Lessons learned from a pilot implementation of physical activity recommendations in axial spondyloarthritis exercise group therapy

Background The Ankylosing Spondylitis Association of Switzerland (SVMB) aimed to implement physical activity recommendations (PAR) within their exercise groups (EGs). The PAR promote exercise in all fitness dimensions at the correct dose. To implement the PAR within EGs, they were translated into a new EG concept with five key activities: (a) training for supervising physiotherapists (PTs), (b) correctly dosed exercises in all fitness dimensions, (c) exercise counselling, (d) bi-annual fitness assessments, and (e) individual exercise training, in addition to EG. All these activities were realized in close coordination with SVMB management. Objectives To analyse the implementation success by evaluating adherence/fidelity, feasibility, and satisfaction at the patient, PTs, and organisational level. Methods The five key activities of the new EG concept were developed, executed, and assessed after 6 months. The primary outcomes for implementation success were adherence of patients to the recommended exercise behaviour, self-reported by electronic diary; fidelity of PTs to the new concept, self-reported by diary; SVMB organisational changes. Secondary outcomes were feasibility and satisfaction with the new EG concept at all three levels. The tertiary outcome, to evaluate the effectiveness of PAR, was patient fitness, assessed through fitness assessments. Results 30 patients with axSpA (ten women, mean age 58 ± 9 years) and four PTs (three women, mean age 46 ± 9 years) participated. The patients' self-reporting of adherence to the PAR was insufficient (43%), possibly due to technical problems with the electronic dairy. The PTs' fidelity to the new EG concept was satisfactory. On all levels, the new concept was generally perceived as feasible and useful for supporting personalised exercise.The frequency of exercise counselling and the fitness assessments was found by patients and PTs to be too high and rigid. Patients' cardiorespiratory fitness [ES 1.21 (95%CI 0.59, 1.89)] and core strength [ES 0.61 (95%CI 0.18, 1.06)] improved over the 6 months. Conclusions The pilot implementation of PAR showed acceptance and satisfaction to be sufficient, thus confirming the need for evidence-based EGs, provided by a patient organisation in order to support active PA behaviour. However, adaptations are necessary to increase its feasibility for nationwide implementation. Trial Registration: SNCTP, SNCTP000002880. Registered 31 May 2018, https://www.kofam.ch/en/snctp-portal/search/0/study/42491.

The PID Life Index: an interactive tool to measure the status of the PID healthcare environment in any given country

Background The “Primary Immunodeficiencies (PIDs) principles of care” were published in 2014 as the gold standard for care of patients with PIDs, setting a common goal for stakeholders to ensure that patients with PID have access to appropriate care and good quality of life. Since then, IPOPI (the International Patient Organisation for Primary Immunodeficiencies), has been working with national PID patient organisations as well as collaborating with scientific and medical institutions and experts to bring these principles closer to the day-to-day life of individuals with PIDs. Method The six PID Principles of Care were revised to consider advances in the field, as well as political developments that had occurred after their initial publication in 2014. Based on this revision the list was updated, and a new principle was added. The six established principles were: diagnosis, treatment, universal health coverage, specialised centres, national patient organisations and registries. Each principle was structured and measured through a series of criteria, and was given the same weight, as they have been considered to all be equally important. Specific weights were attributed to the criteria depending on their relevance and importance to quantify the principle. The index was translated into a survey for data collection: initially involving data from selected countries for a pilot, followed by integration of data from IPOPI’s national member organisations and key countries. Results The PID Life Index was developed in 2020 to assess the status of the PID environment and the implementation of the 6 principles worldwide. The Index allows for benchmarking countries either according to a set of principles and criteria or based on the user’s preferences. This can be displayed in an interactive map or through a data visualisation system. Conclusion The PID Life Index has been developed successfully and has potential to become an important source of information for PID stakeholders, to increase awareness and information as well as support advocacy initiatives on PIDs nationally, regionally or globally.

Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium

New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in developing a treatment for a rare genetic disease. Our unique consortium model incorporated several of the identified developments under one project, DevelopAKUre, truly bringing together academia, industry and patient organisations in clinical drug development. We found that the equal partnership between all parties in our consortium was a key success factor creating a momentum based on a strong organisational culture where all partners had high engagement and taking ownership of the entire programme. With an agreed mutual objective, this provided synergies through connecting the strengths of the individual parties. Another key success factor was the central role of the patient organisation within the management team, and their unique study participants’ advocacy role securing the understanding and meeting the needs of the clinical study participants in real-time. This resulted in an accelerated enrolment into the clinical studies with a high retention rate allowing for delivery of the programme with significantly improved timelines. Our project was partly funded through an external EU research grant, enabling our model with equal partnership. Further attention within the community should be given to establishing a functional framework where sustainable funding and risk sharing between private and public organisations allow for our model to be replicated.

