Patient-reported outcome measures in healthcare

In an increasingly efficient world, a rethink in healthcare must take place sooner or later. Due to the large number of different patient groups and their sensitivities, an orientation towards the individual patient is essential. Data collected from patients, so-called Patient Reported Outcome Measures (PROMs), will play an increasingly central role.

The evaluation and integration of PROMs into healthcare provides the following benefits:

1. Healthcare professionals can identify symptoms more quickly and take targeted initiative ^[1]
2. Additional evidence for market-relevant approval studies (so-called pre-market case studies) and after market approval in so-called post-marketing studies
3. Precise drug therapy monitoring in a real care setting for precision medicine

But what is PRO (Ms) all about and how do they contribute to precision medicine?

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What are PROs/PROMs?

According to the European Medicine Agency (EMA), Patient Reported Outcomes (PROs) are data that is collected directly — by the patient himself ^[2]. In addition to regular medical questions (clinical, genetic and medical questions), subjective data on the personal perception of the disease and the associated therapy are evaluated.

Possible endpoints of the survey include, for example, information about:

• Impact on quality of life
• Symptoms during therapy
• Adherence to therapy
• Satisfaction with the course of treatment

This data is measured using standardized, scientifically developed methods, the so-called Patient Reported Outcome Measures.

PROs are mostly questionnaires that must meet the following quality criteria^[3]:

1. Comparability

The questionnaires ensure that both a comparison between different patients (interpersonal) and a comparison of the same patients over time (intrapersonal) is possible. In addition, different disease groups and cultures can be compared.

2. Reliability and Validity

The applicability of the collected data to the population (validity) is ensured by strict standardized methods. As a result, the results of the study are consistent within themselves and also query the details that are to be queried (reliability).

3. Flexibility

The questions about specific characteristics (items), such as the subjective perception of the therapy, can be freely compiled according to the group to be examined. This gives the questionnaire full reliability and validity.

4. Integrity

People from all segments and groups of the population can complete these questionnaires. Consideration is therefore given to reading ability, language and physical feasibility.

There are currently leading research institutes in Germany, such as the PROMIS Institute at Charité, which develops standardized measurement instruments for patient-reported characteristics and makes them usable in a real care setting.

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Why are PROMs becoming increasingly important before market approval?

PROMs further clarify clinical studies — individual endpoints, such as effects on quality of life, can be determined more precisely than ever and thus form a good basis for future treatment recommendations. The result is a self-learning system which, through more precise evidence, more precise diagnostics and then precise treatment recommendations, enables ^[6]. For example, there has been a significant increase in the number of PROM items in clinical trials in recent years. In 2018, the FDA reported that an increase of > 500% was observed in the number of pre-authorization clinical trials in the USA that contain PROMs within 6 years ^[5].

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PROMs after market approval

In addition to their importance for marketing approval of drugs, PROMs also help to generate relevant outcomes in real care settings. Data on the personal perception of the therapy is collected and collected even after approval. This is often a mandatory component of new drugs that may have received conditional market approval. This has the advantage that patients who are newly prescribed a therapy can read standardized benefit assessments beforehand. PROMs can thus significantly increase adherence to therapy through reliable expectation management.

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How does XO Life contribute to the use of PROMs?

For this purpose, PROM questionnaires are usually used for digital patient surveys. Through a web-based scalable application, such as ImpactMonitor, patients report their subjective psychological and physical feelings directly digitally, which allow conclusions to be drawn about the effectiveness and safety of the therapy in real time. This allows precise and flexible treatment adjustments to be made; but patients can also be managed in terms of their expectations by viewing the treatment processes of other patients. In addition, individual treatment paths can be reviewed with regard to their long-term consequences through long-term support of patients even after treatment.

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conclusion

With regard to the developments of regular medicine towards precision medicine, it will be absolutely necessary to implement PROMs in clinical studies and in everyday care. Precise, individually tailored therapies require precise, individual and, above all, subjective data — reported by the patient — on which they can rely. Through relevant questions (items) and timely recording of these, the monitoring of therapies in the wider society can be ensured. Drug monitoring will be continued.

On the one hand, this requires the expertise of scientific institutes such as PROMIS and the technical know-how of XO Life.

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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: Background. 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. Precision oncology. Urologist 60, 3—7 (2021).