Fit-for-Purpose COA Development: 25 years developing, modifying and validating PROs in more than 60 therapeutic categories. LEARN MORE

Translation Services: 25 years developing linguistically validated translations of COAs, CRFs, protocols, manuals, etc… READ MORE

QualityMetric By The Numbers: 53,000,000+ Global Survey administrations, 48,000+ Peer-reviewed articles across 100+ conditions, 2,000+ Language translations by mode of administrations, 100+ Condition-Specific, Country-Specific, and Insurance Claims Benchmarks General Population Norms. Which survey should you use? SEE OUR SUITE OF PRO SURVEYS

ePRO & EDC Integration: Seamless integration for optimal efficiencies using Web Service Application Programming Interface (API), Single Sign-on Access (SSO) or Electronic-Friendly Survey Administration. FIND OUT HOW



Understanding Rare Cancers Using Patient-Reported Outcome Measures

Many fields in healthcare research benefit from leveraging clinician outcome assessments (COAs) to gain a deeper understanding of patient experiences. Patient-reported outcome (PRO) measures, or PROM, have seen an increased use over the years in clinical trials and other treatment delivery settings. These measurements paint a more complete picture of the spectrum of symptoms and subjective health-related quality of life (HRQoL), which is vital to achieve care initiatives. PROs can help professionals identify and address many aspects of a patient’s healthcare experience. The collection of detailed patient data provides real-world impact data of a particular disease or condition and can help build upon the collective knowledge used to comprehend the burden of diseases.
Although they have proven their worth as validated measures offering substantial benefits across the healthcare ecosystem, PROs still have some room for growth. In the field of oncology, PROs are used to support the study and treatment of rare forms of cancer, including those developed in childhood.
PRO Measures in Rare Cancer Clinical Trials
The European Journal of Cancer revealed in 2020 that only 8 percent of the 711 pediatric oncology clinical trials examined used patient-reported outcome measures as endpoints.1 This low usage persists despite recommendations from regulatory agencies, such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
To conduct high-quality research and offer effective therapies, researchers must begin to adjust their approach to studying rare cancer in children and adolescents. By incorporating the voices of these young patients and embracing more patient-centric methodologies, PROs can pull in data directly from patients to assess disease symptoms and treatment side effects. This approach can lead to better cancer outcomes. In cancer patient populations, this might mean focusing more on quality of life versus longevity; delivering long-term chronic disease care. Patient-reported outcomes have been known to boost communication between patients and physicians, increase patient satisfaction, improve symptom management and result in higher treatment survival rates in several adult cancer research studies.1
The Challenges of Cancer PROs Today
Teams that leverage cancer patient-reported outcome measures will encounter some of the same difficulties as teams using them in rare diseases like AL amyloidosis and Duchenne muscular dystrophy. Rare conditions mean smaller patient populations, which makes it challenging to overcome psychometric issues and manage data collection. Working with adolescents also forces research organizations to plan around protective parents to guide successful studies.


Selecting the right measure, executing an implementation plan and interpreting results include some of the challenges researchers may face when using cancer PROMs. However, these efforts can be simplified when teams follow best practices and partner with an experienced COA solutions provider like QualityMetric.
New Best Practices for Patient-Centric Studies

  • Connecting directly with patient communities and building a robust patient registry.
  • Customizing participant recruitment strategies to accommodate population needs.
  • Investing in direct-to-patient activities and convenient, digital-enabled endpoints.
  • Collecting real-world data and supporting innovative remote data collection.

Learn More About Rare Cancer Research COA Solutions
The strategic use of PROs can help improve the ability to report on health-related quality of life, which will benefit the research teams and industry thought leaders searching for ways to create targeted, patient-centric clinical trials and uphold newer value-based care models.
Learn more about our rare disease expertise to take advantage of custom PRO health surveys in your own oncology research or clinical practice.

  1. Riedl, D, Rothmund, M, Darlington, A. Rare use of patient-reported outcomes in childhood cancer clinical trials – a systematic review of clinical trial registries. European Journal of Cancer. 2021;152:90-99.