Outcomes-driven sustainable healthcare

Outcomes; the key to a Healthier Future 

Along with many stakeholders in healthcare, EFPIA and its member companies believe that an outcomes- or value-based approach to healthcare holds the key to a more sustainable, healthier future for Europe. 

European Healthcare: challenges and opportunity 

By 2025, the global population is expected to increase by 1 billion people, with half a billion more people over 50 years of age. These significant demographic changes are reflected in Europe, where life expectancy has risen by nearly a decade over the last 50 years. According to the Organisation for Economic Co-operation and Development (OECD), the percentage of the European population over 80 years of age has risen from 2% in 1980 to 5% today, and is projected to rise to 7% by 2030.  

As the population continues to age and grow, so does the prevalence of chronic diseases such as diabetes, cardio-vascular disease and cancer. Between the ages of 45 and 65, the incidence of heart disease more than doubles, and over a quarter of people aged 85 years and over lives with dementia.  

The result of these significant demographic changes is that Healthcare systems across Europe are facing unprecedented challenges.  

Despite the challenges, there are many reasons to be optimistic about a healthier future for Europe. With over 7,000 medicines in development, an exciting, new wave of innovation will play a key role in addressing the challenges faced by patients, healthcare systems, and society.  This pharmaceutical innovation is mirrored by developments in medical devices, diagnostics, imaging and data science. 

But patients can only benefit from this innovation if it is affordable now and sustainable in the future. In the context of ageing populations and chronic disease, the adoption of innovation places additional pressure on resources. How we manage the rising healthcare demand and capitalise on the new medical innovation in a sustainable way, is the foremost question for many stakeholders in European healthcare. 

Analysing our current approach to managing healthcare 

Europe provides some of the best healthcare in the world. It has long been a centre of excellence for medical research, education and clinical practice. But despite the progress in public health measures across countries in Europe, significant health inequalities persist. This is not simply a question of economics; the evidence suggests significant variation in outcomes for patients between countries but also within countries, which cannot be explained by different levels of investment in health and healthcare.  

So what lies behind the variance in patient outcomes across Europe and across national healthcare systems within Europe? 

Variations in clinical practice 

The fragmented nature of healthcare systems across Europe means we often see significantly different care pathways for patients and large variations in clinical practice. Healthcare providers may have received different training, use different treatment guidelines, adopt innovation or new clinical developments at varying rates, provide care in multiple settings and have a wide range of political, social and healthcare priorities. Some variation is natural since medical science is constantly in development, and some is desired because certain patients may require a different treatment, for example due to different tolerability. However, too often care practices that deliver a less than optimal outcome continue to be used, despite better methods being available.  

Complex care pathways 

Healthcare systems are often large and complex. A patient with multiple chronic diseases often has to deal with a whole range of providers, from primary care physicians to specialists in hospitals and physiotherapists in rehabilitation centres. Many times these different providers don’t coordinate well with each other, leaving the patient alone to navigate a complicated system. Uncoordinated care can also lead to unnecessary duplication, when the patient has to undergo the same test several times. Similarly, it can result in under-treatment, when patients “fall into the gaps” between providers, and important interventions are not undertaken.   

Data fragmentation 

The fragmentation in care is also mirrored in the data systems intended to capture a patient’s health data. While many systems record data on procedures and interventions, models for measuring and collecting data on health outcomes are under-developed. As such, the data available to scrutinise the effectiveness of different health interventions and identify best practice is limited. Systems that systematically do not track interventions and health outcomes of patients, lack the basis for a root-cause analysis that would tell them why one patient is achieving a better outcome than another. With many systems lacking such a comprehensive evidence base, they struggle to make effective decisions.  

Transaction-based incentives 

In any field of activity, targets and incentives drive behaviours. Healthcare systems have tended to focus on targets, measures and incentives based on inputs to the system rather than the outcomes they deliver. Success is often defined by transactional measures such as the number of tests completed, patient visits to a doctor or waiting times, rather than by the patient’s outcomes that result from these interventions. An inevitable consequence of this approach is that priorities and resources are focused on meeting these input targets with little reference to the impact they have on patients.  

The cumulative effect of variations in clinical practice, complex care pathways, data fragmentation and transaction-based incentives is the variation in outcomes for patients, waste and inefficiency in the system. In the context of ageing populations, increasing prevalence of chronic disease, and constrained resources, this puts the long-term sustainability of healthcare in Europe at risk. It also provides a clue as to how we can re-orientate the management of healthcare to improve outcomes for patients and make them more sustainable.  

