Stimulating development of innovative medicines in the European Union: does a new definition for unmet medical need add value?

The European Commission (EC) has proposed to redefine 'unmet medical need' to steer pharmaceutical innovation and link the definition to incentives and regulatory tools and procedures. A multistakeholder meeting of the Regulatory Science Network Netherlands (RSNN) discussed the impact of this proposal. Four principles were highlighted (flexibility, feasibility, fairness, and sensitivity to risk) that were not considered sufficiently addressed.

Cost-utility analysis of longstanding exercise therapy versus usual care in people with rheumatoid arthritis and severe functional limitations.

Objective: To evaluate the cost-effectiveness of longstanding personalized exercise therapy compared with usual care in people with rheumatoid arthritis (RA) and severe functional disability.

Method: In this cost-utility analysis of a randomized controlled trial (n = 215), with 1 year follow-up, the study population comprised individuals with RA and reported severe difficulties in performing basic daily activities. Assessments were at baseline, 12, 26, and 52 weeks, with measurements of costs including medical and non-medical costs as recorded by patients and healthcare providers.

Work-related support for employed and self-employed people with rheumatoid arthritis or axial spondyloarthritis: a cross-sectional online survey of patients.

Background: Little is known about the provision of work-related support for (self-)employed people with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA) by healthcare providers (HCPs) or employers.

Objective: This study aims to explore the experiences of (self-)employed people with RA or axSpA regarding work-related support from HCPs and employers in the Netherlands.

Methods: This cross-sectional study concerned an online survey for (self-)employed people, aged ≥ 16 years and diagnosed with RA or axSpA.

One-year effectiveness of long-term exercise therapy in people with axial spondyloarthritis and severe functional limitations.

Objective: To evaluate the effectiveness of long-term, personalized, supervised exercise therapy on functional ability compared with usual care in people with axial spondyloarthritis (axSpA) and severe functional limitations.

Methods: Participants were randomly 1:1 assigned to the intervention(maximal 64 sessions, with 14 additional optional sessions of supervised active exercise therapy(e.g.

Development of a Multimodal, Physiotherapist-Led, Vocational Intervention for People with Inflammatory Arthritis and Reduced Work Ability: A Mixed-Methods Design Study.

Purpose: Work ability of people with rheumatoid arthritis (RA) and axial spondyloarthritis (axSpA) is reduced, but underexamined as a clinical treatment target. The evidence on vocational interventions indicates that delivery by a single healthcare professional (HCP) may be beneficial. Physiotherapist (PT)-led interventions have potential because PTs are most commonly consulted by RA/axSpA patients in the Netherlands.

The Extent and Nature of Functional Limitations According to the Health Assessment Questionnaire Disability Index in Patients with Rheumatoid Arthritis and Severe Functional Disability.

Background: For a subgroup of people with rheumatoid arthritis (RA) and severe disability, insight into their limitations is crucial for adequate treatment.

Aim: To describe the extent and nature of functional limitations in people with RA and severe disability and to explore the associations of the extent of the functional limitations with patient characteristics, disease characteristics, and outcome measures.

Methods: Baseline data of 215 participants in an RCT on the (cost-)effectiveness of longstanding physiotherapy were used.

Quality of reporting and nature of harms in clinical trials on supervised exercise in patients with rheumatoid arthritis or axial spondyloarthritis.

To describe the quality of reporting and the nature of reported harms in clinical studies on the effectiveness of supervised exercises in patients with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA). We performed a systematic review, searching eight databases up to February 2023. Randomized controlled trials (RCTs) evaluating supervised exercises in adults with RA or axSpA were considered eligible.

Functional limitations of people with rheumatoid arthritis or axial spondyloarthritis and severe functional disability: a cross-sectional descriptive study.

The objective of the study is to describe the nature of functional limitations in activities and participation in people with Rheumatoid Arthritis (RA) or axial SpondyloArthritis (axSpA) with severe functional disability. Baseline data from people with RA (n = 206) or axSpA (n = 155) and severe functional disability participating in an exercise trial were used. Their three most limited activities were derived from the Patient Specific Complaint (PSC) instrument and linked to the International Classification of Functioning and Health (ICF).

Effectiveness and cost-effectiveness of a multimodal, physiotherapist-led, vocational intervention in people with inflammatory arthritis: study protocol of the Physiotherapy WORKs trial.

