Development of Patient Reported Outcome Measures (PROM)

We have already explained the benefits, importance and relevance of so-called Patient Reported Outcomes (PROs) in a previous article. Back then, we particularly emphasized the benefits of PROs for precision medicine, for randomized controlled trials (RCTs) and for marketing approval of drugs.

Today, however, we want to look at the topic again from a different angle and answer the question of who actually creates the questionnaires, which ask for subjective patient opinions. How are PROMs checked for validity, i.e. the accuracy of the agreement of measurement result with measurement construct, and reliability, i.e. error-free measurement so that the measurements also agree with the population? Are there actually international standards for PROMs?

To answer these questions, we start at the beginning, with the manufacturers of the PROMs. In the following, we will first present the two important players in the industry.

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What is the ICHOM Initiative?

In the book “Redefining Health Care”, 2006, authors Michael E. Porter and Elisabeth O. Teisberg presented the concept of value-based healthcare. This means that healthcare should be designed to create “value” for patients. Value is defined as the end point of health divided by the costs spent on it.^[1] The value calculation also includes endpoints reported by patients, for example. Finally, healthcare should be focused on the value and composition of the endpoint categories.^[2] To date, healthcare has only considered the endpoint categories of mortality, i.e. the number of deaths due to a specific disease per 1000 people per year and morbidity, the number of patients in a population per year.^[2] Based on their own conviction that PROs in particular also have great benefits for healthcare, Porter and Teisberg, together with the Boston Consulting Group, founded ICHOM in 2012 to survey these PROs.

ICHOM — that stands for International Consortium for Health Outcome Measurements, based in Boston, USA, develops the necessary tools to measure patient reported outcomes together with patients, doctors and other industry experts. These are standardized questionnaires, so-called standard sets, which patients fill out themselves, depending on the target group and illness.^[3,4] These standard sets are then used to measure subjective endpoints of therapies on patients' perceived quality of life. The results are then comparable in the same target group or among patients with the same disease.

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What does ICHOM do?

The task of ICHOM is therefore to firstly operationalize these questionnaires, i.e. to transform certain constructs, such as physical and mental well-being, into individual questions, i.e. items, and secondly to standardize them, i.e. to make them comparable. In addition, ICHOM's tasks also include setting standard values for the individual constructs so that they can be used as reference values.^[4] The non-profit organization works with researchers from various disciplines, patients, doctors, medical professionals and other experts to use the latest medical findings to develop the sets. As a result, the standard sets are always up to date and consistently of high quality.^[5]

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conclusion

In summary, it should be said that the international comparability of patients' self-assessment among the individual disease and target groups, along the entire treatment chain, is the central task of ICHOM. ICHOM currently offers 30 evaluated and standardized free kits that cover the most important clinical patterns.^[4]

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What is the PROMIS initiative?

When the Director of the US National Institutes of Health (NHI), in 2002, commissioned a roadmap for medical research in the 21st century over many meetings, the idea of indexing patient-reported outcomes of chronic diseases was developed as a new way of medical research.^[6] This gave rise, two years later, to the group PROMIS, in order to turn exactly this idea into reality. PROMIS stands for Patient Reported Outcome Measurement Information System. Like ICHOM, the international research initiative is developing internationally standardized measurement systems for examining endpoints reported by patients.^[7] The difference is that the PROMIS questionnaires can be applied to all patients, with or without illness or ongoing therapy. This means that these endpoints of evaluation of physical, mental and social health are comparable among all people. Unlike the sets from ICHOM, the PROMIS questionnaires do not matter whether and which illness a person has in order to obtain comparable results.^[1]

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National breakdown of CELEBRITIES

The international PROMIS initiative is divided into national units. In Germany, for example, there is PROMIS Germany, based at Charité Berlin. These national units translate the individual questionnaires so that they remain internationally comparable. PROMIS Germany also validates the sets. That means PROMIS Germany ensures that the questionnaires actually measure the construct they are supposed to measure.

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Benefits and importance of the work of PROMIS and ICHOM

In general, there is a high level of scientific interest in PROMIS items. In the first half of 2021 alone, almost 100 studies were published which either used the items from PROMIS to evaluate treatment endpoints or check the PROMIS system for validity and confirm its usefulness. It is important to develop such instruments because a wide variety of measurement methods have been developed in the various medical fields in the past, including the sets from ICHOM, which measure the same characteristic but do not necessarily provide comparable results due to their disease-specific orientation. This means that physical functioning could, for example, be queried differently in the interrogative sentence for one clinical picture than for another clinical picture. On the one hand, this results in redundancies in the query and low comparability between patients with different clinical patterns. In addition, the quality of the various questionnaires was often very different. For example, it is now recommended to use disease-specific items, such as those from PROMIS, rather than disease-specific items, such as those from ICHOM. In order to support clinical diagnostics in addition to gold standard studies (RCTs), standardized, disease-unspecific measurement systems are therefore needed, which are developed by research initiatives such as the PROMIS.

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conclusion

The self-reported patient results collected by the two initiatives make it possible to make ever more individual, precise treatment controls, forecasts and screenings possible. These measured endpoints have already become increasingly relevant in medical research and treatment evaluation in recent years. For example, the assessment of the benefits of the “quality of life” endpoint has now been firmly anchored in treatment evaluations as a subjective endpoint for several years.^[4] At XO Life, we therefore also rate PROs as a useful tool to help medicine develop even more precisely, patient-oriented and value-oriented in the future.

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sources:

^[1] Terwee C.B., Zuidgeest M., Vonkeman H.E, Cella D., Haverman L., Noorda L.D. (n.d.). Common patient-reported outcomes across ICHOM standard sets — the potential contribution of PROMIS. https://osf.io/fbw2j/download

^[2] Harvard Business School. (n.d.) International Consortium for Health Outcome Measurement. https://www.isc.hbs.edu/about-michael-porter/affiliated-organizations-institutions/Pages/ichom.aspx

^[3] IQWiG. (2020) Concepts for generating care-related data and their evaluation for the purpose of evaluating the benefits of drugs in accordance with Section 35a SGB V. https://www.iqwig.de/download/a19-43_versorgungsnahe-daten-zum-zwecke-der-nutzenbewertung_rapid-report_v1-1.pdf?rev=184598

^[4] Deutsches Ärzteblatt. (2020). Rehabilitation: Measurable patient benefits as a goal. https://www.aerzteblatt.de/archiv/212785/Rehabilitation-Messbarer-Patientennutzen-als-Ziel

^[5] IHOM. (n.d.). About Us. https://www.ichom.org/mission/#founders

^[6] Cella D. et al. (2007). The Patient-Reported Outcomes Measurement Information System (PROMIS). https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2829758/

^[7] CELEBRITIES. (n.d.). background. https://promis-germany.de/ueber-promis-2/hintergrund/

^[8] CELEBRITIES. (n.d.). Publications by Year. https://www.healthmeasures.net/explore-measurement-systems/promis/measure-development-research/publications-promis