Implementing a Delphi Panel to Improve ...

Implementing a Delphi Panel to Improve Understanding of Patient Characteristics of Advanced Parkinson’s Disease

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Angelo Antonini , Per Odin , Leah Kleinman , Anne Skalicky , Thomas Marshall , Kavita Sail , Koray Onuk 1

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Parkinson’s Disease and Movement Disorders Unit, IRCCS Hospital San Camillo, Venice, Italy; 2Department of Neurology, Skåne University Hospital, Lund, Sweden; 3Evidera, Bethesda, Maryland, United States; 4AbbVie Inc., North Chicago, Illinois, United States

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Presented at the 19th International Congress of Parkinson’s Disease and Movement Disorders, June 14–18, 2015, San Diego, California, United States

BACKGROUND • Effective management of Parkinson’s Disease (PD) at all stages is key and often requires individual customization of therapy as the disease progresses1 • Currently there is no clear consensus on how to define the stages of Parkinson’s disease2 and no diagnostic codes available to classify advanced Parkinson’s Disease (APD) • In the absence of a biomarker, diagnostic test or a gold standard index to determine the severity of PD (based on the motor and non-motor symptoms), clinicians most often rely on varied clinical evaluation and medical history to determine staging in PD2 • Lack of consensus on a clear definition of APD may delay the involvement of movement disorder specialist (MDS) and/or introduction of device-aided treatments options for eligible APD patients • Additionally, there is a lack of clear guidance on the appropriate patient profile for the different device-aided treatment options available for treating APD • Recently published systematic reviews and consensus articles acknowledge that there is a growing need to establish guidelines for the different treatment approaches for advanced PD patients3–6

METHODS

RESULTS

CONTINUED

• Stage 2: First Round Delphi – 12 open-ended questions administered via an online survey – Questions were pre-tested for feasibility of usage in an online survey • Stage 3: Second Round Delphi – 6 open-ended questions and 18 close-ended questions with a 4-point response scale were developed based on responses obtained from the first round • Stage 4: Third/Final Round Delphi – In-person meeting of the panelists and the steering committee members – Second round results presented to panel – Anonymous real-time voting in final round to achieve final consensus on the clinical relevance and ranking of key indicators for APD and eligibility for device-aided treatments

OBJECTIVES • To achieve consensus among MDS treating PD patients with regard to the following objectives: 1. Identify the clinically important indicators that define APD 2. Identify patient characteristics that indicate eligibility for device-aided treatment options in APD 3. Identify appropriate patient profile for different device-aided treatment options in APD: Apomorphine, Deep Brain Stimulation (DBS), and Levodopa Carbidopa Intestinal Gel (LCIG)

Round 1 Survey

Stage 2

Questionnaire development and review of the results by a panel of 3 steering committee members. 12 open-ended questions to panel via on-line survey

Analysis Steering Committee review

Round 2 Survey Stage 3

6 open-ended + 18 close-ended questions panel via on-line survey

Analysis Steering Committee review

METHODS

Stage 4

Round 3 Interpretation/Consolidation and Consensus

STUDY DESIGN • The study was conducted using a Delphi panel; a method widely used and accepted involving a structured communication technique for achieving convergence of opinion from a panel of experts7 • The Delphi technique facilitates an efficient group dynamic process and is done in the form of an anonymous, written, multi-stage survey process, where feedback of group opinion is provided after each round • It is expected that, during the Delphi process, the range of the answers decrease and the group will converge towards consensus among experts • There are 4 distinct characteristics of Delphi studies: 1. Anonymity; 2. Iteration; 3. Controlled feedback; 4. Statistical “group response” • For this study, the Delphi process involved 4 stages (Figure 1) and: – Consensus was predefined as ≥70% agreement among the panelists – Near-consensus was predefined as 60–69% agreement among the panelists – No consensus was predefined as a ≤59% agreement among the panelists • Stage 1: Recruitment of panel – 17 MDS from 10 EU countries (Austria, Belgium, Denmark, Germany, Italy, the Netherlands, Norway, Spain, Sweden, and the United Kingdom) were recruited for the panel – Panelists were chosen based on their experience treating PD patients and expertise in treating patients with device-aided therapies – Questionnaire development and review of the results was conducted by a panel of 3 steering committee members

