Patient-Centered Drug Development in Rare Diseases: The Case of Duchenne Muscular Dystrophy

patient preference;patient-centered drug development;patient-centered benefit-risk assessment;rare diseases;Duchenne muscular dystrophy;Public Health Studies

Hollin, Ilene LindsayLehmann, Harold ;

Johns Hopkins University

关键词: patient preference;    patient-centered drug development;    patient-centered benefit-risk assessment;    rare diseases;    Duchenne muscular dystrophy;    Public Health Studies;   

Others  :  https://jscholarship.library.jhu.edu/bitstream/handle/1774.2/60244/HOLLIN-DISSERTATION-2016.pdf?sequence=1&isAllowed=y

瑞士|英语

来源: JOHNS HOPKINS DSpace Repository

【 摘 要 】

Problem StatementPatient-centeredness has gained favor in recent years. Drug development researchers increasingly acknowledge that patient preferences can contribute valuable information to decision-making. The importance of incorporating patient preferences is underscored for rare disease populations in which clinicians and researchers have limited experience with the condition and the disease experience is best understood by patients. This dissertation aims to measure preferences among caregivers and patients with Duchenne muscular dystrophy (DMD).MethodsThis body of work is based on a two online surveys intended for caregivers and patients with DMD. Survey respondents were recruited via a patient registry and snowball sampling. The surveys were designed using a community-engaged approach and included best-worst scaling (BWS) case 1, case 2 and conjoint-analysis. The main analytic methods included latent class analysis, mixed logistic regression and conditional logistic regression.ResultsChapter 3 demonstrates a successful process for community engagement in survey development that affected eligibility criteria, attribute refinement, and revealed the delicate nature of mapping clinical trial endpoints to meaningful survey objects. Chapter 4 findings indicate that caregivers and patients do not differ in their prior- ities for signs and symptoms to be targeted by therapeutic interventions. However, priority heterogeneity does exist and may be related to unobserved characteristics. Chapter 5 findings reveal patients are willing to accept significant risk in order to have the benefit of moderate improvements in pulmonary decline. Finally, methodological findings in Chapter 6 indicate that conjoint analysis combined with BWS provides policy-relevant information about the intention to use therapy and serves to validate the BWS method.ConclusionsThis research contributes to the overall body of knowledge around patient preferences in DMD and as a result may improve the usefulness of future drug development. It also measures patient preferences for a pulmonary benefit, information that will be incorporated into regulatory review. There are implications for the broader rare disease community: this dissertation demonstrates a model for how to undergo this research and underscores the importance of preference work to meet the unique needs of the rare disease community.