2: Standards Associated with Patient-Centeredness

PC-1: Engage people representing the population of interest and other relevant stakeholders in ways that are appropriate and necessary in a given research context

Include individuals affected by the condition and, as relevant, their surrogates and/or caregivers. Other relevant stakeholders may include clinicians, purchasers, payers, industry, hospitals, health systems, policy makers, and training institutions. These stakeholders may be end users of the research, or, be involved in healthcare decision making.

Examples of processes in which patients, caregivers, clinicians, and other healthcare stakeholders can be involved include but are not limited to:

• Formulating research questions;
• Defining essential characteristics of study participants, comparators, and outcomes;
• Identifying and selecting outcomes that the population of interest notices and cares about (e.g., survival, function, symptoms, health-related quality of life) and that inform decision making relevant to the research topic;
• Monitoring study conduct and progress; and
• Designing/suggesting plans for dissemination and implementation activities.

PCORI’s Engagement Rubric provides further guidance.

When applicable, research proposals should describe how these stakeholders will be identified, recruited, and retained. If engagement is not necessary or appropriate in these processes, explain why.

Public comments

PCORI is faced in developing this new set of Standards with the challenge of being prescriptive without being overly directive. When describing the relevant stakeholders within this Standard, PCORI should consider changing the language to say, “Other relevant stakeholders may include but are not limited to clinicians, purchasers…” There are other stakeholder categories that may fall beyond the list identified—one example being community-based organizations working with health care systems—that researchers should also consider when determining the types of individuals who will be involved within the study and their approach to engagement. As currently written, PCORI’s list is more exclusive than illustrative. Beyond this, here PCORI defines the role of consumers or stakeholders in a manner that is appropriate in terms of a particular research project, but it does not speak to the fact that stakeholders should be involved in the research enterprise more broadly. There’s a greater role for researchers to play in engaging consumers and patients in governance and oversight processes, beyond simply the research project. AcademyHealth’s Electronic Data Methods (EDM) Forum authored a paper in 2012 (http://repository.edm-forum.org/cgi/viewcontent.cgi?article=1001&context=edm_briefs) that examines and offers insight into these issues that may be useful to PCORI for incorporating these critical concepts into its revised Methodology Standards. In addition, the final bullet regarding PCORI’s Engagement Rubric is not proportionate in level and scope with the other bullets on processes. This statement should act as a broader note within the Standard, and thereby be removed from the bulleted list. We would also raise that in the last sentence of the Standard— “If engagement is not necessary or appropriate in these processes, explain why”—it is not clear whether the intent is to note where engagement is or is not appropriate for each of the project’s components or for the project overall.

Lisa Simpson, AcademyHealth, Stakeholder - Other, 04/11/2016 - 4:27pm

We applaud the Committee on clarifying how patients and stakeholders should be involved in the prioritization of research, conduct of research, and the dissemination of research findings. As outlined in the standards, a broad set of stakeholders, including the biopharmaceutical industry, contribute to a more robust research process. Purchasers, payers, and industry communities seek to ensure that relevant questions are addressed, research findings are usable, and results are translated and implemented in practice. This engagement is an important aspect towards improving the relevance of CER questions and ultimately the impact of CER on health care decision-making.

National Pharmaceutical Council, Industry, 03/28/2016 - 9:42am

Patient Advocates need to be added to the stakeholders list

Astrazeneca, Industry, 03/21/2016 - 10:29am

The bolded print here refers to "ways that are appropriate and necessary". This is very broad. Essentially, this will result on everyone conceivable being drawn into the process, or the investigator being compelled to offer explanations as to why some segment was not engaged. Can the authors be more specific about how one determines what groups are really appropriate and necessary for such engagement?

Merck & Co Inc, Industry, 02/23/2016 - 2:34pm

While patients' participation in different portions of the research process is certainly of interest to researchers, the benefits from a patient perspective might not be immediately obvious. It is therefore important that their participation be compensated with a reciprocal gain. This gain could be in form of information to be taken back to their community, free lectures provided by researchers with expertise in the field, or anything else that might make this collaboration feel like a fair exchange with mutual gain.

Ricardo Pietrobon [email protected], Health Researcher, 01/29/2016 - 12:53pm

PC-2: Identify, select, recruit, and retain study participants representative of the spectrum of the population of interest and ensure that data are collected thoroughly and systematically from all study participants

Research proposals and subsequent study reports should describe:

1. the plan to ensure representativeness of participants;
2. how participants are identified, selected, recruited, enrolled, and retained in the study to reduce or address the potential impact of selection bias;
3. efforts employed to maximize adherence to agreed-on enrollment practices; and
4. methods used to ensure unbiased and systematic data collection from all participants.

If the population of interest includes people who are more difficult to identify, recruit, and/or retain than other study populations (for example, individuals historically underrepresented in healthcare research such as those with multiple disease conditions, low literacy, low socioeconomic status, or poor healthcare access, as well as racial and ethnic minority groups and people living in rural areas), then specify plans to address population-specific issues for participant identification, recruitment, and retention.