Background: Although reduced work ability is a substantial problem among people with inflammatory arthritis (IA), work ability is an underexposed area in clinical practice. Evidence on vocational interventions in IA is limited, but favourable results of delivery by a physiotherapist (PT) warrant the need for further research. Therefore, we aim to evaluate the (cost-)effectiveness of a multimodal, PT-led, vocational intervention in (self-)employed people with IA compared to usual care.

Physical therapy in patients with rheumatoid arthritis and axial spondyloarthritis: the patients' perspective.

Objective: To assess the duration, frequency, and content of individual physical therapy (PT) in patients with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA).

Method: In this cross-sectional study, an electronic questionnaire aimed at people with RA and axSpA was distributed through various communication channels of the Dutch Arthritis Foundation. It comprised questions on sociodemographic and health characteristics, received PT (currently and/or in the past year) and, if applicable, its duration, frequency, and content (active exercises, manual treatment, physical modalities, and/or counselling/education).

The use of PROMIS measures in clinical studies in patients with inflammatory arthritis: a systematic review.

Purpose: Although the use of Patient-Reported Outcomes Measurement Information System (PROMIS) measures is widely advocated, little is known on their use in patients with inflammatory arthritis. We systematically describe the use and outcomes of PROMIS measures in clinical studies involving people with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA).

Methods: A systematic review was conducted according to the PRISMA guidelines.

Construct validity of the PROMIS PF-10 in patients with inflammatory rheumatic diseases and severe limitations in physical functioning.

Objective: Assessing the construct validity of the Patient-Reported Outcomes Measurement Information System Physical Function 10-Item Short Form (PROMIS PF-10) in a subpopulation of rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA) patients with severe limitations in physical functioning (PF).

Method: RA/axSpA patients with severe functional limitations completed the PROMIS PF-10, Health Assessment Questionnaire - Disability Index (HAQ-DI for RA) or Bath Ankylosing Spondylitis Functional Index (BASFI for axSpA), 36-item Short Form Health Survey (SF-36), EuroQol 5-dimensions 5-level (index score, EQ-VAS), and performed the Six-Minute Walk Test (6MWT). Construct validity was assessed by computing Spearman rank or Pearson correlation coefficients and testing hypotheses about correlations between the PROMIS PF-10 and measures of PF and quality of life.

Clinically Relevant Deficits in Performance Tests in Patients With Axial Spondyloarthritis.

Objective: To assess the association between self-reported and performance-based physical functioning and to evaluate which performance tests are most frequently impaired in patients with axial spondyloarthritis (axSpA).

Methods: Consecutive patients with axSpA underwent standardized assessments including patient and disease characteristics; patient-reported outcomes for disease activity, functioning, depression, mobility, and physical activity; and performance tests. Patients were defined as being impaired if they were not able to perform ≥ 1 of the performance tests.

Reporting quality of interventions using a wearable activity tracker to improve physical activity in patients with inflammatory arthritis or osteoarthritis: a systematic review.

To assess the reporting quality of interventions aiming at promoting physical activity (PA) using a wearable activity tracker (WAT) in patients with inflammatory arthritis (IA) or hip/knee osteoarthritis (OA). A systematic search was performed in eight databases (including PubMed, Embase and Cochrane Library) for studies published between 2000 and 2022. Two reviewers independently selected studies and extracted data on study characteristics and the reporting of the PA intervention using a WAT using the Consensus on Exercise Reporting Template (CERT) (12 items) and Consolidated Standards of Reporting Trials (CONSORT) E-Health checklist (16 items).

Instrument selection for the ASAS core outcome set for axial spondyloarthritis.

Objectives: To define the instruments for the Assessment of SpondyloArthritis international Society-Outcomes Measures in Rheumatology (ASAS-OMERACT) core domain set for axial spondyloarthritis (axSpA).

Methods: An international working group representing key stakeholders selected the core outcome instruments following a predefined process: (1) identifying candidate instruments using a systematic literature review; (2) reducing the list of candidate instruments by the working group, (3) assessing the instruments' psychometric properties following OMERACT filter 2.2, (4) selection of the core instruments by the working group and (5) voting and endorsement by ASAS.

Differences in characteristics, health status and fulfillment of exercise recommendations between axial spondyloarthritis patients with and without supervised group exercise.