Impact of axial spondyloarthritis on mental health in Europe: results from the EMAS study

ObjectiveTo determine the presence of mental disorder risk and associated factors in European patients with axial spondyloarthritis (axSpA).MethodsData from 2,166 patients with axSpA in 12 European countries were collected from 2017 to 2018 through the European Map of Axial Spondyloarthritis online survey. Risk of mental disorders was assessed using the 12-item General Health Questionnaire. Possible predictors included age, gender, relationship status, patient organisation membership, job status, educational level, Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), functional limitation (0–54) and self-reported depression or anxiety. Bivariate analyses were conducted to determine predictors of risk of mental disorders (Mann-Whitney and χ2) and multivariable analysis identified factors associated with risk of mental disorders.Results60.7% of patients reported risk of mental disorders: they were younger (41.7 vs 46.0 years), more likely female (68.2% vs 57.9%), unemployed (7.5% vs 2.7%), on temporary (15.9% vs 5.4%) or permanent sick leave (13.2% vs 8.0%), reported depression (45.2% vs 14.2%) or anxiety (41.3% vs 12.5%), higher disease activity (BASDAI ≥4; 87.6% vs 62.3%) and functional limitation (16.5 vs 10.8). The factors most associated with risk of mental disorders were disease activity (OR=2.80), reported depression (OR=2.42), anxiety (OR=2.39), being unemployed or on sick leave (OR=1.98), functional limitation (OR=1.02) and younger age (OR=0.97).ConclusionsCompared with the general population, patients with axSpA show disproportionately worse mental health associated mainly with disease activity and employment status. Healthcare professionals should pay close attention to patients with high disease activity and address internally or refer to specialist services, where appropriate to ensure optimal patient outcomes.

Fondazione Telethon and Unione Italiana Lotta alla Distrofia Muscolare, a successful partnership for neuromuscular healthcare research of value for patients

In 2001, Fondazione Telethon and the Italian muscular dystrophy patient organisation Unione Italiana Lotta alla Distrofia Muscolare joined their efforts to design and launch a call for grant applications specifically dedicated to clinical projects in the field of neuromuscular disorders. This strategic initiative, run regularly over the years and still ongoing, aims at supporting research with impact on the daily life of people with a neuromuscular condition and is centred on macro-priorities identified by the patient organisation. It is investigator-driven, and all proposals are peer-reviewed for quality and feasibility. Over the years, this funding program contributed to strengthening the activities of the Italian neuromuscular clinical network, reaching many achievements in healthcare research. Moreover, it has been an enabling factor for innovative therapy experimentation at international level and prepared the clinical ground to make therapies available to Italian patients. The ultimate scope of healthcare research is to ameliorate the delivery of care. In this paper, the achievements of the funded studies are analysed also from this viewpoint, to ascertain to which extent they have fulfilled the original goals established by the patient organisation. The evidence presented indicates that this has been a highly fruitful program. Factors that contributed to its success, lessons learned, challenges, and issues that remain to be addressed are discussed to provide practical examples of an experience that could inspire also other organizations active in the field of rare disease research.

‘More than just chitchat’: a qualitative study concerning the need and potential format of a peer mentor programme for patients with early rheumatoid arthritis

BackgroundPatients recently diagnosed with rheumatoid arthritis (RA) have specific educational and supportive needs. These could partly be addressed with mentoring by other patients living with RA. This qualitative study explores stakeholder perceptions towards peer mentoring in early RA care.MethodsTwo focus groups with patients with early RA (n=10), one with patient organisation representatives (n=5), one with rheumatologists (n=8) and one with rheumatology nurses (n=5) were held. Two patient research partners supported analysis and interpretation.ResultsFour overarching themes were found: added value, experience with peer mentoring, concerns and need in daily care. Patients and patient organisation representatives confirmed the potential of peer mentoring especially regarding sensitive topics not easily discussed with professionals. Patients felt it could provide additional understanding and recognition. Nurses and rheumatologists were less convinced of the added value of peer mentoring because patients never mentioned it and they were concerned about the loss of control over correct information provision. The need for peer mentoring was perceived as person and disease phase-dependent and should therefore be optional, rather than a care standard. The requirements for a peer mentorship programme remained challenging to define for stakeholders. However, all expressed the need for supervision by healthcare professionals and that peer mentors should be carefully selected, educated and matched to newly diagnosed patients.ConclusionPeer mentoring and its implementation remain vague concepts, especially for healthcare providers. However, patients are interested in mentoring by peers, and the current results may support in effectively implementing such programmes early in the disease.

