Planning COA strategy for patient focused drug development

When planning a Clinical Outcome Assessment (COA) strategy to generate patient experience data in drug development, you need a systematic approach to ensure your endpoints truly capture what matters to patients. Here are some things to consider when developing an effective strategy:

Different Types of COAs in Drug Development

COAs are measurements that capture how a patient feels, functions, or survives. They are critical for demonstrating relevant and meaningful treatment benefits. The four main types are:

1. Patient-reported outcomes (PROs): Direct reports from patients
2. Clinician-reported outcomes (ClinROs): Assessment performed by a trained  healthcare professional
3. Observer-reported outcomes (ObsROs): Assessment performed by an observer (i.e. not a health care professional), e.g., a parent/caregiver
4. Performance outcomes (PerfOs): Measurement based on a standardized task performed by a patient

Strategic Planning Steps

Create Target Population Profile (TPP)

·      Define intended use of product; target population; delivery mode; efficacy goal; etc.

Understanding Patients’ Disease Experience

• Conduct patient interviews (individual or focus groups)
• Are interviews with, e.g., healthcare professionals needed?
• Review empirical published evidence
• Identify symptoms and impacts that matter most to patients
• Understand the disease burden and treatment goals from the patient perspective

Select Appropriate COA Measures

• Review existing validated instruments
• Assess if available measures adequately capture patient priorities
• Consider developing new measures if gaps exist (time-intensive)
• Evaluate psychometric properties, including if measures are sensitive to change

Engage Regulatory Agencies Early

• Review FDA guidance on patient-focused drug development
• Present COA strategy—including endpoints of interest—early to regulatory and HTA bodies for feedback
• If qualitative interviews will be included in the clinical trial (and if time allows), consider submitting interview manual for feedback

Incorporate COAs Throughout Development

• Include COAs as early as possible to gather preliminary data
• Refine endpoint selection based on early findings
• Design pivotal trials with clear primary and secondary COA endpoints

Establish  Meaningful Treatment  

• Generate  meaningful treatment thresholds using both anchor- and distribution-based approaches
• Link COA results to other clinical outcomes

Implementation Best Practices

• Train site staff on proper COA administration
• Minimize missing data through patient education and reminders
• Consider electronic COA collection to improve data quality
• Plan for cultural and linguistic validation for global studies

Translation to COA Strategy

• Map patient-identified concepts to existing validated instruments
• Identify measurement gaps that may require new instrument development
• Prioritize endpoints based on patient importance and regulatory considerations
• Document the evidence linking patient input to selected measures

Documentation for Regulatory Submission

Create an Evidence Dossier

• Summarize interview methodologies and findings
• Provide clear rationale for how patient input informed endpoint selection
• Include conceptual frameworks derived from patient interviews
• Demonstrate how selected COAs align with patient priorities

This structured approach ensures that your clinical endpoints genuinely reflect outcomes meaningful to patients, which strengthens your regulatory submissions and increases the likelihood of demonstrating clinically meaningful treatment benefits.

Incorporating Patient Interviews into Your COA Strategy

Patient interviews are a foundational element of an effective COA strategy. Here's how to systematically incorporate them:

Planning the Interview Process

Define Interview Objectives

• Identify what specific information you need from patients, e.g.:
  • Is cognitive debriefing of the intended COA instrument needed, i.e. evaluate if the questions and instructions are clear, culturally appropriate, and relevant to the patients' experiences?
  • Is concept elicitation (gathering data directly from patients to understand their experiences, perspectives, and impact of a condition or treatment on their lives) needed to evaluate if the instrument captures all relevant concepts that are meaningful to the target patient population?
  • Should interviews inform endpoint selection?
  • Should interviews be supportive in determining what is meaningful treatment effect?

Develop a Sampling Strategy

• Ensure diversity in disease severity, demographics, and treatment experiences
• Include patients across the disease spectrum and treatment journey
• Consider including caregivers for perspectives on observable impacts

Aim for 20-30 patients for concept elicitation and 10-15 for cognitive debriefing. However, the number of patients needed depends on the diversity of the population.

Conducting Effective Interviews

Create Interview Guides

• Develop semi-structured interview guides with open-ended questions
• Design questions that avoid leading patients to specific answers
• Have guides reviewed by patient advocates and clinical experts

Interview Implementation

• Use trained interviewers familiar with qualitative research methods
• Record and transcribe interviews for thorough analysis
• Consider using journey mapping to understand disease progression
• Document contextual factors that might influence patient experiences

Analyzing and Applying Findings

Qualitative Data Analysis

• Code transcripts systematically to identify key concepts
• Track concept saturation to ensure comprehensive coverage
• Develop conceptual frameworks showing relationships between symptoms and impacts
• Identify potential endpoints based on frequency, importance to patients, and sensitivity to change

CLINIGMA^® provides expert solutions in capturing patient feedback in clinical trials in accordance with regulatory requirements. With the largest global network of researchers on the market, we are able to interview patients worldwide, which enables us to accommodate the needs of global pharmaceutical companies.