Outcomes-driven, sustainable healthcare

What are outcomes? Outcomes are the results of treatment that patients care about most. Outcomes are not “outputs”; they are not lab results; they are not technical details. They’re real-world results, like physical functioning or level of pain. Unfortunately, today, in healthcare systems around the world, evaluation efforts take into account a number of clinical indicators, structural metrics, and even reputation – but they tend to ignore outcomes.

International Consortium for Outcomes Measurement, 2016

By re-orienting healthcare systems towards outcomes, many stakeholders, including EFPIA, believe that we can put healthcare systems in Europe on a more sustainable path. 

The principle behind outcomes-focused healthcare is that healthcare systems should focus on delivering health-outcomes, rather than on delivering interventions. Focusing on outcomes addresses the central problem that healthcare systems today do not incentivise what actually matters: better health for patients.  

Instead of paying for hospital beds, visits to the doctor, pills, screenings and surgical interventions, our focus should be paying for better health and longer lives. By determining exactly what type of intervention brings the best health outcome for each patient, and directing our resources to those specific measures, facilitates better health outcomes and quality of life for patients. A focus on delivering outcomes also results in more value for money and can contribute significantly to healthcare system sustainability by identifying and discontinuing interventions that do not deliver superior or that fail to deliver patient outcomes. By eliminating spending on ineffective interventions, a focus on outcomes can free up the resources required to address the healthcare needs of an ageing population and fund those innovations that deliver positive results for patients and value for systems. The potential for waste reduction is significant. It is estimated that 20% of healthcare spending currently is wasted on ineffective interventions.   

True, value-based healthcare, in which systems guide their decision based on the ratio of outcomes to cost, is still in its infancy. Healthcare systems across Europe are still grappling with how to make the concept a reality and what kinds of tools are needed to make it work. Like other potentially paradigm-shifting concepts, such as big data or mobile health, the initial excitement is followed by the realisation that system-wide change is immensely challenging. Nevertheless, at EFPIA we believe that the goal, a healthier, more sustainable future, can be realised step by step, in partnership with stakeholders across the system.   

This transition will take both time and investment, and most of all political will. Member States need to invest in integrated health information systems for tracking health outcomes – with disease registries and Electronic Health Records as key components – and standardise outcomes metrics that will make it possible to compare health outcomes across providers, regions and even countries. Patient involvement is key when agreeing on these outcomes metrics, but equally so is the involvement of healthcare professionals, since a continuous evolution of evidence-based clinical practice is at the core of an outcomes-focused approach to healthcare.  

The good news is that we don’t have to wait for the perfect system to be in place: change can be implemented step-by-step. Even though reduced waste and better value for money will be one of the rewards, the main driver for change must be better health outcomes and putting the patient at the centre of healthcare management. The key is to learn from each other and spread the use of models that have been proven to work.  

What are the barriers to moving towards an outcomes-focused healthcare system?  

Better outcomes for patients and more sustainable healthcare systems are common goals for all stakeholders in the European healthcare landscape. Outcomes-focused approaches have been around for over a decade, but system-wide change is problematic and notoriously difficult. This section explores some of the barriers to a system-wide transition to a more value- and outcomes-focused approach and can be grouped into technical, structural, financial and political aspects.  

Technical barriers 

Outcomes-focused healthcare relies on delivering value, measured as health outcomes divided by costs. It is based on the ability to capture, analyse and utilise outcomes (and financial) data, with standardised definitions of outcomes at the core. 

Today, the measurement of outcomes is not common practice. Many providers and healthcare systems do not know which outcomes they achieve in which disease area. Those healthcare systems and providers within one system that are measuring outcomes, often use different outcomes measures, making comparisons and evidence-based service design difficult. At a more basic level, the availability and usability of IT systems in clinical settings varies significantly between providers. For clinicians operating under pressure, the capture of the necessary outcomes data can quickly become a burden. Even where outcomes data is captured effectively, the fragmented nature of IT infrastructure and lack of interoperability between IT systems, mean that sharing data across providers and between healthcare services is often not possible. Without a comprehensive evidence base, the analysis of clinical data to identify where best outcomes are being achieved and why, is burdensome and costly at best, and technically-unfeasible at worst.  

