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The Value of a Voice

Incorporating Patient-Reported Outcomes in Research and Practice

This is the fifth piece in our new Patient Engagement series, where Lionbridge Life Sciences experts discuss the history and current status of participants in clinical studies and patients in the healthcare system. Come back every week for more from our team and get in touch if you have ideas of your own to share.

The Patient's Voice

Patients are the experts on their own conditions and health. The patient’s perspective — or the patient’s voice — provides insights into the patient’s wants, needs and preferences and thus presents a critical dimension of health care quality.

“The concept of value is hard to define in health care, unless you are a patient,” said Roger Wilson, a patient advocate and founder of Sarcoma UK. His comment highlights how critical the patient’s perspective is in developing patient-centric health care.

Historically, the medical profession has centered the clinical view when considering relevant outcomes of care. This approach often fails to capture the considerations about the patient’s own experiences of living with the condition, its impact on the patient’s quality of life or their experiences with health care.

The World Health Organization’s 1978 Declaration of Alma Ata affirmed the importance of the patient’s voice: “The people have the right and duty to participate individually and collectively in the planning and implementation of their health care.” From that point onwards, the idea of patient engagement in healthcare provision has been gaining ever-increasing traction and acceptance.

Capturing patients’ perspectives includes various stakeholders, and within the concept of patient engagement the term “patient” may also encompass the patient’s family, caregivers or patient representatives. All these characters can give relevant perspectives.

Patient-Reported Outcomes as Part of the Healthcare Ecosystem

One approach that supports patient engagement is the use of patient-reported outcome (PRO) measures.  Presented in the form of standardized questionnaires, PRO measures are important instruments to help evaluate healthcare interventions from the patient’s perspective, both in clinical research and clinical practice.

For example, in clinical trials, regulatory evaluators can use PRO data derived from PRO measures to determine whether a medical product has been shown to benefit patients. The evidence from the trial PRO data may then be used for downstream decision-making in a product’s pathway to market. In routine clinical care, PRO measures may be used to collect the patient’s input on the impact and quality of care received or on health-related quality of life.

The use of PRO measures truly enables the capture of the patient’s voice and outcomes valued by patients. As the Organization for Economic Co-operation and Development put it in a recent report on patient-reported indicators for assessing health system performance: “That medicine has become quite successful at treating disease should be celebrated. However, continual improvement must include assessment of impact treatments have on people’s lives. This makes outcomes valued by patients a key indicator of success.”

The Patient's Voice in Health Outcome Research and Medical Product Development

A wide range of research communities have created initiatives to increase patient engagement in health outcomes research. Organizations such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), Patient-Centered Outcomes Research Institute (PCORI), International Consortium for Health Outcomes Measurement (ICHOM) and others recognize the benefits of patient engagement in their research activities and actively support various engagement initiatives. They have formal groups and guidelines to define best practices:

  • ISPOR’s Patient Council and Patient Representatives Roundtables serve to facilitate communication among patient representatives and to convene stakeholders with the goal of engaging patient representatives in the research and decision-making processes.

  • PCORI’s Engagement Rubric provides a framework for operationalizing engagement to incorporate patients in all phases of research. It aims to encourage active and sustained engagement of patients as partners in planning, conducting and disseminating research to improve clinical decision-making and outcomes.

  • ICHOM brings together global teams of physician leaders, outcomes researchers and patient advocates to define a standardized list of the outcomes (along with the corresponding measures and methodological guidance) that matter most to patients living with different conditions. Patient involvement ensures that healthcare providers focus their resources on delivering outcomes that make the biggest difference to patients and their experience of care.

The patient’s perspective is equally important in medical product development. Input from patients and caregivers helps to ensure that clinical studies assess outcomes that are meaningful to patients.

The importance of incorporating the patient’s perspective into the development and execution of clinical studies is demonstrated by the broad scope of regulatory and industry framework and guidance documents. To name just a couple:


  • As part of its patient-focused drug development initiative, the U.S. Food and Drug Administration (FDA) has been developing a series of guidance documents to capture and meaningfully incorporate patients’ experiences, perspectives, needs and priorities into drug development and evaluation. The third and fourth guidance documents in this series will specifically focus on the topics related to the identification or development of the fit-for-purpose PRO measures (as well as other types of clinical outcome assessments) and their incorporation into endpoints for regulatory decision-making.

