Identifying disease-specific patient and societal needs to foster needs-driven healthcare and innovation policies in the EU

Eurohealth  —  Vol.29  |  No.3  |  2023 40 A Europe that cares, prepares, protects IDENTIFYING DISEASE-SPECIFIC PATIENT AND SOCIETAL NEEDS TO FOSTER NEEDS-DRIVEN HEALTHCARE AND INNOVATION POLICIES IN THE EU By: Irina Cleemput, Charline Maertens de Noordhout and Wim Goettsch Summary: Healthcare policy decisions in the European Union are  largely supply-driven. To move towards needs-driven healthcare  innovation and policy, patient and societal needs should be defined  upfront and subsequently guide decisions of researchers, research  funders, regulators and health technology assessment bodies.  Evidence-based needs-driven policy assumes the availability of  scientific evidence on disease-specific needs in a large range of  diseases. Collaborative efforts at EU level are necessary to establish  a needs assessment and appraisal framework, gather evidence, and  incorporate these into assessment and decision-making processes.  The EU pharmaceutical regulation, EU HTA regulation and EU research  funding programmes can stimulate this effort. Keywords: Needs Assessment, Biomedical Research, Pricing and Reimbursement, Regulatory Approval Irina Cleemput is Scientific  Programme Director, Charline Maertens de Noordhout is Expert  in Health Services Research,  Belgian Health Care Knowledge  Centre (KCE), Brussels, Belgium;  Wim Goettsch is Professor HTA  of Pharmaceuticals, World Health  Organization (WHO) Collaborating  Centre for Pharmaceutical  Policy and Regulation, Division  of Pharmaceutical Policy and  Regulation, Utrecht University,  Utrecht, The Netherlands and  Special Advisor HTA, National  Health Care Institute, Diemen,  The Netherlands.   Email: Irina.Cleemput@kce.fgov.be Introduction In a patient-centred healthcare system, research and development (R&D) should focus on addressing high-priority patient and societal needs. However, private investments in R&D often fail to target the highest priority needs due to the absence of commercial interest. 1 – 3 Current intellectual property frameworks for innovation and supply of health technologies are failing to stimulate and deliver solutions for priority healthcare needs, such as prevention of multimorbidity, mental health, antimicrobial resistance and treatment of neglected diseases. 4 Industry tends to prioritise innovation that promises significant long-term revenue. In Germany, for example, an empirical study found that pharmaceutical innovation is more closely related to market size than disease burden. 3 A Dutch study found that for about 30% out of 33 selected high need conditions, there was no or little pharmaceutical development (including, among others, stroke, hearing disorders, Eurohealth  —  Vol.29  |  No.3  |  2023 41A Europe that cares, prepares, protects oesophageal cancer, brain cancer, contact eczema, arthrosis). 5 Yet, for other conditions, such as diabetes mellitus, HIV and breast cancer, continuous development has been observed in the pharmaceutical sector over the past 15 years. 5 As a consequence, important amounts of public resources are being invested to bridge these gaps, through research funding, pharmaceutical incentives, early (conditional) access and temporary reimbursement schemes. ‘‘ industry tends to prioritise innovation that promises significant long- term revenue However, there is little discussion about priorities across diseases between different types of decision-makers. Decisions are often made following ad hoc requests for one specific product or related to one specific disease. For example, incentives for R&D are provided following a request of developers (e.g., a pharmaceutical company), rather than granted on the basis of previously defined and prioritised patient and societal needs. Moreover, the focus of decision-makers is commonly on the assessment of single technologies instead of having a broader agenda addressing the health needs of the whole society. This article describes a possible approach to move towards needs-driven healthcare innovation and policy, including a framework for identifying disease-specific societal and patient needs, and proposes ways to implement these principles at the EU and national level. Steering innovation towards areas with high needs Moving towards needs-driven healthcare policy requires the consideration of health- related patient and societal needs * before, during and after the development of any new healthcare intervention. Therefore, the *  Health-related needs are the specific needs related to  a particular health condition, which encompasses the needs  of individuals affected by the condition and possible broader  societal needs that involve the consequences of the condition  which impact segments of society not directly afflicted by  the condition (externalities). patient and societal health-related needs under the current standard of care must be identified in an evidence-based manner. Based on this scientific evidence for a large range of diseases, priorities for innovation can be identified and be used in research and innovation policy decisions. Once interventions for high need diseases are being developed, regulatory and reimbursement assessment processes should take the information about the basic underlying needs that the interventions aim to address into account to allow for a suitable judgment about their value. This life-cycle approach to the use of patient and societal needs evidence is at the core of the statement of the Global Policy Forum of HTA International (HTAi – the global health technology assessment society) saying that the value of a technology at various points in time should drive the focus of resources to areas where they are most needed. 7 An example of a national initiative in this direction includes the Future Affordable and Sustainable Therapies (FAST) initiative in the Netherlands. FAST aims to ensure the affordability of therapeutic innovations targeting healthcare needs of chronically ill patients by allocating public resources for innovation in a less fragmented manner. 