| Literature DB >> 34103902 |
Andrew Thach1, Jessie Sutphin2, Joshua Coulter2, Colton Leach2, Eric Pappert3, Carol Mansfield2.
Abstract
INTRODUCTION: Several on-demand treatments are available for management of "OFF" episodes in patients with Parkinson's disease (PD). We evaluated patients' preferences for features of theoretical on-demand treatment options.Entities:
Keywords: Parkinson’s disease; apomorphine sublingual film; discrete choice experiment; patient preference; “OFF” episode
Year: 2021 PMID: 34103902 PMCID: PMC8179791 DOI: 10.2147/PPA.S301644
Source DB: PubMed Journal: Patient Prefer Adherence ISSN: 1177-889X Impact factor: 2.711
Attributes and Attribute Levels of Theoretical Treatments for DCE Questions
| Attribute | Respondent-Facing Attribute Label | Levels |
|---|---|---|
| Mode of administration; adverse eventsa | How you take the OFF episodeb medicine and possible side effect from the medicine | Inhaled; none |
| Time to FULL “ON”c | How long until you are fully back ON | 15 min |
| Duration of FULL “ON”c | How long the OFF episode medicine treats your symptoms | 1 h |
| Out-of-pocket cost per prescription (30 doses) | Cost for a 30-day supply of the medicine | $0 (no cost) |
Notes: aThe mode of administration and mode-specific adverse events were modeled in the experimental design as 1 attribute with 6 levels. In the DCE questions presented to the respondent, the mode of administration and adverse event were put on 2 rows to make the question easier to read (see for an example of a DCE question). b“OFF” episodes were defined in the survey as times when respondents’ regular, daily PD medication did not work to control PD symptoms. cFULL “ON” was defined in the survey as being back to where symptoms normally were when the respondent’s regular, daily PD medicine was working at its best.
Abbreviations: DCE, discrete choice experiment; PD, Parkinson’s disease.
Respondent Characteristics
| Characteristic | Respondents (N = 300) |
|---|---|
| Age, y, mean (SD) | 59.0 (10.7) |
| Male, n (%) | 180 (60) |
| White, n (%) | 248 (83) |
| Time since PD diagnosis, n (%) | |
| <1 y | 9 (3) |
| 1–2 y | 68 (23) |
| 3–4 y | 80 (27) |
| ≥5 y | 142 (47) |
| Do not know or not sure | 1 (<1) |
| Experience with “OFF” episodes,a n (%) | 294 (98) |
| Frequency of “OFF” episodes,b n (%) | |
| Multiple times a day | 74 (25) |
| Once a day | 77 (26) |
| Every few days | 91 (31) |
| Once a week | 28 (9) |
| Every few weeks | 17 (6) |
| Once a month or less | 7 (2) |
| Current PD medications,c n (%) | |
| Levodopa or combination of carbidopa/levodopa | 300 (100) |
| Dopamine agonists | 93 (31) |
| MAO-B inhibitors | 73 (24) |
| COMT inhibitors | 30 (10) |
| Other | 37 (12) |
| Has caregiver,d n (%) | 112 (37) |
| Education, n (%) | |
| High school/GED | 18 (6) |
| College degree/technical school or some college | 185 (62) |
| Graduate/professional degree or some graduate school | 95 (32) |
| Other/prefer not to answer | 2 (<1) |
| Do you currently take a prescription medicine for memory or thinking concerns?, n (%) | |
| Yes | 77 (26) |
| No | 223 (74) |
| What medicines do you currently take for your memory or thinking concerns? (Please check all that apply),e, f n (%) | |
| Rivastigmine | 25 (32) |
| Donepezil | 20 (26) |
| Memantine | 18 (23) |
| Galantamine | 12 (16) |
| Memantine and donepezil | 10 (13) |
| Other | 9 (12) |
| I do not know the name | 7 (9) |
| On a scale of 1 to 5, where 1 is very easy and 5 is very difficult, how difficult do you think it would be for you to take a medicine that is injected/inhaled/dissolves under the tongue using the instructions provided when you are having an “OFF” episode without help from someone else? | |
| Injection, mean (SD) | 3.4 (1.2) |
| Inhaled medicine, mean (SD) | 2.5 (1.2) |
| Dissolvable sublingual film, mean (SD) | 1.9 (1.0) |
| How important is it to you that an on-demand treatment works well first thing in the morning?, n (%) | |
| Very important | 116 (39) |
| Important | 122 (41) |
| Neither important nor unimportant | 40 (13) |
| Not very important | 20 (7) |
| Not important at all | 2 (1) |
| Are you concerned about increasing the amount of levodopa you take?, n (%) | |
| Yes | 136 (45) |
| No | 86 (29) |
| Do not know or not sure | 78 (26) |
| Is someone helping you use the computer to take this survey?, n (%) | |
| Yes | 76 (25) |
| No | 224 (75) |
Notes: aExperience with “OFF” episodes was determined from respondents’ answers to 4 screening questions: Have you ever experienced a time in the morning when your night-time Parkinson’s medication dose has worn off, your Parkinson’s symptoms reemerge, and you are waiting for your first dose of medicine to start working? Have you ever experienced a time, during the day or night, when your Parkinson’s medication has worn off too early and your Parkinson’s symptoms reemerge, but it is not time to take your next dose of medication yet? Have you ever experienced a time when your Parkinson’s medication takes longer to start working than it normally does or when it does not work at all to control your Parkinson’s symptoms? Do you ever experience unpredictable times in the day, unrelated to when you take your medication, when your Parkinson’s symptoms suddenly reemerge without warning? bBased on 294 respondents who had experience with “OFF” episodes. cRespondent responses to this question were distinct from their responses to questions about experience with currently available on-demand treatments of “OFF” episodes. dIncluded full- or part-time caregiver who was not a spouse. eRespondents could provide multiple responses to this question. fBased on 77 respondents who were currently taking a prescription medicine for memory or thinking concerns.
Abbreviations: COMT, catechol-O-methyltransferase; GED, General Educational Development; MAO-B, monoamine oxidase-B; PD, Parkinson’s disease; SD, standard deviation.
Figure 1Normalized mean preference weight estimates for attribute levels of theoretical treatments. The vertical bars surrounding each mean preference weight denote the 95% confidence intervals (CIs) of the point estimate. The preference weights indicate the ranking of levels within each attribute (ie, a higher preference weight indicates that a level is more preferred).
Figure 2Preference share analysis. Results from the discrete choice experiment were used to predict the probability that the average respondent would choose a theoretical on-demand treatment for “OFF” episodes based on 2 different scenarios of 3 theoretical treatments, as presented in . Theoretical treatment A is a dissolvable sublingual film with possible mouth or lip sores, theoretical treatment B is an injection with possible injection-site reactions, and theoretical treatment C is an inhaled drug with possible cough or mild respiratory infection. In scenario 1, theoretical treatment B works faster (15 min) to reach FULL “ON”; in scenario 2, all 3 theoretical treatments reach FULL “ON” in 30 min. In scenario 2, theoretical treatment A has a longer duration of “ON” (1.5 h); in scenario 1, all 3 theoretical treatments have the same duration of “ON” (1 h). In both scenarios, out-of-pocket cost for a 30-day supply is held constant at $30 for each treatment.