March 15, 2018: New Resource for Understanding Ethical and Regulatory Architecture of Patient-Centered Outcomes Research Data

Using patient-centered outcomes research (PCOR) data requires balancing the need for sufficient private health information to support meaningful research with the need to protect patient privacy and autonomy. In support of this dual goal, The Office of the National Coordinator for Health Information Technology (ONC) has just released a document that provides a collection of tools and resources aimed at helping a broad audience of stakeholders understand the ethical and regulatory requirements related to collecting, using, sharing, and disclosing PCOR data.

“An architecture is necessary to ensure patient privacy is protected and health information is appropriately secured during collection, access, use, and disclosure as required by law, regulation, and/or policy.” —Legal and Ethical Architecture for PCOR Data

PCOR data will help expand the evidence base for therapies and improve health outcomes for individual patients. The document is divided into 5 chapters:

Chapter 1: Overview of Legal and Ethical Architecture for PCOR Data provides background for the project and an overview of key ethical and regulatory requirements.

Chapter 2: Legal and Ethical Significance of Data for PCOR describes fundamental concepts for organizing data into categories such that legal and ethical frameworks can be applied. The chapter includes key considerations and types of data relevant to PCOR, such as clinical, administrative, patient-generated, etc.

Chapter 3: Linking Legal and Ethical Requirements to PCOR Data organizes the relevant legal provisions according to the key data considerations outlined in Chapter 2: identifiability, subject, source, access and use/purpose, consent/authorization, security, and legal status.

Chapter 4: Framework for Navigating Legal and Ethical Requirements for PCOR is designed as a decision tool that builds on the key data considerations described in Chapters 2 and 3. The goal of this chapter is to help researchers determine whether laws apply to particular data and if so, what requirements to attach to their collection and use.

Chapter 5: Mapping Research Flows to Legal Requirements identifies 6 hypothetical use cases, identifies decision trigger points, and maps representative data flows to the relevant legal requirements.

Read the full document: Legal and Ethical Architecture for Patient-Centered Outcomes Research (PCOR) Data (“Architecture”)

March 14, 2018: Public Input on Patient-Reported Outcome Measures Due April 1

The Agency for Healthcare Research and Quality (AHRQ) is requesting information to help inform a competition in the fall to develop and implement user-friendly technical tools for the collection of patient-reported outcome (PRO) data. The AHRQ is interested in learning about experiences with physical function PRO measures that are currently in use in ambulatory care settings, including primary and secondary care, as well as the methods used to collect these data. The information will be used to select physical function measures for the competition, which aims to bolster collection and integration of PRO data in the electronic health record by developing and piloting new, user-friendly tools. Responses can be emailed to and are due April 1, 2018.

March 7, 2018: FDA Offers Workshop on Submitting Draft Guidance on Patient Experience Data

The FDA is conducting a public workshop on Monday, March 19, to obtain input from stakeholders—including patients, patient advocates, academic and medical researchers, expert practitioners, drug developers, and other interested persons—to inform the drafting of a patient-focused drug development guidance as required by the 21st Century Cures Act. Workshop attendees will discuss considerations for development and submission of a proposed draft guidance regarding patient experience data submitted by an external stakeholder. The guidance is intended to help stakeholders continue progress in developing new medicines to respond to patient’s needs.

Registration for the event, either in person or via a live webcast, ends March 12. More meeting details, including background materials, will be posted by FDA as available.

February 28, 2018: New Meeting Summary Examines How to Integrate Patient‐Reported Health Data for Pragmatic Research

A recently released summary from the ADAPTABLE Roundtable Meeting explores ways to better understand the sets of circumstances and considerations that could guide when and how to gather and integrate patient-reported health data with other data sources in pragmatic trials.

For outcomes that represent subjective experiences, such as pain, symptoms, and physical functioning, the patient is the unique and privileged source of information. Other patient-reported health data may not have a clear source of truth, such as co-morbidities and hospitalizations. In such cases, patient-reported health data may supplement, contradict, or agree with EHR and claims data. For example, medication data reported by patients might be a more accurate reflection of what patients are actually taking than medication data in the EHR, especially for over-the-counter medications.

