[Not Available]. / Recommandations en matière de communication et de sollicitation de patients pouvant participer ou participant déjà à des études cliniques.

The active patient participation in clinical trials is key for a competitive clinical research. Given this, the Health Industry Physicians and Actors Association (AMIPS) has set up a working group to make communication recommendations towards patients. The group was made of patients, investigators and industry sponsors representatives. Efficacious communication is rarely obtained because it is not clear what is possible to do ethically and regulatory and because of technical and financial constraints. After having identified the expectations and limitations for every actor category, the group has summarized all types of communication, in a sort of tool box, before and during the whole of a study. The benefits and regulatory prerequisites such as the submission to the Ethical Committee and to the National Data Information and Freedom Commission (CNIL) as well as the practical feasibility are described for each tool.

[Recommendations for Communication and Solicitation of Patients Potentially or Already Involved in Clinical Studies]. / Recommandations en matière de communication et de sollicitation de patients pouvant participer ou participant déjà à des études cliniques.

The active patient participation in clinical trials is key for a competitive clinical research. Given this, the Health Industry Physicians and Actors Association (AMIPS) has set up a working group to make communication recommendations towards patients. The group was made of patients, investigators and industry sponsors representatives. Efficacious communication is rarely obtained because it is not clear what is possible to do ethically and regulatory and because of technical and financial constraints. After having identified the expectations and limitations for every actor category, the group has summarized all types of communication, in a sort of tool box, before and during the whole of a study. The benefits and regulatory prerequisites such as the submission to the Ethical Committee and to the National Data Information and Freedom Commission (CNIL) as well as the practical feasibility are described for each tool.

A patient-derived and patient-reported outcome measure for assessing psoriatic arthritis: elaboration and preliminary validation of the Psoriatic Arthritis Impact of Disease (PsAID) questionnaire, a 13-country EULAR initiative.

INTRODUCTION: The objective was to develop a questionnaire that can be used to calculate a score reflecting the impact of psoriatic arthritis (PsA) from the patients' perspective: the PsA Impact of Disease (PsAID) questionnaire. METHODS: Twelve patient research partners identified important domains (areas of health); 139 patients prioritised them according to importance. Numeric rating scale (NRS) questions were developed, one for each domain. To combine the domains into a single score, relative weights were determined based on the relative importance given by 474 patients with PsA. An international cross-sectional and longitudinal validation study was performed in 13 countries to examine correlations of the PsAID score with other PsA or generic disease measures. Test-retest reliability and responsiveness (3 months after a treatment change) were examined in two subsets of patients. RESULTS: Two PsAID questionnaires were developed with both physical and psychological domains: one for clinical practice (12 domains of health) and one for clinical trials (nine domains). Pain, fatigue and skin problems had the highest relative importance. The PsAID scores correlated well with patient global assessment (N=474, Spearman r=0.82-0.84), reliability was high in stable patients (N=88, intraclass correlation coefficient=0.94-0.95), and sensitivity to change was also acceptable (N=71, standardised response mean=0.90-0.91). CONCLUSIONS: A questionnaire to assess the impact of PsA on patients' lives has been developed and validated. Two versions of the questionnaire are available, one for clinical practice (PsAID-12) and one for clinical trials (PsAID-9). The PsAID questionnaires should allow better assessment of the patient's perspective in PsA. Further validation is needed.

Development and Validation of a Self-Administered Questionnaire Measuring Essential Knowledge in Patients With Axial Spondyloarthritis.

