Cost-effectiveness of Nursing Interventions for Patients With PD
Cost-effectiveness of Nursing Interventions for Patients With PD
Background: Current guidelines recommend that every person with Parkinson's disease (PD)
 should have access to Parkinson's Disease Nurse Specialist (PDNS) care. Thus, hospitals
 increasingly offer PDNS care to their patients with PD. However, there is currently little
 scientific evidence on the cost-effectiveness of PDNS care. Consequently, many hospitals lack
 the nursing capacity to offer PDNS care to all patients, which creates unequal access to care
 and possibly avoidable disability and costs.

 Objective: The investigators aim to study the (cost-)effectiveness of specialized nursing
 care provided by a PDNS as compared to no PDNS care for people with PD in all disease stages.
 To gain more insight into the used interventions and their effects, a subgroup analysis will
 be performed based on disease duration (diagnosis made <5, 5-10, or >10 years ago).

 Methods: The investigators will perform an 18-month, single-blind, randomized controlled
 clinical trial in eight community hospitals in the Netherlands. A total of 240 people with
 idiopathic PD that have not been treated by a PDNS over the past two years will be included,
 independent of disease severity or duration. In each hospital, 30 patients will randomly be
 allocated in a 1:1 ratio to either PDNS care according to the Dutch Guideline on PDNS care or
 no nursing intervention (continuing usual care). For the allocation of participants, a
 computer-generated list of random numbers will be used. The co-primary outcome measures are
 Quality of Life (QoL) and motor symptoms. Secondary outcomes include PD symptoms, mobility,
 non-motor symptoms, health-related quality of life, experienced quality of care,
 self-management, medication adherence, caregiver quality of life, coping skills and caregiver
 burden. Data will be collected after 12 months and 18 months. A healthcare utilization and
 productivity loss questionnaire will be completed every 3 months by both the patient and the
 caregiver.

 Hypothesis: The investigators hypothesize that, by offering more patients access to PDNS
 care, QoL will increase with equal healthcare costs. Increasing direct medical costs (for
 nurse staffing) will be offset by a reduced number of consultations with the general
 practitioner and neurologist. If these outcomes are reached, wide implementation of PDNS care
 is needed.
1. Background The Parkinson's Disease Nurse Specialist (PDNS) can fulfill a pivotal role in
 the multidisciplinary team of people with PD. The PDNS was introduced in 1989 in the
 United Kingdom to bridge the gap between medical management and the unique personal
 needs of patients. To obtain greater uniformity in care delivery by a PDNS and to
 facilitate the efficacy of nursing care in PD, the Dutch Guideline 'Nursing care in
 Parkinson's disease' was published in 2016. The main roles of the PDNS are clearly
 described in the Guideline, and include 1) providing information, education and
 instruction; 2) supporting the patient and caregiver in the promotion of
 self-management; 3) supporting psychosocial care questions; 4) prevention; 5)
 specialized diagnostic strategies and therapeutic nursing interventions; and 6)
 multidisciplinary collaboration.

 Based on expert opinion from healthcare professionals, the Dutch guideline advises that
 every person with PD could benefit from PDNS care, including those in early-stage
 disease where delivery of information, education about medication compliance and support
 in self-management are critical. So far, only three studies have evaluated PDNS care and
 they have found inconsistent results. Overall, they indicate that PDNS care may improve
 patient wellbeing, physical functioning and general health status and reduce anxiety and
 depression, but definite conclusions could not be drawn. Moreover, there is little
 evidence that shows that quality of life actually improves with PDNS care and to date,
 no studies have been performed that evaluated the cost-effectiveness of PDNS care.

 Currently, most hospitals in The Netherlands offer PDNS care, since it is advised in the
 Dutch Multidisciplinary Guideline. However many centers still lack the nursing capacity
 to offer PDNS care to all patients because the scientific evidence is inconclusive. This
 situation creates an undesirable inequality in access to care and presumably leads to
 avoidable disability and costs (e.g. from early admissions to nursing homes or crisis
 admissions to the hospital). Therefore, the investigators aim to study the
 cost-effectiveness of specialized nursing care provided by a PDNS as compared to no PDNS
 care for people with PD.