6 Figure 1: Conceptual framework for a needs-based decision approach  1. Patient and societal needs framework: • criteria to be assessed • type of evidence • evidence sources Scientific work Tool Evidence-based decision making 2. Assessment Evidence database of health- related needs criteriainput inform input inform 3. Appraisal Ranking or classification of health-related needs 4. Use of needs categories R&D programmes, research funding, marketing authorisations, innovation incentives, pricing and reimbursement decisions … Weighing the criteria in the light of the evidence Scoring/Deliberating on relative level of health-related needs • Collecting evidence on the criteria for various health issues • Input in an evidence database Requirements for a life-cycle approach to needs-based policy in healthcare Source: Adapted from NEED (https://www.health-needs.eu/index.php/en/about-en). Eurohealth  —  Vol.29  |  No.3  |  2023 42 Eurohealth 29(3) It is important to consider all types of health interventions when steering innovation towards areas with high needs, as defined needs could potentially be addressed by different types of interventions. Health-related needs are independent of the disease-specific interventions in the pipeline. Besides medicinal products, also medical devices, surgery, (supportive) care, preventive interventions or organisational changes could be efficient solutions to specific patient or societal needs. We prefer to use the term “health-related needs”, rather than “unmet medical needs”, as the latter term is often used in relation to pharmaceutical products or in relation to access to healthcare services. The different terminology also avoids confusion with the mainly individual patient needs- driven interpretation of the term ‘unmet medical needs’. Moreover, it is important to emphasise that health-related needs should be defined ‘given the current standard of care’, irrespective of whether this standard of care involves a pharmaceutical product, a medical device or another type of treatment, care or disease management approach. Distinguishing individual patient needs from societal needs is challenging. However, it is important to acknowledge that needs can be defined from both an individual patient’s point of view (health issues representing a high individual burden of disease, despite current diagnostic, preventive or therapeutic options available) and from the societal point of view (health issues representing a high burden on population health, a high impact on the sustainability of the healthcare system, or with ethically unacceptable consequences). Patient needs may relate, for instance, to increased life expectancy or quality of life. While societal needs may relate, for instance, to the availability of an affordable treatment option or of a preventive treatment for a contagious condition. How can EU policy contribute to a needs-driven approach to healthcare innovation and policy making? Evidence-based, needs-driven innovation and policy making requires a scientific evidence base on patient and societal needs in various diseases. Whilst there is no consensus among stakeholders, both on a national and European level, regarding the definition of (unmet) health-related needs, 8 there is agreement among stakeholders that specific criteria can and should be defined for the assessment of health- related patient and societal needs. 9 10 This is a prerequisite for the use of evidence about health-related needs in decision- making processes at all geographical levels. Therefore, a framework must first be developed with transparent criteria on which evidence must be collected and a database with evidence on each of these criteria for a variety of diseases must be established (“assessment” in Figure 1). This framework should be endorsed at EU level to ensure efficient use of resources for research on disease- specific patient and societal needs. ‘‘ respond to the real needs of patients and society Secondly, stakeholders at different levels (EU, national, regional) should agree to use this evidence to make an appraisal of the disease-specific patient and societal needs, i.e., the determination of the relative importance (or level) of the health-related needs in different disease areas (“appraisal” in Figure 1). How the evidence is used for the appraisal may vary between decision-makers. For decisions at EU level (e.g. regarding research funding programmes, incentives for innovation), Member States should agree on the ranking of patient and societal needs. However, for national decisions (e.g., reimbursement of health interventions, national research funding programmes), the ranking may differ between Member States. A classification in categories representing the level of need (high, medium, low or none) or, alternatively, a complete ranking of needs (“ranking” in Figure 1) could be pursued. Finally, there should be agreement to use the classification or ranking for concrete decisions. At the EU level, the high priority needs can be used to inform or define EU research funding programmes. EU regulators (e.g., the European Medicines Agency, EMA) can use them to inform decisions regarding regulatory incentives for the development of health interventions for major health-related needs (e.g., PRIME †, data protection and conditional marketing authorisation for pharmaceuticals). Similar types of possible uses exist at lower geographical levels. Based on the appraisal of the relative importance of health-related patient and societal needs in different health conditions, like-minded countries could indicate to healthcare technology developers who have an intervention (e.g., medical product, new medical procedure) in the pipeline, where it is worthwhile to pursue efforts and where investments are less likely to offer sufficient returns because of the expected willingness to pay for the intervention. In the context of HTA, health-related need categories or rankings may assist HTA bodies and payers, both in national HTAs and joint clinical assessments on the EU level, to assess whether new treatments respond to the real needs of patients and society. Patient and societal needs framework A framework for identifying, measuring and assessing health-related patient and societal needs should be developed with input from stakeholders, including citizens, patients, healthcare providers, policy makers and industry. Because the societal and patient needs perspectives are difficult to merge into one framework, we propose the development of two separate frameworks, one for societal needs and one for patient needs. As a consequence, health-related needs can be appraised as being ‘high’ because either the patient needs or the societal needs are high, or both are high. This approach also allows †  PRIME is a scheme run by EMA to enhance support for the  development of medicines that target health-related needs.  