Public comments

The research proposals should include a description of how the researchers worked with populations who are anticipated to be hard to recruit and retain to ensure that the study design and practical aspects of the study were adapted and/or additional support given to ensure that barriers to participation are minimised. (ie not just design it for those patient populations, but work with them)

Astrazeneca, Industry, 03/21/2016 - 10:29am

Usually the most challenging aspect related to patient data collection is longitudinal retention. It is therefore important to have researchers emphasize multi-pronged approaches to retention that focus not only on individual patients but also in those close to them. For example, involving families and close friends will not only assist in retention through multiple points of contact, but might also provide the necessary support and incentive for participants to keep themselves motivated throughout the project. These mechanisms should therefore be described in detail. Finally, as stated under PC-1, it is important that these connections to decrease attrition be based on offering patients something that might be of value to them, establishing a connection based on trust and a sense of fairness in the exchange.

Ricardo Pietrobon [email protected], Health Researcher, 01/29/2016 - 12:53pm

PC-3: Use patient-reported outcomes when patients or people at risk of a condition are the best source of information

When patients or people at risk of a condition are the best source of information regarding outcomes of interest, then the study should employ patient-reported outcome (PRO) measures in lieu of, or in addition to, measures derived from other sources. Proposals should describe: 1) the concept(s) underlying each PRO measure (e.g., symptom or impairment) and how it is meaningful to, and noticed by, patients in the population of interest; 2) how the concept relates to the health decisions the study is designed to inform; 3) how the PRO measure was developed, including how patients were involved in the development; and 4) evidence of measurement properties including content validity, construct validity, reliability, responsiveness to change over time, and score interpretability, including meaningfulness of score changes in the population of interest with consideration of important subgroups. If these measurement properties are not known, a plan for establishing the properties must be provided. Caregiver reports may be appropriate if the patient cannot self-report the outcomes of interest. If PROs are not planned for use in the study, justification must be provided.

Public comments

The context of use (ie why the PRO is considered appropriate for patient population) should also be described. In situations where the PRO is available in several languages, the translation and linguistic validation evidence should be provided.

Astrazeneca, Industry, 03/21/2016 - 10:29am

While traditional self-reported scales are essential, technologies such as Computer Adaptive Tests ensure not only the measurement precision will be increased among patients in the two extremes of the domain being measured (very high or very low), but will also allow for other features such as a reduction in time to respond a questionnaire, the ability to respond using a mobile phone, the measurement of multiple dimensions through multidimensional CAT, the enhancement of the measurement model over time through progressively increasing items banks, among a number of other advantages associated with this technology and the underlying Item Response Theory modeling, and multiple other characteristics that are fully aligned with the mission of centering research on individual patients and communities. In addition, with the new open source packages such as mirtCAT https://goo.gl/OTxaEN , the time and cost to generate a CAT system is minimum. Another suggestion would be to emphasize that new scales or item banks should be ideally licensed under Open Access Licenses such as https://creativecommons.org/ , so that researchers and other stakeholders can freely use that resource.

Ricardo Pietrobon [email protected], Health Researcher, 01/29/2016 - 12:53pm

PC-4: Support dissemination and implementation of study results

For study results that are appropriate for dissemination and implementation, involve patients and relevant stakeholders: a) in planning for dissemination from the start of the research study; b) in creating a dissemination plan for the study indicating clinical implications; c) in working with patients or organizations to report results in a manner understandable to and usable by each target audience; and d) in identifying successful strategies for adoption and distribution of study findings to targeted patient and clinical audiences.

Public comments

AcademyHealth was perplexed by the addition of the language qualifying study results as “appropriate for dissemination and implementation” and why such a modifier would be necessary. It is unclear when—or why—PCORI would not want to share a study’s findings with appropriate stakeholder audiences. Finally, similar to concerns raised in previous Standards, the framing of newly added standard clause ‘d’ is too restrictive to be of value to improving health and health care. Specifically, study findings should be adopted and distributed beyond merely ‘patient and clinical audiences.’ We would recommend ending the sentence at ‘findings’ or broadening the listed audiences.

Lisa Simpson, AcademyHealth, Stakeholder - Other, 04/11/2016 - 4:27pm

While dissemination is of utmost importance, if the results being disseminated are not put to use, then the value of the dissemination is significantly decreased. As such, researchers should probably devise the simplest possible methods to measure the impact of their dissemination efforts. As a rule of thumb, these metrics should be simple and easy, perhaps starting with something as mundane as gathering data on number of Web site hits or social network shares a given resource might have reached. The central concept is that without this type of feedback, researchers and patients will know very little on the most effective methods of disseminating information.

Ricardo Pietrobon [email protected], Health Researcher, 01/29/2016 - 12:53pm