would take years to identify enough participants to enroll in a trial. With enough potential participants to choose from trial managers are hoping to to recruit local patients, allowing them to overcome the costs of paying for patient travel and ease the stress patients experience by spending extended time away from home.

To augment the diversity of their database, 23andMe has launched programs to provide free genetic analysis to people whose four grandparents were born in the same country. The Global Genetics Project focuses on underrepresented countries like Mali, Tajikistan, and Paraguay, while the African Genetics Project focuses on the ethnic and tribal groups of countries such as Cameroon, Ghana, Libera, and Senegal. By starting with the genomes of people with clearly known ancestry to better define the genetic differences common to them, 23andMe is hoping it will be able to improve its unravelling of more complicated genetic lineages. A greater diversity of enrollees is sure to appeal to clinical trial recruiters, who are trying to meet FDA guidelines designed to enhance the diversity of clinical trial populations.

It remains to be seen how receptive 23andMe customers will be to being targeted for participation in clinical trials. Many of the 8 million may not have fully realized that consenting to genetic research would potentially extend to drug companies paying for their data. However, for many people, the possibility of playing a role in the discovery of cures for genetic diseases may outweigh privacy concerns and any initial discomfort from being singled-out. 

(Rebecca Robbins, STAT News)

NIH reveals its formula for tracking foreign influences

NIH’s extramural research program is struggling to find the balance between curbing inappropriate information sharing, fostering the international collaboration recognized as necessary for the global pursuit of science and maintaining some level of ease in the grant application process. The grants that are distributed by NIH are won following review by a panel of experts, composed of about 27,000 reviewers recruited to serve on committees by NIH each year. It is believed that some reviewers have violated NIH policy by sharing the confidential contents of grant applications with international colleagues. The data and research approaches described in grant applications is proprietary and often has the potential to result in patents in addition to publications. 

A more complicated scheme concerning officials involves international researchers setting up labs in parallel, one in the US to win grant money, and one in a foreign country where the grant winner wishes to benefit from any emerging intellectual properties stemming from the funded research. Such an arrangement allows US funded discoveries to be transferred to another country without any oversight from the US government. 

It is difficult to clearly spot scientists unfairly leveraging their role in NIH grant giving activities. While grant reviewers are not supposed to share any parts of the application, it is common for researchers to share grants with trainees in their lab, sometimes seeking relief from the burden of review by having junior researchers take the first look, other times viewing it as a needed opportunity for future grant-seekers to become familiar with the process. Grant applicants routinely have joint appointments with international institutions. This is acceptable if clearly disclosed when they seek funding from NIH. 

In the past the issue has been treated as a regulatory hot potato, NIH requires grant seeking institutions certify the grantee listed on the application, institutions rely on the assumption that foreign scientists would be denied visas by the US Department of state if they were thought to have improper ties to their home countries. Of course, citizens that are US citizens are at risk of behaving improperly as well. NIH is taking the next sixth months to develop a risk-based approach to flag potential conflicts with reviewers. As a first step they are considering moving grant reviews to a read-only online portal to prevent any untracked distribution of downloaded applications. 

 (Jeffrey Mervis, Science)

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October 1, 2019 at 11:32 am

Science Policy Around the Web August 20th, 2019

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By Mohor Sengupta PhD

Image by Ryan McGuire from Pixabay 

FDA makes new push for graphic warning labels on cigarettes

According to government records, the percentage of smokers in the U.S. has reduced from 40% of the population in the 1960’s to 14%. Despite this dramatic decrease, approximately 480,000 people in the U.S. die each year from diseases directly related to smoking. In an attempt to educate smokers about the dangerous medical consequences of smoking, in 2009 the FDA proposed 9 graphic labels to be printed on cigarette packets. However the FDA’s plan didn’t see the light of day when five tobacco companies challenged the FDA’s decision in court and in 2012 won on grounds of free speech. The judges who ruled in favor of the tobacco companies said that “[the images were] crafted to evoke a strong emotional response,” rather than educating consumers. 

The FDA then backed out saying that it will work on introducing new images and it was soon to announce new proposed labels. In 2016, a lawsuit was filed by health groups against the FDA for not proposing a new plan soon enough. Last Thursday, the FDA announced 13 new graphic labels to be displayed on cigarette packets that would show cancerous tumors, diseased lungs, and feet with amputated toes. The FDA’s tobacco director Mitch Zeller said that these new labels had been designed while keeping in mind the limited public awareness of lesser-known diseases caused by smoking. 

