Including Patient Representatives in the Design and Modification of PROs: A New Era of Patient-Centric Clinical Research

As clinical research becomes increasingly focused on patient-centricity, the role of patients is evolving from passive study subjects to active collaborators. Nowhere is this shift more critical than in the development and refinement of Patient-Reported Outcomes (PROs).

Including patient representatives — individuals who can speak on behalf of patient populations — in the design and modification of PRO measures is not only an ethical imperative but a scientific one. Their involvement ensures that the tools we use to capture patient experiences are relevant, meaningful, and reflective of what truly matters to those living with the disease.

This article explores why patient involvement in PRO development is vital, the benefits it brings, and best practices to operationalize patient engagement effectively.

I. The Traditional Model and Its Limitations

Historically, PROs were developed by clinicians, researchers, and psychometricians based largely on clinical assumptions about what symptoms and outcomes mattered. While scientifically rigorous, this top-down approach often missed the nuanced, real-world experiences of patients.

As a result:

• Important symptoms or quality-of-life issues may have been overlooked.

• Questionnaire language was sometimes confusing, burdensome, or irrelevant.

• Outcomes measured did not always align with patients' priorities and treatment goals.

This disconnect reduced the validity, acceptance, and regulatory utility of PRO data — ultimately diminishing the patient voice at the heart of clinical evidence.

II. Why Including Patient Representatives Matters

1. Enhancing Relevance and Validity

Patients live with their conditions every day. Their insights can reveal symptoms, side effects, and quality-of-life impacts that clinical teams may not anticipate. By incorporating their perspectives early, PROs can better capture the outcomes that genuinely reflect patient experiences and treatment goals.

This enhances both content validity (the extent to which the instrument measures what it claims to measure) and the overall scientific rigor of the data collected.

2. Improving Patient Engagement and Compliance

When patients see that study instruments ask the "right" questions — those that resonate with their actual experiences — they are more likely to engage with the trial, provide complete and thoughtful responses, and remain committed to the study.

This leads to:

• Higher compliance rates with PRO completion schedules.

• Reduced missing data.

• Richer, more reliable datasets for analysis.

3. Aligning with Regulatory and Ethical Expectations

Regulatory agencies increasingly advocate for meaningful patient engagement in drug development, clinical trial design, and outcomes measurement.

The FDA’s Patient-Focused Drug Development (PFDD) guidance series and EMA’s patient involvement initiatives emphasize that patient input should shape endpoint selection, trial design, and benefit-risk assessments.

Including patient representatives in PRO design aligns trials with evolving regulatory science and enhances the likelihood of label claims based on PRO evidence.

III. Strategic Benefits Beyond the Trial

1. Accelerating Market Access and Reimbursement

In value-based healthcare environments, demonstrating treatment impact on patient-centered outcomes is crucial for payer approval and HTA (Health Technology Assessment) evaluations. PROs designed with patient input are more credible and persuasive during pricing and reimbursement negotiations.

2. Building Public Trust and Corporate Reputation

Sponsors who authentically involve patients in PRO development strengthen their brand as patient advocates. This can drive greater trial participation, public goodwill, and long-term loyalty from patient communities.

Moreover, it mitigates the growing scrutiny from stakeholders demanding greater transparency, inclusivity, and societal value from the biopharma industry.

IV. How to Effectively Include Patient Representatives

1. Early and Continuous Engagement

Patient representatives should be engaged from the earliest phases of PRO development — starting with concept elicitation — and continue through item generation, cognitive debriefing, and validation studies.

Best practice: Form patient advisory boards or councils dedicated to specific trials or therapeutic areas.

2. Diversity of Perspectives

Patients are not a monolithic group. It is critical to ensure diversity across:

• Demographics (age, gender, race, socio-economic status)

• Geography (urban, rural, global regions)

• Disease stages and comorbidities

This ensures PROs are broadly applicable and equitable, reducing bias and improving generalizability.

3. Co-Design, Not Just Feedback

True inclusion means more than token feedback at the end of development. Patients should be invited to co-create, providing input on:

• Which symptoms matter most

• How questions are phrased

• What recall periods are reasonable

• How burdensome the survey experience is

Participatory design methodologies, including workshops, focus groups, and iterative prototyping sessions, are valuable tools.

4. Training and Empowerment

Not all patient representatives are familiar with clinical research processes or psychometric principles. Providing orientation, mentorship, and supportive resources empowers patients to contribute meaningfully.

Likewise, researchers should be trained in patient communication best practices to foster respectful and productive collaboration.

V. Case Study: Patient-Centric PRO Design in Action

In a recent oncology trial, a sponsor partnered with a lung cancer patient advocacy group to redesign its PRO instruments.
Through participatory workshops, patients highlighted that fatigue and cognitive fog were as debilitating as more traditional endpoints like pain or tumor-related symptoms — but were missing from the original tool.

The revised PRO incorporated patient-prioritized domains and was subsequently praised by the FDA during the sponsor's NDA review as an example of patient-focused outcomes evidence.
The trial also recorded higher PRO completion rates (above 90%) than historical norms.

VI. How Cloudbyz Supports Patient-Inclusive PRO Development

At Cloudbyz, we believe the future of clinical research belongs to organizations that listen to and co-create with patients.

Our Cloudbyz ePRO platform:

• Supports patient advisory integration in instrument configuration.

• Provides real-time feedback mechanisms to adjust PRO tools dynamically based on participant experiences.

• Offers easy-to-configure forms enabling rapid incorporation of patient-prioritized domains.

• Ensures multilingual, mobile-first designs to accommodate diverse patient needs.

Cloudbyz empowers sponsors and CROs to build authentic, patient-centric studies — from endpoint development to real-world deployment.

Conclusion

Including patient representatives in the design and modification of PROs is not merely a best practice — it is a necessity for credible, meaningful, and actionable clinical research.

By respecting patients as experts in their own lived experiences, sponsors can create outcome measures that drive scientific innovation, regulatory success, patient trust, and market differentiation.

The modernization of PROs must be driven by partnership, inclusion, and empathy — because in the end, the most important outcomes are those that patients themselves deem meaningful.