The PID Life Index: An Interactive Tool To Measure The Status of The PID Healthcare Environment in Any Given Country

Background: The “Primary Immunodeficiencies (PIDs) principles of care” were published in 2014 as the gold standard for care of patients with PIDs. Since then, IPOPI, the International Patient Organisation for Primary Immunodeficiencies, has been working with national PID patient organisations as well as collaborating with scientific and medical institutions and experts to bring these principles closer to the day-to-day life of individuals with PIDs. Results: The PID Life Index was developed in 2020 to assess the status of the PID environment and the implementation of the 6 principles worldwide. The Index allows for benchmarking countries according to a set of principles and criteria or based on the user’s preferences. This can be displayed in an interactive map or through a data visualisation system. The 6 PID Principles of Care were reviewed to consider advances in the field of health and science, as well as to include political developments that had occurred since their initial publication in 2014. This resulted in the following 6 principles: PID diagnosis, treatments, universal health coverage, specialised centres, national patient organisations and registries for PIDs. All principles are given the same weight while the criteria have a specific weight. PID Life Index also includes documentation on each country, on PID specialised centres taking care of patients with PIDs (children or adults), on immunoglobulin replacement therapies registered in each country as well as a glossary of the terminology used. Conclusion: The PID Life Index aims at becoming a resource for patients with PIDs and their representatives, healthcare professionals, decision-makers and other relevant stakeholders interested in learning more about the concrete situation of patients with PIDs. With this tool IPOPI wants to increase awareness and information as well as support advocacy initiatives on PIDs nationally, regionally or globally.

International BEAT-PCD Consensus Statement for Infection Prevention and Control for Primary Ciliary Dyskinesia in collaboration with ERN-LUNG PCD Core NETWORK and patient representatives

IntroductionIn primary ciliary dyskinesia (PCD) impaired mucociliary clearance leads to recurrent airway infections and progressive lung destruction, and concern over chronic airway infection and patient-to-patient transmission is considerable. So far, there has been no defined consensus on how to control infection across centres caring for patients with PCD. Within the BEAT-PCD Network, COST Action and ERS CRC together with the ERN-Lung PCD core a first initiative was now taken towards creating such consensus statement.MethodsA multidisciplinary international PCD expert panel was set up to create a consensus statement for infection prevention and control (IP&C) for PCD, covering diagnostic microbiology, infection prevention for specific pathogens considered indicated for treatment, and segregation aspects. Using a modified Delphi process, consensus to a statement demanded at least 80% agreement within the PCD expert panel group. Patient organisation representatives were involved throughout the process.ResultsWe present a consensus statement on 20 IP&C statements for PCD including suggested actions for microbiological identification, indications for treatment of Pseudomonas aeruginosa, Burkholderia cepacia and nontuberculous mycobacteria and suggested segregation aspects aimed to minimise patient-to-patient transmission of infections whether in-hospital, in PCD clinics or wards, or out of hospital at meetings between people with PCD. The statement also includes segregation aspects adapted to the current COVID-19 pandemic.ConclusionThe first ever international consensus statement on IP&C intended specifically for PCD is presented and is targeted clinicians managing paediatric and adult patients with PCD, microbiologists, patient organisations and not least the patients and their families.

Assessing social preferences in reimbursement negotiations for new Pharmaceuticals in Oncology: an experimental design to analyse willingness to pay and willingness to accept

Background Price negotiations for specialty pharmaceuticals take place in a complex market setting. The determination of the added value of new treatments and the related societal willingness to pay are of increasing importance in policy reform debates. From a behavioural economics perspective, potential cognitive biases and other-regarding concerns affecting outcomes of reimbursement negotiations are of interest. An experimental setting to investigate social preferences in reimbursement negotiations for novel, oncology pharmaceuticals was used. Of interest were differences in social preferences caused by incremental changes of the patient outcome. Methods An online experiment was conducted in two separate runs (n = 202, n = 404) on the Amazon Mechanical Turk (MTurk) platform. Populations were split into two (run one) and four (run two) equally sized treatment groups for hypothetical reimbursement decisions. Participants were randomly assigned to the role of a public price regulator for pharmaceuticals (buyer) or a representative of a pharmaceutical company (seller). In run two, role groups were further split into two different price magnitude framings (“real world” vs unconverted “real payoff” prices). Decisions had real monetary effects on other participants (in the role of premium payers or investors) and via charitable donations to a patient organisation (patient benefit). Results 56 (run one) and 59 (run two) percent of participants stated strictly monotone preferences for incremental patient benefit. The mean incremental cost-effectiveness ratio (ICER) against standard of care (SoC) was higher than the initial ICER of the SoC against no care. Regulators stated lower reservation prices in the “real world” prices group compared to their colleagues in the unconverted payoff group. No price group showed any reluctance to trade. Overall, regulators rated the relevance of the patient for their decision higher and the relevance of their own role lower compared to sellers. Conclusions The price magnitude of current oncology treatments affects stated preferences for incremental survival, and assigned responsibilities lead to different opinions on the relevance of affected stakeholders. The design is useful to further assess effects of reimbursement negotiations on societal outcomes like affordability (cost) or availability (access) of new pharmaceuticals and test behavioural policy interventions.