Tag: Collaboration

A Fresh Look at the Rules for Returning Research Results

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As the biomedical research enterprise increasingly moves to a more participatory model of research, where research participants are treated more as partners than passive subjects, we can expect greater emphasis on returning individual-level results of research to participants. A prominent example is the All of Us Research Program. All of Us aims to recruit one million people living across the United States, and as a central tenet of the program will also make data available to those participants, as well as the findings of research that use their samples and data. Recent revisions to the Common Rule have anticipated this movement, and the changes that will take effect in 2018 include new provisions that are specific to the return of research results.

As the research enterprise works to determine how and when to provide participants access to their individual-level research results and data, questions have emerged regarding whether the current regulatory environment is conducive to such access. One such example includes the Clinical Laboratory Improvement Amendments (CLIA) which is enforced by the Centers for Medicare and Medicaid Services (CMS). The Secretary’s Advisory Committee for Human Research Protections (SACHRP) has suggested that the requirements under CLIA may, in effect, create a conflict in some situations with the right of access provided by the Health Insurance Portability and Accountability Act (HIPAA) to laboratory test results. The Food and Drug Administration (FDA) device regulations may also require certain additional measures before returning individual-level research results.

In recognition of these important considerations, NIH, FDA, and CMS are collaborating to support a study by the National Academies of Sciences, Engineering, and Medicine who will convene a committee representing a range of diverse perspectives (e.g., clinicians, laboratory directors, researchers, research participants and advocates). This committee will consider whether the current regulatory environment for the return of individual-level results from analyses of biospecimens in research laboratories adequately minimizes risks and maximizes benefits both for individuals and society. This will require the committee to, among other activities, review the current regulatory environment and the evidence of harms and benefits from returning individual-level results from research laboratories, and to consider whether alternative approaches could achieve a more appropriate balance of risks and benefits.

The proceedings of this committee are sure to raise important questions and robust dialogue. I encourage all interested stakeholders to stay tuned to the National Academies for more news on this study.

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In Perfect Harmony: The NIH – FDA Clinical Trials Protocol Template

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One of the most frequent exasperated questions I face when I talk to NIH stakeholders is, “Why can’t federal agencies harmonize more? Don’t you people ever talk to each other?!?” I am happy to share tangible proof that the clear answer is “Yes!”  Today, NIH and FDA released the final version of a template document I blogged about last March that will assist NIH-funded investigators in preparing Phase 2 and 3 IND/IDE trial protocols. In utilizing this template, investigators can prepare documents that will help enable efficient and speedy reviews by both the IRB and the FDA.

The final template was developed by a wide variety of stakeholders.  We received over 200 comments on the draft template, many of which have been incorporated into the final product.  Additionally, NIH and FDA collaborated with the non-profit organization, TransCelerate Biopharma in order to align our template with a template TransCelerate has been developing for industry-sponsored investigators. A shared template format with tailored instructions that fit each sectors’ needs will further facilitate the FDA review and help increase the efficiency of the clinical trial enterprise.

This being the digital age, NIH thought having a web-based platform where investigators could utilize the protocol template in an interactive fashion would be an essential tool.  With that in mind, NIH developed an Electronic Protocol Writing Tool.  We like to think of this as a sort of Turbo TaxTM for clinical trial protocols.  The tool allows for a collaborative approach to writing and reviewing protocols.  Investigators will be able to form a “protocol writing team” and assign different individuals with writing and reviewing roles.  The tool also makes it easy for the investigator to track the progress of the protocol, share comments between team members and keep accurate version control.  NIH is also considering future enhancements to the functionality of the tool as well as additional instructional and example text for other study types, such as behavioral clinical studies. I highly recommend that all investigators who plan on utilizing the NIH-FDA template take the e-Protocol Writer for a spin and provide us with their feedback.

With today’s release of the final protocol template and the e-Protocol Writer, NIH has taken another step towards enhancing our stewardship of NIH-funded clinical trials.  To read some additional perspectives on the importance of this initiative, please read a statement released today by NIH Director Dr. Francis Collins, as well as a blog published by the FDA.

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De-clunking the dbGaP Data Submission and Access Process – We’re All Ears!

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Data. It is the essential output of biomedical research that allows us to move science forward and improve human health. It gets a little trickier however when the conversation turns to how to best provide researchers with access to that data. Especially when you’re trying to balance appropriate protections for human participants in research, who deserve both the maximal use of their data for achieving medical progress and the respectful use of their data in a way that affords privacy protections and consistency with consent. At the NIH, lots of smart people spend a lot of time thinking about human data and how best to manage it. However, we can’t do it alone. We also need help from our stakeholders to solve these difficult issues.