This voluntary scheme is based on enhanced interaction and  early dialogue with developers of promising medicines, to  optimise development plans and speed up evaluation so these  medicines can reach patients earlier, see https://www.ema. europa.eu/en/human-regulatory/research-development/prime- priority-medicines. Eurohealth  —  Vol.29  |  No.3  |  2023 43A Europe that cares, prepares, protects the consideration of specific health objectives, if these have been defined. For example, if reducing inequity in health is a defined health objective, diseases with high inequity between patients will get higher weight in the societal needs appraisal. Societal needs could also include future needs, such as the need to protect future generations. A combined framework for patient or societal needs should start with defining the relevant dimensions and then criteria to operationalise the needs for each dimension. Table 1 provides some examples. After establishing and agreeing on such a framework the next step is to establish a deliberative process that could help to weigh the different criteria to identify the most pressing patient and societal needs. A research infrastructure to inform EU research funding priorities While most healthcare related policy matters fall within national competencies, healthcare research has a significant European dimension. Several large scientific funding programmes, such as Horizon Europe, the Innovative Health Initiative, the Innovative Medicines Initiative, and EU4Health have been established to promote healthcare research in the EU. However, funding is often fragmented, and priorities determined in different ways. For a more coherent prioritisation of topics for health-related needs research, the establishment of a research infrastructure at EU level is needed. This research infrastructure should be responsible for the organisation, coordination and quality assurance of scientific research on health- related patient and societal needs and should create a needs evidence database to support priority setting for research funding programmes. Table 1: Possible dimensions and criteria for societal and patient needs  Societal needs dimensions Possible criteria Impact on population health -  Frequency of the health issue -  Duration of the condition -  Mortality  -  Transmissibility -  Antimicrobial resistance -  Burden on informal caregivers  Impact on healthcare -  Value for money of standard of care  -  Affordability of current management options  Social impact -  Productivity losses -  Environmental impact of standard of care Future impact -  Future burden of disease -  Future economic burden Equity -  Financial accessibility -  Burden of health issue in vulnerable groups (e.g., children, pregnant women) Patient needs dimensions Possible criteria Health -  Mortality -  Quality of life before vs. after onset of disease / experienced burden of the disease Healthcare -  Availability of treatment -  Experienced burden of treatment  -  Experience with healthcare organisation -  Accessibility of care (financial, geographical, waiting times) -  Timely diagnosis Social -  Social support needs -  Impact on work -  Financial consequences  Source: Personal communication NEED team (https://www.unmet-needs.eu/index.php/en/team-en).  Eurohealth  —  Vol.29  |  No.3  |  2023 44 Eurohealth 29(3) Scientific work has been performed at EU level regarding possible research infrastructures that would better address the objective of needs-driven R&D in the EU. 11 12 This research concluded that a public European biomedicines infrastructure should be created to tackle market failures and ensure a proper return on investment, while addressing the pricing of medicines and intellectual property rights. 11 The idea was presented to the European Parliament by the European Parliament’s Panel for the Future of Science and Technology (STOA) in 2022: “Such an initiative could answer the identified issues of shortages and high costs and prices of pharmaceuticals, lack of transparency, and unmet therapeutic needs, by taking back public ownership rights to innovations that are exclusively in the public interest. It would also centralise public investment in public interest areas of low economic return, such as rare medicines and antimicrobials.” 13 Another study highlights that due to the lack of coordination of EU-funded research, there is no coherent investment and long-term strategy that would benefit Europe’s competitiveness and leadership in biomedical innovation. 12 Both studies propose the creation of an independent EU research agency to coordinate EU Health Research and Innovation and address structural weaknesses in the current R&D system. There is a clear link between our proposed approach and these proposed initiatives. Issues such as shortages, and high costs and prices of pharmaceuticals relate to the societal need of wanting to ensure respectively sufficient product availability and affordable treatments, while unmet therapeutic needs refer to patient needs. Both types of needs can be addressed through a European research infrastructure. Furthermore, a prioritisation based on available capacity is foreseen. The issues at stake for an EU research infrastructure focusing on the establishment of an evidence database for patient and societal needs, are similar to the ones described for the biomedicines infrastructure (i.e., available resources, prioritisation, diversification of the agenda: bottom-up approach versus product-driven approach or a combination). Adaptation