  • Similarly, in draft guidance documents on patient engagement in the design and conduct of medical device clinical investigations and principles for selecting, developing, modifying, and adapting PRO instruments for use in medical device evaluation, the FDA has shared methodological considerations to encourage and support the collection, analysis and integration of patient perspectives in the development, evaluation and surveillance of medical devices.

  • The European Medicines Agency's (EMA) "Regulatory Science to 2025" strategy contains a clear message in support of advancing patient-centered access to medicines in partnership with healthcare systems. For instance, the agency aims to reinforce patient relevance in evidence generation; to coordinate its approach to and update relevant clinical guidelines to include reference to PRO measures; to promote the use of core health-related quality-of-life measures; and to enhance international collaboration with regulators that are pioneering initiatives on patient-focused drug development such as the U.S. FDA.

  • As part of its Patient Experience initiative, TransCelerate developed the “patient protocol engagement toolkit” and “participant feedback questionnaire toolkit”. These materials (which are targeted at study sponsors and study participants, respectively) serve as tools to obtain meaningful patient input on clinical study planning and execution and to improve overall patient experience.

"The patient’s perspective is equally important in medical product development. Input from patients and caregivers helps to ensure that clinical studies assess outcomes that are meaningful to patients."

Patient Involvement in the Development of PRO Measures

As only patients themselves can determine which health outcomes are relevant for them and whether a PRO questionnaire captures these outcomes, their involvement in the development of PRO measures is essential.

During the design and validation of a new PRO measure, the patient’s input informs the scope and content of questions or tasks to be included in the draft measure. This input is typically solicited via one-on-one interviews, focus groups or consensus panels. Once a PRO measure has been drafted, patient feedback helps to assess the draft measure’s various aspects, such as whether it is comprehensive and captures the concepts important to the intended patient population; if the patients understand how to complete it; and if it is easy to read and understand.

Patient understanding and ease of PRO measure’s use by the patients are assessed by conducting cognitive interviews, usability tests and readability tests. The patient’s input received from these interviews or tests serves to further refine the draft PRO measure, for example, by deleting or modifying questions, response options, or patient instructions.

If the original PRO measure is modified — such as when it’s translated into another language or adapted from paper to the electronic format — the patient’s input helps to ensure that the modification has not impacted the content validity of the modified PRO measure and that the new format and mode of use are appropriate for the target patient group.

The benefits of patient involvement and participation are clear. Without a doubt, PRO measures that are developed in collaboration with the patients would be more relevant and acceptable to the relevant target patient population than those developed without. In turn, this can increase patient engagement and enhance the quality and regulatory acceptance of PRO data. 

Translating Involvement into Empowerment and Impact

"Patient engagement should not be a case of all or nothing. It should be regarded as an ongoing commitment rather than a box-ticking exercise."

However, engaging patients in outcomes research and development of PRO measures is not without its challenges. Researchers and patients may be deterred from offering the opportunities for involvement and from seeking such opportunities, respectively, by the perceived complexity of the process. The scope of involvement may be limited due to the budget issues on the researchers’ side and time constraints on both the researchers’ and patients’ sides. And in some cases, such as with rare diseases, the target patient population may simply be very small. These challenges may present the idea of patient involvement as something that is desirable in principle but hard to achieve in practice.

Deciding how to involve patients can be difficult since the decision can have implications for the research project. Patient involvement is increasingly recommended by regulators.

The important consideration here is that patient engagement should not be a case of all or nothing. It should be regarded as an ongoing commitment rather than a box-ticking exercise.

There is no universal approach or procedure that fits all research contexts or provides the answers on how to best engage the patients. Patient involvement should be planned and developed for each individual case. Choose the approach which is most suitable for the patient group and the purpose of the research study, using the available methodological recommendations and practical experiences.

The conventional wisdom suggests that the chances of success of patient involvement initiatives are higher if these initiatives are supported by the trained and experienced research staff, open and clear communication and a shared understanding of roles and expectations of what each party is supposed to bring to the table. In turn, the success of patient involvement initiatives can further empower patients in taking a more active role of “partners in research”, helping to conceptualize, design and conduct research and disseminate its findings. Indeed, the partnership approach to decision-making will serve as the foundation of and the differentiator for truly patient-centric health care.

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Nataliya Volohov
Nataliya Volohov