For 35 years, everything that the USA’s cigarette packets have told consumers about the harmfulness of smoking has been contained in a warning statement so tiny that it is missed by most consumers. Research has shown that graphic label inclusion on cigarette packets discourages smokers, and Canada was the first country to adopt this measure in 2000. 

Since then, more data have come to light identifying graphic images as an effect anti-smoking measure. A 2013 study showed that participants tended to respond to graphic labels rather than warning statements on cigarette packets. Another 2013 study estimated that if the FDA had adopted graphic labels in 2012, 5.3 to 8.6 million adults would have quit smoking in a year. They based their results on similar data from Canada where graphic labels are in use. 

It is anticipated that Big Tobacco will challenge the FDA’s move once again. Some of them are already citing the First Amendment as their defense. It is now time to see what is decided if the matter is taken to the courts once again. While thousands of people continue to die because of smoking habits in the USA, and graphic label inclusion on cigarette packets are repeatedly challenged by the tobacco industry, nearly 120 countries across the world have already adopted this measure and are reaping its benefits. 

(Matthew Perrone, STAT)

Experimental Ebola Drugs Saved Lives In Congo Outbreak

Ebola is raging in the Democratic Republic of Congo (DRC). However, a climate of distrust around Ebola clinics compounded with political upheavals in DRC has discouraged patients in the early stages of infection from leaving their community and seeking help in designated Ebola clinics. In this way, the virus has rapidly spread across communities and now threatens people outside of DRC as well. 

Despite these issues, efforts are underway to combat Ebola and develop more effective treatment strategies. A NIAID-funded study that compared four drugs against the Ebola virus recently concluded that two of them showed better results in combating the disease. The study, called ‘Pamoja Tulinde Maisha’ (PALM), is a randomized controlled trial of the four drugs and started on November 2018 as part of the emergency response in DRC.

The four investigational agents were Remdesivir, a commonly used antiviral drug, ZMapp, which showed effectiveness in previous Ebola outbreaks, REGN-EB3, developed by Regeneron and mAb114, a monoclonal antibody jointly developed by NIAID and INRB (in the DRC). 

Having reached a definite conclusion about the effectiveness of the four drugs, the PALM trial stopped earlier than originally scheduled. Preliminary results released on August 9, 2019 show only 30% of Ebola Virus Disease (EVD) patients treated with REGN-EB3 or mAb114 succumbed to the infection, compared to half of those treated with ZMapp or Remdesivir. When analysis was restricted to the relatively healthier patients receiving the more potent treatments, 6% and 11% died on REGN-EB3 and mAb114 treatment regimes respectively. 

With these preliminary results, patients on Remdesivir and ZMapp will now be switched to one of the more effective drugs based on their physician’s discretion. The study will continue to measure the effects of the two drugs from now on. The final analysis of the study data is likely to be made available later this year. 

(Richard Harris, NPR)

 

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August 20, 2019 at 4:56 pm

Science Policy Around the Web August 6th, 2019

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By Allison Dennis B.S.

Image by rawpixel from Pixabay 

Researchers weigh in on Trump’s $500 million plan to share childhood cancer data

Researchers are contemplating developing what they call The Childhood Cancer Data Initiative (CCDI). This approach comes in response to the 10-year $500 million research budget for childhood cancer proposed by President Donald Trump during his January State of the Union Address. Federal officials implementing the proposal have seized on an ongoing effort to transform “big data” into new medical discoveries by looking to expand the sharing of patients’ data to develop new approaches to treat childhood cancers. Currently, patient data can be found spread across state registries, tumor DNA databases, and clinical trial records, obscuring potential insights. 

A symposium held by the National Cancer Institute (NCI) in August 2019, gathered members of the research community to brainstorm the future of the CCDI. Experts made clear the need to first inventory what data already exists, including making efforts to digitally join the five largest existing pediatric cancer databases. Close attention and broad changes will be needed to unify the individual observations being made as children battling cancer make their way through the medical system. Yet despite these big ideas, it is not yet clear that Congress will follow through to appropriate the $50 million down payment needed to kick start CCDI. 

