Opportunities to improve the adoption of health-related quality of life evidence as part of the French Health Technology Assessment process.

OBJECTIVES: Patient's health-related quality of life (HRQoL) is an important outcome measure that is considered by many payers and health technology assessment (HTA) bodies in the evaluation of treatments. We aimed to identify opportunities for HRQoL to be further incorporated into the assessment of the French HTA by comparing three health systems. We put forward recommendations that could bring further innovations to French patients. METHODS: We reviewed methodologies by the French, German and British HTA, and conducted a systematic review of all French (n = 312) and German (n = 175) HTA appraisals from 01 January 2019 to 31 December 2021. We also setup an advisory board of 11 ex-HTA leaders, payers, methodologists, healthcare providers and patient advocates, from France, Britain and Germany, to discuss opportunities to improve acceptance and adoption of HRQoL evidence in France. RESULTS: Our systematic review of HTA appraisals showed a higher HRQoL data rejection rate in France: in > 75% of cases the HRQoL evidence submitted was not accepted for the assessment (usually for methodological reasons, for example, data being considered exploratory; 16-75% of the appraisals mentioned HRQoL evidence, varying by therapeutic area). Overall, we found the French HTA to be more restrictive in its approach than IQWiG. CONCLUSIONS: Based on these findings we articulate collaborative proposals for industry and the HAS to improve acceptance of HRQoL evidence and create a positive feedback loop between HAS and industry along four dimensions (1) patient perception, (2) testing hierarchy, (3) trial design and (4) data collection.

STYLCONNECT Study: An Assessment of Automatic Data Collection Devices by People Living with Diabetes and Using an Insulin Pen.

INTRODUCTION: The use of devices to connect insulin pens could facilitate management and improve glycaemic control in people with type 1 (PwT1D) and type 2 diabetes (PwT2D). However, their acceptance seems little studied. We conducted an online survey with the main objective of assessing the level of interest among insulin-treated people with diabetes (PwD) in a device connected to a disposable pen and secondary objectives of assessing the perceived benefits and important features expected of a connected device and identifying factors associated with interest scores. METHODS: An ad-hoc questionnaire, validated by PwD, was used. Responses from 1798 PwD (975 PwT1D and 823 PwT2D) were analysed. RESULTS: The mean interest rating was 7.4/10 (PwT1D: 7.2 vs PwT2D: 7.7; p < 0.001). PwD perceived that the device would make it easier to record their diabetes-related information (7.7/10) and keep all insulin and diabetes data in a single location (7.7/10). It was particularly important for PwD that this type of device could integrate data from glucose-measuring devices (7.8/10) and could set an alarm when all insulin in the body had been metabolised (7.7/10). CONCLUSION: Our study highlighted PwD's strong interest in automating the collection of their insulin therapy data, with significantly more interest among PwT2D than PwT1D, and the importance of interoperability between glucose measurement devices and interchangeability between the different brands of insulin. More generally, for the first time and on a large scale, our study provided a greater understanding of the expectations of PwD regarding these devices.

Place of patient-reported outcomes & experiences measurements (PROMS/PREMS) in the assessment and pricing of Health technologies in France.

In the context of health technologies assessment, patient-reported outcome measures (PROMs) have become assessment criteria that are expected by evaluation agencies along with the other usual clinical criteria. PROMs instruments measure all aspects of patient experience in connection with their health: symptoms, activities of daily living (physical function, sleep, etc.), various aspects of health-related quality of life (QoL), compliance, global impression of change in wellbeing. PROMs are useful both as 1) a primary or secondary efficacy endpoints, and 2) a tolerability criterion to supplement vigilance data reported by clinicians. Measurement of PROMs must be subject to methodological rigor that is identical to that of other assessment criteria measured by an observer. Scales must be validated, suitable for the objective, and where possible specific to a disease. In addition to standard measures of quality of life, PROMs are taken into consideration in the assessments performed by the HAS, even if their impact on the conclusions is difficult to isolate, as assessments are multifactorial and take into account all data available with regard to the medical context. The CEPS will indirectly take into account PROMs in the fixing of the price or tariff only if they have contributed to the award of the ASA/ASMR by the ad hoc committee of the HAS. The working group has formulated three recommendations which aim to further the implementation of patient-reported outcome measures: (1) Better information for all parties involved in a dossier for technology assessment, (2) Systematization of the collection of PROMs for evaluation of health products, (3) Improved quality of dossiers thanks to the use of relevant and validated tools.