Back in 2007, the National Center for Biotechnology Information (NCBI) developed the database of Phenotypes and Genotypes (dbGaP) to archive and distribute the results of human genome-phenotype studies that fall under NIH’s policies for sharing genomic data.

The dbGaP is a controlled-access data repository and currently serves as a central portal to submit, locate and request access to genomic and associated phenotypic data. It is a highly utilized, valuable, and rapidly growing resource with over 750 studies available for access. Users of dbGaP have access to a wide range of data types such as microarray, genome-wide association study, whole and targeted genomic, transcriptomic, epigenomic, and metagenomic data. As of January 2017, NIH has approved approximately 28,000 Data Access Requests for over 4,500 investigators from 46 countries.

Over the years, users of the dbGaP system have shared their feedback, and many have expressed a number of frustrations relating to the difficulty in navigating the submission process. To address these concerns, NIH has made a number of improvements to dbGaP (see Box 1). To best serve the needs of the research community and enable robust and responsible data sharing, it is imperative that new resources, tools, and data management models be developed to make the system as user-friendly and efficient as possible, as well as increase its utility.

With this in mind, NIH released today a Request for Information (RFI) seeking public comments on the data submission and access processes for dbGaP, and on the management of data within dbGaP, in order to consider options to improve and streamline these processes.

It is vital that we hear from members of the research community on this topic. We want to take your thoughts and ideas into account when attempting to increase the utility of dbGaP. I invite all stakeholders who currently use or may use dbGaP to provide us with their thoughts. Comments will be accepted until April 7, 2017.

                                                   

Box 1: Recent Improvements/Upgrades to dbGAP  

  • Development of standard data use limitations to promote consistent implementation of the consent group categories.
  • Development of fillable Institutional Certification forms to standardize and expedite the Institutional Certification process for institutions.
  • Implementation of user-friendly, electronic study registration, submission, DAR, project renewal, and project close-out forms.
  • Development of the dbGaP Data Browser to enable viewing of controlled-access summary statistics and individual-level genotype and sequence data associated with phenotypic features, by dbGaP approved users, without the need to download datasets.
  • In collaboration with the Global Alliance For Genomics and Health Beacon project, implementation of a simple web interface that allows users to query dbGaP for genomic variants of interest and their presence in the database.
  • Issuance of a Position on the Use of Cloud Computing Services for Storage and Analysis of Controlled-Access Data Subject to the NIH Genomic Data Sharing Policy to allow investigators to request permission to transfer controlled-access genomic data and other associated data obtained from dbGaP to public or private cloud systems for storage and analysis.
  • Creation of search filters for dbGaP datasets (e.g. data use limitations, disease area, data type).
  • Assembly of two data collections that allows investigators to submit a single DAR to gain access to most of the individual-level datasets in dbGaP approved for general research use (currently includes 96 datasets), or only the aggregated data from these datasets.
  • In an effort to promote transparency, the addition of a “Facts & Figures” section on the NIH GDS website to highlight current dbGaP data submission and access statistics, including DAR processing times and data management incidents.
  • Development of a mechanism to establish structured partnerships with external organizations or “trusted partners”.
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The Revised Common Rule: A Tribute to the Past and a Promise for the Future

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As humans living longer, healthier lives than at any point in history, we all owe a great deal of thanks to the countless volunteers who have served as research participants. These are people who have given of their time, of their bodies, who have accepted risks from the very small to the very large, and who have done so knowing that they might receive no personal benefit. Lifesaving vaccines, cancer therapies, cardiovascular treatments, and every drug, diagnostic, and cure in our modern medical pantheon have been made possible thanks to the willingness of research volunteers; real people with real lives and real loved ones.

And we owe it to those willing to volunteer to ensure that we have the best possible safeguards in place to ensure that research involving humans is conducted ethically, safely, and equitably.  In 1978, the Belmont Report was published, outlining the principles and guidelines for protecting human research participants, built on the foundations of respect, beneficence and justice. This led to the regulation now known as the “Common Rule,” whose purpose was to ensure that research involving humans was conducted in line with the highest ethical standards and practices. Today, the U.S. government has announced revisions to the Common Rule, the culmination of nearly a decade of rulemaking aimed at improving our system of oversight and facilitating research.

In the world of science policy geeks, there are few Eureka! moments or end zone dances. It is not every day that landmark rules are released. Furthermore, it is rare for significant policy changes to take place in the absence of a crisis, but rather in recognition of the evolution of science, our increased understanding of what works and what doesn’t in research oversight, and the changing nature of participant engagement in research. The revision of the Common Rule is the endpoint of years of discussion, debate, thousands of public comments, and, most importantly, a dedication to the very people who it is designed to protect and to the researchers who serve as their partners in discovery.

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