(Jocelyn Kaiser, Science)

 

‘Mosaic’ HIV vaccine to be tested in thousands of people across the world

The ‘mosaic” vaccine is the latest innovation in HIV prevention scheduled to start late-stage clinical trials in September. The experimental vaccine is designed to elicit an immune response to protect against more strains of HIV than any developed so far. The phase III trial (termed “Mosaico”) will be conducted in Europe and the Americas to follow its effectiveness at preventing the transmission of HIV in 3,800 participants, divided evenly into groups receiving four injections of vaccine or placebo. The innovative approach taken by researchers started with engineering a disabled common cold virus to carry pieces of DNA encoding synthetic copies of three HIV genes. The synthetic genes help the body to recognize several different global HIV strains. In addition to the DNA sequences, the vaccine is delivered with two synthetic proteins designed to match HIV strains common in Africa, the Americas, Europe, and Australasia.

Only four HIV vaccines have ever been tested for efficacy in humans. One which initially showed promise but whose efficacy waned over time resulted in a modest 31% difference in rates of infection between groups who received the vaccine compared with placebo. By combining DNA sequences and proteins reflecting a broad diversity of globally circulating HIV strains, the Mosaico team is hoping to give the body an immunological snapshot to prepare it to defend against any strain it might be exposed to. 

(Emiliano Rodriguez Mega, Nature)

 

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August 6, 2019 at 4:47 pm

Science Policy Around the Web – November 20, 2018

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By: Andrew Wright, B.S.

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Source: Wikimedia

 

Habitat Loss Threatens All Our Futures, World Leaders Warned

Recent reports have suggested that humanity has only 12 years to avoid catastrophic environmental collapse due to 1.5C of industrial warming. While solutions to the threat of runaway climate change have been given a new sense of urgency by these findings, there exists a commensurate and oft less visited issue, that of rapid declines in global biological diversity. Driven primarily by agricultural land conversion of terrestrial and marine ecosystems (via forest clearing and river damming, respectively), vertebrate species have declined by 60% on average since 1970 according to the World Wildlife Fund’s most recent Living Planet Report . While this decline appears strongest in South and Central America and in freshwater habitats, the report joins a compendium of literature suggesting holistic declines in biodiversity among birds, insects, fish, and terrestrial vertebrates as part of an ongoing anthropogenic mass extinction event.

To address some of the issue, the UN Convention on Biological Diversity (CBD) is currently meeting in Sharm El Sheikh, Egypt to discuss progress on the Aichi biodiversity targets for 2020.  These targets came out of The Convention on Biological Diversity, a multilateral treaty signed in 1992 focused on preserving biodiversity, sustainable use of biological resources, and equitable sharing of resources. The Aichi biodiversity targets specified that people would be aware of risks to biodiversity, and that biodiversity values would be adopted by public, private, and governmental entities by 2020. Given the rapidity, intensity, and ubiquity of the decline in species, most, if not all, of these targets will likely be missed. As such, the delegates from the 196 signatory nations will also work on creating new biodiversity targets to be finalized at the next CBD meeting in China.

Since a comprehensive solution seems necessary given the increasingly global nature of trade, authors of the new targets hope to garner a greater deal of international attention, and intend to make the case that government commitment to reversing or pausing biodiversity loss should receive equivalent weight as action on climate change.

(Jonathan Watts, The Guardian)

The Ethical Quandary of Human Infection Studies

The United States has greatly improved its ability to soundly regulate the ethics of clinical studies since the infamous malfeasance of the Tuskegee syphilis study. Most significantly, the National Research Act of 1974 established the Institutional Review Board to address how to adequately regulate the use of human subjects by adhering to the principles of respect for persons, beneficence, and justice.

The National Research Act provided a substantial step forward and provided a clear requirement for universal informed consent. However, the expansion of clinical studies to new international regions of extreme poverty, due in part to the influx of private money from large charitable organizations, has come with novel ethical considerations. In these newly explored populations where income, education, and literacy levels may be lower, emphasis is now being place on how to recruit volunteers without implicitly taking advantage of their circumstances.

One area of concern is compensation levels. While compensation in a malaria infection study in Kenya was tied to the local minimum wage, the number of volunteers recruited far surpassed expectations. This may have been due to the fact that payment during this study was guaranteed and consistent, in contrast to local work.

Aware of the concern, two of the largest private medical research funding organizations, the Bill and Melinda Gates foundation and the Wellcome Trust have recently instituted ethical guidelines putatively reinforcing the principle of beneficence, placing special emphasis on maximizing benefits over risk. It is an open question whether these protections will be sufficient, but at the very least it is important that rules to be put in place proactively rather than as a reaction.