 2. Study design The NICE-PD study is an 18-month, single-blind randomized controlled
 clinical trial that will be performed in eight community hospitals in the Netherlands. A
 total of 240 people with PD will be included (120 in each group) and equally distributed
 over the participating hospitals. The investigators have selected hospitals where, due
 to lack of sufficient PDNS staff, only a proportion of PD patients currently has access
 to PDNS care. This provides the investigators with a unique opportunity to identify
 patients who at present have no access to PDNS care, and to randomize them within
 hospitals (at the patient level) between PDNS care and no nursing intervention.

 Eligible patients will be allocated randomly to either PDNS care or usual care in a 1:1
 ratio, using a computer-generated list of random numbers. To ascertain an equal
 representation of patients, the investigators will stratify for disease duration
 (according to pre-defined subgroups, i.e. disease duration <5 years, 5-10 years and >10
 years). The PDNS intervention will be carried out according to the Dutch 'Guideline
 Nursing care in PD'. A blinded researcher will perform the clinical assessments at
 baseline (t0), after 12 months (t1) and after 18 months (t2). Patients and caregivers
 will also be asked to complete a set of questionnaires at t0, t1 and t2. Finally, every
 three months patients and their caregivers will complete an online questionnaire about
 healthcare utilization, costs and productivity loss.

 3. Intervention

 The PDNS intervention will be performed according to the Dutch 'Guideline Nursing care
 in PD' published in 2015. The intervention is not standardized, but tailored to the
 patients' and caregivers' needs. This includes the following:

 -  Assessment of individual care needs of people with PD and their caregivers. The
 PDNS performs a specific nursing assessment related to the medical, physical,
 psychological and social domains.

 -  Development of a patient-centered treatment plan that supports the patient and
 caregiver in self-management. The PDNS composes a multidisciplinary plan, based on
 the results of the individual assessment, and as prioritized by the patient and
 caregiver (shared decision making). The treatment plan is developed according to
 the national self-management framework.

 -  Specific nursing interventions. The intervention varies across disease stages and
 is tailored to the specific problems and needs of individual patients and their
 caregivers. The Guideline on PDNS care describes general- and specific nursing
 interventions. General interventions consist of providing information and
 education, disease management (e.g. considering advanced treatment options such as
 DBS) and monitoring (e.g. of caregiver burden). Specific nursing interventions are
 described for the following areas: mental functions, fatigue, sleep, urogenital
 functions, sexuality, medication adherence, orthostatic hypotension, caregiver
 burden, coping, mobility, self-management and dietary issues (table 2 provides
 examples of such interventions).

 -  Collaboration with other healthcare professionals. The PDNS stimulates and supports
 multidisciplinary collaboration between healthcare professionals based on the
 individual patient-centered treatment plan. The PDNS also plays a pivotal role in
 the timely referral to other healthcare professionals.

 The PDNS will keep a pre-defined electronic study report according to a structured
 format for each PD patient, documenting the individual care needs, present symptoms,
 performed interventions and (changes in) the individual care plan. This report will be
 started at the initial assessment and updated at every follow-up contact with the
 patient, e.g. at the outpatient clinic, during a telephone consultation or at a home
 visit. This data will be purposefully collected for a possible process analysis at the
 end of the study.

 Patients will have regular contact with their PDNS about the progress and realization of
 the personal goals, both during face-to-face contacts and by telephone, and sometimes
 during additional home visits. The frequency and type of contact will be optimized for
 each patient depending on disease stage and individual patient needs. The Guideline on
 PDNS care advises that each patient has a minimum of one contact with the PDNS each
 year. Currently in the Netherlands, patients are seen on average twice a year by their
 PDNS, with an additional two interim telephone consultations per year.

 The control group will receive ongoing/ usual care which is otherwise comparable, but
 without a nursing intervention. This involves regular consultations with a neurologist
 in their own community hospital (typically 2-4 times per year, depending on patient
 preferences and health status). In addition, control patients will have no other
 restrictions considering any other medical treatments (e.g. by a psychologist or social
 worker). Importantly, many important elements of care (including in particular the
 treating neurologist) remain comparable between the two intervention arms because of the
 randomization at patient level within hospitals.

