The value of co-creating a clinical outcome assessment strategy for clinical trial research: process and lessons learnt.

BACKGROUND: In support of UCB pharmaceutical research programs, the aim of this research was to implement a novel process for patient involvement in a multidisciplinary research group to co-create a clinical outcome assessment strategy to accurately reflect the experience of people living with early-stage Parkinson's. Patient experts were an integral part of the decision-making process for patient-reported outcome (PRO) research and instrument development. METHODS: In partnership with two patient organizations (Parkinson's UK and the Parkinson's Foundation), 6 patient experts were recruited into a multidisciplinary research group alongside clinical, patient engagement and involvement, regulatory science, and outcome measurement experts. The group was involved across two phases of research; the first phase identified what symptoms are cardinal to the experience of living with early-stage Parkinson's and the second phase involved the development of PRO instruments to better assess the symptoms that are important to people living with early-stage Parkinson's. Patient experts were important in performing a variety of roles, in particular, qualitative study protocol design, conceptual model development, and subsequent co-creation of two PRO instruments. RESULTS: Involving people with Parkinson's in PRO research ensured that the expertise of these representatives from the Parkinson's community shaped and drove the research; as such, PRO instruments were being developed with the patient at the forefront. Working with patient experts required considerable resource and time allocation for planning, communication, document development, and organizing meetings; however, their input enriched the development of PRO instruments and was vital in developing PRO instruments that are more meaningful for people with Parkinson's and clinicians. CONCLUSIONS: Conducting PRO research, in the context of clinical development involving pharmaceutical companies, requires balancing regulatory and scientific rigor with tight time constraints. Incorporating a multi-stakeholder perspective, which included patient experts as joint investigators, had a strong positive impact on our research, despite the logistical complexities of their involvement. Due to the input of patient experts, the innovative clinical outcome assessment strategy and the co-created novel PRO instruments were more relevant and holistic to the patient experience of early-stage Parkinson's.

Patient-reported outcome (PRO) instruments allow people living with a disease and participating in a clinical study to describe the symptoms and experiences that they consider meaningful. PRO instruments use tools such as questionnaires and scales to capture patient perspectives on a treatment that might not be captured by a clinical measurement. It is recommended that the patient community and patient experts are included in the development of PRO instruments to accurately capture information that is important to them. Building on the experience of a recent PRO research project in support of UCB pharmaceutical programs, this article provides recommendations on how pharmaceutical companies can partner with patient organizations and involve patient experts as joint investigators in the co creation of PRO instruments. Despite the additional resource and time required, involving patient experts and patient organizations into the research collaboration had a strong positive impact and ensured that the PROs were meaningful to patients (in this instance, people living with early-stage Parkinson's). Patient organizations facilitated patient engagement and recruitment in research activities, maintained communication with the pharmaceutical company's research team, and built trust between collaborators by implementing patient engagement tools and best practices. Patient experts contributed to several parts of the PRO instrument development process: study design, identifying key symptoms and experiences, and developing individual PRO questions. Co-creation between the pharmaceutical company, patient experts, and patient organizations resulted in considerable improvements to typical PRO instrument development for use in clinical trials and is thus recommended.

Development and early qualitative evidence of two novel patient-reported outcome instruments to assess daily functioning in people with early-stage Parkinson's.

BACKGROUND: Previous research on concepts that are important to people living with early-stage Parkinson's indicated that 'functional' slowness, fine motor skills, and subtle gait abnormalities are cardinal concepts that are not comprehensively captured by existing patient-reported outcome (PRO) instruments that are used in clinical practice and research to assess symptoms and daily functioning within this patient population. We sought to develop novel PRO instruments to address this unmet need. METHODS: PRO instrument development was led by a multidisciplinary research group, including people living with Parkinson's (termed 'patient experts'), as well as patient engagement and involvement, regulatory science, clinical, and outcome measurement experts. A first set of PRO instruments, termed Early Parkinson's Function Slowness (42 items) and Early Parkinson's Mobility (26 items), were drafted to capture 'functional' slowness, fine motor skills, and subtle gait abnormalities. These PRO instruments were used in cognitive debriefing interviews with people living with early-stage Parkinson's (who were not involved with the multidisciplinary research group) to identify issues with relevance, clarity, ease of completion, conceptual overlap, or missing concepts. RESULTS: Sixty people living with early-stage Parkinson's were interviewed, which led to refining the items to 45 for the Early Parkinson's Functional Slowness and 23 for the Early Parkinson's Mobility PRO instruments. Refinement included rewording items to address clarity issues, merging or splitting items to address overlap issues, and adding new items to address missing concepts. The Early Parkinson's Function Slowness PRO instrument resulted in a multidimensional instrument covering upper limb, complex/whole body, general activity, and cognitive functional slowness. The Early Parkinson's Mobility PRO instrument resulted in comprehensive coverage of everyday mobility tasks, with a focus on gait concepts, plus complex/whole body, balance, and lower limb mobility. CONCLUSIONS: The Early Parkinson's Function Slowness and Early Parkinson's Mobility PRO instruments aim to address gaps in existing PRO instruments to measure meaningful symptoms and daily functioning in people living with early-stage Parkinson's. Utilizing a meticulous study design led by a multidisciplinary research group that included patient experts helped to ensure that the PRO instruments were patient-centric, content valid, and meaningful from a clinical and measurement perspective.

Patient Experience in Early-Stage Parkinson's Disease: Using a Mixed Methods Analysis to Identify Which Concepts Are Cardinal for Clinical Trial Outcome Assessment.