 

(Linda Nordling, Undark/Scientific American)

 

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November 20, 2018 at 11:58 am

Science Policy Around the Web – September 19, 2018

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By: Saurav Seshadri, Ph.D

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source: pixabay

Clinical Trials

Universities are worse than drug companies at reporting clinical trial results

Reporting the methods and results of clinical trials, in a timely and accurate manner, is critical to the process of drug development.  Such reporting is described by the World Health Organization as a ‘scientific, ethical, and moral responsibility’.  However, a recent study conducted by Oxford University’s Evidence-Based Medicine (EBM) DataLab has found that less than half of clinical trials conducted in the EU since 2004 actually fulfilled this responsibility.  In fact, an abysmal 11% of trials with non-commercial sponsors had reported their results, with many institutions reporting results for 0% of up to 200 registered trials.  Industry sponsors did a better job, with almost 70% of trial results reported, and higher rates of reporting among companies with more trials.  Of equal concern, inaccurate reporting appeared to be rampant in the EU Clinical Trials Register database.  For example, 30% of trials that were listed as ‘completed’ or ‘terminated’ did not have an end date, and therefore couldn’t be evaluated for timeliness (results are due after 1 year).

Clinical trial data reporting is governed by a similar system in the US: sponsors are required by the FDA to post results on ClinicalTrials.gov within a year of completion.  But the problem of lax reporting is well known, and the current results are in line with previous reviews.  In February, the EBM DataLab launched a tool to monitor trial reporting, aiming to increase public accountability by ranking sponsors, tabulating potential fines, and regularly notifying the FDA of non-compliance.  Legally, investigators can be charged $11,569 per day for late reporting, and according to the tracker, unclaimed fines now add up to more than 650 million dollars.  The Oxford team recently launched a similar tool for the EU.

Many factors are likely to contribute to the problem of non-compliance in academia.  These include an absence of a ‘culture of reporting’, unfamiliarity with data curation or registration procedures, pressure to save data for a high-profile publication, and unwillingness to accept negative results.  In the end, effecting a change in academic reporting habits will require a combination of public scrutiny and institutional support.  The EBM DataLab has at least the first part well in hand.

(Tania Rabesandratana, Science)

Health insurance

Merger of Cigna and Express Scripts Gets Approval From Justice Dept.

In a recent announcement, the Department of Justice (DOJ) appears to have cleared the way for a more consolidated healthcare landscape.  After over a year of review, the DOJ has granted approval for the $52 billion merger between Cigna, a major health insurance company, and Express Scripts, a leading pharmacy benefits manager (PBM).  According to the companies, this union between the entities who pay for and provide prescription drugs will allow them to share patient information and eventually negotiate better drug pricing.  While the details are unclear, the DOJ apparently agrees on the principle; a separate merger between Aetna and CVS Health, valued at $69 billion, is now expected to be greenlit as well.

The decision represents a path forward for insurers, whose previous attempts to merge with each other were blocked by federal antitrust officials.  Overall, the shift towards vertical integration is largely a response to Amazon, which caused many health companies’ stocks to plummet when it revealed its intention to enter the healthcare industry earlier this year.  Amazon’s partnership with investment titans Berkshire Hathaway and JPMorgan Chase & Co, as well as its purchase of online pharmacy Pillpack.com, make it a credible threat even to well-established companies in the notoriously complicated healthcare industry.  Until recently, the Cigna-Express Scripts deal itself was vehemently opposed by activist investor Carl Icahn, who urged shareholders to vote against it on the grounds that competition from Amazon would soon render the PBM model inviable.

The mergers may also be an attempt by established companies to show that they are trying to adapt their business models to address growing public dissatisfaction with drug pricing and access.  However, there is no guarantee that any discounts that these more powerful, merged organizations are able to obtain will be passed on to customers.  Critics ranging from the pharmaceutical lobby to the Trump administration have suggested that PBMs are designed to maximize profit rather than help patients.  On the other hand, the merger may increase competition among insurers (and thereby benefit patients) by enabling Cigna to enter new markets.  With Amazon closing in, healthcare companies’ survival may ultimately depend on their ability to balance putting themselves or their customers first.

(Reed Abelson, The New York Times)

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September 19, 2018 at 11:35 am

Science Policy Around the Web – July 27, 2018

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By: Emily Petrus, Ph.D.

