Ervin C, Thach A, Lee A, Navia B, Evans E, Doward L. Refinement of the treatment preference questionnaire in adults with Parkinson's Disease and off-episodes. Poster presented at the ISPOR 2019 European Conference; November 6, 2019. Copenhagen, Denmark. [abstract] Value Health. 2019 Nov; 22(S3):S756. doi: 10.1016/j.jval.2019.09.1870

OBJECTIVES: Little is known about factors influencing patients’ OFF-episode treatment preference in Parkinson’s disease (PD). Fit-for-purpose preference questionnaires are lacking in the evaluation of PD treatments. The Treatment Preference Questionnaire (TPQ) was developed to explore patients’ experience with and preference for OFF-episode treatment and further evaluate clinical benefits of APL-130277, a sublingual formulation of apomorphine, shown in a pivotal trial (CTH-300; NCT02469090) to be effective and generally well tolerated in treating OFF-episodes in PD patients. This study confirmed the content validity of the TPQ.

METHODS: Three rounds of qualitative interviews were conducted with adults with PD and OFF-episodes, according to a semi-structured interview guide. Rounds 1 and 2 included concept elicitation and debriefing of the 9-item TPQ; Round 3 included debriefing only.

RESULTS: Twenty-six interviews were conducted with 22 adults (4 participated in both Rounds 2 and 3). All participants reported OFF-episodes and current Levodopa/carbidopa use; mean time since diagnosis was 10.4 years. Rapid onset (73%), efficacy (reduced/alleviated symptoms; 59%), lack of side effects (45%), and mode of administration and portability (32%) were most commonly included in participants’ list of top 3 treatment attributes influencing preference. During debriefing, all participants reported that the TPQ assessed concepts most relevant to determining preference for an OFF-episode medication (specifically ease of use, efficacy, speed of onset, durability, and side effects). Illustrative quotes include: “I'm thinking about feeling better, quicker, longer, being more normal.” “I can't think of anything else you'd want to put in there.” Only treatment cost was noted as missing. All participants reported most items were clear and easy to understand and answer. One item was revised during this process and successfully debriefed in Rounds 2 and 3.

CONCLUSIONS: These data support the salience of TPQ concepts, the ease with which patients understood and answered the TPQ, and overall content validity.

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