A Novel Multi-Dimensional Clinical Response Index Dedicated to Improving Global Assessment of Pain in Patients with Persistent Spinal Pain Syndrome after Spinal Surgery, Based on a Real-Life Prospective Multicentric Study (PREDIBACK) and Machine Learning Techniques.

The multidimensionality of chronic pain forces us to look beyond isolated assessment such as pain intensity, which does not consider multiple key parameters, particularly in post-operative Persistent Spinal Pain Syndrome (PSPS-T2) patients. Our ambition was to produce a novel Multi-dimensional Clinical Response Index (MCRI), including not only pain intensity but also functional capacity, anxiety-depression, quality of life and quantitative pain mapping, the objective being to achieve instantaneous assessment using machine learning techniques. Two hundred PSPS-T2 patients were enrolled in the real-life observational prospective PREDIBACK study with 12-month follow-up and received various treatments. From a multitude of questionnaires/scores, specific items were combined, as exploratory factor analyses helped to create a single composite MCRI; using pairwise correlations between measurements, it appeared to more accurately represent all pain dimensions than any previous classical score. It represented the best compromise among all existing indexes, showing the highest sensitivity/specificity related to Patient Global Impression of Change (PGIC). Novel composite indexes could help to refine pain assessment by informing the physician's perception of patient condition on the basis of objective and holistic metrics, and also by providing new insights regarding therapy efficacy/patient outcome assessments, before ultimately being adapted to other pathologies.

Professional Status of Persistent Spinal Pain Syndrome Patients after Spinal Surgery (PSPS-T2): What Really Matters? A Prospective Study Introducing the Concept of "Adapted Professional Activity" Inferred from Clinical, Psychological and Social Influence.

Persistent Spinal Pain Syndrome Type 2 (PSPS-T2) represents a main cause of work disruption. Beyond its societal consequences, occupational inactivity is responsible for a major decrease in physical/mental health in individuals but remains poorly analyzed. We designed a study to prospectively examine Professional Status (PS) evolution and its association with key bio-psychological markers. Data from 151 consecutively included working-age PSPS-T2 patients were analyzed to determine the proportion of professional inactivity and the relationships between PS and Social Gradient of Health (SGH), Numeric Pain Rating Scale (NPRS), EuroQol 5-Dimensional 5-Level (EQ-5D-5L), Oswestry Disability Index (ODI), Hospital Anxiety and Depression Scale (HADS), and Fear-Avoidance Belief Questionnaire work subscale (FABQ-W). Despite optimized medical management, 73.5% of PSPS-T2 patients remained inactive after 1 year of follow-up/p = 0.18. Inactive patients presented a low SGH/p = 0.002, higher NPRS/p = 0.048, lower EQ-5D-5L/p < 0.001, higher ODI/p = 0.018, higher HADS-D/p = 0.019 and higher FABQ-W/p < 0.001. No significant mediation effect of FABQ-W on SGH consequences regarding PS was observed in our structural model/p = 0.057. The link between unemployment and bio-psycho-social pain dimensions appears bidirectional and justifies intense collaboration with social workers. Optimizing therapeutical sequencing towards personalized professional plans implies restoring "Adapted Physical Function" as an initial goal, and tailoring an "Adapted Professional Activity", matching with patient expectations and capabilities, as a final objective.

Finite Mixture Models Based on Pain Intensity, Functional Disability and Psychological Distress Composite Assessment Allow Identification of Two Distinct Classes of Persistent Spinal Pain Syndrome after Surgery Patients Related to Their Quality of Life.