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source: pixabay

Innovation

Artificial Intelligence Has a Bias Problem, and It’s Our Fault

While computer and data scientists are working to create systems which can reason and perform complex analysis, groups of ethicists, lawyers and human rights advocates express increasing concerns about the impact artificial intelligence will have on life. It is becoming apparent that human bias regarding race, gender and socioeconomic position also influence algorithms and data sets used to train machine learning software.

Most artificial intelligence (AI) systems are trained on data sets culled from the internet. This results in skewed data which over-represents images and language from the United States. For example, a white woman in a white dress results in algorithms labeling a picture as “bride” or “wedding”, while an image of a North Indian bride is labeled as “performance art”. If that seems like a harmless hiccup, think about algorithms designed to detect skin cancer from images. A recently published study did a decent job detecting dark moles on light skin, but only 5% of the data set depicted dark skinned people, and the algorithm wasn’t even tested on that data set. This bias could skew accurate diagnoses for already underserved minority populations in the United States. Finally, AI will have a huge impact on financial markets beyond the replacement of humans to do jobs, particularly in manufacturing. Decisions on loan eligibility and job candidate hiring decisions are being filtered through AI technology, which is guided by data which may be biased.

It is apparent that computer scientists must make concerted efforts to un-bias data training sets and increase transparency when they develop new AI systems. Unfortunately, these common-sense suggestions are just that: suggestions. Before Obama left office in Fall 2016, a roadmap was created by the administration to guide research and development of AI systems. There’s no teeth in policy dictating fairness and inclusivity in AI development, but private and academic institutions are making gains in this arena. The Human-Centered AI project at Stanford University and Fairness, Accountability, Transparency, and Ethics (FATE) in AI research group at Microsoft are two examples of these types of efforts. Both groups seek to increase inclusivity in AI algorithms and reduce bias – human and computer generated. AI can also be trained to detect biases in both training data and the models by conducting an AI audit. An effort of developers in academia and private industry will be necessary to produce and prove their AI is unbiased, and it is unlikely that federal regulations would have the power or dexterity to administer any concrete regulations regarding this technology. Like most other scientific advances which bring significant monetary gains, the pace is breakneck but corners should not be cut. Legislation is unlikely to be able to keep up with the technology, but incentives to keep the playing field fair should come from within the AI community itself.

(Ben Dickson, PC Mag)

Scientific oversight

NIH delays controversial clinical trials policy for some studies

How does the brain process images of faces? How do we respond to frustrating situations? What does the mind of a sociopath look like in an MRI? These are all basic science questions in brain research which may discover treatment options for future studies. But for the moment, no drugs or interventions are being tested in many basic research labs funded by the National Institutes of Health (NIH). This means they’re not clinical interventions, or by definition, clinical trials, right? Maybe…

Basic researchers studying the healthy human brain sigh a breath of relief as the NIH decided to delay new rules applying to the classification of human trials. At issue is the re-classification of research which can be considered a clinical trial. The intent of the new guidelines was to increase reproducibility and transparency in government funded human research, for example requiring more rigorous statistical practices.  In practice, investigators will be required to upload their studies to clinicaltrials.gov, take mandatory trainings, and produce significantly more paperwork to continue receiving funding for their basic research. In addition, researchers were concerned that this would create more confusion in the public, as their research would be inaccurately represented as a clinical trial.

After the announcement last year, professional societies and academics sent letters of complaint to NIH, prompting congress to delay the implementation of the requirements to September 2019. This delay also gives leniency to basic researchers who apply to funding opportunity announcements seeking studies labeled as clinical trials, meaning they would not be immediately disqualified from being scored. Although many researchers hoped the NIH would drop all requirements for basic research, the delay is welcome for now. “This delay is progress because it gives them more time to get it right, and in the interim people aren’t going to be in trouble if they get it wrong,” said Jeremy Wolfe, a cognitive psychologist at Harvard Medical School.

(Jocelyn Kaiser, Science)

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July 27, 2018 at 4:51 pm

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Clinical Trials Policy Revision: For Better or Worse

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By: Jenn L. Nguyen, Ph.D., M.P.H.

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source: pixabay

As the largest public funder of biomedical research in the United States, the NIH wants to ensure that conducted trials are relevant to health priorities of the US, trials are conducted efficiently and are not duplicates of previously conducted trials, and trials contribute to scientific knowledge. In an editorial in 2016, NIH leaders noted a need for quality and efficiency improvements to clinical trials. NIH has introduced several initiatives, to enhance clinical trials stewardship by addressing accountability, transparency, efficiency, and dissemination. However, along with the widely acknowledged improvements some recent changes may hinder the pursuit of scientific knowledge.