INTRODUCTION: Qualitative research on patient experiences in early-stage Parkinson's disease (PD) is limited. It is increasingly acknowledged that clinical outcome assessments used in trials do not fully capture the range of symptoms/impacts that are meaningful to people with early-stage PD. We aimed to conceptualize the patient experience in early-stage PD and identify, from the patient perspective, those cardinal symptoms/impacts which might be more useful to measure in clinical trials. METHODS: In a mixed-methods analysis, 50 people with early-stage PD and nine relatives were interviewed. Study design and results interpretation were led by a multidisciplinary group of patient, clinical, regulatory, and outcome measurements experts, and patient organization representatives. Identification of the cardinal concepts was informed by the relative frequency of reported concepts combined with insights from patient experts and movement disorder specialists. RESULTS: A conceptual model of the patient experience of early-stage PD was developed. Concept elicitation generated 145 unique concepts mapped across motor and non-motor symptoms, function, and impacts. Bradykinesia/slowness (notably in the form of "functional slowness"), tremor, rigidity/stiffness, mobility (particularly fine motor dexterity and subtle gait abnormalities), fatigue, depression, sleep/dreams, and pain were identified as cardinal in early-stage PD. "Functional slowness" (related to discrete tasks involving the upper limbs, complex mobility tasks, and general activities) was deemed to be more relevant than "difficulty" to patients with early-stage PD, who report being slower at completing tasks rather than encountering significant impairment with task completion. CONCLUSION: Patient experiences in early-stage PD are complex and wide-ranging, and the currently available patient-reported outcome (PRO) instruments do not evaluate many early-stage PD concepts such as functional slowness, fine motor skills, and subtle gait abnormalities. The development of a new PRO instrument, created in conjunction with people with PD, that fully assesses symptoms and the experience of living with early-stage PD, is required.

We conducted research to find out about the experiences and symptoms that have the greatest impact on everyday living for people with early-stage Parkinson's disease. This research also looked at which symptoms patients think are important to be tracked in clinical trials. The research team running this study included people living with Parkinson's disease (called "patient experts"). The team also included technical experts and representatives of patient organizations. To begin with, people living with early-stage Parkinson's disease and relatives were interviewed. The interviews collected their thoughts on the impact of early-stage Parkinson's disease on their daily lives. These insights revealed which experiences and symptoms were most important. The research team analyzed ideas and quotes from the interviews to create a picture of early-stage Parkinson's disease. The symptoms that mattered the most to people living with early-stage Parkinson's disease were tremor, rigidity/stiffness, fatigue, depression, sleep/dreams, and pain. Another important symptom was slowness of movement (which is called "bradykinesia/slowness"), and in particular "functional slowness," which included tasks involving the upper limbs, complicated movement tasks, and general activities. Effects on mobility were also important, particularly fine motor skills and subtle walking abnormalities. This research shows the wide-ranging effects that early-stage Parkinson's disease has on patients from their perspective. It also shows which effects are important to capture in trials of therapies aimed at this patient group.

What People with Parkinson's Disease Want.

Parkinson's disease is an incurable, progressive neurodegenerative disease. This condition is complicated by the varying symptoms in individuals who differ in age of onset, symptoms, progression of disease, response to treatment and prognosis. In this paper, we focus on quality of life achieved through a combination of comprehensive health care, continuous support, and self care. Determining what people with Parkinson's disease want is like assembling multiple puzzles simultaneously. While we surmise that patient centered care, support programs, access to comprehensive health care, and relevant symptom control are pieces of this puzzle, more longitudinal studies- which are observational in nature and correlate the impact of symptoms with patients' reported needs- are necessary.

Integrated and patient-centred management of Parkinson's disease: a network model for reshaping chronic neurological care.

Chronic neurological diseases are the leading cause of disability globally. Yet, our health-care systems are not designed to meet the needs of many patients with chronic neurological conditions. Care is fragmented with poor interdisciplinary collaboration and lack of timely access to services and therapies. Furthermore, care is typically reactive, and complex problems are managed inadequately because of a scarcity of disease-specific expertise and insufficient use of non-pharmacological interventions. Treatment plans tend to focus on the disease rather than the individual living with it, and patients are often not involved in clinical decision making. By use of Parkinson's disease as a model condition, we show an integrated care concept with a patient-centred perspective that includes evidence-based solutions to improve health-care delivery for people with chronic neurological conditions. We anticipate that this integrated care model will improve the quality of life for patients, create a positive working environment for health-care professionals, and be affordable.

Multidisciplinary care for people with Parkinson's disease: the new kids on the block!

INTRODUCTION: Parkinson's disease (PD) is a chronic multisystem disorder that causes a wide variety of motor and non-motor symptoms. Over time, the progressive nature of the disease increases the risk of complications such as falls and loss of independence, having a profound impact on quality of life. The complexity and heterogeneity of symptoms therefore warrant a holistic, multidisciplinary approach. Specific healthcare professionals, e.g. the movement disorders neurologist and the PD nurse specialist, are considered essential members of this multidisciplinary team. However, with our increasing knowledge about different aspects of the disease, other disciplines are also being recognized as important contributors to the healthcare team. Areas covered: The authors describe a selection of these relatively newly-recognized disciplines, including the specialist in vascular medicine, gastroenterologist, pulmonologist, neuro-ophthalmologist, urologist, geriatrician/elderly care physician, palliative care specialist and the dentist. Furthermore, they share the view of a person with PD on how patients and caregivers should be involved in the multidisciplinary team. Finally, they have included a perspective on the new role of the movement disorder neurologist, with care delivery via 'tele-neurology'. Expert commentary: Increased awareness about the potential role of these 'new' professionals will further improve disease management and quality of life of PD patients.