November 12, 2018: Participate in the NIH Office of Disease Prevention’s Research Expertise Survey

The National Institutes of Health (NIH) Office of Disease Prevention (ODP) needs your help to enhance the quality of research supported by the NIH. The ODP is building a directory of experts in research methods and study designs that can help NIH Scientific Review Officers identify the most appropriate reviewers for NIH research applications. Adding your name and expertise to the directory is easy – simply share your methodological and content area expertise by filling out the ODP’s Prevention Research Expertise Survey (PRES).


 

The survey covers 7 areas related (but not limited) to prevention research:

  • Study Design Topics
  • Research Methods
  • Content Topics
  • Settings
  • Populations
  • Regions
  • Income Categories

The PRES takes approximately 15-25 minutes and is strictly voluntary. Based on your skill set and interest, NIH or Department of Health and Human Services (HHS) staff may invite you to serve as a peer reviewer for research applications, or you may be asked to sit on a panel, committee, or workgroup; or to speak at a seminar or workshop. Your responses and information will not be shared with anyone outside of HHS.

The ODP believes the participation of highly qualified methods experts will enhance the quality of peer review; improve the rigor, reproducibility, and impact of research supported by the NIH; and ultimately lead to stronger clinical practice, health policy, and community health programs.

The ODP is the lead office at the NIH responsible for assessing, facilitating, and stimulating research in disease prevention and disseminating the results of this research to improve public health. For more information about the ODP and its work, visit the ODP website.

Thank you in advance for taking the survey – your participation helps improve the rigor, impact, and value of research supported by the NIH.

April 16, 2018: Registration Now Open for Workshop on Embedded Pragmatic Clinical Trials of Therapeutic A vs. B Interventions

Registration is now open for a one-day workshop sponsored by the NIH Common Fund and organized by NIH Collaboratory thought leaders. The workshop, to be held on May 16, will focus on expanding the understanding of a specific type of pragmatic trial, the A vs. B trial, which compares two or more distinct therapeutic medical interventions. Over the past 6 years, the NIH Collaboratory has launched Demonstration Projects that address questions of major public health importance in real-world settings with usual care as a control arm. Much has been learned about the design, conduct, and dissemination of pragmatic clinical trials through this work. As a next step, there is considerable excitement at the prospect of exploring pragmatic clinical trials for comparing medical interventions.

The workshop will include a series of moderated discussions of opportunities and approaches for partnering with healthcare systems, and other stakeholders, to conduct embedded A vs. B trials. Speakers will include representatives from NIH, FDA, OHRP, health systems, academia, and industry.

The workshop will take place from 8:00 AM – 4:15 PM ET on Wednesday, May 16. Participants can join in person at the Bethesda Marriott in Maryland or via webcast. Choose an option for attendance on the registration page.

The organizing committee for the workshop includes Adrian Hernandez & Richard Platt (Co-Chairs), Laura Dember, Susan Huang, Catherine Meyers, Wendy Weber, and Dave Wendler.

 

 

NIH & FDA seek feedback on new clinical trial protocol template


As part of their ongoing effort to improve the speed and efficiency of conducting clinical trials, the NIH-FDA Joint Leadership Council has created a draft clinical trial protocol template. The template contains instructional and sample text intended to assist NIH-funded investigators in writing protocols for phase 2 or 3 clinical trials that require Investigational New Drug (IND) or Investigational Device Exemption (IDE) applications. Feedback is sought from investigators, investigator-sponsors, institutional review board members, and other stakeholders involved in protocol development and review.

Our goal is to provide an organized way for creative investigators to describe their plans so that others can understand them. – Dr. Pamela McInnes, NIH

Details on the rationale and development of the protocol template are on these blog posts:

Notice Number: NOT-OD-16-043. Responses accepted through April 17, 2016.

You can access the template document as well as a template shell, comment form, and other resources at NIH’s Clinical Research Policy website.

Modernizing the Common Rule for the 21st Century


The New England Journal of Medicine today published a perspective by NIH Deputy Directory Kathy L. Hudson, PhD, and NIH Director Francis S. Collins, MD, PhD, in which they outline the major reforms proposed for regulations governing the ethical conduct of research involving humans, known as the Common Rule (45 CFR 46, Subpart A).

The proposed changes are meant to enhance respect for research participants, calibrate oversight to level of risk, simplify consent documents, streamline IRB review, increase privacy and security safeguards, and facilitate broad participation in research.

