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 Janey.hsiao@ahrq.hhs.gov 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March 7, 2018: FDA Offers Workshop on Submitting Draft Guidance on Patient Experience Data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 Workshop attendees will discuss considerations for development and submission of a proposed draft guidance regarding patient experience data submitted by an external stakeholder.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.

CTTI Releases 2015 Annual Report


The Clinical Trials Transformation Initiative (CTTI) has released its Annual Report for 2015. The report describes major achievements from the previous year, including new recommendations and related tools and checklists for improving the safety, efficiency, and overall quality of clinical research.

Cover page of CTTI Annual Report with embedded link to CTTI webpage containing report.
2015 CTTI Annual Report

Highlights of the 2015 Annual Report include recommendations on topics including:

  • Ethics review processes
  • Good Clinical Practice training for trial investigators
  • Research protocol design
  • Engagement of patient groups as equal partners in clinical research
  • Informed consent processes
  • Safety reporting systems for research participants

A public-private partnership whose many stakeholders include government agencies, advocacy groups, professional societies, academic research organizations, and representatives from the medical products industry, CTTI’s mission is to “identify and promote practices that will increase the quality and efficiency of clinical trials.”

A PDF version of the report is available here. Previous Annual Reports are also available on the CTTI website.


 

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.


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.


 

Systematic Review on Stakeholder Engagement in Comparative Effectiveness and Patient-Centered Outcomes Research


This month’s issue of the Journal of General Internal Medicine features a systematic review of stakeholder engagement in comparative effectiveness research and patient-centered outcomes research. Thomas W. Concannon, PhD, and coauthors identified 70 peer-reviewed articles since 2003 that reported on this topic.

Key results included:

  • Patients were the most commonly engaged group, followed by modest engagement of clinicians, and infrequent engagement of other stakeholders across the healthcare system.
  • Stakeholders were more often engaged in earlier stages of research (evidence prioritization and generation) than in later activities such as evidence interpretation and application.

Overall, reporting of stakeholder activities and the effects of engagement were highly variable in the literature. To address this, the authors developed a 7-item questionnaire for the reporting of stakeholder engagement in research. A suggested plan for future research on stakeholder engagement is also outlined.


Patient Engagement Discussed via Twitter Chat


Recently the journal Health Affairs and the Patient-Centered Outcomes Research Institute (PCORI) conducted a Twitter chat on the topic of patient engagement in research. The chat was an hour-long moderated question-and-answer session with Susan Sheridan, the director of patient engagement at PCORI. Participants joined the conversation via #PatientHC and talked about what patient engagement looks like and concerns about the privacy of patient health data.

The hosts referenced the journal’s 2013 theme issue on patient engagement as well as three videos produced in partnership with PCORI that illustrate ways patients and providers are incorporating patient engagement in healthcare decisions. An archived version of the Twitter chat is here.


FDA Releases Action Plan to Encourage Greater Patient Diversification in Trials


In August 2014, the Food and Drug Administration (FDA) released an action plan (link opens as a PDF) aimed at encouraging more diverse patient participation in drug and medical device clinical trials. The Action Plan to Enhance the Collection and Availability of Demographic Subgroup Data includes 27 responsive and pragmatic actions, divided into 3 overarching priorities:

  • Data quality: improving the completeness and quality of demographic subgroup data collection, reporting, and analysis
  • Participation: identifying barriers to subgroup enrollment in clinical trials and employing strategies to encourage greater participation
  • Transparency: making demographic subgroup data more available and transparent

The plan follows an August 2013 report to Congress on these concerns and reflects the agency’s commitment to encouraging the inclusion of a diverse patient population (with reference to sex, age, race, and ethnicity) in biomedical research that supports applications for FDA-regulated medical products. Increasing representation is a multifaceted challenge that requires a multifaceted approach and collaboration of federal partners, industry, healthcare providers, patients and patient advocacy groups, academicians, and community groups.

message from the Commissioner of the FDA contains background and details.


PCORI Featured in Health Affairs Blog


Joe Selby, MD, MPH, executive director of the Patient-Centered Outcomes Research Institute (PCORI), discusses the promise of PCORI’s research efforts in a recent guest blog post for healthcare policy journal Health Affairs. In his post, Selby explains that PCORI’s mandate is not just to fund comparative effectiveness research (CER) studies that compare multiple therapeutic options, but also to share the results in ways that are meaningful to patients, physicians, and other stakeholders.

Although many of PCORI’s resources are dedicated to supporting CER in reaPatient-Centered Outcomes Research Institute (PCORI) logol-world settings, patient engagement in research is also a cornerstone of PCORI’s efforts. Of the $464 million invested, 62% has been focused on CER, with the rest spread across infrastructure (18%), methods (11%) and communication and dissemination research (8%). PCORI expects to commit $1 billion over the next 2 years to expand their work. As part of their mission, they also hope to improve research methods and build the national capacity for CER.