A consequence of these technical issues and a further barrier to an outcomes-focused approach is that outcomes-based reimbursement models that can help drive a more value-based approach, are often considered too burdensome to administer. A lack of readily available data and analytical capability needed to support an outcomes-focused contract adds to the investment cost and uncertainty for entering into such arrangements. Providers will revert to a more volume-based approach if they do not have the systems and administrative capacity in place to measure and, ultimately, reward outcomes. 

Structural barriers 

The most significant structural barrier is the fragmentation of healthcare systems. Individual organisations within a healthcare system often have different definitions of outcomes, different incentives and targets, and alternative preferred care pathways. They also collect different data within their own IT systems. This is a major barrier to the development of outcomes-focusedhealthcare at national or pan-European level.  

 
Financial barriers 

Instead of rewarding the long-term improvement of a patient’s health, fiscal incentives tend to reward process-related measures, such as adherence to clinical guidelines, the number of times a doctor talks to his or her patients about prevention and healthy lifestyles, the number of patients of a certain category that are referred to a specialist or prescribed a certain medication, or something else related to process rather than actual outcome. Where overall resources are under pressure, it is natural that fiscal incentives will drive action. 

There are additional barriers in terms of how healthcare systems are financed. Budgets are often separate for different types of interventions. For example, there are separate budgets for hospital treatment, medical devices, pharmaceuticals, and outpatient care. This is why downstream savings from an intervention are often realised in a different part of the healthcare budget to where the initial investment in the new treatment was made. Sometimes, these savings even accrue outside healthcare budgets, such as in social care budgets, for example when medical interventions allow a patient to continue to work and contribute to social welfare systems. Similarly, the benefits from a healthcare intervention may be realised over the lifetime of the patient, whereas the costs of the intervention may already be incurred in the year the treatment is administered. Introducing flexible approaches to financing healthcare, such as outcomes-based reimbursement models, overcoming budget siloes and developing systems to allocate savings across financial years, are key milestones in transitioning towards a more outcomes-focused approach to healthcare. 

Political barriers 

System-wide, transformational change is challenging; it requires strong political commitment over a number of years to make it happen. Implementing some outcome-focused decisions such as closing hospitals or transferring care to the community can invoke strong reactions from local stakeholders who are attached to particular local services. The concept of outcomes-focused healthcare is intellectually attractive but its implementation can include some difficult, sometimes politically unpopular decisions.  

In the highly emotive and politicised healthcare sector, policy makers need a strong evidence base, case studies of successful implementation, as well as national and international support to facilitate the change to an outcomes and value-based healthcare system at pace and scale. 

Healthcare Data: The key to outcomes-focused healthcare 

As healthcare begins to embrace the digital revolution, the potential of data to change the way we deliver healthcare, improve patient outcomes and shape future research is an important new frontier.  The availability of healthcare data is increasing exponentially, providing an opportunity to explore new ways to capture and analyse healthcare data and to accelerate the transition to an outcomes-focused approach.  

From a pharmaceutical perspective, for several decades, the clinical trial has been the gold standard of measuring safety and efficacy. In the future, clinical trials will continue to play an important role in generating data and evidence to shape healthcare. Yet with the advent of the digital revolution, encompassing electronic health records, disease registries, patient reported outcome measures, mobile health apps and advances in data analytics, our healthcare eco-systems are generating unprecedented amounts of “real world data” (RWD) that can complement clinical trial data in important ways.  

Real world data is all health data that is generated and collected in real clinical practice right across our healthcare systems. Combining pre-clinical and clinical data generated by industry with real world data collected in clinical settings and beyond, we can drive medical innovation and improve patient care. With more and better data, clinicians can better target interventions to the patients where they will have the most impact, improving patient outcomes but also increasing efficiency. Data collection and analysis can help us better understand the side effects of medicines and improve patient safety. Genomic science is facilitating a revolution in personalised medicines and, crucially, all of this data can inform and shape tomorrow’s breakthrough innovations.   

Maximising the potential of all this data for patients, though, requires a new level of collaboration to address a number of challenges.   

In reality, much of our RWD is scattered across the healthcare system without any possibility of bringing this data together. They are noted down in individual patient’s records, recorded in registries kept by small groups of specialists for tracking a specific disease in a selected number of patients, or collected for reimbursement purposes for a specific product, and then discarded. Often, data privacy regulations limit the ability to re-use existing data, despite its potential to deliver important insight into clinical practice and patient outcomes.  

