Patient-reported outcomes in health care decision making | Takeda Stories
Informing health care decision making with patient-reported outcomes
A unique, publicly available dashboard demonstrates patients’ desire to ‘use their voice’
Health care providers, regulatory agencies, payors, and other decision makers depend on comprehensive information to make informed patient care decisions. They rely on rigorous, standardized data from clinical trials, capturing clinical as well as patient-reported outcomes.i But what about the “real world,” beyond the controlled (and costly) environment of clinical trials? How can we capture patient outcomes in real-world settings to inform decision making? Could we empower patients with tools to share their voices within the context of the care they receive? After all, some effects and outcomes are known only to the patient.
While there's growing recognition that the patient voice offers valuable insights, ii,iii health care systems often face challenges in effectively capturing and integrating this data. Patient-reported outcomes (PROs) are inconsistently or infrequently collected, iv,v,vi and the resulting data are rarely shared with key stakeholders – health care providers, regulatory authorities, policy makers, researchers, patients and pharmaceutical manufacturers – who could learn from these insights. This is where standardized PROs, along with the systematic collection and analysis of the data they yield, come in.
A new standardized collection method and publicly available dashboard has been created to address this challenge. More than 1,600 patients have already contributed their personal data to this novel, anonymized digital dashboard – a strong indicator that patients are willing to use digital tools to capture their outcomes when their privacy is protected, and a proof-of-concept for sharing real-world data.
What it is and why it’s unique
It all began with Takeda Chief Executive Officer Christophe Weber’s passion for the promise of digital tools to capture PROs.
“We need a way to report, collect and analyze standardized health data so that patient outcomes become the basis for decision making,” he said. “Think treatment coverage and reimbursement, prescribing, allocation of research funding, and identifying potential gaps in treatment– all decisions that would be better rooted in data and aligned with the goal of driving better health for patients. This is at the heart of value-based healthcare.”
In 2020, Takeda supported the creation of the non-profit Health Outcomes Observatory (H2O). H2O unites public and private sectors to create a groundbreaking, standardized data governance and infrastructure system across Europe. This initiative gives patients digital tools to measure their outcomes in a standardized manner and helps foster improved communication between patients and their health care providers. Patients keep full control of their data, with the ability to choose whether to share it with their clinicians or for research purposes, or both. By sharing their data for research, patients can help generate real-world evidence. This evidence can enable researchers, regulators and other healthcare decision makers to make more informed choices that may improve patient outcomes.
The online dashboard, known as the H2O Insights Centre, displays the demographics and disease states of patients who have shared their outcomes. Users can explore the dashboard to assess whether the datasets may support answering their questions about disease impact or therapeutic efficacy. If the dataset is relevant, users can contact H2O to request access to the PROs. This type of evidence has the potential to inform health care decision making by various critical stakeholders, including those whose perspectives are highlighted below.
Dashboard facts:
The H2O Insights Centre currently displays information about digitally collected and aggregated PRO and clinical data.
- Diabetes (Types I & II)
- Inflammatory Bowel Disease
- Breast Cancer
- Lung Cancer
- Austria
- Germany
- Netherlands
- Spain
- Karolinska University Hospital
- Medical University of Vienna
- Charité – University Medicine Berlin
- Erasmus University Medical Center Rotterdam
- Catalan Institute of Health – Vall d’Hebron University Hospital
- European Patients’ Forum
- Takeda Pharmaceuticals
The patient perspective – European Patient Forum
We’ve had several patient advisory groups validate that patients will share their data with their health care provider to enable shared decision making. In addition, patients (have indicated they) are willing to share their data in an anonymized way when they know this could help advance research and knowledge. However, they want to remain in control of their data and have the possibility to opt out from a research project if they so choose. And H2O being a non-profit organization with patient organizations included in leadership also plays a significant role. Patients are hesitant to share data for pure commercial benefit. So a robust multi-stakeholder governance is really important for building trust.
Valentina Strammiello, director of strategic initiatives, European Patients’ Forum*
The academic perspective – Karolinska University Hospital
To my knowledge, there is no other (free, publicly available) resource like the H2O Insights Centre. This dashboard offers an overview of data related to the patient’s health and care outcomes by showing aggregated statistics. This gives researchers an understanding of what types of patient-level data could potentially be available to them. Let’s say I’m doing research in IBD and I would like outcomes information on, for example, 600 patients from Austria and 300 patients from Spain. I can see in the dashboard whether these patient profiles are available. This can be a starting point for the researcher to initiate dialogue with H2O and explore access to the patient-level data. The cross-institution, cross-border data in the dashboard opens up new possibilities for us all to learn from each other, make meaningful comparisons and answer questions such as, ‘what are best practices that we can learn from and leverage?’.