 4. Clinical assessments At baseline, t1 and t2 all patients will visit their own hospital
 for the study assessments which are performed by a blinded researcher (PDQ-39,
 MDS-UPDRS, and TUG). Furthermore, the patients and their caregivers will complete
 additional home questionnaires. In addition, every three months, patients will receive a
 questionnaire at home regarding healthcare utilization, costs and productivity loss over
 the past three months. Caregivers will complete a cost questionnaire including
 healthcare utilization, costs and productivity loss specifically related to caregiver
 burden. Patients can choose if they want to fill out digital or paper questionnaires.

 5. Data analysis The economic evaluation investigates, alongside the clinical trial, the
 value for money of full implementation of the PDNS into PD care from a societal and
 healthcare perspective. The investigators will take all relevant costs into account. The
 cost-effectiveness timeframe adheres to the clinical study protocol and evaluates
 cost-effectiveness up to 18 months after randomization. Cost will be measured using a
 healthcare utilization questionnaire (e.g. including medical consultations, hospital
 admissions, medication, travel costs, etc.) and a questionnaire measuring productivity
 loss while working of both patients and caregivers. Per item of healthcare consumption,
 standard cost-prices will be determined using the guideline for performing economic
 evaluations. If standardized prices are not available, full cost prices will be
 determined using activity based costing. Costs will be analyzed using a mixed model
 approach or a general linear model approach with a gamma distribution using a log link
 to account for possible skewness of the cost data.

 The investigators will use a PD-specific quality of life measure (PDQ-39) and a generic
 health-related quality of life scale (EQ5D) to evaluate the quality of the health status
 of patients. The potential difference in Quality-Adjusted Life Years (QALYs) measured
 with the EQ5D will be analyzed with a regression approach. The investigators will use a
 linear mixed model with repeated measurements to test for differences in quality of life
 (measured with the PDQ-39) between both groups. The same analysis will be used to
 measure differences between groups in the secondary outcome measures. The investigators
 will include study center as a random effect and fixed effects for group, time and the
 interaction between group and time. Each of the outcomes will be included as dependent
 variable. Statistical analyses will be performed based on the intention-to-treat
 principle.

 Besides the overall cost-effectiveness evaluation, the investigators will perform a
 pre-planned subgroup analysis based on disease duration (diagnosis made <5 years, 5-10
 years or >10 years ago) to obtain more insight into the nursing interventions used in
 each disease stage and the effects of PDNS care in these different groups of patients.
 This subgroup analysis will be performed because, for example, for the more severely
 affected patients the nursing intervention is expected to become more intensive and
 possibly more effective, but also more expensive. When different patterns of this kind
 are found, this should be investigated further in future trials that are powered
 adequately to address such group differences.

 6. Discussion The investigators hypothesize that offering PDNS care will lead to higher
 quality of life with equal healthcare costs. Increasing direct medical costs (for nurse
 staffing) are expected to be offset by a reduced number of (telephone) consultations
 with the general practitioner and neurologist. These short-term goals are the focus of
 the present NICE-PD proposal. In addition to the short-term effects, the investigators
 also expect long-term benefits, which are out of scope of the present project. Examples
 of potential long-term benefits include a reduction in the number of nursing home
 admissions and fewer emergency visits to the hospital, which would lead to a substantial
 cost reduction.

 In conclusion, this study will generate new insights into the cost-effectiveness of
 specialized PD nursing interventions for people with PD. If positive results are found, a
 large shift in the organization of PD care is needed to warrant equal access to PDNS care for
 every person with PD.
Inclusion Criteria:

 -  A diagnosis of idiopathic PD;

 -  Sufficient knowledge of the Dutch language to fill out questionnaires;

 -  Age 18 years or older at the time of diagnosis;

 -  All disease stages, regardless of disease severity or disease duration;

 -  Not having received care from a PDNS in the past two years;

 -  A score of ≥ 18 on the Mini-Mental State Examination (MMSE13) and ≥ 12 on the Frontal
 Assessment Battery (FAB14).

 Exclusion Criteria:

 -  A type of atypical parkinsonism caused by medication (e.g. neuroleptics), a metabolic
 disorder (e.g. Wilson's disease), encephalitis or a neurodegenerative disorder (e.g.
 multiple system atrophy, progressive supranuclear palsy, corticobasal syndrome).

 -  Residing in a nursing home or another type of residential care facility (because the
 PDNS is not operational there).

 -  Any other medical or psychiatric disorder that, in the opinion of the researcher, may
 compromise participation in the study.