Objective: Since decades, supervised group exercise (SGE) is recommended for people with axial spondyloarthritis (axSpA). This study examines if weekly SGE contributes to fulfillment of exercise recommendations in axSpA patients.

Methods: Cross-sectional data from three studies with axSpA patients in The Netherlands, including two with outpatient populations (n = 196 and n = 153) and one with SGE participants (n = 128), were analysed.

Implementing enhancements in supervised group exercise for people with axial spondyloarthritis: a hybrid effectiveness-implementation study.

Objectives: The content of supervised group exercise (SGE) for axial spondyloarthritis (axSpA) has hardly changed in recent decades, despite new evidence-based insights to improve SGE quality. This pilot implementation study evaluated the effects and feasibility of enhancements in axSpA-specific SGE in four regions in the Netherlands.

Method: The implemented enhancements included: more high-intensity aerobic exercise; exercise personalization with periodic assessments; and patient education on home exercise.

Effectiveness and cost-effectiveness of longstanding exercise therapy versus usual care in patients with axial spondyloarthritis or rheumatoid arthritis and severe limitations: The protocols of two parallel randomized controlled trials.

Objectives: Research on effectiveness and cost-effectiveness of longstanding exercise therapy in patients with axial SpondyloArthritis (axSpA) or Rheumatoid Arthritis (RA) is scarce, and mainly concerned patients with a relatively favorable health status. We aim to evaluate the effectiveness and cost-effectiveness of longstanding exercise therapy compared to usual care in the subgroup of patients with axSpA or RA and severe limitations in functioning.

Methods: In two separate, parallel randomized controlled trials the effectiveness and cost-effectiveness of longstanding, active exercise therapy (52 weeks) compared with usual care (1:1) will be evaluated.

The ASAS-OMERACT core domain set for axial spondyloarthritis.

Background: The current core outcome set for ankylosing spondylitis (AS) has had only minor adaptations since its development 20 years ago. Considering the significant advances in this field during the preceding decades, an update of this core set is necessary.

Objective: To update the ASAS-OMERACT core outcome set for AS into the ASAS-OMERACT core outcome set for axial spondyloarthritis (axSpA).

Organisation and content of supervised group exercise for people with axial spondyloarthritis in The Netherlands.

Supervised group exercise (SGE) is recommended for people with axial spondyloarthritis (axSpA). Recent literature suggests that its contents and dosage must probably be revised. As a first step towards renewal, this study examined the current SGE organisation and content for people with axSpA in The Netherlands.

Adequately dosed aerobic physical activity in people with axial spondyloarthritis: associations with physical therapy.

Introduction: This study aimed to compare the engagement in moderate- and vigorous-intensity PA in axSpA patients with and without current physical therapy (PT).

Methods: In this cross-sectional study, a survey, including current PT treatment (yes/no) and PA, using the 'Short QUestionnaire to ASsess Health-enhancing PA' (SQUASH), was sent to 458 axSpA patients from three Dutch hospitals. From the SQUASH, the proportions meeting aerobic PA recommendations (≥ 150 min/week moderate-, ≥ 75 min/week vigorous-intensity PA or equivalent combination; yes/no) were calculated.

The Ankylosing Spondylitis Performance Index: Reliability and Feasibility of an Objective Test for Physical Functioning.

Objective: Physical function in patients with axial spondyloarthritis (axSpA) is currently evaluated through questionnaires. The Ankylosing Spondylitis Performance Index (ASPI) is a performance-based measure for physical functioning, which has been validated in Dutch patients with radiographic (r-) axSpA. The interrater reliability has not yet been determined.

Responsiveness and Interpretability of 2 Measures of Physical Function in Patients With Spondyloarthritis.

Background: Maintenance or improvement of physical function is an important treatment target in the management of patients with axial spondyloarthritis (axSpA); measurement tools that can detect changes in physical function are therefore important.

Objectives: The objective of this study was to compare responsiveness and interpretability of the patient-reported Bath Ankylosing Spondylitis Functional Index (BASFI) and the Ankylosing Spondylitis Performed-Based Improvement (ASPI) in measuring change in physical function after exercise in patients with axSpA.

Design: This was a sub-study of 58 patients nested within a randomized controlled trial comparing the effect of 12 weeks of exercise with usual care.