Health Outcomes Observatories – empower patients with tools to measure their outcomes in a standardised manner creating transparency of health outcome - 2nd stage

Specific challenges to be addressed
Patient outcomes and overall care could be improved through systematic capture of the patient voice and perspective:

Current conceptualisations and measures of disease and clinically relevant disease outcomes have generally been developed from the perspective of the clinician and often fail to completely capture the totality of the disability, the symptoms of the disease and the impact on a patient's health-related quality of life (HRQOL) from the patient’s perspective. Important patient-to-patient variations in disease presentation and symptomology may also be lost in the effort to develop a generalisable framework for the disease.

It is important to complement existing clinical outcome measurements with patient-generated measures of disease and HRQOL to ensure that the patient perspective of disease and the impact of healthcare interventions are more completely captured and that disease heterogeneity is better understood.

In order to achieve this, it is essential to provide patients with tools that have the ability to better capture the entirety of the impact of a disease and treatments (e.g. signs, symptoms, tolerability), allowing them to document their disease more completely and in a structured manner. To be effective, these tools should be built on the basis of accepted standards, developed in partnership with all relevant stakeholders and accepted and integrated into the existing healthcare ecosystem.

A reward system that truly focuses on value requires measurement and transparency of patient outcomes:

Disease registries have already been established in a wide range of diseases. However, these registries tend to measure a non-standardised set of outcomes, are rarely interoperable, focus on clinical measurements, and have varying terms and conditions for access to the data captured. As a result, they often fall short from providing sufficient transparency of patient outcomes in specific diseases to inform scientific and policy decisions.

At the level of the individual patient, the data thereby generated, once structured and subjected to a degree of standardisation, will enable patients to have more productive interactions with their healthcare provider. At the level of the healthcare system, this data will allow a systematic measurement of health outcomes and the possibility to set up a reward system based on value – which can be defined as the level of health outcomes achieved for a given cost.

There is a lack of models for capturing and managing patient-reported health data in an ethical and sustainable way:

Structured health data is invaluable for all stakeholders, from the individual patient, health care professionals (HCPs), the life science industry, policy makers to the patient advocacy groups. There have been a few successful examples of approaches to integrate patient-reported health data into clinical care. In an era of greater focus on the patient, it is thus critical for a society that patient-reported health data is captured and managed in an ethical manner ensuring broad and appropriate access while safeguarding patients’ privacy and building high levels of trust.

The goals of this topic are:

1. Identify appropriate standards for capturing the patient perspective when measuring health outcomes and obtain the support of these standards by the relevant stakeholders. Where appropriate the partners will give preference to standards already being developed (e.g. International Consortium for Health Outcomes Measurement - ICHOM) and will follow the Observational Medical Outcomes

Partnership Common Data Model (OMOP CDM) developed through Observational Health Data Sciences and Informatics (OHDSI)

2. Implement appropriate technology solutions (including adopting existing technology where appropriate) that would allow individual patients to record and measure their outcomes according to these standards and use the information for a more structured dialogue with their HCPs

3. Establish the appropriate platform to collect, process and manage data in the best interest of patients, patient organisations, health authorities, healthcare professionals, the research community and health care payers, and in compliance with General Data Protection Regulation (GDPR) and other relevant rules and regulations

4. Create a sustainable and ethical model for the continuous collection of data and an appropriate model for providing access to the anonymised or aggregated data to researchers with a legitimate interest in analysing them.

These goals can be achieved through the creation of a consortium whose mission will be to establish Health Outcomes Observatories in three selected disease areas, collecting health data in (at least) three different European countries for each disease area.

The Observatories should be designed according to the following principles:

  • Full integration within the respective countries’ health care systems;
  • Consistency in design across Observatories;
  • A sustainable model for the Observatories;
  • Robust patient consent and engagement;
  • Standardisation and interoperability across countries;

The disease areas selected are:

  • Diabetes Type 1 and Type 2;
  • Inflammatory Bowel Disease (IBD);
  • Cancer (side effects of chemotherapy and immuno-oncology).

Criteria considered for this selection were: (a) their prevalence in the European population; (b) their chronic and progressive nature; (c) their significant impact in patients’ quality of life; (d) their compatibility with patients’ digital literacy; (e) the patients’ sufficient autonomy and motivation to become engaged in selfmanagement of their disease; and (f) the investment in novel medicines and disease management tools for these diseases by EFPIA members and their Associated Partners.

2nd stage Deadline: 26 March 2020

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