Zofia Das-Gupta is a Standardisation Project Leader, based in the London office for the International Consortium for Health Outcomes Measurement (ICHOM). She is the co-lead of IMI’s BigData@Heart work package (WP2) on ‘disease understanding and outcomes definition’.

Q1. Could you explain in a nutshell what ICHOM does?

ICHOM is a non-profit organisation that develops standard sets for specific diseases, including chronic conditions such as kidney and cardiovascular diseases (CVD). Since 2012, we have developed 25 sets, many of which fall within the CVD portfolio, such as heart failure, coronary artery disease and hypertension. We are currently developing one on atrial fibrillation (the BigData@Heart project) and have another one in the pipeline on paediatric congenital heart disease, due to be published in 2019.

ICHOM also supports healthcare providers and organisations in implementing these sets and comparing the outcomes between multiple hospital sites around the world, through our benchmarking work.

Q2. Who do you develop these standards for?

Our mission is to support healthcare systems worldwide to measure patient reported outcomes in a standardised way, in addition to clinical indicators. Our standard sets are designed to be implemented in clinical practice by healthcare organisations and providers (e.g., hospital networks) as well as governments and payers.

All ICHOM sets are publicly available on the website, as well as the reference guidance that specifies ‘what’, ‘how’ and ‘when’ to measure the standard set.

Q3. What are Patient Reported Outcomes (PROs) and Measures (PROMs)?  Can you give us examples?

Each standard set is a mix of clinician, administrative and patient-reported outcomes (PROs) (that represent true success in managing the specified medical condition); case-mix variables (i.e., factors that will affect these outcomes, but which we cannot control as part of management of the condition); measurement tools (PROMs); data sources (be those administrative, clinician or patient-reported); and time points (specific time points for data collection).

PROs are the treatment results that matter the most to patients. They are subjective as they are reported by patients, but are outcomes to which patients attribute a great -if not the greatest- deal of importance. For example, PROs can relate to quality of life, functional status, fatigue, feelings of depression or anxiety, perceived control of the disease, patient empowerment, ability to go back to work, etc. The PROMs are the methods and tools used in that condition’s field to collect and measure patient input, preferences or insights.

Establishing a core set of outcomes that includes PROs and PROMs on a given disease is important to physicians, health providers, payers and patients because it can support shared decision making between patients and physicians, as well as improving the quality and delivery of care and facilitate for the comparison of these outcomes within and between countries.

Q4. How do you go about defining the Patient Reported Outcomes (PROs) and Measures (PROMs) which should be included in the ICHOM standard sets?

We bring together a working group of external experts, such as clinicians, clinical trials investigators, researchers, and, of course, patient advocates (which make up 25% of the group), to hear which outcomes should be included in the core set. The working group then discusses how to measure the selected outcomes and which measurement tools (PROMS) to include in the standard set. In the final stage of the process, we also consult with a wider group of 700 or 800 patients on whether the pre-selected outcomes and tools reflect what matters the most to them, to reach consensus on what should be included in the published standard set.

When choosing the best tools to include in the core set, the working group takes into consideration following criteria: 1) whether the tool really captures patient insights, 2) the psychosomatic quality of the tool (reliability, validity and responsiveness of the tool), 3) the feasibility and assessment, 4) the financing and licensing cost as well as, 5) how well established the tool is (publications, translations, etc.).  

Q5. Why is there so much interest around PROs and PROMs?

The discussion on the use and utility of these outcomes and measurements actually dates back to the 1970s but interest has increased in recent years given that more and more stakeholders are involved in healthcare decision-making.

The main drivers for PRO(M)s are: 1) the clinical data, biomarkers and genetic markers only tell part of the “story”, 2) there’s an increasing trend in paying for performance and not volume, hence the link financial resources-outcomes and, 3) there is growing variation in clinical outcomes and preferences in healthcare. In addition to this, we have much more advanced technology than ever before, which enables us to collect insights and preferences as well as process information very differently. The IMI BigData@Heart project is a perfect example of this.

All of these combined lead to better outcomes on the patient population, better value for treatment, and cost savings for healthcare systems.

Q6. How does big data contribute to PRO(M)s? Why is it relevant to patients?

Access to big data has revolutionised integrated care. It allows us to look at patient care as a full cycle – from electronic health records, to ways patient advocates communicate with providers, to wearable devices that constantly monitor and track vital bodily functions. All of these data can be harnessed to identify how to provide the best care and reach the best possible outcome in a given patient population and/or region. It’s more incisive, specialised and customised for individual patients.  

Q7. What are some of the challenges?

Firstly, we need to involve all stakeholders to ensure that as we move towards personalised care, that equal access to healthcare is maintained. Secondly, in the era of big data, we also need to ensure that all this new information works for the benefit of patients (with the right policies to ensure data protection) and not against them. And thirdly, we need to tackle the wide variation between high and low income countries where technologies are less available.

Q8. What is the aim of Working Package 2 within BigData@Heart, and what is your role?

In WP2 we are working toward better understanding of atrial fibrillation and heart failure and the outcomes that matter, which will help inform clinical practice, so that treatment is more effective, targeted and at a lower cost in the longer-term.

This is where ICHOM comes in: we bring expertise in building consensus around what outcomes matter most to patients.  Also, we are here to learn from the BigData@Heart project and reflect its findings in our standard sets, in order to further disseminate best practice and driving better outcomes for patients globally.

To support the project, we will engage with partners such as the European Heart Network to bring in patient representatives, the European Society of Cardiology to advise on clinical practice, and colleagues from WP7 on data sharing and governance and the other academic and industry partners. Collaboration is certainly key for the work that ICHOM does.