To address accountability, all investigators and staff conducting and overseeing clinical trials must take the Good Clinical Practice (GCP) training. The training is mandatory for individuals involved with the design, conduct, oversight, or management of clinical trials. While the training may not be sufficient by itself, it does provide a standard of knowledge, a base of knowledge, standards, and guidelines for all clinical trials.

The second change requires that all grant applications for clinical trials be submitted under clinical trials specific funding opportunity announcements (FOA). Investigators interested in conducting a clinical trial can no longer submit under parent funding announcements, which made identifying clinical trials more difficult in the past. The FOAs will list specific review criteria for reviewers to consider clinical trials-related information, such as focus on the rationale, design, and operational and analysis plans. This new policy will increase NIH accountability and efficiency, as it will ensure that required information is submitted with each clinical trial application, allow staff to better track clinical trial proposals and study, and allow staff to uniformly apply appropriate review criteria.

A substantial change, however, is the limited eligibility of trainees to conduct interventional social science research, Institutional training (T) awards, which provide money to institutions for workforce training, do not allow money to be given to trainees involved in clinical trials (the exception is for D43s and K12s), Fellowship (F) awards, which support individual trainees,  do not support trainees involved in independently conducted clinical trials, but trainees can propose a research experience with a sponsor/co-sponsor.  For Career Development (K) awards, applicants may apply to either FOAs that specify “clinical trials required” or FOAs that are for “no independent clinical trials.” Scientists are concerned this may limit postdocs and students to get support for their fellowships and adequate career training.

To further address efficiency and accountability, applications must be submitted using a clinical trials protocol template that consolidates information from multiple forms, has structured data fields, and will collect information at the study level. This requirement will ensure that all investigators will submit the same information. In addition, the forms will contain fields forcing investigators to be clear and concise about their analytical and dissemination plans.

Addressing efficiency, NIH now requires use of a single Institutional Review Board (IRB) to review multisite studies. Prior, each institute involved with the study required duplicate or multiple IRB reviews, which involved the redundant assembly of experts to assure that the same proposed study was in line with the rights and protections of human and animal research subjects. Multiple reviews resulted in delays and at times, conflicting reviews. Guidance to establish a single IRB on record has been published.

Finally, there are significant changes for registration and reporting of clinical trials to address accountability, transparency, and dissemination. Investigators are now required to register their clinical trial(s) in the ClinicalTrials.gov database within 21 days of enrollment of the first participant. NIH makes the argument that this effort may help reduce the number of trials that fail, as it will require scientists to disclose their results even if the studies do not support their hypotheses urthermore, all investigators must adhere to the NIH policy on Dissemination of NIH-clinical trials. There have been longstanding concerns that investigators are not reporting all results (especially negative or non-significant results, not reporting results in a timely manner, and even sometimes, deviating from their own research protocol.

Along with these initiatives, The National Institutes of Health (NIH) broadened what was considered a clinical trial: “a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.” Adaption of this updated definition did not take effect until earlier this year and has alarmed some scientists. Clinical trials have been traditionally understood as experiments or observations for/in clinical settings to answer three questions: 1) Does the proposed treatment/intervention work? 2) Is the proposed treatment or intervention more effective than other treatments? 3) Are there side effects?

Scientists critical of the new definition first and foremost recognize and appreciate the motivation for NIH to increase transparency and replicability, specifically efforts for pre-registration, data sharing, and protocol sharing of trials. Yet, many scientists who conduct basic and behavioral research disagree agree that their work and studies should now be considered clinical trials. These scientists, and even scientific associations, remarked that the new clinical trials definition is too broad and traditional criteria to evaluate a trial might be inappropriately applied to their proposal. There is also concern that these changes will increase the administrative and bureaucratic burden for many scientists, specifically for exploratory scientists. To address and alleviate concerns, NIH released a set of case studies to help scientists identify and understand what is considered a clinical trial and must adhere to all the changes in the policy. While this effort provided clarification, many scientists are calling for NIH to hold further conversation with the extramural community.

While scientists recognize the need and laud NIH’s effort to address clinical trials stewardship, many of the same scientists are worried that these benchmarks set the wrong standards for success and rigor. Scientists are also worried about the additional administrative burden these changes will bring. As NIH enforces the policies, they have promised to monitor trouble issues and work with the community to find a solution without compromise.

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February 20, 2018 at 3:52 pm