1. Moderate level of troublesome motor fluctuaons 2. At least 2 hours of the day with off symptoms 3. At least 1 hour of the day with troublesome dyskinesia

4. Moderate level of dyskinesia

Non-motor 1. Mild level of demena 2. Non-transitory troublesome hallucinaons 3. Moderate level of psychosis 4. Non-motor symptom fluctuaons 5. Moderate level of nighme sleep disturbances

Funcon 1. Repeated falls* despite opmal treatment 2. Needs help with ADLs at least some of the me

Final consensus using anonymous real-time voting during a live-in person meeting of the panel

Analysis

4. Moderate impaired mobility

• Panelists reached consensus that individual motor symptoms or non-motor symptoms alone may be considered as clinically relevant indicators of APD • Top 3 clinically relevant most important motor symptom indicators: 1. Moderate level of motor fluctuations 2. At least 2 hours of the waking day with off symptoms 3. At least 1 hour of the day with troublesome dyskinesia • Top 3 clinically relevant most important non-motor symptom indicators: 1. Mild levels of dementia 2. Non-transitory troublesome hallucinations 3. Moderate level of psychosis • Top 3 clinically relevant most important function indicators: 1. Repeated falls despite optimal treatment 2. Needing help with activities of daily living (ADLs) at least some of the time 3. Not being able to perform complex tasks

Figure 3. Characteristics of APD Patients Eligible For Device-aided Treatments

RESULTS • A total of 17 MDS participated in the study panel, 15 (88%) participated in all 3 rounds • Panelists represented different countries across Europe, had extensive years of experience treating PD patients (mean: 24.8 ± 7.2 years) and all (100%) were MDS (Table 1)

Table 1. Baseline Demographic Characteristics of All the Delphi Panelists Variable

N = 17

Male, N (%)

11 (65%)

Countries represented (N)

Spain (n = 4), Italy (n = 4), Denmark (n = 2), the Netherlands (n = 1), Norway (n = 1), United Kingdom (n = 1), Germany (n = 1), Austria (n = 1), Belgium (n = 1), Sweden (n = 1)

Specialty of panelist: Movement Disorders, N (%)

17 (100%)

Years working with PD patients, Mean ± SD

24.8 ± 7.2 (range 14–40) years

PD Patients treated per month, Mean ± SD

84.4 ± 50.9 (range 30–250) patients

Motor

• • • • •

• Most important patient characteristics that determine eligibility for device-aided treatments: – At least 2 hours of off-time – Troublesome dyskinesia • Other possible motor, non-motor, and functional indicators: – Off-period postural instability – Freezing of gait during off periods – Nighttime sleep disturbances – Limited ADL

STRENGTHS: • This is the first study to identify, define and rank the clinically relevant motor, non-motor and functional indicators that define APD and eligibility for device-aided treatments • Inclusion of an international panel of movement disorder specialists with extensive years of experience treating PD patients • Use of Delphi technique combining qualitative (open-ended) questions in Round-1 provided inclusion of diverse clinical perspectives and clinician-driven question development for Round-2 and Round-3 • The Delphi technique allows for anonymous feedback over several rounds strengthening the conclusions • The in-person meeting allowed for discussion but did not sacrifice the anonymity of final voting

3. Not able to perform complex tasks — most of the me

Severity definitions were provided by the panelists — Mild: Detectable to clinician but not interfering with daily life (not or minimally troublesome to the patient); Moderate: Detectable to clinician and Influences daily life (troublesome to the patient); Severe: Detectable to clinician and significantly influences daily life (very troublesome to the patient);*Repeated falls was defined as more than 1 fall.

Recruit panel of experts

Steering Committee review

Motor

6. Daily oral levodopa doses “5 mes a day”

Define research questions

Stage 1

Figure 2. Ranking of Clinically Important Motor, Non-motor and Functional Characteristics That Define a Patient Suspected to Have APD

5. Troublesome dysphagia

Figure 1. Study Design

STRENGTHS AND LIMITATIONS

CONTINUED

Troublesome dyskinesia and off-periods At least 2 hours of off-me Off-period postural instability Dystonia with pain Freezing of gait during off

Table 2. Patient Profiles for Device-aided Treatments Characteristics

Apomorphine

DBS

LCIG

Younger age (