Data should be generated according to the same standards in order to be comparable. E-health systems should be made compatible between hospitals or countries, with a single, electronic health record for individual patients, with all health data collected in one place. There should be a means of linking all data collected for a variety of purposes. Regulation needs to facilitate data being transferred between systems and used for research or quality improvement.   

The situation, though, is changing rapidly. Considerable efforts are now being made both by public and private stakeholders to capture, consolidate and most importantly utilise this real-world data to benefit patients, science and our healthcare systems. Trust is critical in ensuring a new health data ecosystem can thrive. It is vital to be cognisant of the sensitive nature of healthcare data. Patients need clarity on how and why their data will be used. Systems need high standards of technical data protection, recognising that, as we move into a world of many different data sources and opportunities, these standards will need to evolve.  

With more information about how different interventions actually compare in terms of health outcomes for patients, healthcare managers and policymakers will be able to take much more informed decisions on implementing clinical practice and resource allocation, creating not only better health outcomes for patients but also getting more value from every euro spent on healthcare. Progress will require dialogue, collaboration and investment, but as the key to an outcomes-focused, more sustainable future driving the healthcare data agenda, it is a critical success factor. 

Moving along the outcomes-focused spectrum 

No two healthcare systems in Europe are at the same point on the journey to a more outcomes-focused approach. Some have advanced examples of good practice, others are beginning to look at outcomes-based reimbursement models, others simply do not have the infrastructure in place to base clinical decision-making and service design on outcome measures. However, at whatever point a healthcare system is on the journey towards an outcomes-focused approach, there are a number of actions that can be undertaken to support change. 

Understand the healthcare challenges 

Understanding the epidemiological, structural, technical, financial and political challenges can help inform strategies to move to outcomes-focused model of healthcare

System Readiness Assessment 

Using structured analysis of stakeholder awareness, data infrastructure, proof of concepts, and enablers in a local system to obtain insight into areas to develop and invest

Define health outcomes measures 

Defining standardised sets of health outcomes measures for all diseases and conditions, together with patients, that will allow for systematic measurement and comparisons across providers and countries  

Analyse variation 

Data analytics provides the key to identifying variances in care and their impact on outcomes, to detect sources of waste and inefficiencies in the system 

Develop integrated health information systems  

Tools such as electronic health records, disease registries and user-friendly data capture systems all contribute to developing an outcomes-focused system 

Build a health data eco-system  

To spark, develop and deliver change, data needs to be of high quality and shared across the healthcare system for quality improvement and research 

Identify best practice  

Standardised outcomes measures, quality and transparent outcomes data, coupled with the use of data analytics will facilitate the identification of best practice for replication across health systems 

Promote proof of concepts 

Successful pilots of improving outcomes in a specific patient population build trust in the merits of an outcomes-focused approach and provide important clues into the practicalities of implementing outcomes-focused healthcare 

Feedback and learn 

As clinical practice and service delivery changes, real world evidence and data analytics provide a mechanism for real-time learning and continuous development 

Remove budget siloes and reward quality of care 

Establishing flexible and holistic finance systems that promote care integration, and payment models that reward good health outcomes for patients can help facilitate change 

Standardising definitions of outcomes, agreeing measures and putting in place the infrastructure to capture, measure and analyse healthcare data to support outcomes-based decision making is a long and complex process. To facilitate change requires dialogue, partnership and collaboration from across the healthcare spectrum. Often it will require attitudinal change at an individual and institutional level. System-wide transformation of healthcare is a bold and ambitious goal, but central to a more sustainable and Healthier Future. 

Moving the debate forward

There are many stakeholders and thought leaders across European healthcare, engaged in the outcomes agenda.  EFPIA is keen to facilitate and support this dialogue to ensure the long-term sustainability of healthcare for the citizens of Europe.  One practical example of industry supporting and enabling action on the outcomes agenda is Big Data for Better Outcomes (BD4BO).

Big Data for Better Outcomes

BD4BO is an Innovative Medicines Initiative programme designed to catalyse and support the evolution towards outcomes-focused and sustainable healthcare systems in Europe, exploiting the opportunities offered by big and deep data sources.

The umbrella programme provides a platform and resources for defining and developing enablers of the outcomes-focused evolution such as:

  • Definition of outcomes metrics;
  • Protocols, processes and tools to access high quality data;
  • Methodologies and analytics to drive improvements; and
  • Digital and other solutions that increase patient engagement

In the BD4BO programme, pharma and companies from other sectors work together with payers, patients, physicians, regulators and other stakeholders to identify solutions.  More information on BD4BO can be accessed here