For decades, health authorities like the FDA and EMA have been steadily encouraging the
integration of patient voice into clinical development. Clinical Outcome Assessments (COAs), which include Patient-Reported Outcomes (PROs), Clinician-Reported Outcomes (ClinROs), Observer-Reported Outcomes (ObsROs), and Performance Outcomes (PerfOs), have emerged as essential tools in this effort, as they measure not only how patients feel or function but how they live as well.
Thus, identifying fit-for-purpose COAs that both reflect what matters to patients and align with regulatory expectations is a critical component of developing endpoint strategies that pass regulatory scrutiny.
Why endpoint strategy matters
The shift toward patient-focused drug development (PFDD) means that sponsors must go
beyond traditional efficacy measures.1 Regulators now expect endpoints that reflect patients' experiences with disease and treatment to be captured through validated, well-chosen COAs.
But identifying the right COA is rarely straightforward. It requires balancing regulatory guidance, trial design, disease specifics, population characteristics, and the psychometric properties of the assessment tools. A misstep in this process can increase participant burden, delay trial timelines, or jeopardize regulatory success.
As outlined in the webinar, developing a high-impact, regulatory-ready endpoint strategy
requires following a structured path:
- Define the Concept of Interest (COI) - Sponsors must begin by identifying the
symptoms, functional impacts, or quality-of-life concerns that matter most to patients
living with the target condition. - Incorporate regulatory precedent - Then, it is important to review label claims and
guidance documents to understand how similar concepts have been measured and
accepted by agencies like the FDA and EMA. - Evaluate and select COAs - Clinical trial teams must choose instruments that are
validated for the specific context of use, align with regulatory expectations, and
demonstrate strong evidence of reliability and validity. - Translate results into endpoint variables - Lastly, trials teams must convert COA
results into measurable variables and scoring systems that can serve as meaningful
endpoints in the trial.
Accelerating COA selection with the industry’s pre-eminent facilitator of instruments
Mapi Research Trust offers sponsors a suite of proprietary databases and expert services to
streamline the complex process of leveraging fit-for-purpose COAs :
- PROQOLID - A curated solution of 8,000+ COAs, including detailed descriptions,
concepts measured, and supporting validation data. - PROLABELS - Access to more than 2,000 drug labels featuring COA-based claims
granted by the FDA and EMA. Sponsors can use this database to identify precedent and
understand how COAs have supported regulatory success. - PROINSIGHT - With access to more than 300 disease-specific and general COA
guidelines published by global regulatory bodies, PROINSIGHT helps sponsors
understand how agencies define meaningful concepts and which measurement tools
they recommend.
In addition to these databases, the Patient Centered Endpoint Intelligence (PACE) team at
Mapi Research Trust offers targeted or comprehensive landscape reviews, psychometric
evaluations, and insight-driven reports tailored to a sponsor’s development program.
The benefits of fit-for-purpose endpoint strategies
This strategic, evidence-based approach to COA selection offers benefits across the drug
development lifecycle:
- Improved patient outcomes by measuring what truly matters to those living with the condition.
- Greater regulatory alignment and higher likelihood of approval.
- Reduced costs and fewer trial delays by accelerating COA deployment and minimizing protocol amendments.
- Enhanced precision in decision-making, supporting stronger value demonstrations for
payers and health technology assessments.
It is clear that COAs are no longer a “nice to have” but rather, a regulatory and scientific
imperative. With tools like PROQOLID, PROLABELS and PROINSIGHT, and support from
experts at Mapi Research Trust, sponsors can confidently build endpoint strategies that are not only patient-centered but also primed for success.
Learn more about how Mapi Research Trust can support your COA strategy at
www.mapi-trust.org/services.
Watch the full webinar recording here.