Alexander Chakhunashvili, Ph.D., data analyst, Karolinska University Hospital*
The regulatory agency perspective - Healthcare Technology Assessment (HTA)
There are lots of new treatments coming to the market. As an HTA agency, we need to find ways to prioritize and make sure the right treatment gets to the right patient. The evidence created through clinical trials is important but does not give us the whole picture. We need to understand how new treatments compare to the standard of care in the real world and we need evidence on patient outcomes. Traditional evidence generation strategies give us a lot of useful information but often miss a critical piece: The outcome of the treatment on the patient’s life in the real world.
Jørgen Schøler Kristensen, former chairman, Danish Medicines Council*
The industry perspective – Takeda
Takeda’s support for this project is an extension of our 240-year legacy of innovation in service of patients. The H2O Insights Centre demonstrates how, by focusing on the patients’ needs and their desire to engage in shared decision making with their health care providers, we can generate valuable evidence for payers and decision makers that may ultimately improve patient access to therapies.
Julie Spiesser, global head of Pricing & Access, Global Product & Launch Strategy, Takeda
* Quotes originally appeared on The impact of the H2O Insight Centre – How Patients, Academics and Regulators see it - Health Outcomes Observatory
This image represents dashboard data as of May 7, 2025. For real-time data updates, visit the H2O Insights Centre
This is an example set ** of some of the standardized questions designed to capture patient-reported outcomes within the H2O initiative. Patients answer these questions at agreed-upon intervals via digital tools made available by their providers. The H2O Insights Centre dashboard represents the demographics and disease states of the patients who have shared their outcomes.
**Courtesy of H2O
General health
In general, would you say your health is:
5 = Excellent
4 = Very good
3 = Good
2 = Fair
1 = Poor
Quality of life
In general, would you say your quality of life is:
5 = Excellent
4 = Very good
3 = Good
2 = Fair
1 = Poor
Physical health
In general, how would you rate your physical health?
5 = Excellent
4 = Very good
3 = Good
2 = Fair
1 = Poor
Psychological health
In general, how would you rate your mental health, including your mood and your ability to think?
5 = Excellent
4 = Very good
3 = Good
2 = Fair
1 = Poor
Social participation
In general, how would you rate your satisfaction with your social activities and relationships?
5 = Excellent
4 = Very good
3 = Good
2 = Fair
1 = Poor
Social participation
In general, please rate how well you carry out your usual social activities and roles. (This includes activities at home, at work and in your community, and responsibilities as a parent, child, spouse, employee, friend, etc.)
5 = Excellent
4 = Very good
3 = Good
2 = Fair
1 = Poor
Physical function
To what extent are you able to carry out your everyday physical activities such as walking, climbing stairs, carrying groceries, or moving a chair?
5 = Completely
4 = Mostly
3 = Moderately
2 = A little
1 = Not at all
Psychological health
In the past 7 days, how often have you been bothered by emotional problems such as feeling anxious, depressed or irritable?
5 = Never
4 = Rarely
3 = Sometimes
2 = Often
1 = Always
Fatigue
In the past 7 days, how would you rate your fatigue on average?
5 = None
4 = Mild
3 = Moderate
2 = Severe
1 = Very Severe
Pain
In the past 7 days, how would you rate your pain on average?
Scale from 0 – no pain to 10 – worst pain imaginable
References
i. Gnanasakthy A, Russo J, Gnanasakthy K, Harris N, Castro C. A review of patient-reported outcome assessments in registration trials of FDA-approved new oncology drugs (2014-2018). Contemp Clin Trials. 2022 Sep;120:106860. doi: 10.1016/j.cct.2022.106860. Epub 2022 Jul 25. PMID: 35901962. ii. Walsh A, Matini L, Hinds C, Sexton V, Brain O, Keshav S, Geddes J, Goodwin G, Collins G, Travis S, Peters M. Real-time data monitoring for ulcerative colitis: patient perception and qualitative analysis. Intest Res. 2019 Jul;17(3):365-374. doi: 10.5217/ir.2018.00173. Epub 2019 May 31. PMID: 31146510; PMCID: PMC6667366. iii. Basch E, Deal AM, Dueck AC, et al. Overall Survival Results of a Trial Assessing Patient-Reported Outcomes for Symptom Monitoring During Routine Cancer Treatment. JAMA. 2017;318(2):197–198. doi:10.1001/jama.2017.7156 iv. Benson T. Why it is hard to use PROMs and PREMs in routine health and care. BMJ Open Qual. 2023 Dec 21;12(4):e002516. doi: 10.1136/bmjoq-2023-002516. PMID: 38135303; PMCID: PMC10749067. v. Nguyen H, Butow P, Dhillon H, Sundaresan P. A review of the barriers to using Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs) in routine cancer care. J Med Radiat Sci. 2021 Jun;68(2):186-195. doi: 10.1002/jmrs.421. Epub 2020 Aug 19. PMID: 32815314; PMCID: PMC8168064. vi. Robert Bone et al. Frequency of standardized patient-reported outcome measures in routine clinical care of patients with cancer.. JCO Oncol Pract 19, 342-342(2023). DOI:10.1200/OP.2023.19.11_suppl.342
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