Patient-reported health data come from various sources and can be feasibly collected in the conduct of a pragmatic clinical trial, but the optimal approaches for capturing and analyzing these data are unclear. Questions include how to integrate this information with other data collected as part of a study, including data from the EHR.

To better understand patient-reported health data and how to use them in pragmatic trials, 18 experts from 8 institutions convened at the roundtable meeting, coming from a wide variety of backgrounds including biostatistics, epidemiology, oncology, nursing, psychiatry, health policy, and regulation. Representatives from the NIH Collaboratory included Drs. Lesley Curtis and Rachel Richesson from the EHR Core and Dr. Kevin Weinfurt from the Patient-Reported Outcomes Core.

In addition to the meeting summary, two white papers are forthcoming. For more information about using patient-reported data in pragmatic trials, see the Living Textbook Chapter on Endpoints and Outcomes.

This effort was funded by Office of the Assistant Secretary for Planning and Evaluation at the U.S. Department of Health and Human Services through a supplement provided to the NIH Collaboratory Coordinating Center.

February 22, 2017: NIH Collaboratory Launches First Embedded Pragmatic Clinical Trial Training Workshop

On February 20-21, 2018, a group of 27 clinical investigators met in Durham to learn from the NIH Collaboratory about the design and conduct of embedded pragmatic clinical trials (ePCTs). Through the experiences of the NIH Collaboratory, much has been learned about how to launch and implement successful ePCTs. The workshop’s goal was to help investigators advance their ePCT research ideas and build a larger community of researchers capable of conducting high-quality ePCTs. Participants from across the country were selected based on a rigorous application process.

“ePCTS hold the promise of an efficient and powerful way to generate evidence. We’ve learned so much through the Collaboratory, but the knowledge is not helpful if we’re not passing it along to other clinical investigators so they can contribute even more evidence to the knowledge base,” said Kevin Weinfurt, PhD, a co-principal investigator of the NIH Collaboratory’s Coordinating Center who helped develop and organize the workshop along with a planning committee.

Representatives from the Coordinating Center, Demonstration Projects, and Core Groups were on hand to provide information, guidance, and lessons learned from their experience with the NIH Collaboratory ePCTs. The topics included an introduction to ePCTs, engaging stakeholders and aligning with healthcare system partners, designing with implementation in mind, design and analytic considerations, regulatory and ethical challenges, measuring outcomes, dissemination, and ePCT team composition. Representatives from the NIH were also in attendance and presented sessions about the importance of pilot and feasibility testing and developing a compelling application for funding. During the workshop, participants completed hands-on exercises and worked through trial design challenges with the experts and their colleagues.

Wendy Weber, ND, PhD, MPH, Acting Deputy Director of the National Center for Complementary and Integrative Health (NCCIH), presenting on pilot and feasibility testing in ePCTs.

“We’re trying to change the world, and to change the world you need generalizable data and interventions that are scalable. The chasm for implementation of clinical evidence is huge, and PCTs have the capacity to take great interventions and get them to the patients so that they can have impact and improve care,” said Wendy Weber, ND, PhD, MPH, acting deputy director of the National Center for Complementary and Integrative Health (NCCIH), and one of the workshop presenters. “While local improvements can be accomplished through quality improvement activities, if you have outcomes that matter, randomizing interventions and studying them on a large scale can provide the evidence that will make a real difference in the care of people across the United States.”

Another goal of the workshop was to pilot the educational materials and collect feedback on their quality and appropriateness from both attendees and subject matter experts. The Collaboratory Coordinating Center will use the feedback to refine the content for future workshops and educational materials. The NIH Collaboratory’s Living Textbook provided a foundation for much of the workshop, and a host of new information was presented that can be used in future materials and updates to the textbook.

The training was funded as an NIH Roadmap Initiative 3U54AT007748-05S2.


February 20, 2018: National Academies Release Workshop Summary on Real-World Evidence in Medical Product Development

The National Academies of Sciences, Engineering, and Medicine released a summary of the first workshop in a 3-part series on the development and use of real-world evidence in medical product development. The report, Examining the Impact of Real-World Evidence on Medical Product Development: I. Incentives, focuses on the role of incentives in supporting the collection and use of real-world evidence in product review, payment, and delivery.