Persistent Spinal Pain Syndrome Type 2 (PSPS-T2), (Failed Back Surgery Syndrome), dramatically impacts on patient quality of life, as evidenced by Health-Related Quality of Life (HRQoL) assessment tools. However, the importance of functioning, pain perception and psychological status in HRQoL can substantially vary between subjects. Our goal was to extract patient profiles based on HRQoL dimensions in a sample of PSPS-T2 patients and to identify factors associated with these profiles. Two classes were clearly identified using a mixture of mixed effect models from a clinical data set of 200 patients enrolled in "PREDIBACK", a multicenter observational prospective study including PSPS-T2 patients with one-year follow-up. We observed that HRQoL was more impacted by functional disability for first class patients (n = 136), and by pain perception for second class patients (n = 62). Males that perceive their work as physical were more impacted by disability than pain intensity. Lower education level, lack of adaptive coping strategies and higher pain intensity were significantly associated with HRQoL being more impacted by pain perception. The identification of such classes allows for a better understanding of HRQoL dimensions and opens the gate towards optimized health-related quality of life evaluation and personalized pain management.

Persistent Spinal Pain Syndrome Type 2 (PSPS-T2), a Social Pain? Advocacy for a Social Gradient of Health Approach to Chronic Pain.

The Social Gradient of Health (SGH), or position in the social hierarchy, is one of the major determinants of health. It influences the development and evolution of many chronic diseases. Chronic pain dramatically affects individual and social condition. Its medico-economic impact is significant and worldwide. Failed Back Surgery Syndrome or Persistent Spinal Pain Syndrome type 2 (PSPS-T2) represents one of its most fascinating and disabling conditions. However, the influence of SGH on PSPS-T2 has been poorly explored. We designed a prospective multicentric study (PREDIBACK study) to assess the SGH prevalence, and to examine its association with medical and psychological variables, in PSPS-T2 patients. This study included 200 patients to determine the SGH association with pain (NPRS), Quality of life (EQ-5D-5L), kinesiophobia (FABQ-Work), catastrophism (CSQ), and functional capacity (ODI). Around 85.3% of PSPS-T2 patients in our study had low SGH. Low SGH patients had a higher FABQ-Work and CSQ-Catastrophizing score than high SGH patients (p < 0.05). High SGH patients have a higher ODI score than low SGH patients (p < 0.10). Our results suggest that SGH is a relevant factor to guide prevention, research, and ultimately intervention in PSPS-T2 patients and could be more widely transposed to chronic pain.

Optimizing the Management and Outcomes of Failed Back Surgery Syndrome: A Consensus Statement on Definition and Outlines for Patient Assessment.

Failed back surgery syndrome (FBSS) is a controversial term for identifying patients affected by new, recurrent, or persistent pain in the low back and/or legs following spinal surgery. The lack of a comprehensive standardized care pathway compromises the appropriate management of FBSS patients, which is associated with a heavy financial burden. An international panel of spine surgeons, neurosurgeons, and pain specialists with a particular interest in FBSS established the chronic back and leg pain (CBLP) network with the aim of addressing the challenges and barriers in the clinical management of FBSS patients by building a common transdisciplinary vision. Based on literature reviews, additional input from clinical expertise of multiple professional disciplines, and consensus among its members, the network attempted to provide recommendations on the management of patients with FBSS utilizing a multidisciplinary team (MDT) approach. The presentation of this work has been divided in two separate parts to enhance its clarity. This first paper, in favour of selecting appropriate validated tools to improve the FBSS patient assessment, focuses on FBSS taxonomy and its clinical implications for evaluation. Concise recommendations for assessment, treatment, and outcome evaluation using a MDT approach would be an important resource for specialists and nonspecialist clinicians who manage patients with FBSS, to improve decision-making, reduce variation in practice, and optimize treatment outcomes in this difficult-to-treat population.