XO Life is using Patient-Reported-Outcome-Measures

Patient-Reported-Outcome-Measures in health-care

15.02.2021

In an increasingly efficient world, a rethinking of the healthcare system must take place sooner or later. Due to the multitude of different patient groups and their sensitivities, an orientation towards the individual patient is indispensable. In this context, data collected by patients, so-called Patient Reported Outcome Measures (PROMs), will play an increasingly central role.

Evaluating and incorporating PROMs into healthcare enables the following benefits:

  1. Medical professionals can recognize 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 (so-called post-marketing studies)
  3. Precise drug therapy monitoring in a real-world care setting for enabling precision medicine

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

What are PROs / PROMs ?

According to the European Medicine Agency (EMA), Patient Reported Outcomes (PROs) is data collected directly – by the patient [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 gets evaluated.

Possible endpoints of the survey include information on:

  • Impact on quality of life
  • Symptoms during therapy
  • Adherence to therapy
  • Satisfaction with the course of therapy
This data is measured using standardized, scientifically developed methods known as Patient Reported Outcome Measures.
 
The majority of PROMs are questionnaires that must fulfill the following quality characteristics [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 also internally consistent and query those details that are to be queried (reliability).

3. Flexibility

The questions on certain characteristics (items), such as subjective perception of the therapy, can be composed freely according to the group to be examined. The questionnaire maintains full reliability and validity.

4. Integrativity

People from all groups of the population can fill out these questionnaires. Thus, consideration is given to reading ability, language and physical feasibility.

In Germany, there are currently already leading research institutes, such as the PROMIS Institute of the Charité Berlin, which develops standardized measurement instruments for patient-reported characteristics and makes them applicable in real care settings.

Importance of PROMs in clinical pre-market studies

PROMs make clinical studies more precise – individual endpoints, such as impact on quality of life, can be collected more accurately than ever before and thus form a good basis for future therapy recommendations. A self-learning system is emerging which, through more precise evidence, enables more precise diagnostics and subsequently precise treatment recommendations [6]. Thus, a significant increase in PROM items in clinical trials has been observed in recent years. In 2018, the FDA reported that in 6 years, an increase of >500% was observed in the number of pre-authorization clinical trials in the United States that included PROMs [5].

PROMs after market approval

In addition to their importance for the market approval of drugs, PROMs also help to generate relevant outcomes in the real healthcare setting. Data on subjective perception of the therapy is collected even after approval. This is often a mandatory component of new drugs that may have received conditional market approval. In addition to real-time PROM data collection, it has the advantage that patients can obtain transparent information on the therapy progress of similar patients. Thus, PROMs can significantly increase adherence to therapy through reliable expectation management.

What does XO Life contribute to the use of PROMs ? 

For this purpose, PROM questionnaires are mostly used for digital patient surveys. Using a web-based scalable application, such as ImpactMonitor, patients provide their subjective psychological and physical perceptions directly in digital form, which allow conclusions to be drawn about the effectiveness and safety of the therapy in real time. This allows precise and flexible therapy adjustments; patients can also be managed in their expectations by viewing the therapy progress of other patients. In addition, individual therapy paths can be reviewed with regard to their long-term effects by monitoring patients even after therapy.

Conclusion

With regard to the developments of regular medicine towards precision medicine, an implementation of PROMs in clinical studies as well as in everyday care will be imperative. Precise, individually adapted therapies need precise, individual and above all subjective data – which is reported from the patient – on which they can be based. With relevant items and a real-time collection of these, such a monitoring of therapies  in real-care settings can be continued.

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

Sources

[1] Licqurish SM, Cook OY, Pattuwage LP, et al. Tools to facilitate communication during phy- sician-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).

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