PROMs and PREMs: The patient-centered evaluation revolution in the healthcare system

The healthcare system, historically focused on pathology and clinical outcomes measured by professionals, is undergoing a major transformation. The experience and results perceived by patients are becoming essential indicators for evaluating and improving the quality of care. At the heart of this evolution are Patient-Reported Outcome Measures (PROMs) and Patient-Reported Experience Measures (PREMs). These patient self-reported measures offer a unique and indispensable perspective on the effectiveness of treatments and the quality of care. The development of PROMs and PREMs has been intense worldwide for about ten years, with a strong acceleration observed since 2015, responding to a profound evolution of health systems. The objective of this synthesis note is twofold: to determine the scope of legitimacy of pharmaceutical companies in the use of PROMs and PREMs, and to promote the valorization of their use in the life cycle of the drug to promote their deployment. But beyond the drug, PROMs and PREMs are revolutionizing evaluation throughout the healthcare system.

What are PROMs and PREMs? Definitions and key distinctions

To understand this "revolution", it is crucial to define these two distinct but complementary concepts.

PROMs (Patient-Reported Outcomes Measures) are measures of care outcomes reported directly by patients. They assess the patient's perception of their health status, symptoms, functional abilities, quality of life, or well-being. The assessment is based on self-administered questionnaires or other collection tools. PROMs can be generic, exploring issues important to the general population, or specific, focusing on a particular disease, intervention, or specific dimension (pain, fatigue, anxiety, depression). Among the generic PROMs commonly used in international studies are the EQ-5D (with its 3L and 5L versions), the SF-36 or SF-12 (short versions of the SF-36), and the PROMIS (Patient-Reported Outcomes Measurement Information System). The World Health Organization (WHO) has also developed tools such as the WHO-5 (well-being index) or the WHO Disability Assessment Schedule 2.0 (WHO-DAS-12), which are generic PROMs. The ICHOM (International Consortium for Health Outcomes Measurement) recommends standardized sets of PROMs for various pathologies or populations.

PREMs (Patient-Reported Experience Measures), for their part, measure the experience lived by patients with the health system or the care they received. They relate to the quality of the relationship with health professionals, the organization of care, access to information, the environment of the hospital, etc. The definition of PREMs is not always systematically reported or standardized internationally by evaluation agencies, with the exception of some such as the HAS in France. Initiatives such as the Consumer Quality Index (CQI) in the Netherlands or the CAHPS® (Consumer Assessment of Healthcare Providers and Systems) surveys in the United States are examples of standardized PREMs questionnaires used on a large scale. In France, the e-Satis system is an example of a mandatory national collection of PREMs.

The essential distinction lies in what is measured: PROMs assess the outcome (what changes in the patient's health status), while PREMs assess the experience (how the care was delivered and perceived). These two types of measures are complementary to obtain a complete view of the quality of care perceived by patients.

Why are PROMs and PREMs key? Objectives and benefits

The growing interest in PROMs and PREMs is not accidental. They bring a unique and valuable perspective that was previously underrepresented in the evaluation of the quality of care. Their deployment is likely to produce many benefits and affect all actors in the healthcare system.

For patients and users, PROMs and PREMs are a way to be heard and contribute to the improvement of their own care and that of others. Answering these questionnaires allows patients to become actors in their health and the evolution of the system. PROMs can facilitate communication with healthcare professionals in consultation, by providing structured information on their symptoms and quality of life, which can improve follow-up and shared decision-making. Patient experience collection initiatives, such as PREMs surveys, reinforce the feeling of being listened to and considered.

For healthcare professionals and care organizations, the use of PROMs and PREMs can improve daily clinical practice. The results can help to better monitor the evolution of patients, to quickly identify problems not detected otherwise, and to adapt treatments or support. At the organizational level, data aggregation makes it possible to evaluate the performance of services, to carry out internal comparisons (benchmarking) to identify areas for improvement, and to better allocate resources. The use of PROMs results can, for example, improve the quality of life of patients thanks to feedback to doctors on their functional status.

For health authorities and regulators, PROMs and PREMs provide essential data for steering the healthcare system. They are used to evaluate the performance and quality of care providers, to inform regulatory decisions (funding, authorization, reimbursement), and to enhance transparency. The public dissemination of results, although not systematic everywhere, allows users to make informed choices between healthcare providers. The integration of PROMs and PREMs into pay-for-performance (P4P) models aims to incentivize care providers to improve patient-reported outcomes and experience. In addition, these data are valuable for clinical research and the evaluation of treatment effectiveness.