OBJECTIVE: To develop and validate a patient knowledge questionnaire regarding axial spondyloarthritis (axSpA). METHODS: Knowledge considered essential for patients with axSpA was identified through Delphi rounds among rheumatologists, healthcare professionals (HCPs), and patients, then reformulated to develop the knowledge questionnaire. Cross-sectional validation was performed in 14 rheumatology departments to assess internal validity (Kuder-Richardson coefficient), external validity, acceptability, reproducibility (Lin concordance correlation coefficient), and sensitivity to change (knowledge score before vs after patient education sessions and effect size). RESULTS: The Spondyloarthritis Knowledge Questionnaire (SPAKE) is a self-administered 42-item questionnaire with a 32-item short form, both scored 0 to 100, assessing knowledge of disease, comorbidities, pharmacological treatments, nonpharmacological treatments, self-care, and adaptive skills. In the validation study (130 patients; 67 [51.5%] male, mean age 43.5 [SD 12.9] yrs), the mean (SD) score of the long-form questionnaire was 71.6 (15.4), with higher scores (better knowledge) in nonpharmacological treatments and adaptive skills and lower scores in cardiovascular comorbidity and pharmacological treatments. Acceptability was good, with no missing data; the internal validity coefficient was 0.85. Reproducibility was good (0.81, 95% CI 0.72-0.89). SPAKE showed good sensitivity to change; scores were 69.2 (15.3) then 82.7 (14.0) after patient education sessions (Hedges effect size = 0.92, 95% CI 0.52-1.31). CONCLUSION: SPAKE is a knowledge questionnaire for patients with axSpA, developed with the involvement of HCPs and patients and reflecting current recommendations for the management of axSpA. SPAKE will be useful in assessing knowledge acquisition and self-management strategies in routine care and research.

Costs comparison of treating diabetic macular edema with aflibercept, ranibizumab or dexamethasone at 1 year in France (INVICOST study).

PURPOSE: INVICOST, a medico-economic analysis, compared costs of managing treatment-naive patients with diabetic macular edema (DME) receiving intravitreal injections (IVIs) of aflibercept (AFL), dexamethasone implant (DXI) or ranibizumab (RAN) over 1 year. METHODS: Healthcare resource use and associated costs were estimated using individual patient data from INVICTUS, a prospective, open-label, monocentric study. Healthcare costs comprised direct medical costs such as drug acquisition and administration, consultations and ophthalmological procedures. Costs were assessed from the French National Health Insurance perspective using published national tariffs expressed in 2019 euros. RESULTS: Of the 60 treated eyes, 48 had no treatment switch; 14 received AFL, 19 received DXI and 15 received RAN. AFL-treated eyes received an average of 6.5 IVIs, DXI-treated patients received 2 IVIs and RAN-treated received 6.8 IVIs. All treated eyes received an initial prescription for adjunctive ocular medications and 349 follow-up procedures were performed including an average of 3.9 optical coherence tomography and 3.2 retinography procedures per eye. Average total direct cost of per-eye treatment was €4516 (€1128-€8257). Average cost was €5782 for eyes treated with AFL, €2779 with DXI and €5536 with RAN. Drug therapy was the cost driver: €4394 (76%) for AFL, €1915 for DXI (69%) and €4268 (77%) for RAN. CONCLUSION: The difference in total treatment cost is largely explained by the significantly lower frequency of IVI and annual cost of therapy with DXI, compared with AFL and RAN. INVICOST is the first study comparing treatment costs with AFL, DXI and RAN in France in current clinical practice.

Essential knowledge for patients with rheumatoid arthritis or spondyloarthritis: Results of a multicentric survey in France among health professionals and patients.

OBJECTIVE: Information and education are recommended for patients with inflammatory arthritis including rheumatoid arthritis (RA) and spondyloarthritis (SpA). However, there is no consensus on which knowledge is essential to enhance patients' self-management. The aim of this study was to determine such knowledge. METHODS: Based on published knowledge questionnaires (KQs) collected by a systematic literature review, a list of items was elaborated, classified in domains and sub domains. A Delphi process was performed with rheumatologists, healthcare professionals and patients in 2014-2015, selecting the items considered useful. RESULTS: Three published KQs were analysed: 2 for RA; 1 for SpA and 5 unpublished KQs were collected. In the KQs, 90 knowledge items were mentioned for RA and 67 for SpA. The 1st Delphi round enlarged the list to 322 items for RA and 265 items for SpA. The second round selected 69 and 59 knowledge items for RA and SpA respectively, of which 36 (52%) and 34 (57%) were not present or modified from the published KQs. Key domains included treatment strategies, managing cDMARDs and bDMARDs, managing symptomatic medications. Knowledge on non-pharmacological treatment concerned pain and fatigue, physical activity, adaptative skills to personal and professional environment, patient-HP communication and shared decision-making. CONCLUSION: The present study provides a corpus of knowledge considered essential for patients in the self-management of their arthritis. The selection of many items reflects recent emphasis on professional recommendations and the patients' perspective. Future work should lead to the development of new updated KQs for patients with inflammatory arthritis.