of approval and reimbursement processes The implementation of the needs-driven approach both in regulatory processes and at the HTA/reimbursement decision level is crucial to achieve improved healthcare for European citizens based on their needs. Regulatory incentives for innovation in specific disease areas, as for instance provided for in the EU pharmaceutical legislation, should consider the identified disease-specific needs of patients and society, as included in the evidence database created by an independent research infrastructure. The evidence will highlight which specific needs must be met to reduce the burden of the disease to patients and/or society, which helps to direct incentives towards interventions that are most likely to meet these needs (e.g., pharmaceutical, devices or other). Although the organisation and funding of healthcare still primarily fall under the responsibility of individual Member States, new EU legislation like the EU HTA Regulation can offer incentives to support this approach. Within this regulation, activities on horizon- scanning, joint scientific consultation, joint clinical assessment and post-license evidence generation are organised in subgroups under the Coordination Group. We propose that all these activities incorporate evidence-based needs in their methodological guidelines, to promote the development of health technologies that address the predefined patient and societal needs. Including evidence on patient and societal needs in horizon scanning, helps to identify upcoming (potentially) high impact interventions. This has a signalling effect to policy makers to take or prepare action (e.g., on reimbursement), and will, in the long run, stimulate developers to invest in such potentially high impact interventions to benefit from such actions. Needs evidence can inform joint scientific consultations and joint clinical assessments by highlighting the criteria on which an improvement should be demonstrated and post-licence evidence generation allows to measure the actual impact of the intervention on the needs or the remaining patient and/or societal needs. Close alignment between these activities and regulatory processes is essential to ensure that regulatory incentives also support these needs. Proposals for more effective EU engagement A summary of proposals to foster needs- driven healthcare and innovation policies in the EU are summarised in Box 1. Based on what has been described, we can conclude that the creation of a needs-driven approach requires the inclusion of scientific evidence on patient and societal needs before, during and after the development of health interventions, i.e. a life-cycle approach where needs are considered even at the ideation phase of development. This requires (1) the identification of disease- specific health-related needs, both on the individual and on the societal level, in a scientifically robust manner (2) the creation of a needs evidence database by an independent research infrastructure that organises and coordinates scientific research on disease-specific health-related needs, (3) the ranking of diseases based on an appraisal of the relative importance of the disease-specific health-related needs, (4) the creation of smart and predictable incentives towards health intervention developers and researchers to steer R&D activities towards the highest needs, and (5) approval and reimbursement processes that take the performance of new health interventions on these needs into account. A strong collaboration at the EU level is required. We therefore call on the European Commission to draft an EU strategic plan that helps both the EU and the Member States to implement a needs- driven ecosystem in healthcare, taking into account all types of health interventions as possible solutions for (unmet) health-related needs, and aiming at the coordination of public support and incentive mechanisms for R&D in order to adequately and effectively respond to the most pressing patient and societal needs. The development of a general approach to define, assess, appraise and use evidence on patient and societal health-related needs should be initiated, including a common framework with needs criteria, to support Eurohealth  —  Vol.29  |  No.3  |  2023 45A Europe that cares, prepares, protects evidence-based, needs-driven decision- making in innovation and healthcare policy. We recommend the EU to support a research infrastructure for the coordination of health-related needs research in the EU and for the establishment and maintenance of a Needs Evidence Database. This research infrastructure should work closely with Member States and support them in the assessment of disease-specific health- related needs. It could consist of a network of national research agencies coordinated by a central independent research agency at European level. For the needs evidence database to be useful for decision making at EU level (e.g., regarding research funding prioritisation, incentives for pharmaceutical products, public procurement, etc.), the identified needs must be ranked. A Needs Appraisal Committee should be established at the EU level for this purpose. The composition, operating procedures, governance structure and appraisal methods should be specified. Its composition should be multi- disciplinary, covering HTA, pricing and reimbursement, regulatory, and scientific experts. Questions related to when and how to move a need from a higher to a lower category of health-related need should also be addressed by this committee. ‘‘ a life-cycle approach where needs are consideredThe reflection at EU level on the various factors that contribute to health-related patient and societal needs should continue and the EU should commit to addressing these needs through policies and programmes, for instance through the EU Health Policy Forum, which brings together stakeholders across the healthcare sector. A life-cycle approach should be implemented in all activities on regulation and reimbursement at the European level, using existing structures such as EMA and the joint work under the EU HTA regulation. 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