Dataset Information

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

ABSTRACT:

Setting

Phase II trials for bedaquiline (BDQ) and delamanid (DLM) were completed by 2011 and the drugs were approved by stringent regulatory authorities for the treatment of multidrug-resistant tuberculosis (MDR-TB) between 2012 and 2014. Manufacturers established 'early access' mechanisms to provide drugs before local registration.

Objective

To inform improvements in early access, we explored experiences of providers and advocates in accessing BDQ and DLM before the end of 2015 using a mixed-methods design.

Design

We examined barriers and facilitators to early access through an electronic survey. Barriers and facilitators were classified as occurring at the manufacturer- or country-level. We identified themes using inductive content analysis and illustrated themes through case studies.

Results

We analysed 41 survey responses from 36 respondents reporting on 22 countries; early access was attempted in 30 (73%) survey responses. Eligibility restrictions (11/30, 37%) and complicated and slow processes (8/30, 27%) were manufacturer-level barriers; access to companion drugs (10, 33%) and importation difficulties (4, 13%) were country-level barriers. Previous experience with manufacturer (3/30, 10%) and country processes (2/30, 7%) facilitated access. Eight case studies show the human impact of barriers and facilitators.

Conclusion

Manufacturers and countries should develop transparent processes to permit early access, particularly for diseases that largely affect the poor, such as MDR-TB. Developers should plan for this need and rapidly register drugs with proven benefit, prioritizing high-burden settings.

SUBMITTER: Rodriguez CA

PROVIDER: S-EPMC6436488 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature

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Publications

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

Rodriguez C A CA Brooks M B MB Guglielmetti L L Hewison C C Jachym M F MF Lessem E E Varaine F F Mitnick C D CD

Public health action 20190301 1

<h4>Setting</h4>Phase II trials for bedaquiline (BDQ) and delamanid (DLM) were completed by 2011 and the drugs were approved by stringent regulatory authorities for the treatment of multidrug-resistant tuberculosis (MDR-TB) between 2012 and 2014. Manufacturers established 'early access' mechanisms to provide drugs before local registration.<h4>Objective</h4>To inform improvements in early access, we explored experiences of providers and advocates in accessing BDQ and DLM before the end of 2015 u ...[more]

PMID: 30963040

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Project description:BackgroundAt a time when programs were struggling to design effective regimens for the treatment of multidrug-resistant tuberculosis (MDR-TB), the marketing authorization of bedaquiline and delamanid was a critical development in the MDR-TB treatment landscape. However, despite their availability for routine programmatic use, the uptake of these drugs has remained slow; concerns included a lack of evidence on safety and efficacy and the need to protect the new drugs from the development of acquired resistance. As part of the endTB Project, we aimed to address these barriers by generating evidence on safety and efficacy of bedaquiline or delamanid based MDR-TB regimens.MethodsThis is a protocol for a multi-center prospective cohort study to enroll 2600 patients from April 2015 through September 2018 in 17 countries. The protocol describes inclusion of patients started on treatment with bedaquiline- or delamanid- containing regimens under routine care, who consented to participate in the endTB observational study. Patient follow-up was according to routine monitoring schedules recommended for patients receiving bedaquiline or delamanid as implemented at each endTB site. Therefore, no additional tests were performed as a part of the study. Data were to be collected in a customized, open-source electronic medical record (EMR) system developed as a part of the endTB Project across all 17 countries.DiscussionThe endTB observational study will generate evidence on safety and efficacy of bedaquiline- and delamanid-containing regimens in a large, extremely heterogeneous group of MDR-TB patients, from 17 epidemiologically diverse countries. The systematic, prospective data collection of repeated effectiveness and safety measures, and analyses performed on these data, will improve the quality of evidence available to inform MDR-TB treatment and policy decisions. Further, the resources available to countries through implementation of the endTB project will have permitted countries to: gain experience with the use of these drugs in MDR-TB regimens, improve local capacity to record and report adverse events (pharmacovigilance), and enhance significantly the body of data available for safety evaluation of these drugs and other novel treatments.Trial registrationThis study was registered on 24 August 2017 at clincaltrials.gov (Registration number: NCT03259269).

Project description:BackgroundApp-based treatment for urinary incontinence is a proven effective and cost-effective alternative to care as usual, but successful implementation requires that we identify and address the barriers and facilitators associated with app use.ObjectiveThe goal of the research was to explore the factors influencing app-based treatment for urinary incontinence and identify which barriers or facilitators are associated with treatment success or failure.MethodsWe used a sequential explanatory mixed methods design to connect the results of a randomized controlled trial with data from semistructured interviews. This previous RCT had shown the noninferiority of app-based treatment compared with care as usual for urinary incontinence over 4 months. Participants who reported success or failure with app-based treatment, as measured by the change in International Consultation on Incontinence Modular Questionnaire Urinary Incontinence Short Form symptom score, were selected for telephone interview by purposive sampling (n=17). This study reports mainly on the qualitative component of our mixed methods study. Qualitative analyses were conducted in two ways. First, we analyzed the qualitative data of all interviewed participants and discussed the relationships between the main themes. Second, the experiences between the success (n=9) and failure group (n=8) were compared and contrasted to explore factors that were positively or negatively associated with the quantitative effect of app-based treatment. These factors were then interpreted as barriers to and facilitators of successful app-based treatment.ResultsFour interrelated themes were identified as affecting the app based treatment effect: adherence, personal factors, app factors, and awareness. Qualitative analyses of the relationships between the themes showed that adherence-related factors directly influenced treatment effect in both a positive and negative matter. In turn, adherence was also positively and negatively influenced by the other 3 themes. Additionally, awareness was positively influenced by the treatment effect. Within these themes, several factors were identified that acted as barriers (eg, unrealistic expectation of time investment and interfering personal circumstances), facilitators (eg, strict integration of exercises and prior pelvic floor muscle therapy), or both (eg, personality traits and increased awareness of symptoms).ConclusionsThis study shows that the effect of app-based treatment for urinary incontinence is mainly influenced by adherence, which in turn is affected by personal factors, app-based factors, and awareness. The identified factors could function as both facilitators and barriers depending on the user and interaction with other themes. Insight into these facilitators and barriers could lead to improved implementation and increased treatment effectiveness by targeting women most likely to benefit and through further development of the app.International registered report identifier (irrid)RR2-10.1002/nau.23507.

Dataset Information

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

Setting

Objective

Design

Results

Conclusion

Publications

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

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