“These long-overdue reforms will bring the Common Rule into the 21st century. They should help the scientific community take a giant leap forward in showing respect for research participants, without whom the biomedical research enterprise would cease to exist.”

The NIH is encouraging all stakeholders—the public, researchers, and patients—to closely review the proposed changes and participate in the comment process by the December 7, 2015, deadline.

For more information on the proposed revisions:

Grand Rounds Presentation, Kathy Hudson (video)

Department of Health and Human Services' website on the NPRM 

OHRP Webinars on the NPRM

Living Textbook Chapter: Informed Consent: Emerging Issues and Controversies

Mobile Health (mHealth) Research Platform to Launch


In recent health information technology news, the University of California, San Francisco (UCSF), has received a 5-year National Institutes of Health award to support its launch of a cardiovascular mHealth platform. The research platform, to be named Health ePeople, will build on the successes of UCSF’s Health eHeart Study, which began in 2013. That study, with more than 30,000 participants worldwide, uses the power of mobile technologies to collect cardiovascular data and patient-reported outcomes (PROs) from study participants.

The Health ePeople platform will advance mHealth by providing researchers with easy access to a large cohort of volunteers, along with a quick, affordable means for collecting their health data through mobile and wireless technologies. Though the platform will not be ready to enroll new participants for several months, people who want to participate in the cohort can sign up through the Health eHeart Study website.

For information and short videos on mHealth technologies, visit the Living Textbook’s chapter on mHealth and PROs.


Blueprint for Establishing a National Research Cohort Moves Forward


On September 17, the Precision Medicine Initiative Working Group presented to NIH Director Dr. Francis Collins a detailed design framework to advance the creation of a national, large-scale research cohort for developing more effective treatments tailored to individuals. The framework makes recommendations on cohort assembly, participant engagement, data, biological specimens, policy, and governance. The recommendations are based on a set of high-value scientific opportunities that were identified by the working group following extensive stakeholder engagement.

The NIH plans to move quickly to build the infrastructure so that participants can begin enrolling in the cohort in 2016, with a goal of enrolling at least 1 million participants in 3 to 4 years. Visit the NIH News & Events website for more details.


In Nature: The Precision Medicine Initiative & DNA Data Sharing


A recent article in Nature highlights the Precision Medicine Initiative, launched in January 2015 and spearheaded by the National Institutes of Health. Precision medicine is an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person. This initiative will involve collection of data on genomes, electronic health records, and physiological measurements from 1 million participants. A main objective is for participants to be active partners in research.

But a major decision faced by the initiative’s working group is how much information to share with participants about disease risk, particularly genetic data. Though there is much debate in the field, the article suggests that public opinion on data sharing may be shifting toward openness.

The Precision Medicine Initiative working group will be releasing a plan soon. For details on the goals of the Precision Medicine Initiative, read the perspective by NIH Director Dr. Francis Collins in the New England Journal of Medicine.


 

Patient-Reported Outcomes Workshop Report Available


Tools for ResearchIn January of 2015, the NIH HCS Collaboratory’s Patient-Reported Outcomes (PRO) Core Group convened a 2-day workshop in Baltimore devoted to identifying barriers and possible solutions to the use of NIH-supported PRO tools in comparative-effectiveness research (CER).

Findings from the meeting, which include case study presentations and reflections from multiple stakeholders representing the research, clinical, and patient communities, were distilled into a summary document available from the NIH Collaboratory Knowledge Repository at the link below:

The workshop summary is also available on the Living Textbook’s “Tools for Research” section, under “Patient-Reported Outcomes White Paper.


Researchers Find Poor Compliance with Clinical Trials Reporting Law

A new analysis of data from the ClinicalTrials.gov website shows that despite federal laws requiring the public reporting of results from clinical trials, most research sponsors fail to do so in a timely fashion—or, in many cases, at all. The study, published in the March 12, 2015 issue of the New England Journal of Medicine, was conducted by researchers at Duke University and supported by the NIH Collaboratory and the Clinical Trials Transformation Initiative (CTTI). The study’s authors examined trial results as reported to ClinicalTrials.gov and evaluated the degree to which research sponsors were complying with a federal law that requires public reporting of findings from clinical trials of medical products regulated by the U.S. Food and Drug Administration (FDA).

“We thought it would be a great idea to see how compliant investigators are with results reporting, as mandated by law,” said lead author Dr. Monique Anderson, a cardiologist and assistant professor of medicine at Duke University.