In summary, PROMs and PREMs constitute a new and complementary source of information allowing a shift from a purely clinical evaluation to a more global evaluation, integrating the patient's perspective at the center of the process.

Integration of PROMs and PREMs into the drug lifecycle

The use of PROMs and PREMs is particularly relevant and variable throughout the drug life cycle.

Even before the start of clinical trials, pharmaceutical manufacturers can collect indicators from patients, often informally (interviews, focus groups), to better define their needs and priorities. Unstructured indicators or PREMs are mainly collected at this initial stage. Social network analysis, forum verbatim, or "social listening" initiatives can provide new and complementary information to manufacturers for this purpose.

During the research and development phase, particularly during clinical trials (phases II and especially III), PROMs are widely integrated. The majority of clinical trials implemented by pharmaceutical manufacturers include PROMs, whether generic and/or specific. This data collection (mainly PROMs) is a central phase, as it determines the future evaluations that will be made by the health authorities. PROMs are used to evaluate the added value of the treatment or to examine whether the collection can detect significant effects and influence the conclusions of the study, in particular the evaluation of the benefit/risk ratio. However, obtaining a Marketing Authorization (MA) is still little impacted by these data, the evaluation being based mainly on the clinical results measured by professionals (CROMs).

When applying for early access to a drug, the Haute Autorité de Santé (HAS) [French National Authority for Health] recommends the integration of a disease-specific self-questionnaire (PROM), as soon as this measure is deemed relevant in practice.

For reimbursement requests, the HAS stipulates that the absence of quality of life data (which can be measured by PROMs) may, in some cases, negatively impact the evaluation of the Improvement in Actual Benefit (ASMR). However, the systematic integration and the real influence of these data on the reimbursement decision remain difficult to clearly assess.

At the price negotiation stage by the Economic Committee for Health Products (CEPS), there is no formal recommendation regarding the integration of patient indicators (PROMs/PREMs). The negotiation process is mainly based on the assessment previously made by the health authorities.

Finally, in the context of post-marketing authorization (post-MA) follow-up studies, the HAS recommends the integration of a self-questionnaire (PROM) to collect data on quality of life or other relevant measures for patients. The absence of a PROM in the post-registration study protocol must be systematically justified.

The integration of PROMs is therefore formalized and present throughout the life cycle of the drug, from the European to the national level. Conversely, PREMs are less formally integrated into the drug cycle itself, being measured more at the healthcare system level and often collected informally by manufacturers prior to clinical trials.

Using PROMs and PREMs in the healthcare system: an international overview

A preliminary study compared the situation in France with that in four other countries, while a more in-depth report analyzed the use of PROMs and PREMs in 13 countries (7 European and 6 Anglo-Saxon). This overview reveals a wide diversity of approaches and deployment levels.

The levels of PROMs and PREMs collection vary considerably from one country to another. There are initiatives at the national, regional (or provincial) and local levels. Sweden is often cited as a leading country in the routine collection of PROMs and PREMs, particularly through its national quality registers (NQR) covering many pathologies and procedures. The majority of the countries studied have structured national initiatives for PROMs (9 out of 13), sometimes supplemented by local initiatives. For PREMs, the situation is also varied; some countries such as Sweden, Norway or the Netherlands have long-standing or structured national systems, while others such as the United States use standardized systems such as CAHPS® on a large scale. Germany and Denmark do not report national or regional initiatives for routine PROMs, leaving collection to local or research initiatives.

The use of data from PROMs and PREMs is divided into several main applications:

1. Comparison and Benchmarking: This is a very common use for PROMs (10 out of 13 countries) and PREMs. The objective is to compare results between healthcare providers (hospitals, professionals) to identify good practices and stimulate improvement. Countries such as England, the United States, or Sweden actively use this data for comparison. Benchmarking involves a continuous effort to learn from the best performances.
2. Accreditation (or Certification): The use of PROMs in healthcare facility accreditation schemes is rare; none of the countries studied report it. On the other hand, PREMs results are used in these schemes in four countries, including France with the national e-Satis scheme integrated into the certification of healthcare facilities. Some provinces in Canada also use the results of PREMs surveys for accreditation.
3. Public Dissemination of Results: Transparency is an important objective, but public dissemination of PROMs and PREMs results is not systematic everywhere. It is done in different formats: national reports by pathology (Sweden), rankings carried out by insurers or associations (Netherlands), national websites (England with NHS Digital, United States with Hospital Compare). In France, the results of e-Satis are publicly disseminated via the Scope santé website.
4. Pay for Performance (P4P): The integration of PROMs and PREMs into healthcare provider funding models is a developing use, although less frequent than comparison. Some countries link a portion of provider compensation to outcomes on these indicators. Sweden uses PREMs to compensate primary care providers. The Netherlands uses them to inform funding agencies. The United States includes PROMs and PREMs in various P4P programs (e.g., Hospital Value-Based Purchasing program, ESRD QIP for dialysis). Belgium integrated PREMs into its P4P program in 2018. In France, e-Satis results are integrated into the Incentive Financière à l'Amélioration de la Qualité (IFAQ) system.

The international landscape shows that the use of PROMs is more focused on comparison and research, while PREMs are more often integrated into regulatory mechanisms, such as certification or pay-for-performance.

The situation of PROMs and PREMs in France

France is part of this national and international dynamic of taking the patient's perspective into account. The Haute Autorité de Santé (HAS) is a driving force on this subject. A preliminary study explored the perspectives of health authorities, patients and pharmaceutical manufacturers in France.

Regarding PREMs [Patient-Reported Experience Measures], France is among the most advanced countries in terms of collecting and using their results. The national e-Satis system is a mandatory system for collecting patient experience and satisfaction after certain hospitalizations (short-stay MCO, outpatient surgery, SSR). The data collected via e-Satis are used for inter-hospital comparison, integrated into the certification process of healthcare facilities, publicly disseminated via the Scope santé website to inform the public, and taken into account in the quality-based funding mechanism (IFAQ).

Regarding PROMs, France is currently less advanced than with PREMs in terms of a national routine collection system. However, numerous initiatives are under development or being tested.

• Public or associative research platforms allow the collection of PROMs, such as the ComPaRe platform of the AP-HP where patients with chronic diseases answer online questionnaires, or the Moi Patient platform of the Renaloo association for patients with chronic kidney disease.
• As part of the national experiments with innovative organizations provided for by Article 51 of the LFSS 2018, the use of PROMs is being considered or implemented. This is the case for the flat-rate payment for chronic kidney disease care (with the potential integration of PROMIS-29), or for the experiment of payment per episode of surgical care for certain surgeries (hip, knee, colectomy for cancer) where professionals can use existing PROMs. The experiment to incentivize shared care (IPEP) and the project for flat-rate payment to teams of healthcare professionals in urban settings (PEPS) also use patient questionnaires, including PREMs.
• Regional initiatives also exist, such as the collection of PROMs in patients with HIV tested in Aquitaine as part of an ANRS study.
• French healthcare facilities participate in international initiatives such as the ICHOM project, contributing to benchmarking for various pathologies (cancers, cataracts).
• France also participates in the OECD's overview of the collection of PROMs for specific pathologies such as hip arthroplasty or breast cancer.

The HAS plays an active role in supporting these developments. It has planned to continue and expand its work on measuring the quality perceived by patients. It offers practical guides to help with the local deployment of PROMs. The first three guides have been published, one general guide on concepts and criteria, and the other two in specific contexts (hip/knee prosthesis/colorectal cancer and COPD patient pathways). In its policy framework, the HAS also considered formulating a self-referral, following the report, to include the development of the implementation of PROM and PREM measures in a multi-year program.

Despite significant initiatives, France still faces a lack of structured national or regional systems for the routine collection of PROMs, unlike PREMs. PROMs data are often collected as part of research projects or specific experiments.

Challenges and levers for the deployment of PROMs and PREMs

The large-scale deployment and optimal use of PROMs and PREMs face several challenges, identified both in France and abroad.

A major obstacle is the lack of standardization. There is considerable heterogeneity in the instruments (questionnaires) used to measure PROMs and PREMs, the methods of collection and the way in which results are presented. This non-standardization makes it difficult to compare different initiatives or care providers. Even within the same pathology, several PROMs may be used, preventing direct comparison of results between patients or facilities. The lack of consensus on PROMs is particularly notable, with a definition that is not always clear at international level.