Recommendations of the French Society for Rheumatology (SFR) on the everyday management of patients with spondyloarthritis.

UNLABELLED: The management of spondyloarthritis is challenging and has changed with the development of new concepts and treatments. OBJECTIVE: To develop practice guidelines for the everyday management of patients with spondyloarthritis (including psoriatic arthritis), by updating previous national and international recommendations, based on a review of recently published data. METHODS: A task force and a multidisciplinary literature review group were established. The task force identified the issues that remained unresolved. Based on existing recommendations and recent publications, the task force developed practice guidelines, which were revised by the literature review group and graded according to AGREE. RESULTS: Practice guidelines for the management of spondyloarthritis are reported. After a review of the general diagnostic principles, 30 practice guidelines are given: 5 on general principles, 4 on the management strategy, 5 on non-pharmacological treatments, 7 on conventional pharmacological treatments, 6 on biotherapies, and 3 on surgical treatments and follow-up. CONCLUSION: The updated practice guidelines reported here constitute a global framework that can guide physicians in the everyday management of spondyloarthritis.

Estimation of the Bath Ankylosing Spondylitis Disease Activity Index cutoff for perceived symptom relief in patients with spondyloarthropathies.

OBJECTIVE: To estimate the best Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) cutoff based on patients' perceptions of symptom relief collected in a large population of patients with spondyloarthropathies (SpA), in comparison to the BASDAI cutoff determined by experts. METHODS: A survey of patient perceptions about current disease control was conducted among the members of Spondylis, one of the main not-for-profit SpA patient organizations in France. BASDAI was among data collected by the questionnaire. To estimate the best BASDAI cutoff for discriminating between poor and well perceived controlled groups, we plotted the receiver operating characteristic (ROC) curve. We also determined the cutoff separately in male and female patients. RESULTS: Of the 1000 mailed questionnaires, 485 were returned without any missing data regarding perceived disease control and the BASDAI. Of these patients, 55.3% perceived inadequate control of their disease. The mean BASDAI in the overall population was 43.5 +/- 22.9, 30.4 +/- 19.9 in the well controlled group and 54 +/- 19.4 in the poorly controlled group (p < 0.001). The best BASDAI cutoff for discriminating between patients in the 2 groups was 39 (sensitivity 74.6% and specificity 72.4%). According to gender, the best cutoff was 44 for women and 36 for men. CONCLUSION: The best BASDAI cutoff of 39 based on patients' perceptions was very similar to that selected by international experts, i.e., 40. Gender affected the cutoff for perceived symptom relief in our study. These results need to be confirmed by further studies collecting the opinions of both patients and physicians.

Poorly and well controlled spondyloarthropathies: a comparison of 2 groups of patients.

OBJECTIVE: To describe in a large population of patients with spondyloarthropathy (SpA) the proportion and characteristics of individuals whose disease was poorly controlled by their current treatment, compared to patients whose disease was well controlled. METHODS: We conducted a survey among the members of Spondylis, one of the main not-for-profit SpA patient organizations in France. One thousand anonymous questionnaires were sent to patients throughout France. Among collected data were the opinions of patients about control of their symptoms as well as their past and current treatment. RESULTS: Five hundred and seven respondents were included in the study of whom 75.9% were receiving nonsteroidal antiinflammatory drugs (NSAID), 55% reported inadequate control, and 45% good control of their nocturnal pain and morning stiffness. The Bath Ankylosing Spondylitis disease activity index (BASDAI) and functional index (BASFI) scores and the rates of occurrence of main symptoms were significantly higher in the group with poorly controlled disease. All drugs except NSAID were more often used currently and in the past by patients with poor disease control. CONCLUSION: Conventional treatments failed to provide adequate symptom relief in over half the patients with SpA, despite the use of various drugs in the vast majority of them. Although our results were obtained in a selected patient population, they suggest that a rather large proportion of SpA patients might be candidates for biotherapies.