Photograph of study author Monique L. Anderson, MD
Monique L. Anderson, MD.
Photo courtesy of Duke Medicine.

Using a publicly available database developed and maintained at Duke by CTTI, the authors were able to home in on trials registered with ClinicalTrials.gov that were highly likely to have been conducted within a 5-year study window and to be subject to the Food and Drug Administration Amendments Act (FDAAA). This federal law, which was enacted in 2007, includes provisions that obligate sponsors of non-phase 1 clinical trials testing medical products to report study results to ClinicalTrials.gov within 12 months of the trial’s end. It also describes allowable exceptions for failing to meet that timeline.

However, when the authors analyzed the data, they found that relatively few studies overall—just 13 percent—had reported results within the 12-month period prescribed by FDAAA, and less than 40 percent had reported results at any time between the enactment of FDAAA and the 5-year benchmark.

“We were really surprised at how untimely the reporting was—and that more than 66 percent hadn’t reported at all over the 5 years [of the study interval],” said Dr. Anderson, noting that although prior studies have explored the issue of results reporting, they have until now been confined to examinations of reporting rates at 1 year.

Another unexpected result was the finding that industry-sponsored studies were significantly more likely to have reported timely results than were trials sponsored by the National Institutes of Health (NIH) or by other academic or government funding sources. The authors noted that despite a seemingly widespread lack of compliance with both legal and ethical imperatives for reporting trial results, there has so far been no penalty for failing to meet reporting obligations, even though FDAAA spells out punishments that include fines of up to $10,000 per day and, in the case of NIH-sponsored trials, loss of future funding.

“Academia needs to be educated on FDAAA, because enforcement will happen at some point. There’s maybe a sense that ‘this law is for industry,’ but it applies to everyone,” said Anderson, who points out that this study is being published just as the U.S. Department of Health and Human Services and the NIH are in the process of crafting new rules that deal specifically with ensuring compliance with federal reporting laws.

According to Anderson, increased awareness of the law, coupled with stepped-up enforcement and infrastructure designed to inform researchers about their reporting obligations, have the potential to improve compliance with both the letter and the spirit of the regulations. “I think reporting rates will skyrocket after the rulemaking,” she says.

In the end, Anderson notes, reporting clinical trials results in order to contribute to scientific and medical knowledge is as much an ethical obligation for researchers as a legal one: “It’s something we really promise to every patient when they enroll on a trial.”


Read the full article here:

Anderson ML, Chiswell K, Peterson ED, Tasneem A, Topping J, Califf RM. Compliance with results reporting at ClinicalTrials.gov. N Engl J Med. 2015;372:1031-9. DOI: 10.1056/NEJMsa1409364.
Additional reading:

"Results of many clinical trials not being reported" (NPR)

"Clinical trial sponsors fail to publicly disclose report results, research shows" (Forbes.com)

NIH Issues Draft Policy Proposing Use of Single IRB for Multisite Clinical Research Studies


On December 3, 2014, the National Institutes of Health (NIH) issued a draft policy promoting the use of a single institutional review board (IRB) for multisite studies. IRBs play a critical role in assuring the ethical conduct of research, and studies must be reviewed and approved by an IRB before they can begin. Yet over time, the clinical research landscape has become increasingly complex, expanding from studies formerly conducted at single institutions to large, diverse studies across networks and multiple sites. This situation challenges the practicality of using local IRBs to conduct initial and ongoing reviews for such studies.

The goal of permitting use of a single IRB—also called a central IRB or IRB of record—is to enhance and streamline the process of IRB review for multisite studies so that research can proceed efficiently without compromising ethical principles and protections. While both the FDA and Office for Human Research Protections support the use of a single IRB, too few institutions involved in multisite studies are taking advantage of the option.

 Read the NIH draft policy here.

“By using single IRBs in multi-site studies, we reduce duplication of effort, speed the initiation of important research, and save time and taxpayer funds.”
Francis S. Collins, MD, PhD, NIH Director

Among the current NIH programs incorporating the use of a single IRB are:

  • National Cancer Institute’s Central Institutional Review Board (CIRB)
  • National Institute of Neurological Disorders and Stroke’s Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT)
  • Network for Stroke Research (NIH StrokeNet)

Dr. Sally Rockney, NIH deputy director for extramural research, explains the NIH perspective in her blog. Public comment on the draft policy extends for 60 days, through January 29, 2015. When finalized, the policy will apply to all NIH-funded multisite studies carried out in the United States, whether supported through grants, contracts, or the NIH intramural program.