Patient-reported outcome measures in healthcare
In an increasingly efficient world, healthcare will sooner or later have to rethink its approach. Given the wide range of patient groups and their individual sensitivities, a focus on the individual patient is essential. Data collected from patients, known as patient-reported outcome measures (PROMs), will play an increasingly central role in this.
Evaluating PROMs and integrating them into healthcare offers the following benefits:
- Healthcare professionals can identify symptoms more quickly and take targeted action [1]
- Additional evidence for market-relevant approval studies (known as pre-market case studies) and, after marketing authorization, in so-called post-marketing studies
- Precise monitoring of drug therapy in real-world care settings for precision medicine
But what are PRO(M)s all about, and how do they contribute to precision medicine?
What are PROs / PROMs?
According to the European Medicines Agency (EMA), patient-reported outcomes (PROs) are data collected directly from patients themselves [2]. In addition to the standard medical questions (clinical, genetic and physician-related questions), subjective data on the personal perception of the disease and its treatment are evaluated.
Possible endpoints of the survey include, for example, information on:
- Impact on quality of life
- Symptoms during therapy
- Adherence to therapy
- Satisfaction with the course of therapy
These data are measured using standardized, scientifically developed methods known as patient-reported outcome measures.
PROs are mostly questionnaires that must meet the following quality criteria[3]:
1. Comparability
The questionnaires ensure that comparisons are possible both between different patients (interpersonal) and for the same patients over time (intrapersonal). Different disease groups and cultures can also be compared.
2. Reliability and validity
Strictly standardized methods ensure that the collected data can be applied to the overall population (validity). As a result, the study results are also internally consistent and capture exactly the details they are meant to capture (reliability).
3. Flexibility
Questions on specific characteristics (items), such as the subjective experience of therapy, can be freely compiled to suit the group being studied. The questionnaire retains its full reliability and validity in the process.
4. Integrity
People from all social strata and population groups can complete these questionnaires. This means that reading ability, language and physical feasibility are taken into account.
In Germany, there are already leading research institutes, such as the PROMIS institute at Charité, which develops standardized measurement instruments for patient-reported characteristics and makes them usable in real-world care settings.
Why are PROMs becoming increasingly important before marketing authorization?
PROMs make clinical trials even more precise – individual endpoints, such as the impact on quality of life, can be captured more accurately than ever, forming a sound basis for future treatment recommendations. The result is a self-learning system that, through more precise evidence, enables more precise diagnostics and, in turn, precise treatment recommendations [6]. Accordingly, a significant increase in PROM items in clinical trials has been observed in recent years. In 2018, the FDA reported that the number of pre-authorization clinical studies in the US that include PROMs had increased by >500% within six years [5].
PROMs after marketing authorization
In addition to their importance for the marketing authorization of drugs, PROMs also help generate relevant outcomes in real-world care settings. Data on the personal perception of therapy continue to be collected and compiled after approval. This is often a mandatory component for new drugs that may have received conditional market approval. The advantage is that patients who are newly prescribed a therapy can read standardized benefit assessments beforehand. Through reliable expectation management, PROMs can thus significantly increase adherence to therapy.
How does XO Life contribute to the use of PROMs?
For this purpose, PROM questionnaires are usually used for digital patient surveys. Via a scalable web-based application such as the ImpactMonitor, patients report their subjective mental and physical experiences directly in digital form, allowing real-time conclusions to be drawn about the efficacy and safety of the therapy. This enables precise and flexible adjustments to therapy, while patients' expectations can also be managed by letting them view the treatment courses of other patients. In addition, supporting patients over the long term, even after therapy, makes it possible to review individual treatment pathways with regard to their long-term effects.
Conclusion
Given the shift from conventional medicine toward precision medicine, implementing PROMs in clinical trials and in everyday care will be essential. Precise, individually tailored therapies need precise, individual and, above all, subjective data – reported by patients themselves – on which to base them. Relevant questions (items) and their timely collection make it possible to ensure the monitoring of therapies across society at large and to continue drug monitoring.
This requires both the expertise of scientific institutes such as PROMIS and the technical know-how of XO Life.
Sources:
[1] Licqurish SM, Cook OY, Pattuwage LP, et al. Tools to facilitate communication during physician-patient consultations in cancer care: An overview of systematic reviews. CA Cancer J Clin 2019;69(6):497–520
[2] European Medicines Agency: Integrating patients’ views in clinical studies of anticancer medicines. 2016. https://www.ema.europa.eu/en/news/integrating-patients-views-clinical-studies-anticancer-medicines. Last accessed: 15.02.2021
[3] PROMIS Germany: Hintergrund. https://promis-germany.de/ueber-promis-2/hintergrund/ Last accessed: 15.02.2021
[4] Weszl, M., Rencz, F. & Brodszky, V. Is the trend of increasing use of patient-reported outcome measures in medical device studies the sign of shift towards value-based purchasing in Europe?. Eur J Health Econ 20, 133–140 (2019).
[5] U.S. Food and Drug Administration: Value and use of patient-reported outcomes (PROs) in assessing effects of medical devices. CDRH strategic priorities 2016–2017. https://www.fda.gov/media/109626/download. Last accessed: 15.02.2021
[6] Schlomm, T., Rödiger, T. & Graalmann, J. Präzisionsonkologie. Urologe 60, 3–7 (2021).