In clinical research, data from observational studies, registries, claims, safety surveillance, and other sources are increasingly recognized as valuable supplements to randomized controlled trials. The 3-part workshop, sponsored by the US Food and Drug Administration, is intended to “advance discussions and common knowledge about complex issues relating to the generation and utilization of real-world evidence.”

NIH Collaboratory investigators Dr. Gregory Simon and Dr. Richard Platt served on the planning committee for the workshop series. Dr. Simon opened Workshop 1 by summarizing the core qualities of real-world evidence. Dr. Platt presented lessons from the Sentinel Initiative, an example of the successful use of real-world evidence to answer questions of efficacy and effectiveness.

The second workshop in the series, “Practical Approaches,” will explore what types of data are appropriate for which purposes and will recommend practical approaches for data collection. Registration for Workshop 2 is open now.

February 19, 2018: New Podcast: Dr. Andrew Faucett on Considerations for the Return of Genomic Results

The NIH Collaboratory is pleased to announce that the new episode of the Grand Rounds podcast is now available, featuring Dr. Andrew Faucett of Geisinger. In this episode, Dr. Faucett speaks with moderator Dr. Lesley Curtis about the scalability of returning genomic research to clinicians and patients, considering issues like technical validity and sample identity in genetic variants. He also discusses the trend in researchers leaning towards being more transparent and returning pathogenic genetic variants to patients, as this information can ultimately benefit them.

Listen to the episode here:

At least once a month, we will release interviews with Grand Rounds speakers that delve into their topic of interest and give listeners bonus time with these featured experts.

Please let us know what you think by providing your feedback through the podcast page. We also encourage you to listen and share the recordings with your colleagues!

February 15, 2018: Timely Tips from Demonstration Project Principal Investigators

The NIH Collaboratory will soon welcome a new group of Demonstration Projects and guide them through the piloting and implementation phases of their embedded pragmatic clinical trials (ePCTs). We asked three seasoned principal investigators—Drs. Laura Dember, MD (TiME), Lynn DeBar, PhD (PPACT), and Jerry Jarvik, MD, MPH (LIRE)­—to share tips and advice for investigators who are new to ePCTs.

Download the PDF.

“Integrate really well with the key stakeholders from your sites.”
– Lynn DeBar, PhD

“Engage with senior mentors and people who have gone down this road before.” – Jerry Jarvik, MD, MPH

“Recognize that the perspectives of key stakeholders may be different than anticipated by investigators.” – Laura Dember, MD


February 14, 2018: Grand Rounds Podcasts Now Available on iTunes

The NIH Collaboratory is pleased to announce that Grand Rounds podcasts are now available on iTunes. These podcasts aim to give listeners a deeper understanding of topics discussed during Grand Rounds presentations through interviews with the speakers. Topics covered in past episodes include the IMPACT-Afib trial and the role of Sentinel, data and safety monitoring in pragmatic trials, and more. Grand Rounds podcasts are released at least once monthly and can be played or downloaded for free.

We encourage you to subscribe, listen, and share the recordings with your colleagues. The podcast episodes are also available on the Living Textbook and through SoundCloud.


February 7, 2018: In New Video, Drs. Susan Huang and Gloria Coronado Give Advice to Pragmatic Trial Investigators

In the spring of 2018, the NIH Collaboratory will be welcoming a new set of Demonstration Projects and will help shepherd the new pragmatic trials through the piloting and implementation phases of their studies. In a new video in the Living Textbook, two of our seasoned principal investigators—Drs. Susan Huang and Gloria Coronado—give advice to the newcomers and other investigators new to conducting pragmatic trials.

Advice to New Pragmatic Trial Investigators from NIH Research Collaboratory on Vimeo.

“My greatest advice is to gain partners who are operational and have insight into particular areas—whether they be hospitals, or clinics, or nursing homes—who know about how they really work, how to best incorporate the intervention into workflow, how to get the right approvals, and how to get the best information technology support available to them.”—Susan Huang, MD

“One of the key things that we’ve received from being part of the Collaboratory—compared to an R01 grant—is the opportunity to interact across all of the institutes at NIH as well as learn about other projects that are working in pretty diverse health systems, including hospital systems, nursing homes, and dialysis centers.” —Gloria Coronado, PhD