Methodological and operational difficulties are also encountered. The design of relevant questionnaires, adapted to different populations (age, health status, language proficiency) is complex. Response rates to questionnaires, especially online or in routine clinical practice, can be relatively low. The length of some questionnaires can be an obstacle. The implementation of the collection requires in-depth reflection upstream to be successful. It is important to clearly define the objectives of the collection, the target populations, and the collection times.

Technical and organizational barriers exist. The integration of PROMs and PREMs data into existing information systems (electronic medical records, registers) can be complex. Healthcare professionals may lack the training and dedicated time to use these indicators in consultation and discuss the results with patients. The lack of human and financial resources to support data collection and analysis is a hindrance.

Another major challenge, particularly in the context of pharmaceuticals, is the lack of value placed on patient indicators by health authorities in certain decisions. Analysis of concrete examples has shown that authorities have limited flexibility in the use of these data, for example, beyond the classic clinical criteria for marketing authorization. Similarly, patient indicators are not formally required for drug price negotiations in France. In the wider healthcare system, PROMs results are not used for facility accreditation in the countries studied.

Faced with these challenges, several levers can be activated:

• Standardization is crucial, promoting the use of validated and standardized tools, notably via international initiatives (ICHOM, OECD PaRIS) that develop indicator sets. Harmonization at the national or regional level (such as the initiative in Wales) can facilitate comparison and deployment.
• Support for professionals is essential, through training, practical guides (such as those from the HAS), and the integration of PROMs into the clinical workflow with dedicated time for discussion of results.
• The adaptation of collection methods (paper format, online, tablet, mobile application) to make them simple and flexible for patients, and taking into account the specific characteristics of the target populations. Using technologies like Computer Adaptive Testing (CAT) for PROMIS can reduce the burden on the patient.
• The involvement of patients in the co-construction of measurement tools and collection processes reinforces acceptability and relevance.
• The strategic integration of PROMs and PREMs into health policies: including them in evaluation (reimbursement, access), certification, and pay-for-performance schemes. The commitment of public authorities is a major lever.
• The transparent dissemination of results, by standardizing report formats and facilitating access for the public and professionals.

These levers require a strong commitment from all stakeholders in the healthcare system.

What future for PROMs and PREMs? Outlook and development

The development dynamic of PROMs and PREMs is strong and should not be questioned. The future of these patient-reported measures seems promising, with several development prospects.

A key trend is theincreasing integration into clinical routine. The aim is for the collection of PROMs and PREMs no longer to be confined to research projects or experiments, but to become an integral part of patient follow-up, consultations and ongoing quality assessment. There are also plans to extend the collection of data to a greater number of pathologies and care sectors (primary care, chronic diseases, mental health, home care).

Standardization will remain a major challenge to enable reliable comparisons and large-scale data aggregation. International efforts (ICHOM, PaRIS) to define standardized sets of PROMs and PREMs by condition or care pathway will continue to play an essential role.

Technological advances will offer new opportunities for the collection and use of patient data. Mobile applications, connected devices, and online platforms facilitate real-time collection and integration of data into electronic patient records. The analysis of unstructured data from social networks or patient verbatim ("social listening") could also be further structured to extract useful information on the perceived experience and outcomes.

The strategic use of data by health authorities and payers should also increase. Although challenges remain, it is likely that the results of PROMs and PREMs will play an increasing role in the evaluation of health technologies (medicines, medical devices), reimbursement decisions, price negotiation, and the funding of hospitals. The emphasis on "Value-Based Healthcare", which integrates outcomes that are important to patients, reinforces the relevance of PROMs.

In France, the HAS has clearly expressed its intention to continue and amplify its work in this area, with the prospect of a multi-year program dedicated to developing the implementation of PREMs and PROMs. The development of practical guides and support for local pilot projects are concrete steps to facilitate adoption.

The role of patients and their associations will continue to grow. Their involvement in the design, deployment and use of PROMs and PREMs is recognized as a key success factor.

In conclusion, the future of PROMs and PREMs is one of deeper and more systematic integration into the evaluation of the quality of care and decision-making at all levels of the healthcare system. This evolution is essential to truly place the patient's perspective at the heart of the quality approach and to steer healthcare systems based on what really matters to people receiving care. Despite the challenges, the current dynamic suggests that PROMs and PREMs are destined to become essential tools for a more patient-centered, transparent, and efficient healthcare system.